P4A Let's Talk Rare: The Life Science Podcast · Partners4Access

Celebrating 5 Years of P4A: Reflections on Rare Diseases, Cell and Gene Therapies, and Legislation in Europe

·54 min·5 clips

Sophie warns that new EU legislation could force gene therapy launches in all 27 member states within two years.

This special anniversary episode of P4A's Let's Talk Rare features hosts Georgie Wach and Owen Bryant alongside original host Aparna Krishnan and P4A founder Sophie Schmitz. The group reflects on the podcast's five-year journey and its role in the rare disease and cell and gene therapy space. They discuss the show's origins, evolution, and impact on its growing audience of over 35,000 subscribers. The episode revisits the podcast's founding in May 2018, conceived as a platform to give voice to diverse stakeholders including patients, companies, and regulators. Early challenges involved basic logistics like selecting microphones and soundproofing rooms before evolving into a monthly show with external guests. Memorable early episodes included the first external guest, Matthew Harold, and a live recording from an industry conference. A standout moment was an emotional interview with a mother, Danielle, discussing her son's rare disease journey and the burdens of clinical trials. Another favorite was a two-part series on patient empowerment featuring advocates Laurence Willard and Neil Bertelsen. The hosts note the shift from having to recruit guests to now having a roster booked months in advance, with professionals actively seeking to appear on the show. Sophie Schmitz provides an industry outlook, noting that in 2022, 91% of global gene therapy revenue came from a single product, Zolgensma. She predicts the global cell and gene therapy market will grow from an estimated $9 billion in 2023 to $24 billion by 2028. Key future challenges she identifies include adapting payment models for high upfront costs, utilizing real-world evidence, and navigating major European regulatory reforms. These reforms include proposed EU pharmaceutical legislation that could reduce regulatory data protection from ten to eight years and impose a two-year launch conditionality across all member states. The upcoming European joint HTA process, starting in 2025, is also highlighted as a significant future complexity for market access. The conversation underscores that companies must enhance patient engagement, invest in real-world evidence registries, and streamline operations to succeed in this changing landscape. The tone is celebratory, conversational, and reflective, blending personal anecdotes with professional industry analysis. The style is informal and engaging, featuring lighthearted segments like "OB's Quick Fire Round" to balance the serious subject matter. This episode is ideal for long-time listeners, rare disease advocates, and professionals in cell and gene therapy interested in market access trends and the podcast's history. Those seeking a deep dive into a single scientific topic or new clinical data might find the retrospective focus less immediately relevant.

As heard by us

A warm anniversary episode that keeps orphan-drug innovation tied to access.

This anniversary episode from P4A keeps its focus tight: orphan drugs, cell and gene therapy, and the practical question of how those treatments actually reach patients.

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Why you'd press play

A five-year celebration that turns orphan-drug and cell-and-gene-therapy news into plain-English stakes.

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