Podcasts about oncology center

Launched in 2021 by the FDA's Oncology Center of Excellence, Project Optimus seeks to reshape traditional oncology trial designs and dosing paradigms, shifting from maximum tolerated doses to precision dosing strategies. While the FDA's hope was that the added time and cost of collecting comprehensive dose and exposure response data would benefit both patients and sponsors, there is also concern and debate in the biotech community that, if the Optimus formula is closely followed, the additional time, cost, and patient needs may, in fact, be crippling to cash-strapped emerging companies. So what is the reality around Project Optimus? And how best can it be implemented and navigated?Kicking off a new special series on Project Optimus, in this episode Nick Kenny, Chief Scientific Officer at Syneos Health, is joined by Dr. Wael Harb, Vice President on the Syneos Health Medical Oncology Team, to explore this transformative initiative, diving into the implications for biotech companies and discussing the balance between upfront investment and long-term benefits, the operational challenges of implementation, and the critical role of early planning.Stay tuned for upcoming episodes in this series, which will dive deeper into the regulatory, statistical, clinical, and operational implications of this evolving regulatory framework.For more insights from our oncology experts:Syneos Health Podcast | A Site-Specific Approach to Optimizing Diversity in Oncology TrialsWEBINAR | Transforming Oncology Drug Development: Insights from Healthcare Providers on the Frontlines of Cancer CareSyneos Health Podcast | Circulating Tumor DNA: Transforming Oncology Clinical TrialsNavigating Tumor-Agnostic Drug Development: Value, Challenges and Strategic Insights for SponsorsThe views expressed in this podcast belong solely to the speakers and do not represent those of their organization. If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you'll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision-making and investment. You can find it all at insightshub.health. Like what you're hearing? Be sure to rate and review us! We want to hear from you! If there's a topic you'd like us to cover on a future episode, contact us at podcast@syneoshealth.com.

In this episode, Dr Camidge sits down with Jeanne Fourie Zirkelbach, PhD, a clinical pharmacologist in the Office of Clinical Pharmacology in the Center for Drug Evaluation and Research at the FDA.  Drs Camidge and Zirkelbach discuss how Zirkelbach got her start in the pharmacokinetics field; her involvement with Project Optimus, an initiative by the FDA's Oncology Center of Excellence that works to reform dose-optimization and -selection paradigms in oncology drug development; and how her research with patient-reported outcomes emphasizes the importance of keeping patient needs, preferences, and experiences at the forefront of drug development to minimize the effects of cancer treatment on quality of life.

In this JCO Article Insights episode, Emily Zabor interviews Dr. Gregory H. Reaman, the Scientific Director of the Childhood Cancer Data Initiative at the National Cancer Institute, on their paper titled  “The Childhood Cancer Data Initiative: Using the Power of Data to Learn From and Improve Outcomes for Every Child and Young Adult with Pediatric Cancer”. Dr. Reaman introduces us to the initiative, its goals and structure, and what has already been achieved since its launch. TRANSCRIPT The guest on this podcast episode has no disclosures to declare.  Emily Zabor: Welcome to this JCO Article Insights episode for the August issue of JCO. This is Emily Zabor, JCO's Biostatistics Editorial Fellow. And today I am interviewing Dr. Gregory Reaman, the Scientific Director of the Childhood Cancer Data Initiative at the National Cancer Institute, on their paper titled “The Childhood Cancer Data Initiative: Using the Power of Data to Learn from and Improve Outcomes for Every Child and Young Adult with Pediatric Cancer.” Dr. Reaman, welcome to our podcast. Dr. Gregory Reaman: Thanks very much, Emily. Appreciate the invitation. Emily Zabor: Dr. Reaman, could you start by introducing yourself and describing your involvement in the Childhood Cancer Data Initiative? Dr. Gregory Reaman: I'm Gregory Reaman. I'm a Pediatric Oncologist. And I guess my involvement with the CCDI began shortly after the initiative was announced at the State of the Union media address in 2019, which was followed shortly thereafter by the formation of a working group by the NCI's National Cancer Advisory Board Board of Scientific Advisors. Given my role at the FDA at that time as Associate Director for Pediatric Oncology in the Oncology Center of Excellence, and the fact that I was the founding Chair of the Children's Oncology Group, I was an ex-officio member of this working group. So from very early on, I had involvement. I formally joined the NCI in November, left the FDA to assume the position as Scientific Director of CCDI. Emily Zabor: That's great. So you've really been involved from the start. I had not really been familiar with the initiative until I read this paper. And as a cancer biostatistician, I was really excited to learn about this initiative, which sounds like it will ultimately create a very valuable data resource to be used for research purposes, among other things. So I think it's a really interesting project. So for our listeners who may not be familiar, could you describe the motivation for and goals of the Childhood Cancer Data Initiative? Dr. Gregory Reaman: As I mentioned, this really is a very unique initiative, venture, if you will, on the part of the Cancer Institute and in large part driven by this fortunate infusion of funds to support childhood cancer research. And given the fact that pediatric oncology is very much a collaborative enterprise, it really does sort of follow that data sharing and using the power of data, its ability to be used by multiple investigators, irrespective of the source, aspirationally can improve outcomes for children cancer. The three primary objectives– actually, this working group that I mentioned earlier put together a white paper that had 24 specific recommendations to the NCI. But there were three foundational objectives or goals. One was to learn from every child diagnosed with cancer, irrespective of the institution where they were diagnosed to receive therapy, to develop an ecosystem that would enable the submission, aggregation of data, and harmonization in a federated system that could then be accessed and used by investigators and analyzed to ultimately improve outcomes. And then one objective, which was a little bit more specific, and that was to really focus on the opportunity to genomically classify tumors from newly diagnosed pediatric cancer patients, because this was something that obviously is much more widespread in the adult population, given the advent of targeted therapy and precision oncology and its more widespread use in medical oncology than pediatrics. And although many large academic institutions do have resources, the majority of smaller institutions do not. And when it's necessary and preferable to accurately and timely identify or diagnose a child's cancer that may actually provide information on treatment recommendations, the ability to do that and have it covered by insurance is sometimes problematic. So developing a program that would not cost patients or institutions anything and then make that data available to patients, families, and providers, as well as making it available for secondary research use, was a major goal and objective. Emily Zabor: Yeah, that sounds like such an important initiative. The Molecular Characterization Initiative, which I understand has already enrolled and characterized the genomics of 751 participants just in the first year, I think is what the paper reported? Dr. Gregory Reaman: That's correct. That was in the first year. We're now beyond the first year and we're approaching 2000 patients that have had their tumors genotyped and about 1500 results that have been returned to patients and providers. Emily Zabor: That's fantastic. So with this linkage to the clinical data, that's going to be an extremely important data source. And I understand that the participation is currently limited to members of the Children's Oncology Group, which consists of over 200 children's hospitals, universities, and cancer centers. Can you describe in more detail what information it currently provides and how this initiative is going to be advertised and implemented to ensure complete participation across all of these member sites? Because that sounds like a really big challenge. Dr. Gregory Reaman: Limiting the participation to the Children's Oncology Group initially was in no way meant to be exclusionary, but really provided an opportunity for linkage to clinical data. Since the Children's Oncology Group really represents nearly all of the pediatric cancer programs in the United States and some programs even outside the US, in Canada, and a couple of European sites, Australia and New Zealand, it was felt that given the resources that currently exist within the COG for specimen procurement, specimen submission, and then DNA and RNA extraction through the COG's Biopathology Center at the Nationwide Children's Hospital would really facilitate having the sequencing done at a single site, single institution, using a single platform. And also it provided an opportunity for some clinical data, including demographics, diagnosis, radiographic data, and treatment data that could be collected somewhat longitudinally from patients enrolled on the MCI. Looking to make this as broad as possible since the objective of the CCDI is to learn from every patient, and every patient that we're concerned about not being able to capture adequately within the Children's Oncology Group are older adolescents and young adults with cancers that are more frequently seen in the pediatric population. So we are looking at ways to work with the COG's Biopathology Center to see if we can create systems that we can actually have specimens submitted from patients seen at institutions outside of the COG and molecularly characterized the same way. And that will be important as we launch another new planned initiative called the Coordinated National Initiative for the Treatment of Rare Pediatric and Young Adult Cancers. Emily Zabor: Okay, that makes sense. So those adolescents and young adults are harder to capture since they're not being seen at those COG member institutions. Okay, well, that sounds like a big challenge to find those patients at their institutions and get them involved, but I think it's an important piece of this for sure. Dr. Gregory Reaman: I should also point out that there were opportunities for some of the larger well, for all of the NCI-designated cancer centers, the pediatric programs associated with those cancer centers, to submit genomic data on newly diagnosed patients. That was something that actually transpired early on in the history of CCDI. So those data are in the CCDI's ecosystem. Emily Zabor: Oh, that's great. So you collected the existing data. Dr. Gregory Reaman: Right.  Emily Zabor: That kind of leads into my next question about aggregating data sources. With these disparate sources of pediatric cancer data, it seems like the aggregation is a lofty and important goal, but once that's complete, you're going to have this data ecosystem, which you said was one of the main goals of this initiative. I was wondering if you could tell us who will have access to this data ecosystem and what will be required for individuals to gain access. Dr. Gregory Reaman: All of CCDI was predicated on this really being a community initiative if you will, so multidisciplinary and community-based. So patients, families, advocates, clinical researchers, physician providers, basic and translational researchers, researchers in public health and epidemiology. So there will be different levels of data that will be available to specific individuals. Patient-level data will be deidentified through a system of APIs that will be used that will enable the association of clinical data to existing molecular data and outcome data that might be available in the ecosystem. Those data will be- there are many data in the ecosystem that will be open source and available to anyone who is interested. This includes data from the NCCR in the Childhood Cancer Data Catalog, which is basically a listing of some close to 300 pediatric cancer databases that are available. The patient-level data will be sort of a controlled access. So there will be a requirement for individuals, investigators who wish to access that data, to sort of be certified, if you will, utilizing NCI and NIH data sharing requirements. Emily Zabor: That makes sense. Yeah, you mentioned deidentification, but especially when we're dealing with these kinds of rare diseases, patient privacy does seem like it could be a concern. So what exactly are you doing to ensure that that is not something that gets violated through this process? Dr. Gregory Reaman: I think there's every attempt to eliminate any PPI, HPI, obviously. So, again, most of the clinical data that are being provided currently are data that's coming from the Children's Oncology Group, where for every patient enrolled or registered through the COG and enrolled on a clinical trial, there is a COG ID number that is associated and that will be available only to the NCI and the CCDI to link it to unique specimen identification numbers, which are the only numbers that will be available to any investigator. So no one will be able to make the connection from the specimen identifier to the unique patient identifier in the COG. Emily Zabor: That's great. And that way, you can really get access to all of the detailed data without concerns about privacy. Dr. Gregory Reaman: Correct. And then being able to link all of these disparate data sets will really require the identification or the development, I should say, of a participant index. So that is one of our highest priorities right now in developing a CCDI participant index so that we would be able to link the identifier or clinical data with any research data or biologic data that may be available on patients to facilitate research plans and programs. Emily Zabor: And through that process, is there also some method involved for identifying duplicated data? Because I assume some of these patients may get seen at different institutions over time, and that could be a concern that they end up in the database multiple times. Dr. Gregory Reaman: That's exactly why I think developing the participant index is so critical to, number one, link, and number two, to avoid, prevent duplication, because you're absolutely right. There may well be the same patient data in multiple data sets, which are, of course, disparate. And the only way that they're going to be really utilizable and made interoperable is by linking them to the specific patient or individual patient. Emily Zabor: Great. And do you have an idea of the timeline when that part would be complete and this data ecosystem would be available to researchers?  Dr. Gregory Reaman: The ecosystem is already available to researchers. We launched several months ago the CCDI hub, which is sort of the entryway or entry point, if you will, for access to the ecosystem. We hope to actually have the participant index up and running, and it's something that we've been working on for over a year, but actually available and utilizable within the next several months.  Emily Zabor: That's fantastic. We'll have to go check out the CCDI hub that's already out there then. Before we end, is there anything you'd like to share with our listeners that we haven't already discussed? Dr. Gregory Reaman: Well, I think the one program that I mentioned just briefly, the Coordinated National Initiative for Rare Pediatric and Young Adult Cancers, we see there's a real opportunity to address a major unmet need. Fortunately, all pediatric cancer is rare, but there are some cancers that are extremely rare and for which there are, in many cases, no defining standard of care, and in many cases, there are no treatment protocols because of the difficulty mounting studies with such small patient numbers. So we see this as an opportunity to actually develop a registry that will provide, hopefully, natural history data that will inform clinical trials. All of these patients will be enrolled on the Molecular Characterization Initiative. So there will be the opportunity to hopefully learn if there are specific molecular drivers of some of these cancers that could inform the use of targeted drugs in a therapeutic approach to some of these. And we're looking to do this international collaboration with colleagues in the EU as well. So that is something that we just launched a task force  to develop a listing of core critical data elements to collect on patients and then developing the registries for a number of these rare cancers. Emily Zabor: That sounds like it's going to be a really valuable resource for planning and designing future clinical trials, so I'm glad to hear about that. Dr. Gregory Reaman: And we would invite anyone who's interested to find out about the CCDI, to find out more about the CCDI, which they can do through cancer.gov/ccdi. There is an opportunity for people to register for newsletters. We have a series of webinars, many of which are designed now to actually provide training on some of the resources and platforms that are available currently through the ecosystem and things that we have all planned for future developments and use. So as I said, this is a community venture and we look to expand the community in every way possible. Emily Zabor: That sounds great. So hopefully our listeners will take note of some of those resources in addition to this paper being out there, which will guide some people in the right direction to learn about this really great initiative for childhood cancer.  So, Dr. Reaman, it has been a pleasure speaking with you. And thank you so much for joining me today on this episode of JCO Article Insights. Dr. Gregory Reaman: Thank you very much. It's been great to be here. Appreciate the opportunity.  Emily Zabor: This concludes this episode on the article, “The Childhood Cancer Data Initiative Using the Power of Data to Learn from and Improve Outcomes for Every Child and Young Adult with Pediatric Cancer.” Thank you all for listening and please tune in for the next issue of JCO Article Insights.  The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions.   Guests on this podcast express their own opinions, experiences, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement.  

In this episode, we hear from Sean Khozin, Senior Partner at the investment firm Braven Partners. He focuses on advancing in innovations at the intersection of biology, technology and AI. Sean was previously the CEO of CancerLinQ and the Global Head of Data Strategy and Data Science at Johnson & Johnson. He had a successful tenure in the US federal government as the associate director of FDA's Oncology Center of Excellence and the founding executive director of FDA INFORMED, a data science and technology incubator he established under special authorities from the US department of health and human services. Topics include: How are Braven Partners incubating and supporting minimally viable ecosystems of companies that can make a difference? What are the goals of the Alliance for Artificial Intelligence in Healthcare? How can digital therapeutics developers ensure effective reimbursement for their products in the healthcare industry? How is the ethos of AI in healthcare speaking to a culture that is focused on safety without compromising innovation? How do we demystify new tools and technologies such as AI? How does the FDA think about regulating AI? What can digital therapeutic manufacturers companies, entrepreneurs and trailblazers learn from the Alliance for Artificial Intelligence in Healthcare (AAIH)? Guest Links and Resources: Connect with Sean Khozin on LinkedIn Visit Braven Partners Episode PDF Host Links: Connect with Eugene Borukhovich: Twitter | LinkedIn Connect with Chandana Fitzgerald, MD: Twitter | LinkedIn Connect with YourCoach.health: Website | Twitter Check out Shot of Digital Health with Eugene and Jim Joyce: Website | Podcast App HealthXL: Website | Twitter | Join an Event Digital Therapeutics Podcast would not be possible without the support of leading DTx organizations. Thank you to: > Presenting Partner: Amalgam Rx > Contributing Partners and Sponsors: LSI | Bayer G4A | Lindus Health Follow Digital Health Today: Browse Episodes | Twitter | LinkedIn | Facebook | Instagram Follow Health Podcast Network: Browse Shows | LinkedIn | Twitter | Facebook | Instagram

ASCO: You're listening to a podcast from Cancer.Net. This cancer information website is produced by the American Society of Clinical Oncology, known as ASCO, the voice of the world's oncology professionals. The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. Guests' statements on this podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement. Cancer research discussed in this podcast is ongoing, so data described here may change as research progresses. Brielle Collins: Hi everyone, I'm Brielle Gregory Collins, a member of the Cancer.Net content team, and I'll be your host for today's Cancer.Net podcast. Cancer.Net is the patient information website of ASCO, the American Society of Clinical Oncology. June 15th to June 21st, 2023, marks the third annual National Black Family Cancer Awareness Week, an initiative led by the U.S. Food and Drug Administration's, or FDA's, Oncology Center of Excellence to increase cancer awareness within the Black community. Today we're going to be talking about cancer disparities in the Black community, the importance of cancer screening and prevention for Black families, and resources available to Black families for support. Our guests today are Dr. Luckson Mathieu and Rea Blakey. Dr. Mathieu is a thoracic oncologist at the FDA in the Division of Oncology 2. Thanks for joining us today, Dr. Mathieu. Dr. Luckson Mathieu: Happy to be here. Thank you for inviting me. Brielle Collins: Ms. Blakey is the Associate Director for External Outreach and Engagement at the Oncology Center of Excellence and leads the National Black Family Cancer Awareness Initiative for the Oncology Center of Excellence Project Community. Thanks for joining us today, Ms. Blakey. Rea Blakey: Thank you, happy to be here. Brielle Collins: Before we begin, we should mention that Dr. Mathieu and Ms. Blakey do not have any relationships to disclose related to this podcast, but you can find their full disclosure statements on Cancer.Net. Now to begin, Dr. Mathieu, research has shown that Black people are more adversely affected by cancer than other racial and ethnic groups in the U.S. Can you describe some of the cancer disparities that exist in the Black community? Luckson Mathieu: Sure, thank you for that question. Before providing a description, I would like to first define cancer health disparities. The National Cancer Institute, or the NCI, defines cancer health disparities as adverse differences that exist among certain population groups and cancer measures, such as numbers of cases, the number of deaths, cancer-related health complications, and quality of life after cancer treatment. Black and African American people have higher rates of acquiring and dying from cancer compared to members of other races. For many of the most common types of cancer, including breast, lung, prostate, and colorectal, the incidence and deaths are higher among African Americans than any other racial and ethnic groups. Furthermore, despite having similar rates of breast cancer, African American women are more likely than White women to die of this disease. African American men have a prostate cancer death rate more than double than that of men of other racial groups. Unfortunately, my description is a brief depiction of an alarming and expansive reality. Brielle Collins: Thank you for walking through that, Dr. Mathieu. And thank you, too, for providing that definition of disparities. And Ms. Blakey, can you describe the purpose of National Black Family Cancer Awareness Week and its role in addressing these disparities and raising cancer awareness in the Black community? Rea Blakey: Sure, happy to. The purpose of the National Black Family Cancer Awareness initiative and the dedicated social media week is to increase cancer awareness in one of the most vulnerable segments of the U.S. population, as you just heard described. OCE's Project Community appreciates ASCO's Cancer.Net involvement, absolutely. We also aim to marshal community-based stakeholders, faith-based organizations, historically Black colleges and universities, Black sororities and fraternities, all of this to increase cancer awareness and to build knowledge surrounding cancer clinical trial participation, as well as minority population donations to national genetic databases for cancer research. So OCE's Project Community is the hub of the social media campaign for the National Black Family Cancer Awareness Week via our hashtag, #BlackFamCan. Project Community's intent is to enlist and encourage a wide array of public and private community-focused engagement entities, organizations, families even, throughout the U.S. and beyond, to support efforts to increase cancer clinical trial awareness. So with a common and concerted mission, organizers are urged to focus their supportive endeavors and activities to occur during National Black Family Cancer Awareness Week. That's also in conjunction with the White House Cancer Moonshot Goals. Brielle Collins: Wonderful, and it sounds like that hashtag, #BlackFamCan, is a good place for people to go if they want to learn more as the week progresses. Rea Blakey: Absolutely. Brielle Collins: Wonderful. And Ms. Blakey, what do you think is most important for Black families to know about their cancer risk? Rea Blakey: The hashtag, #BlackFamCan, and there's also a tagline that's called “Engaging the Generations.” So we know African Americans have the highest mortality rate of any racial and ethnic groups for all cancers combined and for most major cancers. That contributes to a lower life expectancy, obviously, for African American men and women. And so with that, the real effort here is to get people to talk to their families. All too often, families don't really know their own cancer history, and some find it just too difficult to talk about, especially with older generations who may have associated a stigma with a cancer diagnosis, or those who just don't tell to avoid being perceived as a burden to others in their family. So engaging the generations is one of the key aspects of personal or familial cancer awareness and understanding risk and mapping out preventive strategies and pursuing cancer screening. Not only does it take a village, but it requires every generation. Brielle Collins: Absolutely. And I want to build on that piece of cancer screening. So Dr. Mathieu, can you talk a little bit about why cancer screening is so important and some of the hurdles that Black families may face in getting regular screening? Luckson Mathieu: Yeah, absolutely. So cancer screening tests, such as Pap smears, mammogram, low-dose CT scans, and colonoscopy, can help find cancer at an early stage before symptoms appear. When abnormal tissue and cancer is found early, it may be the best time or the more easier time to treat. And treatment may even result in cure. By the time symptoms appear, cancers may have grown and spread, thereby making it more challenging to treat and/or cure. Black people are at the highest risk for cancer deaths. This increased mortality risk may reflect a later stage disease at the time of diagnosis among Black patients. Cancer screening is so important for everyone, especially Black people, because it helps identify cancer early and thereby allows for better clinical outcomes. Regarding the hurdles for cancer screening, I believe the hurdles varies for each family. There may be many complex and interrelated factors that can stand in the way of screening for Black families. Each family should directly address that question of what is in the way of getting appropriate screening for cancer. Identifying and overcoming the hurdles may be the best way to address this cancer screening disparity. Brielle Collins: Got it. Thank you for walking through that. And in terms of resources, Dr. Mathieu, what resources are available to help Black people access this screening? Luckson Mathieu: So your primary care physician can serve as a good start to discover cancer screening resources. Earlier this year, the Department of Health and Human Services announced the Accelerating Cancer Screening Program. The program's goal is to accelerate access to cancer screening as part of the Cancer Moonshot Initiative. I would encourage everyone to go online and consider the role a local HRSA-supported health center can play in the process of getting screened for cancer. In addition, NCCN.org, CDC.gov, American Cancer Society, and the FDA are great online resources to obtain more information on cancer screening. Brielle Collins: Perfect. And I really want to talk about that cancer prevention piece, too, which is another incredibly important element of this. So why is cancer prevention in particular so important, and what measures can Black families take to prevent cancer? Luckson Mathieu: Yeah, cancer prevention is important because the best treatment for cancer is to prevent it from occurring at all or catch it at the earliest and most curable stage. In addition, cancer prevention offers the most cost-effective long-term strategy to manage cancer's devastating societal impact. As previously mentioned, screening tests can find cancers early when they are treatable and it has the prospect of cure. In addition to regular screening tests, everyday behaviors such as not smoking cigarettes, maintaining a healthy weight, and being vaccinated against certain cancer-causing viruses can all help prevent cancer from developing. Brielle Collins: Thank you. And Ms. Blakey, going back to some resources, what resources are available to help Black families as they navigate cancer screening and prevention? Rea Blakey: Well, I'll start with the prevention aspect first. The Centers for Disease Control and Prevention, the CDC, has great resource information for reducing cancer risk. Anyone can share the web page links or even print out materials to hand out to their communities. There's information on the importance of family health history and cancer, on the correlation between alcohol consumption and cancer, cancers related to obesity, smoking cessation resources. And often these resources are culturally curated to meet the needs of a variety of communities, including African Americans, Latinos, LGBTQ. For resources to help families navigate cancer screening, I would recommend that all Americans become familiar with the recommended cancer screening tests that are listed on the National Cancer Institute webpage. It's specific to screening tests. So along with becoming aware of the cancer screening possibilities, you would also want to find a health care provider that you trust. We know trust is a huge issue. And ask questions to help you understand the best cancer screening plan for you. NCI's resource, for example, recommends you ask questions like, are any cancer screening tests recommended for me and which ones? And what's the purpose of the test? Does the test require preparation? How do I do that? These are questions you should be comfortable asking your health care provider and know more about so that you are well equipped to do as much as you can, to make sure that your screening options are actually taken advantage of, and that you do what you can to reduce your risk. You might also want to ask, how often should I have the test and at what age should I stop having that test? Brielle Collins: Got it. Thank you for that. And I know we touched on this earlier in the podcast, but I just want to circle it back here with National Black Family Cancer Awareness Week. But where can people go online throughout this week? We mentioned that hashtag, #BlackFamCan, but are there other resources available during the week that people can go to online? Rea Blakey: Absolutely. We have a webpage and a dedicated social media toolkit. So anyone who uses an online search engine and looks up National Black Family Cancer Awareness should see our webpage. On that page, you will find all kinds of resources that include, for example, in the social media toolkit, videos and graphics, as well as a customizable selfie frame for those who are, please do use the hashtag #BlackFamCan. Thank you. Brielle Collins: Wonderful. It sounds like it's going to be a very engaging week. Thank you for that. And thank you both so much for your time and for sharing your expertise today, Dr. Mathieu and Ms. Blakey. It was so great having you both. Rea Blakey: Thank you, Brielle! Luckson Mathieu: Thank you very much. Brielle Collins: For more information, you can view this podcast on Cancer.Net, where you can also find a link to all the resources mentioned around National Black Family Cancer Awareness Week. ASCO: Cancer.Net Podcasts feature trusted, timely, and compassionate information for people with cancer, survivors, and their families and loved ones. Subscribe wherever you listen to podcasts for expert information and tips on coping with cancer, recaps of the latest research advances, and thoughtful discussions on cancer care. And check out other ASCO Podcasts to hear the latest interviews and insights from thought leaders, innovators, experts, and pioneers in oncology. Cancer.Net is supported by Conquer Cancer, the ASCO Foundation, which funds lifesaving research for every type of cancer, helping people with cancer everywhere. To help fund Cancer.Net and programs like it, donate at CONQUER.ORG/Donate.

There are an estimated 1.6 million transgender people in the U.S. alone, with about 25% identifying as nonbinary. However, based on our research for the Beyond the Binary: Navigating Gender Diversity in Clinical Research report, we found that of the 141,000+ articles published on PubMed (a database of biomedical and life sciences literature) reporting interventional clinical trial results from 2018-2022, fewer than 1% reported study participation of transgender and nonbinary patients. We then set out to determine why this patient population is not being accurately represented in clinical research.      In this episode, Parexel subject matter experts – Rosamund Round, Vice President, Patient Engagement Center; Liam Paschall, Senior Consultant, Management Development; Amy McKee, M.D., Chief Medical Officer & Global Head, Oncology Center of Excellence – discuss the barriers faced by the transgender and nonbinary communities and provide recommendations for more gender-inclusive clinical trials.   To view the Discussions on Diversity report, click here.      

Megan-Claire Chase is a breast cancer survivor who deals with a lot of effects from treatment and being diagnosed with lobular breast cancer at a very young age. But she excitedly begins our conversation by saying her pain melted away at the San Antonio Breast Cancer Symposium when she was able to meet patient advocates in person for the first time. Social media connects many of us but the conference we both attended in early December of 2023 allowed us to sit down together to share intimate conversations, ask each other how we are doing, and share our advocacy work. I was fortunate to be one of those advocates to finally meet her in person at the meeting. Megan-Claire experienced strange symptoms for about two years that were left without a diagnosis. Her mother had ovarian cancer when she was pregnant with Megan-Claire. She states she felt this made her a miracle baby for her parents. Megan-Claire felt certain at some point in her lifetime she would get cancer because she has a strong history in her family. Because of this she was able to get a mammogram at age thirty-five and have it covered by insurance. Being hyper aware of her body due to family history of cancer, the signs she saw were concerning. She noticed tiny green bruising on her legs, weight gain, loss of hair on the left side of her head, and a “zit” on the outside of her left breast. She knew something was not right. Megan-Claire shares with us she would like the medical community to be more aware that black women and other women of color will present with different symptoms that her white counterparts. This leads us to a more in-depth conversation on health disparities, implicit biases in breast cancer care and what Megan-Claire accomplishes in her community and advocacy work to improve the communication between the patient and physician. Megan-Claire will be participating in a community event to connect patients and provide these resources June 15-21 of 2023. The event and other ways to join Megan-Claire on social media can be found here. National Black Family Cancer Awareness Week started by the FDA through the Oncology Center of Excellence. Megan-Claire's Linked Account: https://www.linkedin.com/in/megan-claire-chase/ Breast Cancer Program Director: Share Cancer Support          

What factors could be excluding transgender people from oncology clinical trials, and what can we do to make them more inclusive? Dr. Westin discusses this important issue with her guests, Dr. Ash Alpert and Dr. Lola Fashoyin-Aje. TRANSCRIPT Dr. Shannon Westin: Hello, everyone, and welcome to another episode of the JCO After Hours podcast. This is when we get down and dirty into manuscripts that are published in the Journal of Clinical Oncology. And I am so excited about our topic today. We are going to be discussing a Comments and Controversies article that was published online in JCO October 27, 2022, and it's entitled “Addressing Barriers to Clinical Trial Participation for Transgender People With Cancer to Improve Access and Generate Data.” And please note all authors do not have any conflicts of interest.  It is my great honor to be accompanied today by two of the co-authors on this important manuscript. First is Dr. Ash Alpert, and they are an Agency for Healthcare Research and Quality T32 Postdoctoral Fellow in Health Services Research at Brown University. And I'm so excited to note that they will be joining Yale as an Instructor of Medicine quite soon.  Welcome, Dr. Alpert. Dr. Ash Alpert: Thank you. Dr. Shannon Westin: And I'm also accompanied by Dr. Lola Fashoyin-Aje, and she's the Associate Director for the Oncology Center for Excellence at the Food and Drug Administration. Welcome. Dr. Lola Fashoyin-Aje: Thank you so much. It's my pleasure to be here. Dr. Shannon Westin: So we'll get right to it. I'm so excited. I think this is a topic that many of us, almost all of us, are needing a lot of support, needing a lot of education, and I think let's start by level setting. So what we're going to be speaking today is about transgender people. These are persons whose gender identity does not correspond with what is commonly expected for them based on the sex registered for them at birth. Do we have information about how many transgender people will be diagnosed with cancer and what are the most common cancers they face? Dr. Ash Alpert: So, given many barriers to data collection about transgender people, we actually have very little quality data about transgender people's health outcomes in general and specifically around cancer incidence and outcomes. But what we do know is that at least 0.7% of the US population is trans. And the limited data that we have suggests that transgender people don't have higher rates of cancer than anyone else. So if you look at the numbers for the US, that translates into there being about 2 million transgender people in the US and about 900,000 transgender people who are diagnosed with cancer in their lifetimes.   The other data that we have is that it looks like transgender people who are on hormone therapy and have surgeries, therefore less of a chance of being diagnosed with prostate cancer. If people have had bilateral mastectomy, they have less of a chance of being diagnosed with breast cancer. And it does seem like from retrospective data that it's possible that people on estrogen therapy are more likely to be diagnosed with a breast cancer. So basically, all things that we would expect. And given that about one in 10 transgender people in the US are living with HIV, it's likely that trans people also have higher rates of HIV-associated malignancies.  Dr. Shannon Westin: And I wonder, what about screening? I would think that this could be a real difficulty. If people are misgendered, they may not be offered the appropriate cancer screening. I know I'm getting a little bit away from the crux of your talk, but I think this is so important. Dr. Ash Alpert: Yeah. So there's a number of barriers to screening, and two of the ones that come to mind immediately are that trans people, in general, have negative experiences with physicians. So one study suggested that one in three transgender people had had a negative experience with a physician in the last year and that given this, about one in four trans people avoid necessary health care. So that automatically means that trans people aren't getting screened. And then I think the other important thing that you're bringing up is that because of the ways that certain types of health care are associated with gender—so, in other words, getting a cervical PAP smear is associated with being a woman in the ways that we talk about those tests—there are many other barriers to trans people getting cancer screenings. And we do have some data from the literature that suggest that trans people have lower rates of cancer screenings than the general population, likely because of these two, if not more, reasons. Dr. Shannon Westin: I would anticipate that this could potentially impact, obviously, diagnosis, but then also cancer-related outcomes. Do we have data on that? I know that a lot of this is a data-free zone, so I appreciate you kind of just reviewing what we do know.  Dr. Ash Alpert: So Sarah Jackson published a paper that suggested that in some types of cancer, trans people are diagnosed later with more advanced-stage disease and have poorer outcomes. But, again, it's very limited data. Dr. Shannon Westin: Anything else that you want to talk about on that second question before I move on to the next one? Dr. Ash Alpert: Yeah. I mean, I think that the question you raised about cancer screening is also true about cancer treatment. And I think we may get into this in the paper as well, that certain types of cancer—like ovarian cancer, prostate cancer, testicular cancer, and endometrial cancer—are often, in the ways that we talk about them and the ways that we write about them in guidelines, associated with specific genders. So it's not just individual oncologists, but all oncologists are steeped in this language that associates specific types of cancer with gender. And so then it becomes not just something that's happening in the language between two people, an oncologist and a patient, but also in the optics of the clinics, the names of our clinics, our titles as physicians, that all may inadvertently and implicitly exclude transgender people from care.  Dr. Shannon Westin: It's interesting. As a gynecologic oncologist, I feel like I was just talking about this with someone, about this idea of women's cancer care areas and these very specific gendered kind of comprehensive cancer centers that are incredibly exclusive to this group of people. So I'm really glad that you highlighted that. It's very timely, too, because I think our group is talking about how do we want to name our center. So I appreciate this.   So this is an area of interest greatly to me is clinical trials just in general and participation in clinical trials and encouraging inclusivity and improving representation in clinical trials. So we've seen this. We've seen this in gynecologic cancers and other cancers that, obviously, clinical trials change the standard of care, but they also provide better outcomes for patients and people with cancer. So do we know—are there any data about the current state of participation of transgender people in clinical trials? Dr. Lola Fashoyin-Aje: Yeah, I mean, thank you for that question. The fact of the matter is that we don't really know what the current state of participation in clinical trials in oncology is. This is really part of the reason why the Oncology Center of Excellence at the FDA convened the mini-symposium that we're describing in the paper and the reason why we wrote the paper. I think the assumption that we make is often that the numbers are probably not great. But since we do not routinely collect the data that would help us to identify transgender individuals as part of clinical research, even if transgender individuals did participate in a trial, we would have no way, really, of knowing.  We also do not really have great data regarding what the benchmarks for participation would be because, as Dr. Alpert mentioned before, we don't routinely collect this data as part of the epidemiologic surveys that inform our understanding regarding the populations that are affected by cancer or even at the point of care. It's really very heterogeneous, the types of data that we collect and whether the way that it's collected is optimal. So we really have an information and data deficit that is quite significant for this population that really is needed to be addressed in both sort of systemic ways, but also in our individual settings in the way that we collect data and the way we engage with this population. Dr. Shannon Westin: Are there any interventions or outreach efforts right now ongoing to start collecting these data or even to start raising awareness that these data should be included? Because I'm just thinking of all the NCI trials we do and industry trials and the data that are collected, and you just don't see this, right? This isn't part of the data dictionary typically, right?  Dr. Lola Fashoyin-Aje: Dr. Alpert can speak to some of this as well. We reviewed this as part of the paper-writing process. But there are ongoing efforts to identify the best ways to collect data and identify the opportunities where we could improve upon where some data is already being collected but also making sure that there's sort of structural measures are being taken to ensure that as we collect those data, that we are handling those data appropriately. Because this is a population that really suffers disproportionately bias, discrimination, violence. And so we want to make sure that as we are encouraging folks to provide this kind of information, because we really think that it will help improve their clinical care outcomes as well as clinical trial participation access, but that we are also, at institutional levels, addressing some of the areas that are either explicitly or inadvertently creating barriers and creating environments that are not supportive or that are not safe for this population.  And so there are ongoing efforts. I can say that at a federal level, there's increasing recognition that we really are not doing a great job. And I think there are some recommendations for how to collect these data in surveys, but it may not necessarily be applicable to what we need to know in the clinical trial setting. And so there's so much work to be done still. Dr. Shannon Westin: Great. Dr. Alpert, do you have anything to add there? Dr. Ash Alpert: This could be a very long conversation, but I'll briefly say that there are a couple of things that are happening right now that suggest that things are moving along. One is that NCI put out an administrative supplement for NCI-designated cancer centers to apply for, through which they can begin to collect sexual orientation and gender identity data at the cancer centers. So that means that now that that's happened, cancer centers across the country are testing measures with which to collect sexual orientation and gender identity data.  And at Yale, I'm a part of a research team that is beginning to collect data, both based on some national recommendations and also based on some of our individual research and ideas about how to collect this data in a way that really provides transgender people the autonomy to identify and describe themselves in ways that really feel right to them and to us. I think there are exciting things in the works. But I also really appreciate the conversation we just had about safety because in my qualitative research, what I've found over and over again is that often transgender people are put into a very difficult situation where if they come out to their clinicians, they experience stigma and sometimes violence, and when they do not tell clinicians certain things about their medical histories, including that they are on hormone therapy or that they've had particular surgeries or specific information about their anatomy, then they're put in the position where they may not be receiving the best care because their clinicians don't have all the appropriate information. It's a very difficult situation for individual patients and also for those of us who are thinking systemically and structurally about how to improve what we know about cancer epidemiology and how to provide the best care for transgender people with cancer. Dr. Shannon Westin: I mean, I think that kind of dovetails nicely with my next question, is really trying to understand how some of these structural barriers might impact participation in clinical trials. So what are the barriers that are keeping these people off of these trials? And maybe how can we start to strip those barriers away? All in that, I think, highlighting that highlight of safety and inclusion. Dr. Fashoyin-Aje: I can start by touching upon some of this. I think one important area has to do with language, the language that we use, the signage that we use. And sometimes it's not even just what is explicitly stated but also what is omitted, what is silent, which may have adverse consequences as well. And that could be even worse because then you're sort of rendering a population invisible or basically really kind of reinforcing this idea that they're just not seen.  So I think one area that is really quite relevant in the regulatory setting has to do with eligibility criteria. And I think often, those are silent with respect to transgender individuals because, number one, people just may not know, so they just could fall off, and they just are not thinking that this is a population that I need to explicitly ensure that they are invited to participate in this trial. And then I think other times, there are a lot of assumptions made about whether or not it is safe, and some of those assumptions may be supported, and some of them are not supported. So I don't want to trivialize whether or not an individual is receiving hormonal therapy, whether or not that may have adverse impact on their outcomes in the clinical trial; that could very well be the case, but we just don't know most of the time, and so we don't explicitly state one way or the other. I think the other way is just sort of the kind of language that we use. Calling individuals women may exclude certain people; they may not be sure. I think a lot of people when they hear women, they're not thinking about transgender women. So, in the paper, we really highlight opportunities where we could be more specific about the language that we use so that it is clear what we're referring to. So if you are designing a study that is meant to test a drug in patients who have ovarian cancer, say that. You don't need to say “females with ovarian cancer” if you're not really explicitly—there would really typically not be any reason to exclude people just based on that without more information. And often that more information isn't actually collected.  So I think that's sort of part of what we are trying to highlight here, that there are things that can be done at a micro level, but there are things that can be done at a macro level, and really culture change is a really important part of this, this sort of thinking like which of my neighbors might want to be included in this trial? Which one of my children's friends might want to be enrolled in this pediatric trial? And just kind of thinking a little bit out of the box and thinking of ways to be more inclusive and then have reason to exclude rather than to start from a baseline of exclusion and then inviting people. Dr. Ash Alpert: Yeah, I think we highlighted three main areas of eligibility criteria that may inadvertently exclude transgender people. One was mentioning sex or gender in the inclusion or exclusion criteria when it's unnecessary. The second is mentioning hormone therapy but not specifically and explicitly stating whether hormone therapy used for gender-related purposes would be included in that. So, for example, we, I think, in the paper, describe a prostate cancer trial that excluded people with prior hormone therapy for prostate cancer, but I think it would be very confusing for me if I were a transgender woman on estrogen therapy, whether or not that excluded me from the trial because it's not specifically called out. And then we still see blanket exclusions for people living with HIV despite all the discussion and commentary about this. And that necessarily excludes many trans people because one in 10 transgender people in the US is living with HIV.  And then I think we also, in the paper, highlight that there's actually been qualitative research done with transgender people looking into the facilitators and barriers to participating in clinical trials, and we summarize them just saying that trans people suggest that they're more likely to participate in trials that are led by or staffed by transgender researchers, that explicitly benefit transgender communities, that provide resources, that address financial barriers, barriers to transportation, and that are integrated into health care that transgender people are already receiving.  So I think there are clear ways for cancer centers, principal investigators, to think about revising their trials to ensure that they are accessible to transgender people. And I think one thing that's hinted at in those suggestions is community-based participatory research in order to really ensure that the trials that we're writing are meeting the needs of community members. Dr. Shannon Westin: I think there's always a huge opportunity to have advocates at every level reviewing our trials and our grants. And I don't know that we always do the best job of being incredibly inclusive of who we invite to the table to review those. So that part of the paper really spoke to me as someone who sits on different—task force for ovarian cancer and through the NIH and others, making sure that we really have a representative group that is reviewing these trials and ensuring that they are appropriately inclusive. So I really always like true action items because I think we all get really frustrated when we talk about a problem and say there's a problem and we wave our hands at the problem, but what we need are really goal-based solutions. And I think that was one of the parts of your paper that really I felt like elevated this paper, and now I think we just need to get it out so that it moves on beyond this workforce, this task force, and actually gets implemented on a day-to-day basis as we're developing these trials.  So I think I have one more question that I think we've kind of highlighted, but I want to make sure to put a pretty fine point on it. And you've talked a little bit about this, but we know that many transgender people are taking hormones so they can align their anatomy and their physiology with their gender. And we talked a little bit about exclusion around this, but I'd love to hear your thoughts about how can we better address this particular issue. And I'm just thinking of gynecologic cancers. We're using hormones constantly as a treatment. Breast cancer, same. So is there a way to align the drug development in this space but allow people that are taking these hormones for a different reason, a non-cancer-related reason? Dr. Lola Fashoyin-Aje: Yeah, I think, as we discussed, these exclusions can be both explicitly stated in the protocol, like Dr. Ash mentioned before, or it can be silent, where there just isn't information that would direct a provider or investigator one way or the other as to whether or not it is safe to enroll participants on the trial who are transgender and who are receiving hormonal therapy in that context. So addressing this really takes a lot of education for each one of us. And, as I mentioned before, it's a data-free zone. And I think, ultimately, what's going to make some of these recommendations that we made in the paper sustainable is really having data.   When we were preparing the symposium and when we held the symposium, it was quite clear that some of the available data regarding the safety of hormonal therapy—like does it increase the risk of cancer, what are the impacts on an investigational therapy—there's just a lot of inconsistent information, incomplete information. And so we really need a lot of research to be done to fill those data gaps. And that's why in the FDA Oncology Center of Excellence, we actually have an active funding opportunity right now where we're requesting proposals for applied regulatory science research to really understand the factors that affect safety and efficacy of underrepresented populations in oncology therapeutic development. And we specifically call out sexual and gender minorities as part of that because we recognize that that's sort of an area where safety is always sort of invoked. But we just don't know many times if that's actually supported. And so we really do need data.   I don't have a best-practices approach. I think it's important that an investigator or provider ask their patient if they are receiving this and do preliminary basic research about whether or not there's even opportunity for drug interactions, which is something you would be concerned about, or for increasing the risk of developing tumor or tumor progression, depending on the disease. But again, we just don't know. So I think it's really challenging to offer—as a representative of a regulatory agency, it's really challenging for me to offer a best-practices approach here, other than we should just collect the information and do some research to really better understand. Dr. Ash Alpert: Another thing that complicates this whole conversation is that historically, hormone therapy and surgeries have been used as a surrogate target for transphobia. Oftentimes, trans people have the experience of presenting to care for some symptom that we're having and having that symptom blamed on hormone therapy or surgeries when it's not related. So I think that complicates the ways that we're describing our research. That complicates national conversations about the safety of continuing hormone therapy in the context of a cancer diagnosis and treatment. And it definitely complicates conversations that individual transgender people and their oncologists have about whether or not to continue hormone therapy or how to manage the timing of surgeries in the context of cancer treatment. So that's not to stop or halt these really important conversations in this data gathering, but I think those are important considerations to keep in mind as we describe these questions, collect our data, and describe our findings.   Dr. Shannon Westin: Well, great. Well, this has been amazing, and I think that we wanted to put this podcast together so that we could get your very important findings out. How else can we get this out there? What else do we need to do? What are you all doing at the FDA? Dr. Lola Fashoyin-Aje: The symposium that we organized was a huge first step, and we are having those conversations internally about what the best approach is that is data-driven approach. But to be quite honest, I think that there are so many barriers to changing the status quo. And I think what's really important is the continued highlighting of these issues at every opportunity to not leave out this population when we're talking about equity and underrepresented populations and to keep making those changes in our own particular settings about how we use language, the recommendations that we give to sponsors. I'm speaking from the FDA now about inclusion.  And our research and policy priorities really have to reflect this as well. And so, at an organizational level, what are you doing to ensure that this population is safe, has access, and that you're really engaging them in determining what—there are so many issues to prioritize. Where do you start? What are the things that are more short term, and what are the things that are longer term? And these are conversations that we have with patients at the FDA, inviting them to talk to us about them in different product development contexts, but also more generally. Dr. Shannon Westin: Any last thoughts, Dr. Alpert? Dr. Ash Alpert: I really urge readers to take these issues into consideration in their clinical trials office, to their cooperative groups, and to continue to think about them as they're writing trials. I really appreciate—I think we really appreciate the opportunity to speak on this podcast and to potentially have more people hear about these concerns and think about them. Dr. Shannon Westin: Well, I really appreciate all the time that you spent, knowing that you are both very, very busy researchers.  Thank you all for tuning in today to JCO After Hours. We've been discussing the Comments and Controversies manuscript “Addressing Barriers to Clinical Trial Participation for Transgender People With Cancer to Improve Access and Generate Data.” I am very grateful to all of you for listening. Hope you'll check out other episodes of the podcast, and we'll see you very soon. The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions.   Guests on this podcast express their own opinions, experience, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement.      

Dr. Patrick Collier provides an overview of the multidisciplinary approach to Cleveland Clinic's Cardiovascular Oncology Center.

A cancer diagnosis and treatment can be difficult on both patients and their families emotionally. But support is available for people who need it. In this podcast, Robin Valpey, MD, medical director, Center for Counseling and Support, UPMC Hillman Cancer Center, and Elizabeth Hale, MD, clinical assistant professor, Psychiatry, University of Pittsburgh School of Medicine, discuss the mental health care available for cancer patients and their loved ones from the beginning of treatment onward. They also discuss the role of psychiatrists in the comprehensive cancer care that UPMC provides.

Steve was out golfing with a few of his friends on a cold December day on the Cape. During his round, he noticed a bump on his left side that he showed to his wife when he got home. He booked an appointment with his primary care doctor shortly thereafter, just before Christmas in 2016. After a few tests, Steve was completely floored to discover he had stage 4 breast cancer. He underwent a mastectomy shortly thereafter. He also came to Dana-Farber and to Dr. Erica Mayer for a second opinion. The subsequent treatment plan that Dr. Mayer sketched out for Steve just felt right to him and he decided to switch his care to Dana-Farber. Breast cancer in men is quite rare, only 1 out of 100 cases of breast cancer in the US are found in men. While he is still living with a serious illness Steve is doing great today and “like a million bucks.” He comes to Dana-Farber from Sandwich every three weeks for treatment. Steve travels a lot now with his wife visiting national parks and other spots he wishes he got to sooner.

Amy Cheung was originally diagnosed with Stage II breast cancer in September 2015, at the age of 32. At that point, she was feeling the healthiest she had ever been and was training for a marathon. She happened to cough one day and in the process of clutching herself, felt a lump. Amy went to her primary doctor a few days later and from there, things moved quickly to a biopsy that confirmed cancer. She was treated then with chemotherapy, then surgery, and radiation and presumed to be cancer free. She continued with anti-hormone therapy to keep the cancer from coming back. In late 2019, Amy started experiencing some back pain that she chalked up to exercise. Then from Jan - April 2020, Amy started having regular, low-grade fevers. Finally, after feeling miserable for so long, she e-mailed her oncologist Dr. Hal Burstein and told him of her symptoms. Without delay, Dr. Burstein ordered scans for Amy, which unfortunately revealed extensive cancer metastases in her liver as well as metastases in her bones. Amy has undergone several types of treatments for her cancer including surgery, radiation, and targeted therapies. She is currently part of a clinical trial that involves a targeted therapy combined with an immunotherapy drug, which enables the body's own immune system to recognize and destroy cancer cells. Having grown up in the Boston area, Amy felt like she had always known about DanaFarber. In high school during the summers, she used to volunteer for the Jimmy Fund at movie theaters, fundraising from movie patrons.

Dr Axel Walter, Consultant Medical Oncologist at Bristol Haematology and Oncology Center, kicks off the series about how to manage ovarian cancer.  He discusses in depth the staging system and how women with initial ovarian cancer should be treated, including both those with stage 1 and stage 3 disease.  This includes an explanation regarding BRCA and HRD (homologous recombination deficiency) testing and treatment with adjuvant PARP inhibitors. Your host is Dr Alison Cameron, Bristol, UK.

In April 2022, FDA issued new draft guidance on Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Subgroups in Clinical Trials. Because this guidance applies to all medical products, CDER, CBER, and CDRH all contributed, but this draft was led by Project Equity from FDA's Oncology Center of Excellence (OCE). “It is important that we be able to evaluate new therapies in the context of a diverse population that will use these medical products because the diseases for which these products are intended present in variable fashion across the population and because populations respond variably to medical products,” explains OCE Project Equity Lead Lola Fashoyin-Aje. “But we should reconsider the question regarding why diversity in clinical trials is important. Because the question really ought to be: What are we missing when trials are not diverse, when the study population in the clinical trial is not diverse? What are the missed opportunities for advancing science or advancing clinical medicine and, ultimately, for driving improvements in population outcomes?”

In part two this ASCO Education Podcast episode, hosts Dr. David Johnson and Dr. Patrick Loehrer continue their conversation with Dr. Richard Pazdur, director of the U.S. Food and Drug Administration's Oncology Center of Excellence, focusing on his leadership and vision for improving cancer care worldwide. The conversation includes reflection on drug toxicities, approval processes, and complexity of clinical trials. If you liked this episode, please subscribe. Learn more at https://education.asco.org, or email us at education@asco.org.   TRANSCRIPT Dr. Pat Loehrer: Hi. I'm Pat Loehrer, the Director of Global Oncology and Health Equity at Indiana University. Dr. Dave Johnson: And hi. I'm Dave Johnson at UT Southwestern in Dallas, Texas. Dr. Pat Loehrer: This is the second half of our Oncology, Etc. conversation with Dr. Rick Pazdur, who's the director of the FDA's Oncology Center of Excellence. In Part 1, we chatted with Dr. Pazdur about his upbringing and his early career. Today, we're going to focus on his leadership and vision for improving cancer care worldwide. But first, we'll discuss how cancer has impacted his life personally.   I want to flash-forward. I had the pleasure of knowing Mary. And there was no question, if you had a problem in oncology, you would go to Mary and not Rick Pazdur when you were a house staff member. But moving forward a bit, I'm not sure if all the listeners know that Mary came down with ovarian cancer. Dave himself had cancer. My wife had breast cancer. It is incredibly hard to be an oncologist I think when your spouse or someone who's close to you has cancer, and particularly, being married to a medical oncology nurse. Maybe just share a little bit about that journey of being a husband of a- Dr. Richard Pazdur: Yeah. It is interesting because going back to the Rush story, the first patient that my wife and I had in common, and this is so ironic, was a patient with ovarian cancer. The last patient that we had in common was her, which is some ironic fate, so to speak. And the story began of her illness was right around Labor Day. We had gone to Chicago in February driving back from Chicago. I noticed that she kept on taking a whole bunch of Tums and then saying - Oh, I just got a lot of GI symptoms, and she went to see her gastroenterologist or GP and he said, ‘Oh, this is just, you know, indigestion.' And two weeks after that or not even that, she was in the hospital with a massive amount of ascites, needed an intensive care unit. It was readily apparent just on getting her CA 125 what she had and she wound up one day in debulking surgery and then IP chemotherapy, etc. I think something that I learned, and I think we knew from the very beginning that this was not going to be a curable illness, and how to deal with that on an emotional level. And I have to give my wife credit. She spared me a lot of the emotion because she was such a strong person. She made all of her own calls as far as what she wanted. She would ask me what I thought, but she would do her own research, she would go to her own doctors' appointments. She said, ‘You don't really need to come with me. I'm self-sufficient.' She was very much interested in helping other cancer patients, and after she died, I think one of the most cherished conversations I had was a group of women that came to me and said how much she helped them during their support group because she was a nurse. She knew she was dying. She had emotional maturity not to fall apart but to accept the inevitable in a very strong way. My wife was a very religious person, had gone to Catholic schools, really embraced religion during those terminal years basically. And I think that was a great sense of comfort to her. But it did teach me a lot of lessons when you take care of somebody that has cancer, and that is, what a bad job we do with drug toxicities. Drug toxicities to medical oncologists and especially the people at the FDA are numbered, Grade 3, Grade 4, Grade 1. These toxicities are tolerable, tolerable to who, so to speak. And how to manage these toxicities and how they interrupt your life is one of the lasting experience I have, which I always will remember. And that has been one of my roles recently is forming several programs that we have in the OCE to look at dosing, to look at what is this definition if the drug is tolerable or well-tolerated or if the toxicities could be managed. I always say, yeah, every toxicity could be managed, even death. You call the undertaker to manage it. So what do you really mean by that statement. But I think the issue of toxicity is an important one. And then also going on clinical trials and having people considering what you want to go on, what risk you want to take, and what is actually in the informed consent and how meaningful that is. Dr. Dave Johnson: Really glad you brought that up, Rick. That matches my own experience with lymphoma and going through chemotherapy. And as an oncologist, one would think I would know what the side effects are. I'd recounted them dozens and dozens of times to people over the years, but until you've actually experienced them either personally or up close as you did with Mary, it's impossible to fully understand. I'll give you one example. Fatigue. Everybody thinks they know what fatigue is, but until you've had chemotherapy-induced fatigue, the fatigue that never abates, you just don't understand what it is. It's debilitating in ways that are unimaginable to most people. So I'm sure that experience certainly shaped your view and your role at the FDA. Dr. Richard Pazdur: Correct. Dr. Dave Johnson: I wonder, if you might share, you initiated a number of programs recently, including programs to try to improve coordination and co-operation amongst the pharmaceutical companies. Could you speak to some of those programs for us? Dr. Richard Pazdur: I think one of my favorite programs is Project Orbis. Project Orbis is an idea I had when I was walking down the street. It just hit me. When I came to the FDA, one of the things I rapidly noticed is how isolated the FDA was, even from the rest from the regulatory agencies throughout the world. There was very little cross-fertilization there. So one of the very first things that I did was set up a monthly tele-conference first with the EMA, the European Medicines Agency, and then we ended on Health Canada, Australia, Japan, Singapore, you name it. And one of the things that became really apparent to me, we at the FDA got applications always first—always. That's obvious. You know why they had given it to us first? The money. That's where the finances are going to be. So we got the application first, and it could be 2 years, 18 months, 12 months, that these other countries, Canada, Australia, Switzerland, and Brazil, Israel, would get these applications. And I said, well, this isn't right, really, because these people, they have cancer. They have every right to get these medications as soon as possible, and also we have such a large agency. We have 80 to 100 oncologists that work there, and most of these agencies have one or two oncologists. So our expertise in oncology at the FDA is so much greater than these other regulatory agencies. How can we leverage that to help these other countries? So we started Project Orbis, and what it was is that companies come in and they submit an application and they simultaneously submit the same application to the countries that want to participate in the program. They are all preselected and have confidentiality agreements with each other. And we worked together on the applications, basically reviewing the applications. So we had many meetings, tele-conversations, telephone conversations with countries. So that expedites these drugs. This has really had a lasting impact because from a worldwide perspective, it's really promoted more rapid development of drugs and rapid approval of drugs, and that's important because that establishes sooner new standards of care that will impact future trials. So in addition to the humanitarian issue of improving healthcare for patients in these countries, it has an impact on the global clinical trial system by having new standards approved much faster and accepted by world authorities. Dr. Pat Loehrer: Let me just jump on that for a second, just to make a comment. Back when we were growing up, there would be like three to five drugs approved- Dr. Richard Pazdur: Yeah. Dr. Pat Loehrer: And today, it's like once a week, there's a new indication for oncologists. Dr. Dave Johnson: Our listeners have another question that might be appropriate to ask at this time. What is the most common mistake that drug companies make in their applications to the FDA or in the process of trying to get their drug approved? Is there a frequent mistake that you can advise them? Dr. Richard Pazdur: Well, they don't come and talk to us. That's number one. They want, not necessarily what the best registration pathway is, but what the quickest registration pathway is. And sometimes the quickest registration pathway, especially single arm trials, are not the best registrations pathway. So my advice is rather than playing games with the FDA, to put it in the vernacular, just do the right thing and say, what is the optimal information that patients need when I develop this drug. We're seeing a lot of problems now with various drugs where people are developing in a refractory disease setting a drug, and they plan on getting accelerated approval on a response rate. So they push and push the dose. And with a single arm study, you can't really evaluate safety that well. Everything is attributed to the drug, and they want to get the highest response rate. And they get it, and there's a confirmatory study, and the arm of the confirmatory study, the control arm may not be as toxic as theirs, and we're seeing a wave of drugs that now have inferior survival compared to controls, which probably is predicated upon, they got the wrong dose. And I think that is one of the major programs that we have, that we need to address is dosing in oncology, this ‘More is better, more is better,' and ‘Let's push the dose as high as possible.' More isn't even probably good in cytotoxic days, but certainly, not a good idea in targeted therapies and certainly not a good idea in biological therapy. So we've really got to think about dosing more, penetration of targets, what's the optimal dose rather than what's the highest dose. You know as well as I do, pharmaceutical companies want to go with the highest dose because the major hurdle is the demonstration of efficacy, even in a randomized study. So nobody wants to be blamed by saying, well, you spent $100 million on a Phase 3 study and it's negative because you used too low of a dose. But then at the end of the day, we don't have a really good tolerable dose, and it's really hard to go backwards and look at dosing after a drug was already approved because the efficacy study has already been done. Dr. Pat Loehrer: The other aspect of drug companies is not only getting the dosage there, but also the duration. There is motivation for money, and so patients are going to and- Dr. Richard Pazdur: Oh, count on that. Dr. Pat Loehrer: So it begs a question, and I know the FDA can't do this, but in other countries, there is a monetary review together with the toxicity review. Can you reflect a little bit about that to the best of your ability? Dr. Richard Pazdur: Well, even within our simpler agencies, they may communicate more than we do with CMS, but all oncology drugs that when they're approved are then paid for by CMS, okay? In these other countries, that is not so. They may get approval and then they have to go to these health assessment agencies that will decide and argue with the companies what the pricing of the drug is. I think it's a mistake, honestly, for the FDA to get into pricing. We have a hard enough time with efficacy and safety, and pricing is a very ephemeral concept because it could change on a dialing. Somebody could promise you, you should approve their drug because it's much cheaper on Monday, and on Friday, they could say, 'Oops, we made a mistake. We really think that this dose has to be X number of dollars.' And you could see competition hasn't worked well in oncology with seven PD-1 drugs approved, pricing has not really been of any movement here. Dr. Pat Loehrer: I'm sorry. Dave may have another question, but let me ask you this. Going back to the clinical trials and what industry asked you- the complexity of clinical trials is going up logarithmically compared to the way they were in the ‘70s and ‘80s. In many of the trials where we have to get all this data in order to soothe the FDA, what are your thoughts about simplifying clinical trials? Dr. Richard Pazdur: Oh yeah. I'm for it. I am for it. If you really look at it, these are not FDA requirements for the most part. The companies want them, all of this data because it's controlled. They don't want to be blamed at the end of the day for not capturing this data or that data. They have developed complicated bureaucracies, going back in my sociology days there, complicated bureaucracies to gather all of this data, the whole CRO industry to go out and pester you guys in practice by doing site visits. It's a complicated situation and it's really predicated a lot on the history and bureaucracies that have been built up and not money to strip away those bureaucracies for fear of failure, so to speak, of not catching something. Dr. Dave Johnson: So Rick, we're coming to the end of our time that we've scheduled. I actually have two questions for you. We've asked all of our previous guests, the first of which would be if you could talk to your 21-year-old self today, what advice would you give yourself? After you've done that, we'd like to know what books have you been reading lately or is there some documentary that you've seen that you would recommend to us and our listeners? Dr. Richard Pazdur: I would tell myself, when I was 21 years old, relax and be less anxious. All things pass. I think we get so anxious when we're young about relatively small things. I remember my first ASCO presentation, I was petrified. My heart was beating out of my chest. I was sweating. And like relax. It's one of a thousand presentations at ASCO. We tend to magnify things, and I think age puts things in perspective. This in the reality of the world is a small thing, and people probably won't even remember it. Dr. Dave Johnson: Excellent advice. Dr. Richard Pazdur: My favorite author that I'm reading now for the last couple of months is a presidential historical author, Doris Kearns Goodwin. I think many of you know, she's written many books. I love her writing style. And I like non-fiction. I like biographies and I like history books, history stories rather than mysteries or things like that, fantasy books. The two books that I really enjoyed, the first one was No Ordinary Time: Franklin and Eleanor Roosevelt: The Home Front in World War II. I don't know if anybody's read that. It's an excellent book. Most of our attention in World War II is directed toward Europe and what was going on in Europe, the battlefields, etc., which I'm not a big fan of reading about battles and stuff, but this was what was going on in The White House and the relationships of all of the people that came there. It was like a hotel almost with the personalities that were flowing through including Churchill and various princes and queens, etc. But also the interesting relation, the fascinating relationship that Eleanor and Franklin Roosevelt had, I don't know how to describe the relationship. It truly was an unconventional relationship based on some past history that they had of affairs etc., but it was just a fascinating one. The best book, though, again I'm reading now, is written also by Doris Kearns Goodwin, and it is Lyndon Johnson and the American Dream. Doris Kearns Johnson was his biographer and spent a great deal of time with him in The White House as well as when he left The White House. But it's an excellent book on management and reading people, success. One of the things that is most interesting about Lyndon Johnson, and especially when he was running the senate before he became president, was his ability to know what motivated people and how to use that to form a consensus. Does this person want to go on this trip. I'll give it to him and then he could help me with this. Does this person want to go to this party or get on this position in congress? So it was really a skillset that he had, which I think most leaders need to know. You have to motivate people. You can't lead by an autocratic masthead, but you've got to lead from what do people want and to make sure that they feel you have a personal relationship with them. As I say to my staff, everything in life is personal—everything. Dr. Dave Johnson: Well, it's been a great session, Rick. We so much appreciate your willingness to spend time with us. We wish we had twice as much time. I'm sure we could go on for hours. Thanks again, and we appreciate all you do at the FDA. You've been a fabulous leader, and we hope you continue on for many years to come. Dr. Richard Pazdur: Thank you so much, Dave, and thank you so much, Pat. Dr. Pat Loehrer: Great to see you. Dr. Dave Johnson: Pat, before we leave, any idea why our patients seem to get sick on Saturday and Sunday? Dr. Pat Loehrer: I have no clue. Do you know the answer, Dave? Dr. Dave Johnson: Yes. They have a weekend immunity. Thank you for listening to the ASCO Education Podcast. To stay up to date with the latest episodes, please click subscribe. Let us know what you think by leaving a review. For more information, visit the Comprehensive Education Center at education.asco.org. The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement.    

In part one of this ASCO Education Podcast episode, director of the U.S. Food and Drug Administration's Oncology Center of Excellence Dr. Richard Pazdur talks with hosts Dr. David Johnson and Dr. Patrick Loehrer about his upbringing in Indiana, his family, and his circuitous route to oncology. If you liked this episode, please subscribe. Learn more at https://education.asco.org, or email us at education@asco.org.   TRANSCRIPT Dr. Pat Loehrer: Hi, I'm Pat Loehrer. I'm the director of the Center of Global Oncology and Health Equity at Indiana University. Dr. David Johnson: I'm Dave Johnson at UT Southwestern in Dallas, Texas. Pat, great to be back with another episode of Oncology, Etc, an ASCO educational podcast. We have a very special guest today, Dr. Richard Pazdur, from the FDA Oncology Center of Excellence. I'm really looking forward to this conversation. Dr. Pat Loehrer: This will be terrific. Dr. David Johnson: Yeah. You were telling me before we got started about a little event that occurred this week, maybe you want to elaborate on that for us. Dr. Pat Loehrer: Yeah, we always start out this by talking about books, and one of the books I'm drawn to today is a book called, The Emperor of All Maladies, which was written by Dr. Mukherjee several years ago. I want to read a little bit from this because it has pertinence. This is about a guy named John Cleveland. Dr. Mukherjee writes, he goes, ‘In 1973 Cleveland was a 22-year-old veterinary student in Indiana. In August that year, two months after his marriage, he discovered a rapidly enlarging lump in his right testis. He was whisked off to the operating room for surgery and returned with a scar and he was diagnosed with metastatic testis cancer. This was right around the time that Larry Einhorn came to Indiana University where he was treated with a three-drug cocktail of actinomycin-D, bleomycin, and oncovin ABO. And then he had a brief remission progressing and was treated with mithramycin mithrymicin. And then in October of 1974, he once again developed progressive disease, and Larry approached him about a new cocktail with the drug cisplatin, that had never been used before in combination, and Larry's thought was to put it together with another couple of drugs.” So, I'll just finish reading this. “In October 7, 1974, Cleveland took the gamble, he enrolled as patient zero for BVP, an acronym for the new regimen containing Bleomycin, Vinblastine and cisplatin. 10 days later, he returned for routine scans and the tumors in his lungs had vanished. He was ecstatic and mystified. He called his wife from the hospital phone. I can't remember what he said, but I told her the results. So, John was the first one cured of testis cancer. Back then it was a 5% cure rate. Today, it's 95%. He is really the hero of heroes. Last week, at this time, John had asked me to come to his hospital room because he was diagnosed with metastatic cancer of a different type. He knew that this was basically the final hours of it. And so he wanted to say goodbye to me, and it was the most touching reunion I had. Two days ago, John passed away. So, my thoughts are with him, especially his family. But also, when we think about heroes, John was one of them, and if it wasn't for him, and his first treatment, Larry might not have gone on and treated other patients with this regimen. This drug cisplatin was experimental back then it caused a lot of nausea and vomiting and didn't work in many tumors, but this was a drug that was really highlighted and approved for the treatment of bladder cancer so Hubert Humphrey could get treated, and then in testis cancer, and it's really one the really success stories of all success stories in terms of oncology, and it started out with this experimental drug from the NCI that was approved by the FDA.” So, this leads us to our guest today, Rick Pazdur who I've known for many years. He grew up in Calumet City, Illinois, which is famous as the home of the Blues Brothers and Rick Pazdur. He got his bachelor's degree from Northwestern, his medical degree from Loyola Stritch Medicine, and then did his hematology-oncology fellowship, initially his oncology fellowship at Rush Presbyterian St. Luke's, where I first met Rick, and then later finished his Heme-Onc fellowship at the University of Chicago. From there, he went to Wayne State, served on the faculty there for about five or six years, and then joined the faculty at MD Anderson. He was there from 1988 to 1999. Then, we'd like to hear about this, but for some reason, it got in his head that he wanted to go to the FDA and so he moved to the FDA where he was Director of the Division of Oncology Drug Products, from 1999 to 2005, when they created the Office of Hematology Oncology Products, and he became the Director of this. More recently, he's been made the director of the FDA's Oncology Center of Excellence. He still serves as the acting director for this OHOP. Rick is an extraordinary individual. He's been awarded a number of awards. From ASCO, he got the Service Recognition Award and the Public Service Award in 2013. And the AACR also awarded him the Public Service Award in 2015. He's received numerous awards and he is probably one of the most respected oncologists that I know in this society. It's such a great pleasure to have you here today, Rick. Dr. Richard Pazdur: My pleasure, Pat. My pleasure entirely. I look forward to the conversation. Dr. David Johnson: Pat, you left out one award. He got so many awards that you can't list them all, but I was impressed that he got this award for the Massachusetts General Hospital Cancer Center's – The 100 list. Dr. Pat Loehrer: Yeah, we made the cut-off. Dr. David Johnson: I have no idea what that is. Dr. Pat Loehrer: We were on the 1001 list. We made that one. The only thing I want to throw into is that when I first met Rick, when I was a resident or an intern, I think, he was on the service with Phil Bonomi, who is very important for me for my thymoma research, but there was an oncology nurse on the service, Mary Lind, who was a terrific oncology nurse as they all are. But it turned out there was more chemistry that went on. So, Rick ended up marrying Mary, and I'm sure he'll tell some stories. We'll come back to that in a little bit, too. Dr. David Johnson: Yeah, this is really exciting. So, let's get started. Rick, maybe you could tell us a little bit about your background and what got you into medicine in the early part of your career. Dr. Richard Pazdur: I had an interesting story. Pat had mentioned Calumet City home as a Blues Brothers. That was the orphanage in the movie. It was located in Calumet City. But what you don't know about Calumet City is that the real kind of nickname for Calumet City in the 50s 60s 40s was 'Sin City'. It has the honor of having the greatest number of liquor licenses in the United States. And in Calumet City, which was on the border of Hammond, Indiana, and Calumet City, they're kind of Sister Cities. They're one in Illinois, Calumet City. And Hammond is in Indiana, obviously, there was this strip, and it was called ‘The Strip' and it had all of these bars and floor shows with Sally Rand and gambling. If you walk down there, which I never did as a kid, but drove down there with my father some time just traversing the city, those lights were so bright, it was just like Las Vegas, basically. So, you had that Strip and that went on for maybe three, four, or five blocks. And then you have the rest of the city that I grew up in, which was primarily a Polish Catholic city, which I am kind of a representative of being 100% polish. There were scattered bars throughout the city. In fact, my fondest memory is sleeping in bed at 3 o'clock in the morning or 2 o'clock in the morning, awakening to people running out of a bar, which was 50 feet or 100 feet from my bedroom window. They were screaming and yelling and cursing and everything and throwing beer bottles at each other. And in those days, obviously, as you remember, in the 60s, there was no air conditioning. So, the bar was called The Tropical Inn. Let me tell you this, there was nothing tropical about it and there was nothing 'in' about it. Dr. David Johnson: That background might have driven…. Dr. Richard Pazdur: That was not what I would call a highbrow area, but I enjoyed it. It was a fabulous childhood that I had. Most people don't know this. So, I will share this with you because it really had an indelible mark on my life and something that really transformed my childhood and my high school years and my college years, and my medical school years. When I was about in seventh or eighth grade, my father who was basically a blue-collar factory worker, developed glaucoma, and he went blind at a very early age. And that threw the family into not only emotional turmoil but also economic turmoil. I survived basically by getting Social Security dependent income and had to work at a very early age. I started as a dishwasher. My mother lied about my age so I could start working at a crummy restaurant in Calumet City as a dishwasher and I worked throughout school, but that experience really made me grow up really fast if I could say so. You didn't have a childhood, you had to be responsible because there was really no security blanket to fall back on. Although my parents didn't have a lot of money, they really had very good emotional support for me and both my father and my mother came from very large, Polish families. My mother had nine children in her family. She was right in the middle. And so, I had many cousins and many aunts and uncles and my father had seven children in his family and he was the oldest boy. So, there was a lot of support there, but obviously not a lot of financial support. So, it really made me grow up relatively quickly and really come to some of life's lessons, relatively fast like discrimination against disabled people, which I will always remember. When I was a boy, I remember going with my father walking and obviously he had a white cane, but walking down the street, people would actually cross the street to avoid us. They just didn't want to confront that anxiety. I don't know if it was discrimination or if they just felt uncomfortable dealing with it. When you go to a restaurant, people raise their voices, like the man was mentally impaired and that wasn't the case. Dr. Pat Loehrer: I'm sorry to interrupt you just for a second because I read this somewhere and I think it's important to throw in. So, you graduated from high school in three years? Dr. Richard Pazdur: Yes, that's what I was gonna say. I graduated and I worked during that time also. These jobs were not like jobs that kids have now as consultants or internships, these were like real jobs as a janitor, a gardener, or a packer in a grocery store. You had to do it and you had to compartmentalize your life to get things done, basically. But you were driven to do things and I'm thankful for that experience really. I even use it now when I'm facing turmoil in my life. I look back at that time and say to myself, Rick, if you did it then as a 12-year-old kid, a 14-year-old kid, you could do anything. So, it really fostered a sense of responsibility, self-awareness, and the need to do things for yourself and get going on yourself. because nobody's going to help you in a sense. I'm very grateful for that. Dr. David Johnson: Rick, at what point did you decide to become a physician? How did that experience really drive you into that field? Dr. Richard Pazdur: Well, I spent a great deal of time in the lobby of the University of Chicago hospital with my father. I was the primary caregiver and went with him to his doctor's appointments. I can't tell you the number of hours I spent in the lobby of that hospital. I was very interested in science. I was very interested in really helping people because of that background. I really had a great deal of clarity, though. I remember, when I was maybe a freshman or sophomore in high school, I wrote for the Northwestern Medical School catalog, because I thought I would be going to Northwestern undergraduate, I already decided in my own mind that that's where I wanted to go. I just got their medical school catalog and I was thumbing through it, and I remember this vividly sitting on my bedroom floor next to the window that was 50 feet away from the bar, basically, and was looking through the medical school catalog and seeing all these names of this doctor, head of neurology, assistant professor, associate professor. I said, “Hell, if I'm going to be a doctor, I want to be one of these people. I want to be the best doctor. I want to be teaching the physician here and doing research.” So, unlike most kids today that have to find themselves, like, ‘I'm going to take 8 years to complete college or something like that and take a year off to find myself.' I had to be very, very focused on what I wanted to do. So, I really worked very hard during it through time. I don't regret it, as I say. I went to Northwestern undergraduate and had a fantastic experience there. I graduated in three years as I mentioned. I had a special interest there that most people don't know about. The people at the FDA know about it. I did these pre-med courses but I had a really special interest in the field of sociology, and actually was toying with the idea if I didn't get into medical school to pursue a Ph.D. in Sociology and become an academic sociologist. If you remember and both of you are of this age. Dr. Pat Loehrer: David is, not me. Dr. Richard Pazdur: Okay. David might know of the Vietnam War. This was right around the time of Kent State and closing down the schools, I'm sure you remember that, for the colleges, etc. Nobody went into business or anything like that. It was a sociology, psychology, and anthropology type of things people were interested in. My love was this field, and I did a lot of research on it. I remember one of the professors that probably had the most influence on me, a woman called Zena Smith Blau, who was a sociologist, and I did multiple research projects and independent studies. The first thing when I took her first course, she assigned us a paper and she said, write about yourself, ‘What makes you unique? I went to see her and I said, ‘I really don't know exactly what you mean by this.' She said, ‘Well, how different are you?' And I said, ‘Well, my parents are children of Polish immigrants, and I really know the Polish community in Chicago fairly well.' She said, ‘That's it Rick, that's what your specialty will be.' I did multiple research projects on this area with her. Some of them were like the assimilation of Polish immigrants with regard to urban-rural origin. Another one was working-class youth in Chicago, and mobility based on education and high school part-time employment. So, that was a fantastic experience that is totally outside of what I did in my future. Dr. David Johnson: We asked our listeners to submit questions because we knew you were coming on. We did get a question that perhaps is appropriate to ask at this time. It comes from a younger trainee, who wants to know what advice you would give to a trainee aspiring to have a clinical investigative career in oncology? What sort of preparation should they have? Obviously, you've got to learn all of the techniques of clinical trial designs, statistics, etc. But what other advice would you give a trainee hoping to pursue a career in clinical investigation? Dr. Richard Pazdur: I think one's career always has to go back to the basics and have a patient focus. So, what is your interest in the patient, so to speak? And that is what advice I would give them. Are you interested in a supportive care issue? Are you interested in a specific disease? I think you have to follow a passion and that is what is most important to me. What is your passion in life? Because as physicians, we spend a great deal of time preparing for our careers and then subsequently afterward, in our designated careers, and you really can't approach this as a job. It has to be a passion. So, if you do have this, what are the questions that you really want to answer? What is the field that you want to go into and make an indelible mark in? So, that's what I come from, and that's something that I tell our staff is: what do you really want to do? What makes you happy? What would make you a success in your mind? Not defining yourself by somebody else. Dr. Pat Loehrer: Finish the story a little bit. Was it a patient then that turned you on to oncology? What brought you into oncology? Dr. Richard Pazdur: I had a very circuitous circular route to oncology. Oh my God! I don't know if you have enough time to hear this story, but let me start though. I originally went to medical school because of my background in sociology. I wanted to become a psychiatrist because here again, I thought psychiatry, sociology, and psychology, these are kinds of things I was interested in until I took my first rotation in psychiatry. It was nothing like I thought it was going to be. I saw my first patient that got ECT (Electroconvulsive Therapy) and I said, 'I ain't doing this.' So, I was then interested in cardiology. Loyola at that time where I was going to medical school and where I did my internship and residency, had a huge cardiology program that was like CAST city, USA. Everybody was just coming into the hospital getting CAST and going out of the hospital. Dr. Pat Loehrer: [Unintelligible 18:13] was there, right? Dr. Richard Puzdar: Yes. Mary Kate [Unintelligible 18:18]'s father was the head of medicine there. And it had a huge cardiology program and an excellent cardiology program. I really did like Clinical Oncology, and I was all signed up for a cardiology fellowship, the Ts were crossed, the dots were placed, the contract signed, and everything. And somebody said, ‘Rick, why don't you take a rotation while you're a resident in the cath lab? We have an opening and this would be a great time and opportunity for you to jumpstart your fellowship. So, you can come and work for us in the middle of your third year of residency for about two or three months.' So, I did that and I hated it. I just dreaded it. I couldn't stand it. It was the most boring thing. I would think of every opportunity to escape the Cath lab. It was the same thing over and over and over again, inject the dye, turn on the [Unintelligible19:16] machine, and follow the coronary arteries. So, I happened to go to a community hospital, St. Francis in Evanston, Illinois, and I met an oncologist there that had just finished her fellowship. I really was very impressed by her clinical skills. She had finished her fellowship at the University of Chicago and worked with her there and was just amazed by really the quality of care that the oncologist gave patients with cancer. Previous to that, I had an interest also in oncology. At Loyola at that time, we had an oncologist his name was Ketty Badrinath, and he was an excellent clinical teacher. Now that I decided that I'm not going into cardiology, the first problem I have is to find a job and to find a fellowship in oncology. So, I started investigating oncology programs at St. Francis Hospital there. I went down to the gift shop and I said, I want $10 worth of quarters. I went to a payphone, closed the door of the payphone, dialed information, and got every oncology program that I could think of from Mayo Clinic to all of the programs in Chicago. One of my last phone calls was to Rush Presbyterian. I found that program director, Jules Harris, I don't know if you remember him, and he said, “We have an opening.” So, I accepted the position. At the end of June, whatever it was, June 27, 1979. I started my oncology fellowship program. Now Oncology at that time in Chicago, to give you a picture of it, the largest program in oncology was at Rush. It had a total of 12 people. And the therapy started at Rush around solid tumors really in the endocrine therapies of prostate cancer and breast cancer. So, it was a different program than most programs throughout the United States that were offshoots of Hematology programs and the treatment of acute leukemia and lymphomas. So, it had a really kind of different orientation. So, I started my fellowship. And on the first day, I met Phil Bonomi, who had a tremendous influence and still has an influence over me. I know no doctor that I respect most more than Phil. I think the greatest compliment that one could give a doctor is to refer your own family to him. And I've done it on numerous occasions with various cousins and aunts and uncles, etc. But as you mentioned, Pat, I also had the opportunity to meet my wife. And I met her and at first, we were very good friends and there was no romantic relationship. And then, as time proceeded, we knew that there was something special there. My wife was just a wonderful person. Like myself, my wife was pretty much a self-made person. She was one of eight children, the oldest daughter. As the oldest daughter, she had to assume a lot of childcare responsibilities, cooking, etc, for all of her brothers and sisters and took care of the younger children. But she was an excellent student. She graduated first in her class. She was a national merit finalist. I often asked my wife, ‘Mary, you're a nurse, are you interested in going into medicine? I'll be happy to work with you to get you into medical school.' And she said, ‘No, you want to go into medicine, I want to go into nursing.' That was her orientation toward other people. It wasn't about the buck. It wasn't about the title. It was about the work of helping other people. I really have to honor her mother and father who gave her that orientation. Of interest, all of her sisters are nurses. Her mother was actually a school nurse, and an original graduate of Rush. She went to Rush Nursing School in the 50s but had to leave because at that time in the 1950s, if you got married, you can't be in nursing school, they kicked you out. So, it shows you how times have changed.   Dr. Pat Loehrer: Well, this concludes part one of our interview with Dr. Richard Pazdur who is the director of the US Food and Drug Administration's Oncology Center of Excellence. Stay tuned for part two of this conversation where we'll hear how cancer has touched his life personally and will explore the initiatives and programs, he started to improve patient care globally.   Thank you to all of our listeners for tuning into Oncology, Etc. This is an ASCO education podcast where we'll talk about just about everything and anything. If you have an idea for a topic or guests you'd like to see on the show, please email us at education@asco.org. Have a good week.   Unknown Speaker: Thank you for listening to the ASCO Education podcast. To stay up to date with the latest episodes. Please click subscribe. Let us know what you think by leaving a review. For more information, visit the comprehensive education center at education.asco.org.   The purpose of this podcast is to educate and inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. Guest statements on the podcast do not express the opinions of ASCO. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement.    

Dr Charlie Comins, Consultant Clinical Oncologist at Bristol Haematology and Oncology Center, UK joins us again to discuss how to manage the care of a woman with metastatic HER2+ breast cancer, including the management of her brain metastases,  multiple lines of potential treatments and if she was diagnosed post adjuvant therapy, during adjuvant therapy or at inital diagnosis of breast cancer. Your host is Dr Alison Cameron, Bristol, UK.

Listen Now As more young people survive cancer, the issue of fertility preservation is front and center. Dr. Karen Albritton and her team explore the challenges of preserving fertility in children, teens and young adults undergoing cancer treatment and the latest breakthroughs in oncofertility that are delivering promise for the future.Dr. Karen Albritton Related InformtationOncofertilityAdolescent/Young Adult (AYA) ProgramLife After CancerOncologyHematology and OncologyHematology and Oncology ResearchFort Worth Adolescent and Young Adult Onoclogy Coalition Transcript 00:00:02 Host: Hello and welcome to Cook Children's Doc Talk. A while back we talked with Dr. Karen Albritton the medical director of Cook Children’s adolescent and young adult program in the Hematology and Oncology Center, and she oversees the Oncofertility Program. We also spoke with Olivia Prebus who was instrumental in developing the role of fertility navigator for the program. More recently, we talked with Toni Leavitt who we’ll be introducing later in the program. Toni is the current fertility navigator and we'll be sharing updates about the Oncofertility Program. Dr. Albritton is trained in both pediatric and medical oncology and specializes in the care of adolescents and young adults with cancer. She recognizes the unique needs of these young patients and the challenging decisions they face about day to day living that most people take for granted. One of the biggest and often overlooked as how fertility is impacted by the cancer treatment they receive. As an active advocate for AYA cancer patients, doctor Albritton was instrumental in founding the AYA program here at Cook Children’s as well as the Fort Worth AYA Oncology Coalition, which provides resources for cancer patients and survivors, health professionals and community members. The Fort Worth AYA Oncology Coalition launched the first community supported AYA oncology inpatient unit in the nation and offers young adults diagnosed with cancer age relevant resources and specialized care designed to improve their lives before, during, and after cancer. It is through her experience with AYAs that doctor Albritton has seen a need to consider fertility as part of all pediatric cancer care and establishing the oncofertility program here at Cook Children’s. Welcome. 00:01:48 Dr. Albritton: Thanks for having us. 00:01:51 Host: So let me start by asking what oncofertility is and why it's important? 00:01:55 Dr. Albritton: Oncofertility is a relatively new term in the cancer world and it is a combination of 2 fields, reproductive endocrinology which deals with fertility for people with and without cancer for many different reasons who are seeking to either preserve their fertility or attempt to have children to use fertility methods to have biologic children, and many years ago it was realized that cancer patients have their fertility affected by the cancer treatments and that these 2 fields cam

In 2020, ASCO went virtual for the first time. While the format was different, one thing was clear: despite all that is happening in the world, there are still many patients in need. Todd Shuster, MD, Head of Parexel’s Oncology Center of Excellence; Matthew Cooney, MD, co-lead of Parexel’s Cell and Gene Therapy initiative; and Keith Donovan, an expert in Early Phase Development with a focus on oncology, join Alberto Grignolo, Corporate Vice President, Regulatory and Access, to discuss the progress made in cell and gene therapy as presented at ASCO and how the industry can continue to move these developments forward today and in the future.

Kimberly Richardson - seven year cancer survivor of ovarian cancer, Granulosa Cell Tumor, Stage 3A. Prior to diagnosis, Ms. Richardson has 25 years of urban planning and community economic development experience. Accomplishments included creating a TIF district for the development of a major hotel and restaurants; rehabilitation and new construction of single family and multi-family housing units; and revising suburban business and housing code enforcement zoning ordinances. Ms. Richardson has been active since treatment in various forms of advocacy. As an advocate leader for the Ovarian Cancer Research Alliance (OCRA), she speaks with legislators on the importance of funding for ovarian cancer research, has crafted state legislation for an Illinois gynecologic cancer commission and has spoken at the national conference on patient advocacy. In her advocacy role with DoD/CDMRP/OCRP, she is both a pre and peer reviewer and contributing writer to the agency's patient advocacy blog GLOBELTHON. She shares her perspective on disparities in genetic testing and clinical trials through multiple platforms and hosted citywide events in partnership with FDA's Oncology Center of Excellence and CISCRP to increase minority participation. Ms. Richardson currently works with local elected officials and community based groups on health equity surrounding COVID19 and has shared her perspective as a cancer patient and advocacy partner with WCG. She has developed a program model called Survivors Advising Scientists that include training modules on basic scientific inquiry for survivors and new patient advocates that will be showcased at this year's virtual ASCO conference.

Richard Pazdur, MD, director of the U.S. Food and Drug Administration's Oncology Center of Excellence (OCE), joins Lisa Kennedy Sheldon, PhD, APRN, AOCNP®, FAAN, ONS's clinical and scientific affairs liaison, to discuss oncology nurses' contributions to cancer care, OCE's initiatives, and the COVID-19 coronavirus pandemic's implications for oncology drug approvals.   Music Credit: "Fireflies and Stardust" by Kevin MacLeod Licensed under Creative Commons by Attribution 3.0    Episode Notes  Check out these resources from today's episode:  FDA new drug approval announcements on ONS Voice Richard Pazdur's biography Oncology Center of Excellence FDA Guidance on Conduct of Clinical Trials During COVID-19 FDA Community Outreach and Education Programs Project Facilitate Project Orbis Register for the Mosaic of Oncology Nursing webinar on May 19 from 2–3 pm EST. COVID-19 articleson ONS Voice  ONS Information Regarding the Coronavirus (COVID-19) ONS Interim PPE Guidelines During the COVID-19 Pandemic

The US FDA oncology chief celebrates his 20th year at the agency with a wide-ranging podcast interview in which he addresses the next two phases of the Oncology Center of Excellence, the new “Project Avatar” and whether advisory committees still give good advice

Dr. Roohi Ismail-Khan and Dr. Michael Fradley discuss their work in establishing the joint Cardio Oncology program at the University of South Florida and the H. Lee Moffitt Cancer Center and Research Institute, in Tampa, FL. They discuss the many advantages for patients who have access to programs like this.

Subscribe to the podcast through iTunes and Google Play. Disclaimer: The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care, and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement. Dr. Clifford A. Hudis (CH): Welcome to this ASCO in Action podcast. This is ASCO's monthly podcast, series where we explore policy and practice issues that can impact oncologists, the entire cancer care delivery team, and the individuals we care for, people with cancer.  My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action podcast series. For today's podcast, I am delighted to have as my guest, Dr. Richard Pazdur, the Director of the Food and Drug Administration's Oncology Center of Excellence. The OCE was established to expedite the review of novel cancer therapies and products by bringing together expertise from across the FDA. And we'll touch on this a little bit during our conversation. Dr. Pazdur, welcome and thank you for joining me today. Dr. Richard Pazdur (RP): It's a pleasure Dr. Hudis. CH: Thanks. So I want to kick off our discussion by diving right into a hot button issue, expanded access. Can you provide our listeners with some background on this, and explain what the FDA's expanded access program is, and why an oncologist might want to pursue expanded access for an individual patient? RP: Of course. The FDA's expanded access program provides a way that patients with serious or life-threatening diseases or conditions such as cancer can try investigational medical products for treatment when no satisfactory therapies are available, and when there is no opportunity for the patient to enroll in a clinical trial. The process-- to make a request, the patient's physicians will approach the pharmaceutical company to ask for its agreement that the company will provide the medical product. The company has the right to approve or disapprove the physician's request. Then the physician needs to send the request to the FDA. This process can be complex to navigate, particularly for oncologists or physicians who don't have experience working with the clinical trials or these types of requests. FDA allows the vast majority of these requests to proceed. And the FDA has been working to improve the expanded access programs for a number of years, including the development of a more streamlined application process, a more streamlined form. But for many key health care professionals, especially those not familiar with the expanded access program, this process may appear confusing or somewhat burdensome. CH: And so is this a segue to Project Facilitate, which you announced at our annual meeting a few weeks ago? Can you talk a little bit about that and, its practical implications? RP: Yes. The Project Facilitate call center is a pilot program only for oncology that will serve a single point of contact. We have FDA oncology staff there, oncology nurses, oncology pharmacists who will assist the physician and their health care team throughout the process to submit and expanded access request for an individual cancer patient. This is a concierge service to support the patient's medical team throughout the process. It ranges from the initiation of the FDA form 3926. The process will also provide information about IRBs, particularly central IRBs, and really will also follow up on the status of a given patient to determine if that patient has received any benefit from the therapy and if there were any adverse events that need to be reported to the FDA. CH: So imagine that Project Facilitate works as hoped for. What's the thumbnail before and after experience? That is, how will things appear to be different to the physicians and to the patients? RP: It should make the process easier for physicians to get information that they need to submit an expanded access request. As I said before, it's often somewhat complicated, especially for physicians don't have experience with either the drug or with the process. And it's obviously easier to talk to somebody over the phone to ask specific questions rather than just being directed to a website. We're also working in conjunction with Reagan-Udall Foundation for the FDA, which started the expanded access navigator website to educate patients and health care professionals about the expanded access process. This navigator approach offers information provided by companies about their expanded access policy, and now includes the expanded access programs listed in ClinicalTrials.gov. Patients and physicians can look for treatment options. They could discuss clinical trials, and company information could be provided at the navigator at Navigator.Reagan-Udall.org. So this is really to give patients and their physicians information about what is out there. Once the patient obviously has this information and their doctor, then the doctor can utilize the Project Facilitate, which allows easier access to actually submitting these forms and going through the actual process. I'd like to emphasize that companies are now required by the 21st Century Cures Act to publicly list their expanded access policy. And the Reagan-Udall Navigator website helps them comply with that requirement. Again, so once the physician and the patient have identified the investigational therapy they want to try, the physician or other members of the health care team then can contact Project Facillitate for assistance in locating IRB resources and help with the FDA form 3926. CH: So I think you mentioned this when you launched this or announced it at the annual meeting just now, that physicians do already-- or at least before project facilitate often would successfully go straight to pharmaceutical companies and ask for treatments. And I guess in some cases they'd be denied, and in some cases they would be approved. And that would be through the company's expanded access programs. Obviously, that means that regulators wouldn't necessarily know the full extent of expanded access use. So assuming that Project Facilitate will allow the FDA to collect much more data on expanded use, how will the data be useful? And obviously, I'm hinting at the fact that some fear that it will be actually a negative. RP: Well, prior to launching Project Facilitate, the expanded access requests for cancer patients arrived at multiple places within the FDA and were forwarded separately to FDA oncology or hematology   divisions. Sometimes these requests could be delayed, being sent from one place to another in the agency. So this gives a focus point for physicians to contact. In addition, we're seeing that most of the expanded access requests were coming from patients and physicians at larger academic centers. The patients who don't live near these cancer centers and may not be able to get on clinical trials can also hopefully have access to investigational agents by having a more facile and easier process to use here. We're also seeing that many companies have turned down requests from patients, and we have no idea what really the number of requests a company may get if they're turning down these requests. Because generally, they don't come to the FDA. So really, by having the initial contact at the FDA we'll be able to determine number one, the number of patients that are requesting a single patient access. We'll also be able to determine and discuss with the companies their reasons for denying these requests. And there could be multiple reasons. And we also have a process in place that can follow up with what are the benefits that an individual patient may have from this therapy or, as I stated before, were there any adverse events. We have also heard this kind of urban myth-- and I label that in quotations, "urban myth," that companies fear that perhaps adverse events may be held against them when their drug is coming for drug approval. We have not done that. We take into context where the adverse event reporting is coming from. And there really are no instances that I am aware of in oncology where a report of an adverse event has delayed or curtailed an approval of a drug. CH: So really, this is a bright ray of sunshine on a dark corner of drug access. And if it works right, you'll just have much more understanding of the overall use of expanded access. Right? RP: Yeah. I think that gives some clarity to the process here. Here again, we don't know the numbers at this time of actually the number of patients. We only know the numbers of patients that receive a affirmative position from the drug company regarding that the process can continue. But we don't know the numbers of patients that may be requesting single patient access and are denied by an individual drug company. And also, the reasons. And, as I stated before, there can be very legitimate reasons, including inadequate supply of the drug, lack of support staff to follow up on these drug requests, potential interference with clinical trials that the patient may be eligible for. CH: You just used a phrase about patients requesting. And I thought as you described this process you were referring to physicians requesting on behalf of patients. And so I do want to ask, are there resources that are aimed directly at patients or is it really solely aimed at the oncologists in this case? RP: Well, here again, this is a two-prong process. Project Facilitate, the FDA portion of this, is for physicians to call up for assistance in filling out the form and also navigating the process once the decision is made. The other prong of this is, as I stated before, by Reagan-Udall foundation, which patients can call to look at what our options available to them that are potentially listed on ClinicalTrials.gov. And that is also for patients and physicians. However, the portion of the program that is Project Facilitate is for the requesting physician. CH: All right. Well, that's clear. So once we talk about patient's involvement, and even many physicians I think for that matter, we quickly can drift towards the very heated discussion that took place in public over the last year in the area or that we call Right to Try. And I wonder if you could talk for a minute and help us, for the listeners, make this distinction between expanded access and Right to Try. RP: Of course. These programs, Right to Try and single patient INDs are really mutually exclusive programs. The main difference between these programs are first, that under Right to Try the drugs have to complete a Phase I trial. For single patient INDs, it could be done anywhere, even within the context that the drug is being conducted in a Phase I trial. However, the major difference is that the FDA and the IRB does not review Right to Try applications, whereas under a single patient IND, the FDA obviously has to give permission for the patient to proceed as well as an IRB has to review these requests. CH: So to be very clear, Project Facilitate is supporting the single patient INDs, and Right to Try is a separate matter entirely. They are distinctly different programs. Project Facilitate does not apply to Right to Try. That is an independent, separate program. CH: Great. So, you know, one of the problems for a busy clinician is figuring out how to do all this under pressure with a sick patient, and the other pressures of clinic and administration and research. If our listeners want to learn about this more casually, where can they go not under duress, just to start reading up and learning about how to access the program? RP: They could go-- physicians can go and learn more about the program at our website, www.FDA.gov/oce. The Project Facilitate phone number is 240-402-0004. That's 240-402-0004. And the email address is ONCProjectFacilitate@FDA.HHS.gov. CH: That's great. So hopefully, some of our listeners will take advantage of that and learn about this when they're not under pressure so that they're familiar with it if they have to turn to it some months later. Now you mentioned that the host is the Oncology Center of Excellence. And I mentioned in my introduction that we would want to talk a little bit about that. CH: You've been at the helm of the OCE since it was established a little over two years ago, I think. Now that you've been in the role a while, I wonder if you could talk a little bit about your view of what the OCE should be accomplishing, and maybe how that aim has evolved over these two years. RP: Yes. The OCE basically was an offshoot of the Moonshot Program several years ago, and was aimed to be the first center that coordinates activity among the therapeutic center. Obviously, at the FDA there is a center for drugs, a center for biologics, and a center for radiologic health and devices. And they all can review oncology products. The OCE has a designation to really coordinate the activities, particularly in the clinical review of the products that involve the treatment of cancer. So, this is a unique center within the FDA, and is somewhat of an experiment at the FDA to see how we can really coordinate the activities of drugs that affect cancer patients. And here again, the oncology center is primarily designated for the clinical review. And we don't really get into the manufacturing of drugs. That's handled in the individual centers, whether it be a biologic and CBER, the Center for Biological Evaluation and Research or CDER, the Center for Drug Evaluation and Research. With that given said, in addition to the actual bread and butter of reviewing applications, we have many research projects that we're doing. We have a big project looking at real world data. We have a project looking at updating labels called Project Renewal. We have, as I stated before, this project that we launched at this year's ASCO, Project Facilitate. We also have a project aimed at really improving our relationships with international drug regulators. We have monthly meetings, teleconferences with five different regulatory agencies throughout the world to go over applications and discuss different regulatory policies. We have a host of a symposium that we conduct both here at the FDA, inviting external stakeholders including physicians, leading academics, patients to come to the FDA really to discuss important topics to our drug reviewers and the entire discipline of regulatory and oncology, so to speak, how we make decisions in medicine. We have a whole, also, program that we're developing aimed at educating physicians and other health care professionals for educating other health care professionals on how we evaluate drugs, what our thought processes are here at the FDA. So, in addition to the regulatory work, there is a whole body of scientific work that we're also doing, including independent research on different databases, looking at patient populations more likely to respond to different drugs, ways of evaluating and describing toxicities, ways of really looking at patient experiences while they're getting drugs, and different ways of reporting patient reported outcomes. We'd like to thank ASCO, obviously, for their assistance during and helping us with many of these projects throughout the year, especially the educational projects involving fellows, involving different topics that we've found of interest that needed to really have a public disclosure in the community, really, to get input from leading academics, as well as treating physicians. CH: Wow. You are busy. And there's a lot we could unpack there. But I do want to pick up on a couple of things. First of all, you described this as an experiment, so I'm curious. And not to put you on the spot, but if you have an experiment, I presume that just some metric that you would use to call it a success or failure. And I wonder where you think you are right now in that regard. It sounds like you've gotten a tremendous amount done. But are you satisfied, for example? Have you covered the ground you wanted to or do you think that you could be doing more? RP: Well, people who know me realize that I'm never satisfied. So, I think we're in the middle of this experiment. I think it's going quite well. And I think that this is really going to be aimed at-- and the evaluation of the success or failure of this is going to be really how the individuals that work here at the FDA really evaluate drugs and how we facilitate the evaluation of drugs. And also the really important of retention of staff here at the FDA is a major issue, also. And I think many of the projects that we have ongoing really develop our reviewers in really having a real world approach to how oncology drugs are used. So it's very difficult to say what success and failure will ultimately be. But I think we're on, really, the correct path, and pretty much a straight path of looking at a successful venture here. CH: You know, one of the things you said reminded me of another urban myth. And I don't know if you realize this. But when you describe the careful coordination with, I think you said five regulatory agencies around the world, it raises the myth, I believe, but you can address this with some facts, that many people in the United States believe that others around the world have faster access to a broader range of effective therapies. I wonder if you want to expand on that or comment on that at all before we move on. RP: Well, that is an urban myth, and probably was generated 20, 30, 40 years ago when that may have been the fact. Obviously, that antedated my coming to the FDA. But I can say the vast majority of drugs are approved first in the United States. And those include very important drugs such as the PD1 drugs, the targeted drugs, et cetera. They are approved first in the United States. We have taken a very active approach to really rapid approvals of our drugs without sacrificing quality, by having a smarter approach to how we review these drugs, with putting multiple reviewers on particular applications, by cutting down on unnecessary paperwork that many of our reviewers had to do, and really focusing on really the core material that we have at hand, and really emphasizing does this drug really demonstrate safety and efficacy. At the end of the day, I charge all of our reviewers with the following statement. Would the American public be better with this drug than without it? And that's the ultimate decision that we have to make at the time of approval. CH: Well, that's another perfect segue to a hot topic, which you and I have discussed actually offline before this. But I'm going to come back to it. The expedited approval of anticancer therapies was recently the subject of a paper in The Journal of the American Medical Association. And if I remember correctly, they looked at 93 cancer drugs that had been approved through accelerated approval process. But what they claimed is that only 19 of the 93 clearly extended the lives of the patients taking them. That's a value judgment, obviously, about why drugs are approved and introduced to the market. But I wonder if you would want to talk a little bit about your view of some of the complexities and challenges that are inherent in accelerated drug approval, and what your view is of this particular study of the approval outcomes. RP: I think many times people don't understand that it isn't just about overall survival. Obviously, that's the gold standard. But we've had very careful discussions throughout the years that there are many ways to evaluate benefit to the patient. And that includes reduction of the size of the tumor, delay in the progression of the disease, the establishment of complete response rates in hematological diseases. So we have to have some flexibility, both in terms of how we approve drugs and what clinical trials we're going to ask for after drugs have been approved on the accelerated approval pathway. Although overall survival is a very important end point, it's an important efficacy endpoint as well as a safety endpoint, it does have limitations. As we move more toward a targeted therapy and subsegment common diseases into molecular subtypes, many times we find that we have very limited populations. And simply, we don't have the size of a population that we approve the drug on to really do a large, randomized trial. So we have to weigh that issue with what type of trial we're going to ask for, both with the initial approval of the drug as well as with, perhaps, the subsequent studies that we ask for after an accelerated approval. In addition to that, many times we find that we have situations where the disease itself may have a very long natural history, such as CLL or other diseases that may have very long natural histories, where one cannot really do a long-term survival study because it would extend many, many, many years. And many times-- and I think we have to be realistic about this, that there may not be equipoise here to allow a randomized trial to be done looking at overall survival as a primary end point. For example, if we already have information that a drug may have a response rate of 50% or 60% and the comparator drug may have a response rate of 10%, patients will not want to go on a study that looks at overall survival as the primary end point. And many times, we have to take a look at time to progression or progression free survival and those end points, and actually allow for a switch in therapies or crossover at the time of disease progression, which renders the evaluation of overall survival somewhat difficult, and may confound that evaluation. So, there are many reasons why overall survival, although a gold standard, may not be applicable to all situations. And I think that's going to be increasingly so as we get into a more targeted therapy approach and have better definitions of who is going to respond. So here again, it's long natural history of diseases either by its natural history or by the therapies that have been approved that prolong disease. It could be due to the limited populations, which preclude a randomized trial. And it could be due to the lack of equipoise, which really bands that patients have access during the course of disease. I think a much more important question, and one that we are constantly looking at, is not so much what does an individual drug do to the natural history of the disease and prolonging survival in patients that have metastatic disease, but what is the impact over the years of multiple drugs being approved on the basis of progression-free survival or response rates when they are used either in combination or sequentially. And we could see that, for example, in multiple myeloma, where the course of that disease has been significantly changed, and patients' lives have been prolonged. And the vast majority of the drugs that have been approved have been on non-survival endpoints. And this is true not only for multiple myeloma, but also probably for renal cell cancer. CH: Yeah. That's interesting. It's a challenging analysis, of course. But that would be a very interesting, essentially public health roll up of all of these incremental decisions. Right? RP: Correct. CH: Yeah. So, as I said before, the OCE has been in operation just over two years. During that time, more than 80 therapies and products have been approved, I think. Right? And there've been more than a dozen guidance documents approved, 60 workshops and symposia for oncologists and for patients. And there were several of those workshops that we at ASCO were privileged to co-sponsor along with you. This is the favorite child question. But what's your proudest achievement so far? RP: A difficult question, but an easy question, too. It's about the people that work here and the patients that we serve. And I think my brightest moments are when we see the development of our people coming in and taking leadership positions both within the agency in a regulatory context of their job, but also in the academic fields and participating in conferences, publishing papers, and really finding enjoyment in the job that they have outside of the day-to-day regulatory activity. One of the things that I have always emphasized since I came here 20 years ago from an academic medicine position at M.D. Anderson is really to give the agency a much more academic perspective. And I've always stated that I think we do much more academic work here at the FDA than many academic centers. And I'm not talking about the generation simply of papers or research grants. I'm talking about actually critical thinking of what goes on at an application, since we have a multi-disciplinary team of statisticians, clinicians, clinical pharmacologists, toxicologists, manufacturing people that all work together. So it's really about-- my greatest accomplishment is really about the young people that have come in that I've mentored, and really have assumed roles, and really will be my lasting legacy here. But I also want to emphasize that one of the things that I have repeatedly highlighted to this staff is really to consider the patient in really any regulatory decision. Here again, it's not about a P value. It's not about a primary end point. Granted, those things are important, but we really have to bring together the whole body of information about a drug in making a regulatory decision and making that a patient-focused thing. And as I stated before, at the end of the day will the American patient-- will the American public be better off with this drug than without it? CH: Well, Rick, I got to say that's an inspiring description. It makes me wish I were younger, and maybe I could come and be mentored. But alas, it may be too late for me. But we really are proud to work with you, and to work with so many of your staff in many productive collaborations. I want to thank you again for joining me today for this ASCO in Action podcast. We always appreciate your expertise and your perspectives. And we look forward to continuing to work with you to ensure that patients with cancer have access to safe and effective treatments. RP: And thank you, Cliff. It's been a pleasure. And here again, I really think ASCO for providing a lot of resources to us in conducting symposium, and really in fostering better cancer care for patients. I think that's the ultimate goal of both organizations. CH: It sure is. And I want to remind our listeners that you can follow the FDA Oncology Center of Excellence on Twitter. Their handle is @FDAOncology. That's one word. You can follow me @CliffordHudis, and you can follow ASCO @ASCO. For more information on the latest cancer policy news and updates, visit ASCOAction.ASCO.org. And Rick, I'm going to ask you once more to remind the listeners of the way they can access Project Facilitate. RP: They can learn about Project Facilitate from our website at www.FDA.gov/OCE. And our project facilitate phone number is 240-402-0004. And the email address is ONCProjectFacili tate@FDA.HHS.gov. ONCProjectFacilitate is spelled O N C P R O J E C T F A C I L I T A T E @FDA.HHS.gov. CH: That's great. So until next time, I want to thank everyone for listening to this ASCO in Action podcast.  

The treatment of cancer has undergone a sea change in the span of a generation.  In this two-part episode for World Cancer Day, we discuss the increasingly complex oncology landscape, from both a clinical and clinician perspective.  First, Bob Millham, Executive Vice President of Clinical Development and head of our Oncology Center of Excellence, addresses the potential of clinical research in driving toward a world where cancer is treated more like a chronic condition than a death sentence – and the ongoing, critical importance of engaging patients in the process.We then hear from Dave Querry, President of The Navicor Group, on the evolving role of the oncologist and how manufacturers need to think about engaging differently with this increasingly specialized practitioner.Interested in reading more?  Follow these links for a deeper dive into a broad range of oncology topics from these and other Syneos Health experts:Trends In Oncology Trial Terminations Due To Toxicity ESMO 2018 Congress: Some Good Answers and Quite a Few New Questions Pursuing Accelerated Approval in Oncology IndicationsThe Importance of Real World Evidence in OncologyBIO18: Oncology Surplus Could Drive Down ValuesA New Reality for Small Oncology Trial Sites: A Need to Adopt a Federated Approach?

US FDA's new Oncology Center for Excellence brought in key experts to help review and approve Endomag's new Magtrace and Sentimag System combination product to help surgeons trace and remove breast tumors. The case highlights how disease-specific centers could change the way the agency reviews products.

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Oncologists Dr. Sanjeeve Bala and Dr. Abhilasha Nair from FDA’s Oncology Center of Excellence discuss the recent approval of bevacizumab-awwb, a biosimilar to US-licensed Avastin, marketed as MVASI. MVASI is the first biosimilar approved in the US for the treatment of cancer. Released on December 21, 2017

Yvonne Ruddy-Stein, APRN, of the Breast & Oncology Center in Southbury, Connecticut, joins host Robin Sills, RN, to talk about genetic testing. How do you know if certain types of cancers are in your genes? A family history can help point you in the right direction, and testing can help you make the right decision for your health. For more about Saint Mary's Hospital, visit stmh.org.

Three months after the official launch of the Oncology Center for Excellence, applications for cancer products are now being reviewed by OCE medical teams. OCE Director Richard Pazdur provides more details on the process and procedures of the newly established center in a podcast interview.

Friends of Cancer Research CEO Jeff Allen sits down with Ramsey Baghdadi and discusses the passage of the 21st Century Cures Act, the implementation of the bill and FDA’s Oncology Center of Excellence, what could be added to PDUFA VI, working with the Trump Administration, and the ideal profile of the next FDA Commissioner.