Patient-Centered Clinical Trial Design: Using Patient Experience Research to Improve the Patient-Centeredness of Patient-Reported Outcomes Measures and Patient Preference Research to Prioritize Patient-Centered Endpoints
Moderator
Christine Poulos, PhD, RTI- Health Solutions, Research Triangle Park, NC, United States
Speakers
Holly Peay, MS, PhD, Faegre Drinker Biddle & Reath, Washington, DC, United States; Jason Roberts, PhD, US Food and Drug Administration, Silver Spring, MD, United States
Purpose: Discuss challenges and considerations in using patient experience and patient preference research to:
1. assess the patient-centeredness of patient reported outcome measures (PROMs) used in clinical trials and
2. understand how patients prioritize among meaningful aspects of health (MAH).
Description: The FDA’s patient focused drug development (PFDD) program encourages use of patient reported outcomes (PRO) and patient preference information (PPI) throughout the product development lifecycle. The identification of MAH can inform endpoint selection in patient-centered clinical trial design.
In an FDA-sponsored study, we assessed symptoms and function and PROMs used for genitourinary syndrome of menopause (GSM). Qualitative interviews followed by a best-worst scaling (BWS) survey were conducted to examine MAH in GSM. The findings describe patient priorities among MAH, and can inform selection of patient-centered trial endpoints and future adaptations of existing PROMs.
The GSM study will highlight challenges and lessons learned. The session will start with an overview of, and practical considerations for, use of patient experience evidence in endpoint selection (Poulos, 8 minutes). Dr. Peay will describe qualitative research to identify gaps in PROs used for GSM and identify ways to improve patient-centeredness (14 minutes). Dr. Poulos will describe the application of Case 1 BWS to prioritize MAH and the conceptual and methodological challenges in this application of PPI (14 minutes). Dr. Roberts will describe the FDA’s perspective on the value of this type of patient-centered research and how it may be used in FDA decision making and advice (14 minutes). Speakers will attend to challenges including a common but heterogeneous syndrome, underinformed patients, poorly defined symptoms and symptom impacts, and lack of specific PROMs. Interactive polling will be used to engage participants.
The session will conclude with an interactive discussion on pragmatic approaches to ensuring that the potential for patient-centered clinical trial design is realized (10 minutes).
Topic
Methodological & Statistical Research, Patient-Centered Research, Study Approaches