Improving Alignment In Drug Development

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 RareThis 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 landscapeWhat those numbers mean for patients and familiesGaps in diagnosis, access, and treatmentWhy Rare Disease Day remains an important global awareness momentThis 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.