Guidance 4 is the final guidance in a series of patient-focused drug development (PFDD) guidances meant to help sponsors incorporate patient experience information into drug development.
The US Food and Drug Administration (FDA) has released a draft of Guidance 4: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making in its patient-focused drug development (PFDD) guidance series. These series aim to describe ways to incorporate the patient's voice and experiences into drug development, which can then be used to inform regulatory decisions. The guidance recommends using personalized outcome measures, including Goal Attainment Scaling (GAS), as a means to assess what’s important to each patient. GAS is a highly patient-centric and inherently clinically meaningful outcome that is sensitive to change, even in heterogeneous groups