DIA 2020 Virtual Global Annual Meeting

Component Type: Session
Level: Intermediate

Patient preference studies are an increasingly important means of assessing patient perspective on the benefit-risk trade-offs and the importance of properties of medical treatments. Regulator, industry, academic, health technology assessment, and patient –stakeholders are conducting these studies to inform development, regulatory, healthcare and reimbursement decisions across the medical product lifecycle. To enable well-designed studies and informative results, it is important to understand when patient preference studies best inform decision making and the needs and concerns of stakeholders using the results. Additionally, understanding methods of use with clinical trial data enables clear communication and appropriate application of results to inform decision making. This session will present a critical assessment of results from the first phase of PREFER, an Innovative Medicines Initiative (IMI) research project developing recommendations on how and when to use patient preference study for informing medical product development. It will describe three broad classes of approaches to applying patient preference results to clinical trials/data and key trade-offs between these approaches. The session will also demonstrate practical use of patient preferences in regulatory applications at FDA CBER, including roles in osteoarthritis, sickle cell anemia and type I diabetes islet cell transplant.

Learning Objectives

Identify where in the medical product lifecycle (MPLC) patient preference studies can best inform decision making and related stakeholder needs and concerns; Describe different approaches, with related advantages/disadvantages, for applying preference study results for informing MPLC decisions; Describe current and future FDA CBER regulatory applications for preference studies.

Chair

Kristin E Bullok, PhD

Speaker

Critical Assessment of PREFER's First Phase Results: Methods and Challenges
Rosanne Janssens, MSc

Applications of Patent Preferences Studies in Drug Development: How Preference Data Can Inform Decisions
Bennett Levitan, MD, PhD

Capturing Patient Preferences to Inform Regulation of Medical Products
Telba Irony, PhD