Rare Voices Podcast

On this episode, we hear from a variety of voices in the front lines of rare patient-first care. With pandemic uncertainty looming, there are a lot of questions that are constantly emerging for people with rare conditions and their families. How can potential challenges be seen as opportunities for improved quality of care? What are the creative ways that pharma companies are creating awareness for rare conditions? Be inspired and be challenged to rethink your 2022 strategic plans. I can tell you that this one really inspired me! Here are the bios for the awesome Optime Care team that we had on this episode: Dr. Brandon Salke, general manager Dr. Brandon Salke serves as the pharmacist-in-charge and General Manager at Optime Care in Earth City, MO. He previously served as a team pharmacist for Dohmen Life Science Services, where he helped launch several new care programs. He is specialized in specialty pharmaceuticals, particularly ultra-orphan, orphan, and rare disease. Dr. Salke has been involved in all aspects of operations (planning, process integration, project management, etc.) for pharmaceutical manufacturers. This includes clinical trials to commercialization and assisting in commercial launches (and relaunch) of specialty pharmaceuticals. Dr. Salke completed his PharmD from St. Louis College of Pharmacy (StLCOP) in St. Louis, MO and holds the Certified Specialty Pharmacist (CSP) credential from the National Association of Specialty Pharmacy. He is currently an active member of the National Association of Specialty Pharmacy (NASP), American College of Clinical Pharmacy (ACCP), American Society of Hospital Pharmacists (ASHP), and the Missouri Pharmacists Association (MPA). Dr. Salke currently holds a pharmacist license in 20 states and the US Territory, Guam, allowing him a deep understanding of state and federal pharmacy. Stephanie Wasilewski, vice president Stephanie Wasilewski is a VP, Care Director at Optime Care. Stephanie has been a pharmacist for over 15 years and is passionate about providing best-in-class patient care. She started her journey in the retail pharmacy setting, eventually managing a pharmacy for 5 years. Her career path led her to the mail order setting and then she was called to Optime Care where she now manages a program for patients with Hereditary Angioedema (HAE). Jessy Broughton, care director Jessy Broughton is a Care Director at Optime Care. She has been at Optime Care for 2 years and loves the chance to help make a difference in the lives of our patients. Prior to Optime Care, Jessy worked in Care Coordination at St. Luke's Hospital. There she lead a medication adherence program and was instrumental in receiving a 5 star rating for Medicare measures. She also has a background in retail pharmacy, as well as attended pharmacy school. Jessy works hard to make sure her team provides the best experiences to our patients. Hannah Morgan, pharmacist Hannah is a team pharmacist at Optime Care and has been with the company since 2018. She has worked as a pharmacist for the last 8 years and has a passion for helping others. After years in community pharmacy, Hannah found her way to Optime Care where she takes pride in her job as pharmacist. Hannah is committed to providing quality patient care and is enthusiastic about applying her insight, creativity to help make a difference in the lives of others.

21- year-old Doug Lindsay seemingly had his whole life in front of him. However, in college, that future was turned upside down, leaving him bedridden and homebound dealing with his autonomic-adrenal condition. After countless treatments and strategizing with the best in the medical field, Doug sought to have a surgery done that would blur ethical lines in the medical field, but would allow him to recover from his rare ailment. Now, Doug continues to improve medicine. Having worked with 35 senior faculty at 28 institutions, developing new uses for 5 existing prescription drugs, winning a national first court case protecting patients' rights, and developing the concept for the two successful, innovative adrenal surgeries used to treat his case, Doug continues to be an advocate for patient care. On this episode, Doug tells his story, highlighting the highs and lows of the journey and how he looks to help others facing rare medical challenges. Please subscribe and keep listening to Rare Voices! Music Credits: Westpoint Instrumental by  Sun Shapes

The families of people with rare conditions often feel like they have to live two lives. For the person they love and care about, their son or daughter or their parent, they want to put on a tough and sunny face. When they take their loved one out in public, they want everyone to see just how strong the family is and how everyone should be grateful. They grit and smile through the treatment side-effects, through awkward glances, through long nights. Then there is the other life. The one where they just break down. The one that they try to keep hidden from the world and maybe even that person they care about. They keep it hidden because they may come across as weak or ungrateful or even self-centered. But, of course, they are humans. And they are bound to feel the weight of despair, the confusion of unpredictability, and the odd joy of loving someone through tough times. Unless you have had to live this dual life, everything that I just said can still just remain a theory – something that you can turn off now and go about your day. But for the people with rare conditions and the families around them, this is a reality – day-in and day-out. Effie Parks is someone who has lived that dual life and come out the other side. And she is someone who has merged those two lives and offered an alternative for caregivers and advocates. She is the proud mother of Ford, her son born with an extremely rare condition. Her desire to create the best life for Ford drove her into advocacy and to create “Once Upon a Gene,” an inspiring podcast that reveals the stories of the people living dealing with that dual life. On this episode, we both get real and swap stories about the despair of diagnosis day, the hopes we have for our families, and our challenge to a health care industry still blind to the nuances of life with rare disease. It is an unflinching look at real life, and I know that you will enjoy it. Please subscribe and keep listening to Rare Voices! Effie Parks Bio Effie Parks was born in the magical land of Montana, where she was raised with her 12 loving siblings.  After moving to Washington and marrying her husband, they were blessed with the birth of their son, Ford Canon Parks.  When she learned that Ford had been born with an extremely rare genetic condition – CTNNB1 syndrome – she dove into the world of advocacy.  Now, she is the host of her own podcast, Once Upon a Gene, where she speaks to others about their journey through life with rare disease. Music Credits: Westpoint Instrumental by  Sun Shapes

Being rare is, by definition, a lonely and hopeless thing. Loving and caring for someone with a rare condition can sometimes feel even more lonely and hopeless. You spend most of your free time thinking how can I help them or influence the health care industry to find a cure for the rare condition my father, mother, child, brother, sister, husband or wife have? Our guest today is a visionary and inspiration for anyone who has had that lonely hopeless feeling. Sanath Kumar Ramesh is the father of a two-year-old boy who is one of only 9 kids worldwide with an ultra-rare genetic disease. It is hard to find conditions that are more unique. However, Sanath and his wife have found an antidote. Not to the condition for their son, but for the loneliness and hopelessness. And it comes down to two simple things. One – Sharing their story. Sanath has chronicled the good, the bad, and the ugly truths about the journey he is taking with his family on his podcast “Raising Rare.” Two – Crowdsourcing for a cure. Sanath has applied his software wizardry to the creation of the Open Treatments Foundation, an open-source software platform to enable treatments to millions of patients worldwide. By unapologetically sharing his story, Sanath is giving others the inspiration to do the same. By sharing these stories and giving people hope are enough to change our industry. And it is change we need. Please subscribe and keep listening to Rare Voices! Listen now: https://www.optimecare.com/rare-voices/category/podcast/ More About Sanath Kumar Ramesh Connect: Sanath's Linkedin: https://www.linkedin.com/in/sanathkr/ OpenTreatments Foundation Linkedin: https://www.linkedin.com/company/opentreatments/ Resources: Raising Rare Podcast: https://www.raisingrare.fm/ OpenTreatments Foundation: https://www.opentreatments.org/ Sanath Kumar Ramesh Bio Sanath is a bold and visionary software leader, open source pioneer and a rare disease drug developer. In his career, he has built and launched several successful open source software products. He is now using open source software to enable treatments for 400 million patients worldwide affected with rare genetic diseases through his non-profit organization, OpenTreatments Foundation. He is building the world's first software platform to decentralize drug development and empower anyone in the world to create a treatment for a genetic disease. He is also the father of a 2 year old boy who is one of 9 kids worldwide with an ultra-rare genetic disease called SSMD (curegpx4.org) Music Credits: Westpoint Instrumental by  Sun Shapes

In the years that I have worked in health care, I have seen the term “patient first” plastered on so many company websites. More often than not, though, that promise does not hold up when you look under the hood. They say patient first. But what they really mean is: We thought about a mass we call patients. The thing they are missing out on, however, is the powerful results that come from understanding patients as people - whole people. The guest for today's podcast, Dr. Jennifer Mulle, is the real deal when it comes to patient first. She is a groundbreaking researcher for the rare 3q29 microdeletion and its implications for a number of conditions. But, more importantly, she is someone who has designed her innovative research around intimate understanding of her patients as people with families, with dreams, and with deep questions. I won't spoil it for you, but her story, like mine, is personal, and her intellect is only outmatched by her heart for serving people. Please subscribe and keep listening to Rare Voices! Listen now: https://www.optimecare.com/rare-voices/category/podcast/ More About Dr. Jennifer Mulle Dr. Mulle Twitter: https://twitter.com/shotgungladys The Emory 3q29 Project: https://genome.emory.edu/3q29/​ Emory Profile: https://med.emory.edu/directory/profile/?u=JMULLE Dr. Jennifer Mulle Bio Dr. Mulle is an Associate Professor in the Departments of Human Genetics and Epidemiology at Emory University. As PI for The Emory 3q29 Project, she leads an interdisciplinary team to understand the phenotypic spectrum, molecular mechanism, and natural history of 3q29 deletion and 3q29 duplication syndromes. Music Credits: Westpoint Instrumental by  Sun Shapes: https://www.marmosetmusic.com/artists/sun-shapes

The past year has shaken so many of us to our core. I remember, about a year ago, sending everyone home from our offices at Optime Care. And everything felt uncertain. When could we bring people back? What about our patients? They count on us…daily. How will COVID-19 impact them? Not just their health, but their access to other care givers? I went back to my office and fired up my computer. On my LinkedIn feed, there was a familiar face. One of my most respected friends had posted a video he called “A Concerned Doctor.” I hit play. And, in the midst of chaos, I felt like I had found a confidant and ally. Here was someone on the front lines, caring for patients, and doing the most important work a physician can do – adding a voice of reason to an otherwise turbulent time. Over the past year, I have kept my friend, Dr. Jeffrey Miskoff, pulmonologist and critical care physician, as a constant presence. A good portion of his patients also have rare conditions, and it is why this episode is so important for anyone connected to the rare and orphan patient communities. His story over the past year involves loss, heartache, hope, and victories. It has something for you no matter where you are in the spectrum of patient care, and, as with all of our episodes this season, it has an outlook for where health care is going next. Please subscribe and keep listening to Rare Voices!   Dr. Jeffrey A. Miskoff, D.O. Bio Dr. Miskoff specializes in Critical Care Medicine, Pulmonary Medicine and Sleep Medicine. He completed his Residency at Philadelphia College of Osteopathic Medicine and Fellowship at Beth Israel Medical Center. Dr. Miskoff specializes in rare disorders such as Alpha-1 Antitrypsin Deficiency and narcolepsy. He also hosts his own video series on YouTube called, “A Concerned Doctor”, where he discusses the COVID-10 pandemic, treatment, and concerns around the virus. Subscribe here: iTunes   •   Soundcloud   •   Stitcher    •    Youtube   Music Credits: Westpoint Instrumental by  Sun Shapes

Almost all of us in the world of rare and orphan have one of these stories. For me, it was my father and my family, of course. But, almost without exception, everyone I know who is leading an effort to improve the lives of people with rare or orphan conditions has had a catalytic moment with a patient. Maybe it was during clinical trials or at a patient advocacy meetup or even your neighbor, but, at some point, you got to know someone as a person. You learned about their humanity, their aspirations, their families. And that is what led you to working with those populations. And why you listen to a podcast like this one. If, perhaps, you have forgotten what that moment was like for you, we have something very special for you in this episode. This is the story of Jessica Ballinger's family. And this story will remind you what your first time was like. Because this episode is the up-front, raw, and unfiltered voice of a parent in the midst of developing the best possible futures for all her kids — including a toddler with a rare condition. You may know Jessica or, at least, you may know about various members of her family. Nearly each one of them has achieved their own level of notoriety on YouTube or comedy shows. But, today, we are getting a rare, behind-the-scenes look at what it takes to be a mother of a child with a rare condition. Pay close attention to her courageous point-of-view and how she is guiding her family forward one day at a time. Jessica Bio: Jessica Ballinger and her husband, Chris Ballinger, run a family and lifestyle channel on YouTube. They create content across multiple platforms, including Instagram, TikTok, and Twitter. They homeschool their five children, ages 1-13, together and love to share their parenting experiences. They also recently started a podcast called, “Let's Talk About What We Learned Today”, where they discuss family matters and relationships. Music Credits: Westpoint Instrumental by  Sun Shapes

One of our guest we bring you in this episode is a self-described industry dinosaur. The reason he can make a claim like that, of course, is his longevity. But also, perhaps without knowing it, he can make that claim because of the long-term impact he has had on health care. In fact, if you look back over the past 30 years of pharmaceuticals, you can see his fingerprints on so many of the advancements that we take for granted today. This dinosaur is Steve Aselage. He is a member of the board of directors of Travere Therapeutics where he previously was CEO. He is joined on this episode by his daughter, Beth Aselage, Associate Director of Advocacy Relations for Gossamer Bio. Beth speaks with a wisdom that combines analytical insights with an abiding desire to advocate on behalf of patients. She has a unique ability to harvest what is most important to patients and to fuse them into an ongoing development for pharmaceutical companies. Of course the insight that they offer is second-to-none. However, we wanted to bring you the two of them together for an even bigger reason. We wanted you to know about the depth of the legacy that a passion for serving patients can have. When that passion is more than just a saying, patient-first mentalities have the power to change our industry for years to come. Please subscribe and keep listening to Rare Voices! Music Credits: Westpoint Instrumental by  Sun Shapes

We started this show a few months ago. It during the height of the pandemic. While that may seem strange to start something new when health care was getting turned upside down, we felt compelled to deliver something to people who — like us — are committed to creating a path to fulfillment for patients with orphan conditions. So we felt a tug. A call. And, if you have listened to season one, we believe we have delivered. You heard from industry, from patient advocates, from insurance experts, from entrepreneurs. You heard from voices that were likely a lot like yours just how we could make sense of such a volatile world. Not because we are smarter or more virtuous. But, because we have to. For the lives of our patients. And today – I am excited to tell you that we are full steam ahead for another season of Rare Voices. And this year, in addition to the unique perspectives, we are offering you the chance to hear how the brightest minds in health care are looking forward. Where do we go from here now that we have survived the pandemic? What is next for us? We already have a roster of heavy hitters that will provoke your mind and motivate you into action. We have a mother of five and her journey into advocacy after receiving a rare diagnosis for her infant son. We also have a daughter / father team who have decades of experience in reshaping the way that pharma can have a more direct and meaningful impact on the lives of patients. And – of course – so many more voices that will be sure to inspire. I'm honored to host as we move forward and upward out of our crisis and onto a better life for patients. Please subscribe and keep listening to Rare Voices!

For the final episode of our first season, we take a look back at the year that was. Despite our best-laid plans, 2020 had the health care industry on its heels. The pandemic created so much uncertainty and disrupted the familiar pathways to care for patients with rare and orphan conditions. Given the volatility of our past year, what could be our hope for moving forward? To best tackle this question, we moved our host to the interview side of the microphone and pulled in Michelle Hefley, the cofounder of Optime Care and a wise counsel to so many when it comes to rare and orphan patient populations. During this episode, we dive into what brought these two thought leaders together, what the passion is at the core of their partnership, how they have converted that passion into a legacy of improved patient care. Highlights: - The secret to business success for Optime Care? Remaining unapologetically patient-first…no matter what. - How some of the disruptions to health care during the pandemic could have long-term benefits for patients with rare and orphan conditions. - A preview of the rare voices you can expect to hear in Season Two!

After years of tests, after countless of late-night WebMD searches, and a sea of referrals … after hours on the phone with insurance companies and payment plans. After all of that. That is when you FINALLY receive the words: We think your son has a rare condition. And just when you thought you would get answers. That — for patients with rare and orphan conditions — is only where your journey begins. At least that is where the journey began for Monica Weldon, our guest for this episode of Rare Voices. Monica was able to absorb those words and the emotional roller coaster attached to them and create a network of support and a vision for what advocacy can mean for patients with rare and orphan conditions. In 2014, Monica founded Bridge the Gap: SYNGAP Education and Research Foundation. While most patient advocacy groups focus on an illness, Monica focused her foundation on the research involving a rare gene mutation. As a result, she has been able to build a coalition with other advocacy groups, research institutions, and, most impressively, with manufacturers. Today, Monica is a force for change in patient advocacy, an author, and keynote speaker at countless conferences even beyond health care. I'm excited to bring you her story during National Caregivers Month, because, most importantly, Monica has redefined what it means to be a caregiver with what she has accomplished on behalf of her son, Beckett.

Anyone who has invested their time in creating improved outcomes for patients becomes familiar with a kind of balancing act. You must be committed to the science and precision involved in diagnosis and therapy — the research, the development, the trials, the care plan. You must also be devoted to the art of the therapy — the patient's well-being, the expectations, the lives impacted. After all, the patients we care for are … people. And so, anyone who has invested time in creating actual, improved outcomes knows: it's a balance. Our guest on this episode of Rare Voices is an example of what happens when science meets art. He is a master at understanding how to balance what seem like two competing ideas. Dave Penake, CEO of Saol Therapeutics, combines his training as an engineer and systems designer with his passion for serving patients with rare and orphan diseases. Previous to Saol, Dave served as Vice President, Commercial Operations at Corcept Therapeutics and held roles of increasing responsibility at Genentech. In addition to his experience in biotechnology and pharmaceuticals, Dave has designed and launched devices and has experience in a number of different technologies. As you listen to our conversation, be prepared to be provoked by big questions, the kind that will check your balance and set you on a new course — one where you can really put the patient first.

In healthcare, it is easy to become cynical. We are surrounded by the shortcomings of our own industry — it's easy to criticize and point out where we fall short. Everyone… frontline workers, patients, doctors, pharmacists, researchers, administrators… we all have, at some time or another, felt powerless to make things better. This powerlessness is made worse by our pandemic and the complications and uncertainties that surround us. But, once in a while, we meet people who inspire us to pick up our burden and move forward. Rather than preaching from some lofty hilltop, they do this by their example. This episode of Rare Voices features one of those examples. At least, he has been one for me. Our guest today is Bill Jollie, an account executive for a global drug manufacturing company. But, more importantly, Bill is a husband, a foster parent, a cancer survivor, a nonprofit founder, a Marine, and an inspiring human.

Some people are the unique combination of visionary genius and thoughtful character. They connect the head and the heart. Our guest for Rare Voices Episode 3 is one such person, Ken Greathouse. Ken is a veteran commercialization guru and a great friend. Ken has a decades-long point-of-view on developing innovative therapies with a proven track record in commercialization, sales and marketing for pharmaceuticals. When Ken sold Manchester Pharmaceuticals, he created the Greathouse Family Foundation with his wife, three sons, and daughter-in-law, in an effort to fund research for patients with orphan and rare disorders. The phrase, “That's the way we have always done it,” does not apply to Ken. If you are ready to be provoked into a new way of thinking about how we could bring new therapies to market for people who need them the most, you won't be disappointed with this conversation in Episode 3.

One of the least understood components of commercialization is the role of the payer. For manufacturers, it is hard to know how to set the right strategy. For specialty pharmacies, lack of clarity in payer strategy can create challenges with consistent patient experience. For patients, insurance is a black box that they often feel like they have to navigate alone. Our guest for the second episode of Rare Voices, Jim Clement, a partner at Coeus Consulting, demystifies the assumptions that many of us make about payers. Jim has been there. He has seen the good, the bad, and the ugly of commercialization from every vantage point in the system. Simply put: If you want to be successful in bringing new therapies to patients with rare and orphan disorders, then you need to know Jim.

You never get a second chance to introduce your first episode. So, we wanted to start Rare Voices with someone who has granted opportunities for being heard to so many patients, Candace Lerman. Candace, a self-identified disease warrior, is an unconventional advocate for creating a more fulfilling life experience for patients with chronic and rare illnesses. Her work is sprawling — from courtrooms, to policy-maker offices, to living rooms and the virtual world, she has empowered so many, exposed the shortcomings of our healthcare systems, and created new pathways to improved care.

Introducing Rare Voices. The show that reveals the wisest path to a fulfilled life for patients with rare and orphan disorders. Brought to you by the people at Optime Care, a pioneering specialty pharmacy. Hosted by Donovan Quill. Subscribe at www.optimecare.com/rare-voices or anywhere you listen to podcasts.