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Integrating the Patient's Voice Across the Development Program of Rare Diseases: At the Table - Where to Sit?
Session Chair(s)
David H. Schubert
DH Schubert Regulatory Solutions LLC, United States
Patient advocacy groups are increasingly recognized as partners in drug development for the various important roles they can play in improving the clinical trial enterprise. With the renewed and heightened commitment to incorporating the patient voice into drug development and regulatory decision making under the 21st Century Cures Act and PDUFA VI, there is a significant opportunity to enhance participation by patient groups in the work of drug developers and the FDA review process. This session will discuss how as a result, there is value in all stakeholders understanding these opportunities and emerging models for incorporating the voice of the patient, from providing drug developers and regulators a unified understanding of the patient experience with the rare disease, to helping ensure that patient-centered information about outcomes in clinical trials are collected, such as through patient-reported outcomes measures.
Learning Objective : Discuss the regulatory advantages of understanding the patient experience with a rare disease, including new opportunities under the 21st Century Cures Act and PDUFA VI; Describe how the patient’s perspective profoundly affects all dimensions of orphan drug development; Identify how engagement with a small patient population can aid in study endpoint selection that reflects outcomes meaningful to patients.
Speaker(s)
The Importance of the Patient Voice and Patient-Industry Partnerships in Rare Disease
Christine McSherry, BSN, RN
Casimir LLC, United States
Chief Executive Officer
The Rare Disease Patient Voice: Not One-Size-Fits-All
James Valentine, JD, MHS
Hyman, Phelps & McNamara, PC, United States
Director
Mobile App for Pulmonary Arterial Hypertension
Wayne Amchin
FDA, United States
Senior Consumer Safety Officer, ORO, DRCHEN, OND, CDER
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