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Integrating the Patient's Voice Across the Development Program of Rare Diseases: Translation Into Meaningful Outcomes
Session Chair(s)
David H. Schubert
United States
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 Objective : 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.
Speaker(s)
The Importance of Meaningful Endpoints for Rare Disease Clinicians and Patients
Lisa Dilworth, MS
Synteract, United States
Vice President, Rare and Orphan Diseases
Developing the PKU-QOL: A Long Journey to Integrate the Patients’ Voice into Phenylketonuria Drug Development Programs
Benoit Arnould, PHD, MSC
Mapi, France
Senior Director, Global, Patient-Centered Outcomes, Patient Centered Sciences
Rare Disease Drug Development: Incorporating Patients' Perspective Through the Use of Patient Exit Interviews
Lili Garrard, PHD
FDA, United States
Mathematical Statistician, Office of Biostatistics, OTS, CDER