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Tuesday, June 20 • 2:00pm - 3:15pm
#255: Integrating the Patient's Voice Across the Development Program of Rare Diseases: Translation Into Meaningful Outcomes

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-542-L04-P; CME 1.25; IACET 1.25; RN 1.25

Patients living with a rare disease have a direct stake in the end-benefit, and as a result should have their perspective included in the process of developing drugs. The development of clinically meaningful “patient focused” outcome measures has advanced substantially in recent decades. However, several challenges remain when designing and implementing meaningful outcome measures, including heterogeneity of outcomes, availability of suitable, reliable and valid measures, recruitment, and selection of appropriate data collection methods. This session will discuss how the inclusion of the patient's knowledge and experience, the Patient’s Voice, throughout the investigational continuum can help address the many drug development challenges, yield better clinically meaningful outcomes, and get therapies to patient's faster.

Learning Objectives

Discuss the various ways of collecting data and various types of patient perspective data and how such data can be used to create patient-centered alignment on regulatory decisions regarding safety and effectiveness; Describe potential benefits and risks of integrating the patient voice throughout clinical development; Discuss examples of how patient engagement at various stages of clinical development has positively impacted clinical trials.


David H. Schubert


The Importance of Meaningful Endpoints for Rare Disease Clinicians and Patients
Lisa Marie Dilworth, MS

Developing the PKU-QOL: A Long Journey to Integrate the Patients’ Voice into Phenylketonuria Drug Development Programs
Benoit Arnould, PhD, MSc

Rare Disease Drug Development: Incorporating Patients' Perspective Through the Use of Patient Exit Interviews
Lili Garrard, PhD

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 Benoit Arnould

Benoit Arnould

Senior Director, Global, Patient-Centered Outcomes, Patient Centered Sciences, Mapi
Benoit Arnould PhD is Senior Director Global, Patient-Centered Outcomes at Mapi, and has conducted studies to develop and validate Patient-reported outcome (PRO) instruments for more than 15 years. His primary interests are Endpoint Strategy and the application of Mixed Methods Research... Read More →
avatar for Lisa Dilworth

Lisa Dilworth

Director of Therapeutic Expertise, Scientific Affairs, Rare Diseases, PRA Health Sciences
Ms. Dilworth is dedicated to Rare Diseases with over 16 years of experience. She consults with clients to develop global therapeutic product strategies. Ms. Dilworth supports clinical operations with regard to patient recruitment, investigator relations, global feasibility, and site... Read More →

Lili Garrard

Mathematical Statistician, Office of Biostatistics, OTS, CDER, FDA
Lili Garrard is a statistical reviewer on the Clinical Outcome Assessment (COA) Statistical Support Team at the Division of Biometrics III, Office of Biostatistics (OB), Office of Translational Sciences (OTS), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration... Read More →

Tuesday June 20, 2017 2:00pm - 3:15pm CDT
S505ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  07: SpecialPops, Session