Podcasts about expanded access

In this episode of The Psychedelic Podcast, Paul F. Austin speaks with Dr. Raymond Turpin, a psychologist and psychedelic researcher who has been studying these medicines since 1984. Find full show notes and links here: https://thethirdwave.co/podcasts/episode-299/?ref=278 Dr. Turpin shares his journey from discovering Timothy Leary's writings as a college student to founding the Pearl Psychedelic Institute in North Carolina. He discusses his experience as one of only two sites nationwide to provide MDMA-assisted therapy through the FDA's expanded access program, the profound healing he witnessed in patients with treatment-resistant PTSD, and the challenges faced after the FDA's rejection of MDMA therapy in 2024.  Through stories of clinical breakthroughs, educational initiatives, and recovery efforts following Hurricane Helene, Dr. Turpin offers insights into the evolving landscape of psychedelic medicine and his hope for its future integration into mainstream mental healthcare. Dr. Raymond Turpin is the Executive Director and Clinical Director of the Pearl Psychedelic Institute in western North Carolina. He has been studying and researching psychedelics since 1984 and earned his doctorate in clinical psychology from the California Institute of Integral Studies. Dr. Turpin served as an investigator in an Expanded Access program providing MDMA-assisted psychotherapy for treatment-resistant PTSD and has worked with ketamine-assisted therapy while providing psychedelic integration services. Since 2022, he has been a Mentor for the Certificate in Psychedelic Therapy and Research program at CIIS. His clinical experience spans psychiatric hospitals, emergency units, residential treatment facilities, schools, and community mental health clinics. Highlights: A psychedelic calling that began in 1984 How a mushroom trip changed his career path When conventional mental health tools fall short MDMA's remarkable results with treatment-resistant PTSD From 71 to 18: Dramatic PTSD symptom reduction The FDA rejection: A devastating setback Hurricane Helene's impact on Asheville's healing community Why psychedelics threaten traditional mental health paradigms Turning trauma's irritant into a pearl of wisdom Education as resistance against misinformation Episode links:  The Pearl Psychedelic Institute The Pearl Institute YouTube channel California Institute of Integral Studies (CIIS) Certificate in Psychedelic Therapy and Research program Webinar: MDMA-Assisted Therapy for PTSD: Expanded Access & Beyond Timothy Leary's autobiography "Flashbacks" (1984) Episode Sponsor: The Intensive for Psychedelic Professionals in Costa Rica - a transformative retreat for personal and professional growth.

George Canyon, a local country music artist and long-time advocate for diabetes awareness

welcome to wall-e's tech briefing for tuesday, december 17th! explore the latest tech developments: meta's threads milestone: ceo mark zuckerberg shares that threads has hit 100 million daily active users, with monthly users rising to 300 million, spurred by bluesky's growing popularity and new features like custom feeds. openai expands chatgpt search: chatgpt search now available to all users, offering swift search capabilities and extensive information access, amidst ongoing concerns about its impact on publisher web traffic. tiktok's legal battle: tiktok appeals to the u.s. supreme court against a potential sale or ban, arguing free speech, as the deadline looms; hints of changing perspectives following a meeting between tiktok ceo and president-elect trump. waymo's robotaxi launch: waymo is set to debut robotaxis in tokyo by early 2025, in partnership with nihon kotsu and go, having taken into account gm's halted cruise program. softbank's u.s. investment: ceo masayoshi son announces a historic $100 billion investment, aiming to create 100,000 jobs, which aligns with shared goals in advancing ai and tech in collaboration with president-elect trump. stay tuned for tomorrow's tech updates!

Trinkal Patel, PharmD shares insights on compassionate use and investigational medicine.   For more pharmacy content, follow Mayo Clinic Pharmacy Residency Programs @MayoPharmRes.  You can also connect with the Mayo Clinic's School of Continuous Professional Development online at https://ce.mayo.edu/ or on X @MayoMedE 

The BC government rejected Dr. Bonnie Henry's call for expanded access to replacements for hard street drugs. Guest: Jess Ketchum - Co-founder Save our Streets Learn more about your ad choices. Visit megaphone.fm/adchoices

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Show notes FDA Approves High-Concentration Cyltezo Eye on Pharma: Simlandi US Launch; Ranibizumab Partnership; Expanded Access for Adalimumab Biosimilars DDW 2024 Posters Evaluate Safety, Efficacy, Impact of BMI on Zymfentra Use Posters Demonstrate Switching Safety Between Originator, Biosimilar Infliximab in IBD New Evidence Confirms Safe Biosimilar-to-Biosimilar Switching Spanish Real-World Study: Adalimumab Biosimilar MSB11022 Safe, Effective in IBD Data Show Promise for Adalimumab Biosimilars to Deliver on Safety, Cost Savings Patients With IBD Experience Nocebo Effect Post Mandatory Switch to Biosimilar Review: Product Attributes Relevant to Injection-Site Pain, Adalimumab Treatment

Corn Congress Wants Expanded Access to Foreign Markets

Indigenous teens traumatized by years of flooding and fire in Hawaii are leading a legal battle for climate justice. Two of the plaintiffs join us to reflect on a year of tragedy and hope. And, in 2023, voting was a fraught topic. Residents of some states saw increased measures to make voting more accessible, but those in others saw restrictions like bans on ballot drop boxes which served to restrict voting access. Liz Avore of the Voting Rights Lab joins us. Then, the cultural reach of video games has never been greater than in 2023. But working conditions for the people who make games have been dire, too. Here & Now's James Perkins Mastromarino unpacks the top games that came out this year along with the state of the gaming industry.

Dr. Guillermo Garcia Manero explains the new authorization for use of the drug, Reblozyl®.

March 22, 2023 - Assemblymember Jessica González-Rojas, a Queens Democrat, explains her legislation requiring public universities and colleges make emergency contraception available in vending machines on their campuses.

In this episode, Holly Fernandez Lynch and I continue our discussion of clinical research ethics with co-hosts Rahima Ghafoori and Caroline Gozigian (UVA Law '23). In this Part 2 of our interview, we focus on questions of payment, exploitation, and trust. As a reminder, in Part I, Holly introduced the basic regulatory framework governing clinical trials, with a focus on laws and rules impacting payment. She also discussed the benefits of and concerns about human challenge studies, and shared some historical examples. Holly Fernandez Lynch, JD, MBE, is Assistant Professor of Medical Ethics in the Department of Medical Ethics and Health Policy at the Perelman School of Medicine (PSOM), University of Pennsylvania. She has a secondary appointment as an Assistant Professor of Law at the University of Pennsylvania Carey Law School.A lawyer and bioethicist by training, Professor Fernandez Lynch's scholarly work focuses on Food and Drug Administration (FDA) pharmaceutical policy, access to investigational medicines outside clinical trials, clinical research ethics, and the ethics of gatekeeping in health care. Her specific areas of expertise include Institutional Review Board (IRB) quality, payment to research participants, research prioritization, pre-approval access pathways (e.g., Expanded Access, Emergency Use Authorization, and Right to Try), and efforts to balance speed and certainty in drug approvals, including pathways that rely on post-approval trials such as accelerated approval. Links:Lynch HF, Darton TC, Levy J, McCormick F, Ogbogu U, Payne RO, Roth AE, Shah AJ, Smiley T, Largent EA. Promoting Ethical Payment in Human Infection Challenge Studies. Am J Bioeth. 2021 Mar;21(3):11-31. doi: 10.1080/15265161.2020.1854368. Epub 2021 Feb 4. PubMed PMID: 33541252.Shah SK, Miller FG, Darton TC, Duenas D, Emerson C, Lynch HF, Jamrozik E, Jecker NS, Kamuya D, Kapulu M, Kimmelman J, MacKay D, Memoli MJ, Murphy SC, Palacios R, Richie TL, Roestenberg M, Saxena A, Saylor K, Selgelid MJ, Vaswani V, Rid A. Ethics of controlled human infection to address COVID-19. Science. 2020 May 22;368(6493):832-834. doi: 10.1126/science.abc1076. Epub 2020 May 7. PubMed PMID: 32381590.Largent EA, Heffernan KG, Joffe S, Lynch HF. Paying Clinical Trial Participants: Legal Risks and Mitigation Strategies. J Clin Oncol. 2020 Feb 20;38(6):532-537. doi: 10.1200/JCO.19.00250. Epub 2019 Jun 14. PubMed PMID: 31199697.

Holly Fernandez Lynch and I discuss clinical research ethics, including challenge trials, research subject payment, and diversity in medical research with co-hosts Rahima          Ghafoori and Caroline Gozigian (UVA Law '23). In this episode, Holly introduces the basic regulatory framework governing clinical trials, with a focus on laws and rules impacting payment. She also discusses the benefits of and concerns about human challenge studies, and shares some historical examples. In the next episode, Part II of our interview, we explore issues of coercion, inducement, and exploitation more explicitly.Holly Fernandez Lynch, JD, MBE, is Assistant Professor of Medical Ethics in the Department of Medical Ethics and Health Policy at the Perelman School of Medicine (PSOM), University of Pennsylvania. She co-chairs the PSOM Research Ethics and Policy Series (REPS) and serves as Assistant Faculty Director of Online Educational Initiatives in the Department, where she helps lead the Master of Health Care Innovation. She has a secondary appointment as an Assistant Professor of Law at the University of Pennsylvania Carey Law School.A lawyer and bioethicist by training, Professor Fernandez Lynch's scholarly work focuses on Food and Drug Administration (FDA) pharmaceutical policy, access to investigational medicines outside clinical trials, clinical research ethics, and the ethics of gatekeeping in health care. Her specific areas of expertise include Institutional Review Board (IRB) quality, payment to research participants, research prioritization, pre-approval access pathways (e.g., Expanded Access, Emergency Use Authorization, and Right to Try), and efforts to balance speed and certainty in drug approvals, including pathways that rely on post-approval trials such as accelerated approval.Links:Lynch HF, Darton TC, Levy J, McCormick F, Ogbogu U, Payne RO, Roth AE, Shah AJ, Smiley T, Largent EA. Promoting Ethical Payment in Human Infection Challenge Studies. Am J Bioeth. 2021 Mar;21(3):11-31. doi: 10.1080/15265161.2020.1854368. Epub 2021 Feb 4. PubMed PMID: 33541252.Shah SK, Miller FG, Darton TC, Duenas D, Emerson C, Lynch HF, Jamrozik E, Jecker NS, Kamuya D, Kapulu M, Kimmelman J, MacKay D, Memoli MJ, Murphy SC, Palacios R, Richie TL, Roestenberg M, Saxena A, Saylor K, Selgelid MJ, Vaswani V, Rid A. Ethics of controlled human infection to address COVID-19. Science. 2020 May 22;368(6493):832-834. doi: 10.1126/science.abc1076. Epub 2020 May 7. PubMed PMID: 32381590.Largent EA, Heffernan KG, Joffe S, Lynch HF. Paying Clinical Trial Participants: Legal Risks and Mitigation Strategies. J Clin Oncol. 2020 Feb 20;38(6):532-537. doi: 10.1200/JCO.19.00250. Epub 2019 Jun 14. PubMed PMID: 31199697.

Pink Sheet reporters and editor discuss the impact of the midterm elections on the US Food and Drug Administration, the Inflation Reduction Act's effects on drug development, and adjustments to the FDA's expanded access guidance.

Listen in as Mass. State Rep. Lindsay Sabadosa takes us through the omnibus abortion legislation recently signed into law. Rep. Sabadosa has helped lead the way to make public campuses safer for students by expanding access to reproductive healthcare, including medication abortion. Learn more about highlights from this legislative session, tips for talking about abortion with young people, and why it is important to share our stories.

Episode 46 of the How We Do Digital Ministry podcast features Tom Trenney, Minister of Music to First-Plymouth Congregational Church in Lincoln, Nebraska and Assistant Professor of Music and Conductor of Choirs at Nebraska Wesleyan University. Tom and Charlotte Elia discuss music ministry in the hybrid environment, the impact of the digital sphere on sacred song, and encouragement for our future together. "It doesn't have to be perfect to be good, and it sure doesn't have to be perfect to be meaningful and heartfelt." - Tom Trenney Listen to the full episode as Tom talks about these topics: (0:54) Checking in with Church Musicians (6:25) Affirmations from a Broader Reach (10:54) Singing Apart and Singing Together (20:09) Expanded Access to Sacred Music (23:28) Recent Lessons for Musicians (28:35) Encouragement for This Moment You can find Tom online here: https://www.firstplymouth.org https://www.youtube.com/c/FirstPlymouthChurchLincolnNebraskaVideos tomtrenney@gmail.com How We Do Digital Ministry is brought to you by Faith Growth, a small business dedicated to helping churches do digital ministry through coaching, consulting and website development. When you're ready, sign up for a free consult at www.faithgrowth.com/call For now, join pastors and church communications from around the world in our How We Do Digital Ministry Facebook group. Thanks to Presbyterian Media Mission for spreading the word about How We Do Digital Ministry.

April 14, 2022 - Alliance for Quality Education Policy & Operations Director Marina Marcou-O'Malley tallies up the state's commitment for pre-kindergarten classes and what it means for access across New York.

Google is back swinging for the fences with 9 updates this week, and we are back always bringing you the latest updates! Special thanks to our viewers who commented on Youtube letting us know they were having issues with our sound, glad to hear that we resolved them for you, hopefully, we can keep it up and keep bringing you the best Google Workspace and tabGeeks content! Here are this week's updates: Published Releases Google Sheets doubles cell limit View additional Calendar statuses in Google Chat Background noise reduction now available for Google Meet Hardware devices New email draft template in Google Docs Create surveys, quizzes, and more using the Google Forms API, now generally available Expanded Access Management Controls help support your information governance goals Schedule posts for multiple classes in Google Classroom Built-in Webex interoperability on Google Meet hardware expanded to devices with remote control View more information on email delegate activity in the Security Investigation Tool Other Topics GCP Pricing increasing Chrome OS is rebranding to ChromeOS Participate in an upcoming Google Usability study Workspace Recap is the only show dedicated to and discussing all of the changes happening in Google Workspace on a weekly basis, as well as how all these changes affect our users and our businesses. Google Workspace is innovating at a breakneck pace, making it difficult to keep up and keep track. Join us each week as we discuss What's New in Google Workspace, Upcoming Google Workspace releases, and answer your questions. Hit the subscribe button, engage with us on Twitter at @WorkspaceRecap and on our website at workspacerecap.com --- Send in a voice message: https://anchor.fm/workspacerecap/message

This week Jeremy talks to Dr. Alison Bateman-House, a medical ethicist and member of NYU Langone's Working Group on Compassionate Use and Preapproval Access, to uncover the work being done to expand the ethical allocation of drugs that are currently in development.Learn more about the Working Group on Compassionate Use and Preapproval Access at https://med.nyu.edu/departments-institutes/population-health/divisions-sections-centers/medical-ethics/research/working-group-compassionate-use-preapproval-access/frequently-asked-questions

Through the global virtual internship program, one DePaul student assisted a Spanish film company in producing a documentary about the pandemic. Another helped an Irish nonprofit analyze social and emotional support needs for the refugees they serve. They did this without having to fly around the world. GianMario Besana, associate provost for global engagement, joins DePaul Download to discuss how new programs born out of expediency during the pandemic have expanded access and transformed global learning opportunities.

As the omicron variant takes hold, there are growing calls for expanded access to COVID-19 booster shots. Epidemiologist Dr. Prabhat Jha talks about the best way to curb the spread of the variant leading up to the holidays.

Join the advocacy effort! Freda and I talked today about H.R. 2168 - The Expanded Access to Telehealth Act of 2021. Listen to hear about Freda's exciting conversation with ASHA's Monica Sampson - Director of Healthcare Services in Speech-Language Pathology. And please click and share the links below to urge your representatives to cosponsor H.R. 2168 https://www.congress.gov/bill/117th-congress/house-bill/2168/texthttps://takeaction.asha.org/asha/Telehealthhttps://www.apta.org/advocacy/take-action/patient-action-centerhttps://cqrcengage.com/aota/app/onestep-write-a-letter?0&engagementId=510602

Safe & Just Michigan has been working in partnership with several organizations and municipalities to advocate for legislation that passes and implements automatic expungement in Michigan. Safe & Just Michigan's Executive Director John S. Cooper will be joined by Amanda O'Boyle, Assistant City Attorney of Lansing, and Frances Walters, Director of the Conviction Integrity and Expungement Unit of Washtenaw County Prosecutor's Office (WCPO). They will discuss how municipalities can support and benefit from expanded access to expungement and how they are implementing these programs throughout the state. Presenters: John S. Cooper, Executive Director, Safe & Just Michigan Frances Walters, Director of the Conviction Integrity and Expungement Unit, Washtenaw County Prosecutor's Office (WCPO) Amanda O'Boyle, Assistant City Attorney, City of Lansing

For patients who have run out of approved treatment options, hopes of fighting disease can feel diminished. Fortunately, through existing and new programs, patients have access to alternative drugs. Educating physicians more on the program availability can lead to an increase in options for patients and a more positive outlook to recovery. Dr. Ajeet Gajra, Chief Medical Officer at Cardinal Health Specialty Solutions, joins the show to discuss experimental oncology drug use through the Expanded Access Program & the Right To Try Law. Here are the show highlights: - Expanded Access Program vs. The Right To Try Law - Hesitancy & Barriers to Use Programs - First Steps & How To Stay Up-To-Date on Programs - Tips for Office Staff w/ These Programs - Success & Future Use of Drugs Check out these resources we mentioned during the podcast: - Dr. Gajra's LinkedIn (https://www.linkedin.com/in/ajeet-gajra-md-mbbs-facp-77059748/ - Dr. Gajra's Research (https://ascopubs.org/doi/full/10.1200/OP.20.00569) - Cardinal Health (https://www.cardinalhealth.com/en.html) To hear more interviews like this one, subscribe to the Working In Oncology Podcast on Apple Podcasts, Spotify, or your preferred podcast platform.

In this episode, we discuss the basics of stem cells, medical tourism, false claims about stem cells as an autism treatment, bioethical issues within the field of stem cells and methodological issues in autism research—with discussion of Duke University's Marcus Center for Autism and The Stem Cell Institute of Panama among others. This is such … Continue reading “I worry FDA Expanded Access will become a new way of bringing products to market”: Talking with Jeremy Snyder and Leigh Turner about “stem cells for autism”

https://rumble.com/vepk51-520-cpcs-liberal-ad-keilbros-testimony-az-expanded-access-in-cad-and-much-m.html https://www.bitchute.com/video/oIySBNnyBHMa/ Join The Conversation! home Me on Rumble https://rumble.com/user/CanadaPoli Me on Odysee https://odysee.com/@CanadaPoli:f My Show Backcatalog on YouTube https://www.youtube.com/channel/UCCd_S-VxU0vz7tu29FiT_wg Me on Twitch https://www.twitch.tv/canadapoli Me on Periscope https://www.pscp.tv/MarkParalovos/1vOGwkzNPdLGB Headlines and More! https://canadareport.co/ Discussion Based https://speakingmoistly.co/ Me on Twitter Tweets by MarkParalovos Podcast https://play.google.com/music/listen?u=0#/ps/Ihhe33job6uxmjrrws4znzl4eq4 Who Am I? https://markparalovos.com Copyright Disclaimer under section 107 of the Copyright Act of 1976, allowance is ... Read More The post 520 – CPC’s Liberal Ad, Keilbros Testimony, AZ Expanded Access in CAD and Much More! appeared first on CanadaPoli.

NUBC has announced two new condition codes to be added to claims starting February 1, 2021 for Expanded Access and Emergency Use Authorization services.

Adnan Jaigirdar, MD, FACS, provided an overview of the U.S. Food and Drug Administration (FDA) expanded access program (compassionate use) for investigative therapies for mesothelioma patients.

Dec. 31, 2020 - As the fight against COVID-19 continues, many of New York's students are learning from home. Senate Education Chair Shelley Mayer (D-Yonkers) joined the program to explain her legislation that would help expand access to high speed internet. (originally aired 12/11/20)

Dec. 11, 2020 - As the fight against COVID-19 continues, many of New York's students are learning from home. Senate Education Chair Shelley Mayer (D-Yonkers) joined the program to explain her legislation that would help expand access to high speed internet.

The PayPod host Cyrielle Chiron is joined by Anne Butler, Chief Legal Officer and Vice President of Policy and Research at Payments Canada and Jason Young, Head of Legal and Company Secretary at PayPay Canada to discuss why Canada needs to expand membership to payments systems and how ongoing efforts to update existing payments legislation for new entrants and Paytechs is more important than ever to stay competitive. The group dives into the status of the Retail Payment Oversight Framework (RPOF) and other ongoing work to advance payments modernization efforts.

This episode was recorded May 21, 2020Visit the program site here:  https://www.uscovidplasma.orgTo claim credit visit: https://ce.mayo.edu/covid19podcastGuest: Michael J. Joyner, M.D. (@DrMJoyner)Host: Amit K. Ghosh, M.D., M.B.A. (@AmitGhosh006) Convalescent plasma as a passive immunity strategy is not new. What nuances apply for it’s application to treating COVID-19? Is it safe? Is it efficacious? Dr. Michael Joyner, Principal Investigator for Expanded Access to Convalescent Plasma for the Treatment of Patients with COVID-19, discusses what’s known about this treatment option, information that’s currently being gathered, and how you can get involved.AABB Coronavirus Resources: http://www.aabb.org/advocacy/regulatorygovernment/Pages/AABB-Coronavirus-Resources.aspxAskMayoExpert COVID-19 Resources: https://askmayoexpert.mayoclinic.org/navigator/COVID-19Connect with the Mayo Clinic’s School of Continuous Professional Development online at https://ce.mayo.edu/ or on Twitter @MayoMedEd. 

Due to the financial impacts of the Coronavirus, the Government has expanded access to JobSeeker Payment and introduced a new Coronavirus supplement of $550 per fortnight See acast.com/privacy for privacy and opt-out information.

Are you curious about what goes on in the FDA? Natasha Kormanik, MSN, RN, OCN, Senior Regulatory Health Project Manager at the FDA describes her day-to-day job and debunks myths about the expanded access program. Reporting by A. Kate MacDougall, editor and writer.

Are you familiar with FDA's progressive programs for medical device companies? Do you know about Expanded Access, the Expedited Access Pathway (EAP) Program, or Breakthrough Devices Program (BDP)? Jon Speer discusses these programs with Mike Drues of Vascular Sciences. The FDA has created such programs to encourage companies to develop products that meet unmet clinical needs, and reduce the time and cost of bringing new or high-risk products from development to market without actually changing the approval standards. These programs are not shortcuts and do not involve less work; it's about businesses implementing a more efficient process. SOME OF THE HIGHLIGHTS OF THE SHOW INCLUDE: ● Requirement 1: Allows any device from all pathway categories, if it provides a more effective treatment/diagnosis of a life-threatening or irreversibly debilitating disease/condition. ● Requirement 2: Device must represent breakthrough technology, no approved/cleared alternative exists, offers clinical meaningful advantage over existing/approved alternatives, or availability is in best interest of patients. ● One advantage of BDP program is that it encourages people to come to the FDA very early during the development process; basically just need proof of concept. ● A question never asked in the pre-submission is, “Have we confirmed that we qualify for the BDP.” Questions that were asked were technical in nature. ● 2-Step Process: Take pre-submission to FDA for a BDP designation, and then submit second pre-submission that covers additional items. ● Expedite access of device to market by shifting clinical data requirements from pre-market to post-market. ● FDA is trying to be more progressive and innovative in giving clear and more expedited options of pathways to get medical products to market sooner. ● Average regulatory professionals know the rules, the best regulatory professionals know the exceptions.

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.  

Shannon joined us to discuss some of the basic design and published results surrounding MDMA-assisted psychotherapy for PTSD as well as the MDMA Therapy Training Program and how to become an MDMA therapist plus plans for Expanded Access.

Download In this Episode, Joe interviews Brad Burge, Director of Strategic Communications at MAPS. In this episode they discuss the Phase 3 Trial for MDMA Assisted Psychotherapy, contradictions and Expanded Access. 3 Key Points: MAPS is about to run Phase 3 Trials of MDMA Assisted Psychotherapy If MDMA passes this third phase, it will still be tricky to get insurance involved. But the cost of one series of MDMA Therapy is much cheaper than a lifetime of typical pharmaceutical drugs and therapy sessions to heal PTSD. The only reason for-profit companies haven't gotten involved before was because there wasn't a promise on their investment. Finally, for-profit companies (like Compass Pathways) are interested in advancing these medicines (Psilocybin and MDMA). Support the show Patreon Leave us a review on iTunes Share us with your friends – favorite podcast, etc Join our Facebook group - Psychedelics Today group – Find the others and create community. Navigating Psychedelics Show Notes MAPS Brad Burge is the Director of Strategic Communications at MAPS, the Multidisciplinary Association for Psychedelic Studies MAPS started out as just a few employees in 2009 and has grown to over 40 now Phase 3 Study They are now in Phase 3 Trials They started recruiting at 14 sites (US, Canada and Israel) and are recruiting 150 volunteers with severe PTSD Participation The Future of MDMA Assisted Therapy Breakthrough Therapy Designation The FDA categorized MDMA as a breakthrough drug for PTSD After phase 3 trials, if all goes well, it would mean that MDMA would be the drug to be used (only) alongside Psychotherapy MAPS is training therapists, counselors and social workers One way to get more people educated who are interested in this would be taking therapy interns in and having them gain credits for interning and learning alongside trained therapists Access Expanded Access is known as ‘compassionate use’, a program by the FDA that allows people to receive a treatment that is still in trials In order to administer the therapy you are required to get a DEA schedule 1 license “If there’s one thing that changes public perspective on psychedelic therapy, its individual stories of people who have been healed, transformed by or positively or even negatively affected by them in some way” - Brad They have published many studies of the trials The most recent was the Boulder study, 76% of people didn't have PTSD a year after MDMA assisted therapy Insurance won't cover expanded access, it will have to pass Phase 3 trials until insurance can be used in MDMA Assisted Psychotherapy The MDMA is a very small cost (fraction) of the total cost, it’s the hours on hours of psychotherapy that cost so much But the cost of one MDMA Therapy Session process is much cheaper than a lifetime of pharmaceutical drugs and therapy sessions to heal PTSD Argument Joe says he hears this strange argument that people say “giving soldiers MDMA just makes war easier” Brad says it's not about putting these people back into war, it's about healing the retired veterans to help them adapt back into their everyday life “MDMA Assisted Psychotherapy is going to make them a better lover not a better fighter” - Brad “If there's one commonality in psychedelic experiences, its that things are connected.” - Brad Compass Pathways Joe mentions that people are scared to see a business come in that's acting like a normal pharmaceutical company MAPS is not tied at all with Compass Pathways Out of the top two things Americans are mad about, at least one of them is the Pharmaceutical Industry Finally, for-profit companies are interested in advancing these medicines (MDMA) The only reason for-profit companies haven't gotten involved before was because there wasn't a promise on their investment Capitalism has a tendency to put profit first “Money can be used for good as well as evil” - Brad MAPS has raised over 70 billion dollars all from donations Compass owns its own safety data Part of the goal of a patent is to protect the investment Zendo Project MAPS Psychedelic Harm Reduction and Peer Support resource Tim Ferriss has volunteered for Zendo They are always looking for new volunteers They offer trainings on site at the events They will be hosting a harm reduction webinar right before festival season Rave Act The Department of Justice announced that providing free water and harm reduction education are not violations of the rave act Amend the Rave Act Pharmaceutical MDMA The pharmaceutical grade MDMA costs 800,000 for one kilogram It won't be available in bottles, it will be available in bubble packs More than one is never needed Involvement 2021 or 2022 is the next Psychedelic Science Conference Joe is starting a Psychedelic Club in Breckenridge, CO Links Twitter Facebook Website About Brad Brad Burge is Director of Strategic Communications at the non-profit Multidisciplinary Association for Psychedelic Studies (MAPS). Brad earned his B.A. in Communication and Psychology from Stanford University in 2005 and his M.A. in Communication from the University of California, San Diego in 2009. He began working with MAPS in 2009, where he engages daily with journalists and media producers around the world to enhance public knowledge about psychedelic research, while also helping develop and evolve MAPS' brand and outreach strategy. Brad is passionate about finding healthier, more effective, and more compassionate ways for humans to work with the pharmaceutical and digital communications technologies of the 21st century. When he’s not plugged in, you’ll find him in the mountains, carrying a backpack, somewhere down a long trail.

Subscribe to the ASCO in Action Podcast through iTunes and Google Play. "Welcome to this ASCO In Action podcast. This is ASCO's monthly podcast series where we explore policy and practice issues that impact oncologists, the entire cancer care delivery team, and the individuals we take care of, people with cancer. My name is Clifford Hudis, and I am 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. Scott Gottlieb, the commissioner of the United States Food and Drug Administration. The FDA of course plays a critical role in the delivery of high quality cancer care by reviewing and approving cancer treatments. This continues to generate discussions about the pace of scientific advances, and indeed the regulatory role of the FDA. Given that, we are really lucky to be able to talk today with Dr. Gottlieb about the FDA's efforts to increase overall efficiency by updating or modernizing aspects of our clinical trials conduct and expediting the end to end drug development process. I will admit that I'm going to take advantage of this opportunity to also ask how his agency is tackling the issue of tobacco control for the next generation of tobacco products, another area of deep concern from our community and others. Dr. Gottlieb, welcome and thank you for joining me today. Thanks for having me here. Great. Now as a professional society, we are very focused on the intersection of science and society. Given that, and noting that you've been at the FDA for more than a year following a long career in public service and in private industry, I have to start by asking, for you, has your current experience changed or evolved your view of the FDA's role or functions, and if so, how. I've been at the agency three previous times. I had a good sense of what the agency's public health mandate was and what its mission was. I think that the nature of the market and the science that we're grappling with has certainly forced or compelled the agency's mandate to evolve. I think what we're seeing right now is, I feel like we're at the inflection point with respect to a lot of new opportunities from technology. We look at things like gene therapy and cell-based regenerative medicine. Those fields largely didn't exist last time I was at the agency, and now we're an inflection point where we're going to see gene therapies approved to the market, and we saw three CAR-T's approved already, that are going to fundamentally transform the treatment of disease. When I was last here, we were talking about the ability to advance genomically derived drugs and have more targeted approach to the treatment of patients where you can get the right drug to the right patient at the right time with sort of a drug diagnostic system, yet seeing some early examples of that. But now that is a much more routine development pathway. With respect even to tobacco, you mentioned tobacco at the beginning, we're at a point right now where we have the opportunity to use new technology potentially to help currently addicted adult smokers transition away from combustible tobacco products onto products that we presume don't have all the same risks associated with them. And so using new authorities we have to regulate tobacco, including regulate nicotine levels in combustible cigarettes to render them minimally and not addictive, and allowing for a regulatory pathway that puts some of the new technologies like e-cigarettes through an appropriate series of regulatory gates, we have the opportunity to transition adult smokers off of combustible tobacco with all their morbidity and mortality associated with combustible tobacco use more rapidly than we did in the past. And so across the board, I think we're seeing technological inflection points. Digital health tools are another example that are not only creating significant new opportunities, but I think compelling the agency to rethink its traditional approach to regulation in order to accommodate the opportunities that these new technology platforms create. So is it fair to sum all that up as, the agency is recognizing that technology in many domains is defining and driving the need for new regulatory frameworks, and in turn, we have to educate Congress and others to make sure that the agency actually has the appropriate authorities. Is that the virtuous cycle you're describing, you think? I think that's exactly right. I think that there are areas of profound technological change where the traditional approach to regulation doesn't apply well. Digital health tools, probably a very obvious example where you can have sort of practical incentives, where you have a digital health tool, you might evolve it almost on a daily if not weekly basis in the marketplace. It's a medical product that's a digital health tool, like a medical lab, for example. And a regulatory process that requires you to come in and file for premarket approval every time you want to make a modification or have to file a 510(k) supplement, that is antithetical to the rapid cycle of innovation and evolution that those kinds of products undergo. So we had to think of a new regulatory paradigm for how we would treat these products, and that's where we move towards the Pre-Cert model, where effectively what we're doing is validating the underlying architecture of the platform, of the software platform, and validating the SOPs, how good is the company at certifying its own software and validating its own software products, and then we would allow them after an initial approval to come to market with modifications as well as subsequent approvals without having to seek premarket clearance from the FDA every time. And we would shift toward the postmarket regulatory regime for subsequent products. So basically, instead of regulating the individual products, in essence we're regulating the firm and taking a firm based approach. That's an example of where we've had to rethink our regulatory model in order to accommodate a much different approach to innovation. Another example is with cell-based regenerative medicine, where we have very clearly said that we think that there's a lot of opportunity with cell-based regenerative medicine, but we also see a lot of clinics promulgating products based on what we think is incomplete if not poor science and creating substantial risk of patients using cell-based products intrathecally, or for injections into the eye, where they're creating substantial risk and don't have really a scientific basis to argue that there's convincing evidence of a benefit. And these products are clearly subject to FDA regulation. They cross the line between what is and isn't regulated by FDA, but subject to enforcement discretion of the agency over many, many years. Prior to when I came in here, the agency didn't actively regulate these products. We have said very clearly, we're going to actively step in to regulate this field. In fact, it's going to take a number of enforcement actions, and we'll be taking others. At the same time, we also recognize that a lot of these technologies are being promulgated by small developers, and there's a lot of promise here. And so we've had to again come up with a more accommodative approach. But how do we regulate a field where a lot of the really interesting innovations are being brought forward, for example, by academic investigators working in small clinics. And so what we've said in that case is that we'll allow investigators to pool their data so long as they follow common manufacturing protocols that are doing similar things with cells. And it can file a common BLA, common Biologics License Application. And then we'll give individual licenses to the individual institutions or individual investigators. That's a much different approach to regulation than what we've traditionally done where you think of, we regulate companies, we regulate a biotech or a pharmaceutical company. We had to ask ourselves, how do we regulate small clinics or even institutions, academic investigators in institutions, who want to promulgate these technologies. And we've come up with an approach to do that. So at the same time that we've said we're going to be taking more enforcement action to make sure patients aren't being put under duress, we're also going to take a more accommodative approach to allow for regulatory approval for products that are being, in many cases, promulgated by smaller entities and individuals. So that actually lets us pivot, I think, to an area of traditional focus for at least a large group of our members at ASCO. And that would be drug development. And it's clear that you've made it a priority to streamline how new drugs specifically are reviewed and approved. And I think as part of that effort, recently you announced a new office. It's the office of Drug Evaluation Science. And my understanding is the goal is to centralize performance metrics like biomarkers, patient reported outcomes. How do you see this new office specifically helping to support this goal of a more standardized and ultimately more efficient and faster review process? Well, the Office of Drug Development Science, the goal there is to create an infrastructure here that will help better validate scientific tools that are being used to help advance drug development, like patient reported outcomes, like bioinformatics. What we've seen is that these tools now, there's a lot of hard science behind these tools. And they've become much more commonly used in drug development programs. And so we need an infrastructure here that not only provides for a more standardized approach to assessing these modalities when they're incorporated into applications, but also helping to advance the science of how to use these tools. I compare it to what happened in 2003 with modeling and simulation. And I was here during the time period. What we were seeing over that time period was we were seeing more drug developers starting to use modeling and simulation as a component of their overall drug development programs. And we saw modeling being included in applications. Early on, it was often used for dose finding because you wanted to give the dose finding trials. But then you wanted to use the data that you derived from the dose finding trials to simulate what would happen if you picked a dose in between the two doses that you might have tested. And so we said to ourselves, well, this is very interesting. This could really help inform drug review better, give us more information about safety and benefit. We need some standard approach to how we're going to both evaluate these tools as well as help develop them into a harder science so it could be more rigorously used in drug development. We created a Modeling and Simulation Office when I was here. Mark McClellan was involved in doing it. He helped recruit the guy who stood it up, him and Janet Woodcock. It was Larry Lesko. And it started as a two-man office. Now, we've got probably 30 or 40 people in the Modeling and Simulation Group in Cedar. And well more than 95% of all applications that we get for new drugs contain a component of modeling and simulation. It's now a routine part of drug development. And we have a rigorous approach to evaluating these components of the applications as well as helping to evolve the field through multiple guidance documents that we've put out. I see the Drug Science Office, this new office that we created within the Office of New Drugs, working in a similar way where it's going to be a holding office, if you will, for new areas of science that can help improve tools used to inform us about the risks and benefits of new products. So I want to pivot from that to one of the big societal issues. And you and I have been discussing this informally, I think, for more than a year-- drug price. The Trump administration has made it a priority to address the cost of drugs, price specifically. And I guess the question from many will be how confident can we be that ultimately a faster and more effective drug development process will itself actually and favorably impact the costs of drugs. What's your feeling about that, knowing everything that you're doing that's supportive in that way? Well, I see my role in the drug price debate to be focused around creating product competition. You have price competition. But you can't price competition without product variety and product competition. And we're focused on creating the product competition by facilitating entry into the market of generic drugs, but also, facilitating a pathway that allows for follow on innovation within categories. And what we've seen-- and we've analyzed this. We're going to be publishing this data soon. But what we've seen over time is that second to market innovation within a category is coming to market much more slowly. We looked at a cohort of approvals from the early 2000s. And then we looked at a more recent cohort of approvals over like a five-year period. And it's very clear that when you look at areas of unmet medical needs, orphan drugs, first in class drugs and oncologies, the second to market innovation is taking much longer to come to market and more categories of drugs are remaining sole source drugs in perpetuity. So to the extent that you're not getting that second to market innovation for new drugs, that is thwarting the opportunity for price competition within those categories. Because you do see price reductions. Oftentimes those price reductions come in the form of rebates that aren't transparent to the consumer. But there are price reductions or discounting nonetheless when you have a second and third to market drug within a category. And the hepatitis C category was the best example of that. We saw a very dramatic price reductions in negotiations once you had multiple entrants in that category. So we're focused on that. And we're asking questions about why it's become harder to bring second to market innovation to patients. And I think that there are some very specific reasons and there's things we can do to help address that, to make it less costly to bring second to market innovation in an area of unmet medical need. If the trials are onerous or very costly to bring that second to market drug to the market, sometimes the economic opportunity might not be robust enough after this one entrant, especially when you're talking about very narrow niche categories of unmet medical need, they're might not be enough economic opportunity to incentivize that second to market innovation. I think where-- just to sort of close, I think where this might be most evident is in some of these inherited diseases. You've seen this play out in gene therapy, for example, where once you come to market with a treatment and you treat the prevalent population, the people who already have the disease, the incidence population, the number of people who are going to get it on an annualized basis, that might not be a big enough market to support a second entrant that's going to split the market with the first in class product. And so I am quite literally seeing investors pull out of these opportunities which we see what we think are applications being slowed down. We see people pull out if they think they're going to be third to market. And that's ultimately bad for patients. Because it's not just robbing patients of product competition, but it's also robbing them of potential product variety. And we know not every patient responds to a treatment the same way. So you want differentiation in the market. And also if there's a horse race between being first, second, third to market and the third to market pulls out because they don't think they're going to be first or second, sometimes the first or second doesn't pan out. And then you're stuck with nothing. So this is not a healthy development. And if there's things we can do to make the development process more efficient to create more entrants, that's something we're focused on. So that's great. You raised at least two issues that I want to pursue a little further. Let's start with the first one, which is technology based. Recently you announced specific plans to keep pace with the influx of applications for selling gene therapies. And you've referred to that already in your comments. But you've raised concerns specifically around, I think, if I understand it, reimbursement environment for CAR-T therapies and a fear that that reimbursement challenge may, in fact, stifle innovation that's needed. Is there more to say about that? Or is that pretty much the issue right there? Well, I think that that's one of the issues. I think the issue, obviously, is-- and many people who are in this space are acutely aware of this-- is that there's different pay structures on the inpatient and the outpatient side to the extent that some of these products are being labeled for use in inpatient only. The reimbursement on the inpatient side is much lower and more difficult than reimbursement on the outpatient side right now. So that's an unusual situation and something that's artificial. I mean, a drug shouldn't be reimbursed diametrically differently just because it's delivered in one setting versus another and the reason why you're pushing it into an inpatient setting is for safety considerations. And so I think it's something we need to address. We can't allow that sort of artificial differentiation to persist. There's a lot of late stage CAR-T development. But we're not seeing a lot of early stage CAR-T development. And if you talk to people in the field, they'll say, well, it's because CAR-T hasn't demonstrated its ability to really potentially be effective in solid tumors. And a lot of the liquid tumor opportunities are already being pursued. I'm not sure that's true. I think that there is a reluctance. At least part of it is a reluctance to make significant investments right now because the reimbursement environment is so uncertain for these products. So that ultimately needs to be resolved by others, including CMS. But I think that there are things we can do as well here at FDA. So for example, we say that a product should be labeled for inpatient use because we believe that with certain risks associated with the delivery of some of these products-- and you're very familiar with those risks and so are all your listeners-- require the ability to deliver intensive care or significant medical services if a patient does have a reaction on an infusion of this product. But that doesn't necessarily mean that you need to be in an inpatient facility. What it means is you need to have within a reasonable period of time-- and you can define that period of time-- access to significant supportive care. I know institutions where the inpatient infusion center is further away from the medical intensive care unit than the outpatient infusion center. So that makes no sense. Why would you say it has to be in an inpatient setting when the inpatient setting actually is further away from the kinds of medical resources that we want accessible to the patient? So really what we should be considering is defining and labeling the kinds of services that need to be available within a certain period of time, and not necessarily inpatient or outpatient. Because there are a lot of outpatient infusion centers that are adjacent to academic institutions where you can have a significant amount of supportive care delivered very quickly. And the patient is very accessible to a medical intensive care unit if they do have an adverse reaction. So we're rethinking that, how we label these products. But that's a-- it might solve the proximate challenge. But ultimately, I think you need a fundamental solution to the pay structure so there's not an artificial divide between the inpatient and the outpatient with respect to these products. So just in the interest of time, I want to make sure we cover the monetization of clinical trials because that's the other issue, I think, rightly raised. And you've for a while made this a priority, I know. And I'll just jump ahead and say, and I think if I remember correctly, your agency the FDA issued two draft guidance documents on innovative trial design within the last year. The first focused on master protocols. And the second on specific advice in terms of design and conduct for adaptive trial designs. That's a compressing approach in terms of the phases of traditional studies. Can you talk a little bit about why this is important and what kind of savings you think this could actually deliver as a practical matter? I think it's important because, first of all, I think a lot of the drugs that are being put into development can't be developed efficiently with the traditional approaches to drug development. So for example, you think of a drug where it's targeting molecular change that's apparent in multiple disease states. This is most obvious in cancer where you have tissue agnostic approvals where you might want to do a basket trial where you test a drug in multiple tumor types where what's driving the tumor is the same genetic alteration, molecular change. And you want to be able demonstrate that it works across multiple tumor types, especially with rare tumors where you might not-- if you said, well, you have to prove it first in lung cancer and then you go on and prove it in liver cancer. But it might be such a rare genetic change that you're not going to be able to efficiently enroll just in lung cancer and liver cancer. So you want to pool the data across multiple tumor types to demonstrate statistically significant evidence of benefit. I think because more drugs are being designed that way, we have to rethink how we allow sponsors to conduct clinical trials, structured clinical trials. And so things like basket trials and master protocols and tissue agnostic approvals become very important in this paradigm. It also can allow for a lot more efficiency. A master protocol can allow you to test multiple drugs within the context of the same trial. If you have a situation where you're looking at targeting a rare disease or a rare subpopulation of a disease, where it's hard to recruit people, if you have a master protocol set up, you can test multiple drugs in the same population much more efficiently. So as we develop drugs that are targeting smaller and smaller populations and delivering, in many cases, outsized benefit and demonstrating earlier evidence of benefit, we need to rethink how we structure trials to take advantage of those opportunities. I think one of the-- we approved a record number of novel drugs this year by a long margin, 59 approvals. The second best year, which was last year, in modern times, I think was 46. We approved 19 new NDA and BLA products focused on cancer and had 38 supplements this year. If I was to point to one thing that's driving that innovation, it's the fact that more of the drugs, many more of the drugs that are being put into development now not only have a very plausible biologic rationale for why they're going to deliver benefits, but they're so well targeted, so the underlying disease state is so well understood, that we're seeing much more significant benefit much earlier in drug development in much more compelling disease situations. And so, proof of concept is established very early. And you can establish statistically significant evidence of benefit in a very small series. And that's accelerating these products through development. And more of these cases are situations where you're targeting such significant unmet medical needs that even if there is uncertainty around the full scope of the safety profile, the outsized benefit in that clinical setting overwhelms any of that uncertainty. And so you can move these products through development much more efficiently. That's the nature of the science that we're seeing right now. And I think it's going to be the way we see the field move forward, at least for the foreseeable future. You know, one of the issues that this raises is the issue of targeting and niche subpopulations which you've referred to. We've tried to deal with this within ASCO by launching TAPUR, which takes next gen sequencing, by and large, and matches patients who are theoretically scientifically appropriate for off label use with drugs that are in the market but where the indication doesn't include their histology. And I know your agency is familiar with it. But that, in turn, generates prospective evidence. The vast majority of patients, as you know, in the United States simply don't have the opportunity for various reasons to participate in clinical research. And that has raised questions about the utility of so-called real world evidence and real world data. You know that the FDA has been working closely with ASCO, especially with our big data project CancerLinQ, so that your agency has access to our growing big data repository. And that in turn, we all hope, will inform certain aspects of regulatory review, I guess mostly in the area of label extensions. So your agency recently released a framework providing detail on how the FDA is going to develop guidance for real world data in drug regulation. And we're especially excited by this. We're invested in this, in a sense. And we look forward to working on it as it rolls out. How do you see this framework being implemented specifically? How is it going to benefit patients? I think it's going to address one of the things you said right up at the top, which is patients don't have access to clinical trials. I think as we make more rigorous use of real world evidence in the development process and in the regulatory review process, that's hopefully going to open up the opportunity for data collection and clinical trials to move out into the community. Real world evidence isn't just evidence collected after the fact. You can have real world evidence collected in randomized settings. You can have real world evidence collected in prospective settings where you have large, simple registries and other kinds of constructs. And so, as we're able to make more rigorous use of these kinds of data constructs, I think it's going to push clinical data collection further out into the community so more patients are going to be able to access experimental protocols where the evidence is being generated that's going to help inform regulatory review, either in the pre- or post-market setting. And we're clearly making widespread use of real world evidence in post market setting, particularly for confirmatory studies post approval. And you're seeing situations where it's also informing decisions on the premarket side as well. So I guess, since we're talking about access, one has to at least address the question of very ill patients and access to investigational drugs outside of the clinical trial system, what's been called expanded access. And this has been a topic of great discussion and debate for the last couple of years. Can you talk about some of the specific changes that the agency is making and how you see this helping patients and physicians navigate the new expanded access program? Well, the one that we announced recently is that we're going to create a service here at FDA where we're going to staff it. Initially it's going to be sort of a pilot. And we'll focus it on oncology where we'll help patients navigate the expanded access process soup to nuts where effectively they will be able-- if someone identifies an expanded access protocol that they want to get entry into with their physician, their physician is going to be able to call FDA. And FDA is going to help guide them through the process, soup to nuts. FDA will have people who will make the outreach to the sponsor and do the interface with the patient and provider to make sure the documentation is done in a timely fashion. This is also going to have the advantage of allowing us to be on the phone with the drug sponsor to understand why drug sponsors might not give access in certain settings. And so what we find is, in some cases, we're willing, we approve the ability for a patient to get access to a product, but the drug sponsor might turn it down. And so this is going to allow us to collect more information about why it might be turned down. It's also going to allow us to identify situations where there might be a lot of requests of one drug company so that we can intervene to help encourage the development of a true expanded access protocol. If there's a lot of compassionate use requests, for example, of a single sponsor or a single drug, those are situations we might pick up the phone and say, hey, we're approving or we're getting requests for a lot of compassionate use. Why don't you think of starting an expanded access protocol? We can work with you on that. So I think that having FDA be an interface there is not only going to make it more efficient for the patient and provider to access the system, but hopefully will also allow us to interface better with sponsors to sort of create the conditions where drugs can be made more widely available under appropriate conditions. And just for clarity, I assume that there is a 800 number or web URL for that. Is that right? Well, we stand it up. It's still in process. So it's something that we're going to do soon. But yeah, this will be widely disseminated to folks. Great. So the last thing I want to talk about, which brings us in some ways back to our roots, is tobacco. And I said at the top of this that we would touch on this. This is an area where our field saw slow but ultimately critical progress starting in the 1960s. And all of this feels like it might be jeopardized by a recent and alarming uptick in tobacco use in children, essentially kids and young adults. And this is just setting off, as I say, alarm bells across our field. I think there's data from the FDA and the CDC that in 2018, 3.6 million students were e-cigarette users. And this was compared to just 1.5 million about a year earlier. Now there's still not a lot of research on Electronic Nicotine Delivery Systems or so-called ENDS. But there is at least some reason to believe that they might increase the likelihood of nonsmokers or former smokers converting to combustible tobacco with their known risks. So last year, I know that the agency announced the Youth Tobacco Prevention Plan to address this alarming trend. And it'd be great if you could talk a little bit about the plan and what you intend to do and update it, as I know you've been talking at least on social media about this issue in particular. Well, we think that the non-combustible products like e-cigarettes provide a potential opportunity for currently addicted adult smokers to transition off of combustible tobacco onto modified risk products. These products, the e-cigarettes, need to be put through an appropriate series of regulatory gates. But I've said many times, if we can transition every adult smoker off of cigarettes, traditional cigarettes, onto e-cigarettes, that's going to provide a significant public health advantage, public health opportunity. The e-cigarettes are certainly not risk free. Those risks need to be properly defined through a regulatory process. But there is an opportunity there. And what we announced early on last summer of 2017 was that we are seeking to-- and we've advanced the rulemaking to do this. We're seeking to regulate nicotine levels in combustible cigarettes to render them minimally and not addictive so they can no longer sustain addiction. At the same time, we allow the e-cigarettes to remain on the market while we put them through an appropriate series of regulatory gates with the notion being that if regular cigarettes no longer have nicotine, smokers would more rapidly migrate off of traditional cigarettes, hopefully off of nicotine altogether. But if not off of nicotine, onto either medicinal nicotine products, the safest form of nicotine delivery. Or if they want inhaled forms of nicotine delivery, onto e-cigarettes. Again, recognizing that e-cigarettes aren't risk free. But on a risk continuum, nicotine exists on a risk continuum, they are lower risk than combustible tobacco. But what I said all along was that that opportunity and that policy framework couldn't come at the expense of addicting a whole generation of young kids onto nicotine through these same products. And that's, in fact, what we're seeing. We are seeing an epidemic growth. And this is what we spoke to last fall in the use of e-cigarettes by children with fully a 78% rise among high school aged kids in e-cigarettes in over one year, from 2017 to 2018. And really no indication that it's going to abate very quickly in the coming year. So what we set out to do was implement a series of regulatory steps to try to address the access and appeal that these products have to kids. So we are putting in place significantly heightened age verification requirements for the purchase of products in convenience stores. We're particularly targeting the flavored products because we think the flavored products are a primary vehicle by which these products are appealing to children. At the same time, we launched a series of public education campaigns that we think are very effective to try to educate youth about the risks of e-cigarettes. But I'll say in conclusion that if these actions don't have a very immediate effect on these trends-- and you're not going to reverse these trends overnight. These trends are underway. This has become sort of a fashionable item among kids. You're not going to just reverse that overnight. But if we don't see this growth leveling off and starting to reverse, I think that this is an existential threat for the entire e-cigarette industry. You know, I find myself stuck in conversations where I'm debating with them the merits of selling cherry flavored e-cigarettes at convenience stores or gas stations where it's readily accessible to a kid. And I think what they really should be contemplating is, boy, if these trends go up another year, my entire product's going to be taken off the market. Because that is the cold, hard reality. We are going to-- whether it's FDA acting to change its enforcement policy or it's Congress stepping in, if you see another year of 50%, 60% growth in e-cigarette use among minors and you see fully 45% of American kids using some form of tobacco products and you see combustible smoking rates trying to go back up again, that's going to be a public health catastrophe. Nobody is going to have patience to tolerate that for another second. And there is going to be dramatic steps taken. And so I think that the industry ought to wake up to that fact. We've certainly woken up to that fact and recognized it. And it would be a shame. It would be a shame because the e-cigarettes do represent an opportunity for currently addicted adult smokers in a properly regulated market. We don't want to foreclose that opportunity entirely. And we don't want to impede adults unnecessarily from getting access to these products. But we are not-- collectively, we haven't done all we can and all we should to address the youth use. You're going to see us take more steps going into this year. We have more enforcement activity underway. But the manufacturers also need to stop fighting some of these steps. And they need to start addressing this more seriously. And, you know, it's one big manufacturer in particular that's driving a lot of the youth initiation on these products. Well, it's great to hear the vigor that is being brought to bear on this. And I know that in our community there's tremendous support for threading this needle just right, as you describe. So thank you for that. I want to just take a moment now and say, in general, to Dr. Gottlieb, thanks for joining me today for this ASCO In Action podcast. We are really grateful at ASCO for the strong collaboration that exists between us, the entire oncology community, and the FDA. And we look forward to continuing our work together to make sure that patients with cancer have access to safe and ever more effective treatments. As a reminder to listeners, you can follow Dr. Gottlieb on Twitter @sgottliebfda. That's one word. You can follow me, a little less exciting I think, @cliffordhudis. And you can follow ASCO @asco. To stay connected with the latest updates on the FDA's work, visit fda.gov. And as always, we will continue to provide here updates on relevant FDA activities at asco.org/ascoaction. Until next time, thanks again to Dr. Gottlieb and thanks to all of you for listening to this ASCO In Action podcast.

Are you familiar with FDA’s progressive programs for medical device companies? Do you know about Expanded Access, the Expedited Access Pathway (EAP) Program, or Breakthrough Devices Program (BDP)? Jon Speer discusses these programs with Mike Drues of Vascular Sciences. The FDA has created such programs to encourage companies to develop products that meet unmet clinical needs, and reduce the time and cost of bringing new or high-risk products from development to market without actually changing the approval standards. These programs are not shortcuts and do not involve less work; it’s about businesses implementing a more efficient process. SOME OF THE HIGHLIGHTS OF THE SHOW INCLUDE: ● Requirement 1: Allows any device from all pathway categories, if it provides a more effective treatment/diagnosis of a life-threatening or irreversibly debilitating disease/condition. ● Requirement 2: Device must represent breakthrough technology, no approved/cleared alternative exists, offers clinical meaningful advantage over existing/approved alternatives, or availability is in best interest of patients. ● One advantage of BDP program is that it encourages people to come to the FDA very early during the development process; basically just need proof of concept. ● A question never asked in the pre-submission is, “Have we confirmed that we qualify for the BDP.” Questions that were asked were technical in nature. ● 2-Step Process: Take pre-submission to FDA for a BDP designation, and then submit second pre-submission that covers additional items. ● Expedite access of device to market by shifting clinical data requirements from pre-market to post-market. ● FDA is trying to be more progressive and innovative in giving clear and more expedited options of pathways to get medical products to market sooner. ● Average regulatory professionals know the rules, the best regulatory professionals know the exceptions.

Among the great promises of biosimilar therapies is their ability to not only reduce costs for patients and healthcare systems, but to make room in budgets for spending on other high-cost drugs, especially in treating cancer.  This week on “Not So Different,” our guest is Ali McBride, PharmD, MS, BCPS. McBride is pharmacist who serves as the Clinical Coordinator for Hematology and Oncology at The University of Arizona Cancer Center. He’s also the lead author of a recent study that showed that using biosimilar filgrastim can expand patients’ access to treatment with obinutuzumab.

Dr Gospodarowicz speaks with ecancertv at the 2016 World Cancer Congress about the Global Task Force for Expanded Access to Cancer Care and Control, and her work with increasing the availability of radiotherapy in low and middle income countries. She describes the essential benefit to patients of radiotherapy as a treatment modality, and the economic benefit to society through longer survival of citizens. Considering proton therapy, which was discussed further at the Proton Therapy Congress, Dr Gospodarowicz sees it as a useful tool, but not an immediate replacement for most radiotherapy.

Date Posted: 03/31/2010Size: 6.64MBLength: 00:14:32Thought Leader: Nicky Wisener, Biopharmaceutical Account Manager, IdisIn this episode, Ms. Wisener describes how expanded access and named-patient programs can help successfully meet patients' needs while addressing the unique challenges faced by smaller and emerging companies.Play PodcastDownload Whitepaper For more information on how you can be featured in a podcast, contact Dan Limbach at dlimbach@pharmavoice.com or call him at (847) 594-0157.