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 t...

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...

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 medi...

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 landscap...

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 h...

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 s...

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 multipl...

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...

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 30...

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...

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 & Georgi...

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 regul...

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...

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...

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Aurelija Luko , the CFO, and Darren Callanan , the Global Digital Lead at Partners4Access . Join them as they discuss the recent job cuts in the pharma and biotech industry and the reasons behind them. Aurelia explains that redundancies are not limited to...

Welcome to this month's episode of the P4A Let’s Talk Rare podcast by Partners4Acess. Today, Georgie and Owen are joined by their colleagues Pina Haberl , Senior Director, and Sam Morrison , Executive Director at Partners4Access . Join us as we discuss the crucial role of patient experience data in drug development.  The conversation explores the need for drug developers to understand patient...

Welcome to this month's episode of Let’s Talk Rare: The Life Science Podcast brought to you by Partners4Access. Host Georgie records this episode at the World EPA Conference in Amsterdam. She holds a panel discussion with Juliette Sinclair-Spence , Sandrine Ruiz ,  Neil Grubert , and Seema Sondhi , and together we shared our experiences at the EPA conference.  We also shared our excitement for...

Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines. Together they explore the EU joint HTA legislation coming into force in January 2025, and the importance of patient experience data....

Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bottom of our hearts, we would not have gotten here wi...

In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a friendly and engaging tone, this podcast offers val...

Welcome to this month's episode of the Let’s Talk Rare podcast by Partners4Acess. Georgie and Owen are joined by Iola Forster , Head of Publications and Portfolio at Karger Publishers , to discuss the future of patient engagement and the role of technology in healthcare.  Together, they cover the future of HCPs (healthcare practitioners) and patient engagement, highlighting the importance of t...

Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of proactive preparation for the upcoming changes. ...

In this episode we will be talking to  Alejandro (Alex) Dorenbaum, M.D. , CMO of  Reneo Pharmaceuticals . Alex discusses how the company is developing drugs for patients with  #rare  mitochondrial diseases, a high unmet disease with no current treatments available. Their lead candidate  #Mavodelpar  has recently completed enrolment for their pivotal STRIDE clinical trial. STRIDE is a global, rando...

P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their novel therapy XRX-008 to treat patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). Allen discusses the company vision for developing novel therapies for rare progressive kidney disorders, and tells us about the launch journey for their m...