PURPOSE:

• Understand how patient preference information (PPI) informs benefit-risk assessment at the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER)
• Learn the methodological lessons from three PPI studies sponsored by CBER and how to apply them to PPI studies for regulatory application
• Learn how to report the findings from these studies and discuss their potential impact on regulatory decision-making

DESCRIPTION: CBER is committed to better listening and capturing patients’ voices by advancing the science of measuring patient inputs to inform benefit‐risk assessments. CBER has sponsored three PPI studies to address key considerations of applying PPI research in regulatory contexts. These studies have elicited patients’ quantitative benefit-risk tradeoff preferences for biological products that intend to treat sickle-cell disease (SCD), osteoarthrosis of the knee (KOA), and hard-to-control type 1 diabetes mellitus (T1DM).

A speaker from the FDA will provide context on how PPI is used in the regulatory decision-making process, how regulatory considerations impacted the studies’ designs, and how the results can inform regulatory decision-making. Individual investigators of these studies will present for 10-minutes and share key findings, discuss lessons learned and show patients’ evaluation of the preference-elicitation instruments. The presentation will include:

• Adaptation of a PROM as PPI study attributes in two different ways
• Recruitment of participants by self-reported diagnosis vs. physician confirmed diagnoses and whether these impact patient preferences
• Use of hypothetical baseline severity in patients with mild vs. those with actual severe SCD status
• Impact of patient symptom severity on their tolerance of SCD treatment-related risks
• Preferences and preference heterogeneity for islet cell therapy in patients with hard-to-control T1DM