Advertise on podcast: P4A Let's Talk Rare: The Life Science Podcast

One of the most persistent barriers in rare disease drug development isn’t the science — it’s identifying and reaching the small number of patients who could benefit from emerging therapies. Without solving this, even the most promising innovations risk never reaching the people who need them most. In our latest podcast episode, we explore how the industry can better connect patients to clinical trials — and why this is critical to accelerating progress in rare disease. We’re delighted to welcome Dr Jennifer Levin, CEO of Medzown, who shares how her team is working to redefine the healthcare ecosystem by improving patient access to advanced therapies and clinical trials across cancer, rare, and complex diseases. If you’re working in clinical development, patient recruitment, or rare disease, this is a conversation worth your time.

Rare Disease Day Special | Let’s Talk Rare This Rare Disease Day, Let’s Talk Rare releases a special episode focused on the data and real-world context behind rare diseases. The episode features Iro Malekous, consultant at Partners4Access, who discusses: Key statistics shaping the rare disease landscape What those numbers mean for patients and families Gaps in diagnosis, access, and treatment Why Rare Disease Day remains an important global awareness moment This episode takes a clear, evidence-based look at rare diseases while grounding the discussion in lived experience.

26 isn’t just another year in market access — it’s a turning point. In this episode of Let’s Talk Rare, P4A dives into the trends that are about to reshape how rare disease therapies reach patients across Europe — and trust us, the ground is moving. Policy is back in the spotlight. With the EU Pharmaceutical Strategy resurfacing, the big question is:How do we reward innovation and keep medicines affordable? The balance is shifting, and everyone — manufacturers, regulators, patients — will feel it. Meanwhile, AI is no longer hype. It’s influencing research, pricing models, even HTA decisions. Faster insights, smarter systems… but also tough questions about transparency, trust, and who — or what — is really making the call. And patients? They’re not sitting on the sidelines anymore. Patient engagement is becoming central to access decisions, pushing HTA bodies toward more inclusive frameworks, real-world perspectives, and maybe even a rethink of traditional ICER thresholds. Bottom line:2026 is a year of change — and serious opportunity. The winners will be the ones who adapt early. 🎧 Tune in to P4A’s Let’s Talk Rare to get ahead of the trends shaping the future of market access in Europe.

In this episode of Let’s Talk Rare, we looked back at P4A’s recent trip to Glasgow, where our colleagues, Sam Morrison & Iro Malekou were on the ground for ISPOR activities leading up to ISPOR 2025. They weren’t just observing—they were actively contributing, presenting a poster abstract, connecting with experts, and taking the pulse of what’s shaping the HEOR and rare disease landscape. We reflected on their experiences: the conversations that stood out, the insights gained from engaging with stakeholders, and the early signals about where health economics is heading in 2025. From lively discussions in the exhibition hall to the themes emerging in scientific sessions, our colleagues returned with a clear sense of the momentum—and the challenges—facing rare disease access. This episode highlights their key takeaways: what we learned from presenting our work, how the community reacted, and what the atmosphere in Glasgow suggested about the priorities and pressures that will influence ISPOR 2025. Whether you attended, followed from afar, or simply want an insider’s view of what’s happening in HEOR and rare disease, this recap brings you right into the room. 🎧 Tune in now to hear P4A’s colleagues share their insights from Glasgow—the people they met, the conversations that mattered, and the signals shaping ISPOR 2025.

Recorded live from ESMO 2025, Conversations Beyond the Clinic explores the different perspectives of the attendees and stakeholders at the ESMO conference in Berlin. We asked a variety of medical stakeholders including A.I experts, nurses, researchers, patients and more to share their experiences of the conference and the work that brought them to ESMO. Through candid conversations we uncovers how medicine, emotion, and meaning intersect in the world of medicine and how connection can shape an evolving healthcare industry.

In this episode, we tune into the unfiltered voices of patients and caregivers navigating life with rare conditions. Through the power of social listening, we uncover the hidden struggles around mental health—stories often left unsaid in clinics but shared openly online. Joined by Lauren Roberts from Rareminds, we explore what these conversations reveal, why they matter, and how they can reshape support for rare disease communities.

How do we make sure patients receive health information that is clear, trustworthy, and truly helpful? We sit down with Catherine Richards Golini from Karger Publishers to explore the power of collaboration in creating impactful patient resources. From working with healthcare professionals and patient advocates to partnering with researchers and publishers, Catherine shares how involving multiple stakeholders ensures information is not only accurate but also accessible and relevant to those who need it most. We dive into practical strategies, real-world examples, and the lessons learned from bridging perspectives across the healthcare ecosystem. Whether you’re a healthcare provider, patient advocate, or simply curious about health communication, this conversation reveals why collaboration is key to building patient information that empowers.

Join us as we sit down with Paul Romness of OS Therapies to explore an exciting breakthrough in OST-HER2 targeted therapies for osteosarcoma—a rare and aggressive bone cancer. Learn how this innovative approach is offering new hope to patients, advancing precision medicine, and pushing the boundaries of rare disease treatment, not just for humans but for dogs too. Joining the discussion as always is host Owen Bryant, along with George Rack and guest presenter and P4A's MA Oncology expert, Rosa Faria Matthews. Don’t miss this powerful conversation on the frontlines of rare cancer research. Tune in now—because every rare voice matters.  #LetsTalkRare #Osteosarcoma #OSTHER2 #RareDisease #OSTherapies

Discover how artificial intelligence is transforming the future of rare disease research in the latest episode of Let’s Talk Rare, the official podcast from Partners4Access (P4A). Join us as we sit down with Ilya Burkov, AI expert at Nebius to explore how cutting-edge machine learning tools are accelerating the discovery, diagnosis, and development of treatments for rare conditions. With over 300 million people affected by rare diseases worldwide, the need for faster, smarter solutions has never been greater. Ilya sheds light on the real-world applications of AI in identifying new genetic links, optimizing clinical trial data, and personalising care pathways. Whether you're a biotech professional, healthcare provider, or advocate, this episode offers critical insights into how AI is reshaping the rare disease landscape. Tune in now to understand the promises, challenges, and future possibilities of AI in rare disease development—only on Let’s Talk Rare, brought to you by P4A.

In the latest episode of Let's Talk Rare, Owen Bryant & Georgie Rack delve into the world of drug development in rare diseases. They are joined by the brilliant Rob Freishtat, president of Uncommon Cures. Rob brings unique insights on who the key stakeholder are in developing drugs and the many ways that we can bring them together in order to improve and enhance the delivery of much-needed therapies.

Brain cancers are notoriously difficult to manage with current treatments offering limited effectiveness. But what if there was a way to change that? Michael Roberts from Adaptin Bio joins Owen Bryant to explore how a pioneering new treatment called BRiTE is driving hope for improved outcomes for patients.

Dive into P4A's annual appraisal of the year ahead. In this episode of Let's Talk Rare we examine the most important trends as identified and voted for by the P4A team. Ranging from Donald Trump's tariff impact on EU's ability to price orphan drugs to the change of mindset in HTA bodies to include patient experience data in their decision making calculations. Join Owen & Georgie, along with Sophie Schmitz and Pina Haberl for a wide ranging and dynamic discussion on the year ahead.

After3 years in the making, the Joint Clinical Assessment for the EU is in effect. From 12th January 2025 new cancer medicines and advanced therapy medicinal products (ATMPs) will require a JCA across all 27 member states. Join Owen and Georgie, along with guests Darren Callanan and Sam Morrison to discuss the main talking points of the JCA, and what to look out for when dealing with the new regulations.

Welcome to this month's P4A Let’s Talk Rare podcast episode by Partners4Acess. Today, Georgie and Owen are joined by Leonard Mazur, CEO of Citius Pharmaceuticals, to discuss their innovative therapy, Lymphir, for cutaneous T-cell lymphoma (CTCL). Leonard shares his journey with Citius, from its founding in 2013 to the recent resubmission of Lymphirto the FDA, and discusses the drug's potential to alleviate severe itching for the 21,000 annual CTCL patients in the U.S. The conversation also covers Citius's commercialisation plans and ongoing trials exploring Lymphir’s use for other conditions. Join Georgie, Leonard, and co-host Owen for an insightful discussion on how Citius is committed to improving patient quality of life through groundbreaking treatments! Leonard explains how Citius acquired Lymphir in 2021, a drug facing regulatory challenges that they’ve addressed for FDA resubmission, with potential approval expected. They discuss Lymphir’s benefits, especially for CTCL patients, including its potential to relieve severe itching and outline the company's commercialisation plans with Eversana. Leonard emphasises the team’s extensive expertise as vital to navigating the regulatory process. They also explore Lymphir’s off-label potential for other cancers, with ongoing trials at major universities. The conversation closes on the rewarding impact of pharmaceutical work on patient lives, underscoring Citius's commitment to addressing unmet medical needs through innovative treatments. Leonard Mazur Bio: Leonard Mazur is a seasoned entrepreneur and executive with over five decades of experience in the pharmaceutical industry. He is known for his skill in founding and growing multiple healthcare companies. Currently CEO of Citius Pharmaceuticals, Leonard previously co-founded Leonard-Meron Biosciences and Akrimax Pharmaceuticals. He also led Triax Pharmaceuticals as COO, specialising in dermatology, and successfully sold his dermatological company, Genesis Pharmaceuticals, to Pierre Fabre in 2003. His extensive background spans roles in sales, marketing, and business development at Medicis, ICN, Knoll Pharma, and Cooper Labs. Born in Germany, Mazur holds an MBA from Temple University, where he also earned his undergraduate degree. He is an Ellis Island Medal of Honor recipient. Episode Resources: Leonard Mazur on LinkedIn Citius Pharmaceuticals Website Owen Bryant on LinkedIn Georgina Rack on LinkedIn Partners4Access Website  P4A Let’s Talk Rare podcast on Apple Podcasts

In this episode of P4A Let's Talk Rare, hosts Georgie and Owen Bryant from Partners For Access are joined by Wes Michael, founder and president of Rare Patient Voice LLC. Wes shares the organization’s journey from its beginnings as a community project for hemophilia patients to a global platform that connects rare disease patients and caregivers with healthcare professionals. Rare Patient Voice has awarded over $30 million to participants and completed more than 210,000 projects, with Wes highlighting the company’s organic growth through patient referrals and advocacy partnerships. Wes discusses the challenges of reaching patients nationwide, the rewarding impact of patient involvement in healthcare, and the essential role of patient-centricity in shaping drug development and health technology assessments. He emphasizes that patients' voices, as the end-users, are critical for creating effective healthcare solutions. Looking ahead, Wes shares his enthusiasm for expanding Rare Patient Voice into clinical trials and increasing its presence in Europe. The episode concludes with a call to action for patients and caregivers to join the Rare Patient Voice community and share personal stories to further understanding and awareness of rare diseases. Wes Michael Bio: A market research industry veteran of over 40 years, Wes earned his MBA from the University of Chicago Booth School of Business and a BA in English from the University of Pennsylvania. In 2013, Wes launched Rare Patient Voice, based on a novel approach to building a market research panel that enables patients and caregivers to share their voices with researchers and companies developing products to improve patients’ lives. He structured the company on the core values of being a good patient resource, a good client partner, a good employer to staff, and a good citizen in our community. Since then, Wes has seen the company grow, expanding from the US to Canada, the United Kingdom, Spain, Italy, France, and Germany, completing thousands of projects, and providing customized service to hundreds of clients. He hopes that RPV’s work can help the lives of patients and caregivers who have participated in our studies and those who come after. Episode Resources: Wes Michael on LinkedIn Rare Patient Voice LLC Website Owen Bryant on LinkedIn Georgina Rack on LinkedIn Partners4Access Website  P4A Let’s Talk Rare podcast on Apple Podcasts