EP 234: Inside rare disease trial operations: The role of CROs in an evolving landscape with Derek Ansel of Worldwide Clinical Trials

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!