EP 234: Inside rare disease trial operations: The role of CROs in an evolving landscape with Derek Ansel of Worldwide Clinical Trials
The Genetics PodcastShow Notes
This week on The Genetics Podcast, Patrick is joined by Derek Ansel, Global Vice President and Therapeutic Strategy Lead for Rare Disease and Oncology at Worldwide Clinical Trials. They discuss how contract research organizations design and run rare disease trials, the challenges of selecting endpoints and patient populations, and how emerging approaches like n-of-1 therapies, regulatory flexibility, and AI are reshaping clinical development. Show Notes 0:00 Intro to The Genetics Podcast 00:59 Welcome to Derek 01:38 The role of Contract Research Organizations (CROs) in clinical development 02:41 Why endpoints are the hardest problem in rare disease trial design 05:24 How Derek’s team chooses and operationalizes endpoints in rare disease trials 08:14 Balancing patient selection, signal detection, and trial feasibility in rare disease 11:55 How the FDA’s new guidance for personalized therapies could accelerate rare disease drug development 14:20 Patient engagement, genetic counseling, and decision-making in genetic trials 19:30 Patient and clinician dynamics in genetic subtypes of common disease 23:05 What needs to change to make n-of-1 therapies scalable and accessible 26:12 Where AI is delivering real impact in clinical trials today and where it is heading 31:24 Operational bottlenecks, risk tolerance, and the limits of AI adoption in clinical trials 34:55 Derek’s path from early clinical research roles to genetic counseling and rare disease drug development 38:51 Key areas driving progress in rare disease over the next five years 41:42 Closing remarks Please consider rating and reviewing us on your chosen podcast listening platform!
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