Expediting Impact: Leveraging the Patient Voice for Enhanced Clinical Trial Design and Accelerated Regulatory Approvals
June 19, 2025
Breakout Session

In recent years, "patient-centric"has emerged as a buzzword in the healthcare world. However, the reality is that our industry has often overlooked the patient voice, which has, at times, impeded the ability to deliver needed therapies. This is particularly evident in the rare disease sector, where patients’ most apparent symptoms may not always be the most distressing, leading to some of the most burdensome symptoms being overlooked in drug development and clinical trial design. As we embark on a new era of drug development and accelerated approvals - exemplified by initiatives like the FDA’s START program - it is crucial to incorporate the patient perspective early in the clinical development process.
In this discussion, panelists will emphasize the significance of involving patients and their caregivers from the outset to refine study designs and shape protocols so that they can more effectively bring a meaningful therapy to market. Speakers will share insights on how establishing patient ambassador programs, conducting patient advisory boards, and collaborating closely with patient advocacy groups have enabled them to identify endpoints that truly reflect patients’ real-world needs.
Moderator
Speakers