Tuesday, June 20 • 4:00pm - 5:15pm
#281: Integrating the Patient's Voice Across the Development Program of Rare Diseases: At the Table - Where to Sit?

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-546-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

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.


David H. Schubert


The Importance of the Patient Voice and Patient-Industry Partnerships in Rare Disease
Christine McSherry, RN

The Rare Disease Patient Voice: Not One-Size-Fits-All
James E. Valentine, JD, MHS

Mobile App for Pulmonary Arterial Hypertension
Wayne Amchin, MA, MPA, RAC

avatar for David Schubert

David Schubert

Vice President of Regulatory and Quality, Stealth BioTherapeutics
David Schubert is the Vice President of Regulatory and Quality at Stealth BioTherapeutics Inc. Previously, Mr. Schubert held positions at MediVector Inc., Logical Therapeutics Inc., Coley Pharmaceutical Group, Genzyme and Cubist Pharmaceuticals. Mr. Schubert has over 30 years of experience... Read More →

avatar for Wayne Amchin

Wayne Amchin

Senior Consumer Safety Officer, DCRP, ODE I, OND, CDER, FDA
Wayne manages the pulmonary arterial hypertension (PAH) portfolio, which includes many drug-device combination products, along with other therapeutic areas. He is the winner of the FDA’s Innovation Lab 1st Prototype Challenge for Mobile Apps, and he is developing the first mobile... Read More →
avatar for Christine McSherry

Christine McSherry

Executive Director, Jett Foundation
Christine McSherry is the executive director of Jett Foundation, a non-profit dedicated to Duchenne muscular dystrophy, and the co-founder of Casimir Trials, a CRO that designs patient, caregiver, and observer reported outcomes that support regulatory approval and reimbursement. Christine... Read More →
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Tuesday June 20, 2017 4:00pm - 5:15pm
S505ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616