The Genetics Podcast
EP 236: Fixing access and design in rare disease drug development: Insights from experts and patient advocates
Published: 23 April 2026 at 12:00 Europe/London
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Episode notes
This week on The Genetics Podcast, Patrick is joined by Lindsey Wahlstrom, Co-Founder and Chief Momatologist of Rona’s FUN LAB, Jimi Olaghere, sickle cell disease patient advocate and early CRISPR gene therapy trial participant, and Rachel Smith, Vice President and Head of Rare and Genetic Diseases at Parexel. They discuss the realities of developing and delivering advanced therapies in rare disease, how funding models, regulation, and trial design shape access and outcomes, and why embedding patient experience early is critical to building therapies that are not only effective but scalable, accessible, and meaningful for patients and families.
Show Notes:
0:00 Intro to The Genetics Podcast
01:00 Welcome to guests and what Rare Disease Day means to them
08:09 Balancing hope with funding, pricing, and access in advanced therapies
11:48 Why patient access must be built into drug development from day one
14:20 Patient engagement, community readiness, and the realities of trial participation
17:49 Why early patient input is still inconsistent and often treated as a checkbox
23:20 Designing trials around what actually matters to patients and families
26:53 Navigating regulators, payers, and trial design constraints in rare disease therapies
36:02 Redefining success in gene therapy around access, scalability, and real patient benefit
43:27 Closing remarks
