Optimizing Clinical Trial Design Through a Patient-Centric Approach

Daniel Lewi, Founder of the CATS Foundation, and Kathleen Flynn, CEO of the National Tay-Sachs & Allied Diseases Association (NTSAD), discuss an industry-advocacy collaboration to optimize clinical trial design through a patient-centric approach.

An estimated 30% of clinical trials fail due to design flaws such as inappropriate endpoints, poorly defined patient populations, and unaddressed patient needs. This is especially a challenge in the rare disease space where patient populations are smaller and more diverse, as well as difficulties defining adequate trial endpoints. This study aimed to collaborate with global advocacy groups in order to optimize clinical trial design.

By gathering data on patient perspectives, the goal was to effectively integrate this feedback into clinical trial design to ensure both unmet patient needs and scientific validity. Relationships were built through focus groups with patients and meetings with advocacy leaders to discuss real world experiences with trial eligibility, endpoints, and duration.

The collaborative approach of this study aided in increasing the transparency and relationship between companies and the patient community, better aligning mutual goals. By considering and integrating patient perspectives, the risk of clinical trial failing can be reduced, therefore improving the chances of successful outcomes.

Chapters:
Introduction 00:00
Tay-Sachs Disease Overview 1:04
Challenges of Treatment 2:26
WORLDSymposium Presentation 3:41
Take Home Message 7:52
Advice for Newly Diagnosed Families 8:41