The US Food and Drug Administration (FDA) has published its fourth and final guidance in a series of patient-focused drug development (PFDD) guidances meant to help sponsors collect and incorporate patient experience information that can factor into regulatory decision making. The latest guidance focuses on how clinical outcomes assessments (COA) can be used as endpoints to support a product.
In line with recent legislation, FDA has published several guidances covering various aspects of patient-focused drug development, from collecting patient input to choosing and developing “fit-for-purpose” COAs. The latest guidance describes how stakeholders, including healthcare providers, researchers and sponsors can select and construct COA-based endpoints for use in clinical trials, as well as recommendations for evaluating the meaningfulness of results of COA-based endpoints and other study design considerations.
FDA said sponsors should talk to its staff early about the type of patient experience data they plan on collecting related to the burden of disease and the benefits, burdens and harms of treatment. The agency also said sponsors should talk to stakeholders, including patients and subject matter experts when designing their trials to understand the burden of the disease and the benefits and risks of a treatment…
