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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.

Chair

David H. Schubert

Speaker

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



Moderators
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... Read More →

Speakers
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 mobi... Read More →
avatar for Christine McSherry

Christine McSherry

Executive Director, The Jett Foundation
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... Read More →
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine is an associate at Hyman, Phelps & McNamara, P.C., a law firm in Washington, D.C. There he assists medical product industry clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Prior to joining the firm, h... 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