EP 236: Fixing access and design in rare disease drug development: Insights from experts and patient advocates

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 Podcast01:00 Welcome to guests and what Rare Disease Day means to them08:09 Balancing hope with funding, pricing, and access in advanced therapies11:48 Why patient access must be built into drug development from day one14:20 Patient engagement, community readiness, and the realities of trial participation17:49 Why early patient input is still inconsistent and often treated as a checkbox23:20 Designing trials around what actually matters to patients and families26:53 Navigating regulators, payers, and trial design constraints in rare disease therapies36:02 Redefining success in gene therapy around access, scalability, and real patient benefit43:27 Closing remarks