PURPOSE

:

1. Discuss how patient preference information (PPI) informs benefit-risk assessment at the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER)

2. Explain the methodological lessons learned from three demonstrative studies sponsored by CBER and how to apply them to PPI research studies for regulatory applications.

DESCRIPTION

: CBER has committed to better hearing and capturing patients’ voices and advancing scientific methods to achieve that goal. PPI is a quantitative approach to incorporate patient input in FDA decision-making, including to inform benefit‐risk assessments of biological products.

The FDA has sponsored three PPI studies in biological product areas to develop examples of studies that inform benefit-risk assessment and address key considerations of PPI research for regulatory applications. These studies were designed to inform benefit-risk assessments of products that intend to treat sickle-cell disease, osteoarthrosis of the knee, and hard-to-control type 1 diabetes.

In this workshop, a speaker from the FDA will provide context about how PPI is used in the regulatory decision-making process, why these three conditions were selected, and how regulatory considerations affected the design of the three studies. Next, a researcher from each of the studies will discuss the lessons learned in designing and conducting the studies. These lessons learned include:

• planning for subgroup analysis,
• developing studies for pediatric conditions, leveraging patient-reported outcomes (PROs) to create PPI attributes,
• discriminating between pain and function,
• and including quality of life attributes in PPI studies.

Each speaker will present for 10 minutes. Subsequent discussions with the audience will provide critical insights for those who are interested in collecting PPI to contextualize benefit-risk assessments of biological products for regulatory applications.