FDA finalizes guidance on patient-focused drug development
The US Food and Drug Administration (FDA) has finalized guidance on patient-focused drug development (PFDD), outlining approaches for sponsors to select, modify, and evaluate clinical outcome assessments (COAs) to support regulatory decisions and medical product development.The guidance is the third in a series of four documents that the agency is developing to advance the PFDD field. This guidance responds to a mandate from the 21st Century Cures Act, which requires the inclusion of such patient experience data in clinical research.
“This guidance is intended to help sponsors use high-quality measures of patients’ health in medical product development programs. Ensuring high-quality measurement is important for several reasons: measuring what matters to patients; being clear about what was measured; and appropriately evaluating the effectiveness, tolerability, and safety of medical products. Findings from high-quality measures may help support regulatory decision-making in a variety of contexts,” FDA officials wrote in the guidance.
The finalized version incorporates changes made from the draft that was issued in June 2022. (RELATED: PFDD: Draft guidance seeks to demystify clinical outcomes assessments, Regulatory Focus 30 June 2022).
The guidance offers an overview of COAs in clinical trials. It describes the four types of COAs, outlines their role in evaluating clinical benefits, and specifies what each COA assesses. Additionally, it explains the purpose and context of the COAs and determines whether a COA has enough evidence to support its intended use or is suitable for its purpose.
The four different types of COA measurements include patient-reported outcomes (PROs), observer-reported outcomes (ObsROs), clinician-reported outcomes (ClinROs), and performance outcomes (PerfOs).
FDA discourages the use of proxy-reported outcome measures as COAs. “FDA acknowledges that there are instances when it is impossible to collect valid and reliable self-report data from the patient. In these instances, it is recommended that an ObsRO be used to assess the patient’s observable behavior(s) rather than a proxy-reported measure to report on the patient’s experience,” the guidance notes.
The guidance also features a “roadmap to patient-focused outcome measurement” to help guide sponsors in selecting, modifying or developing a COA.
“The Roadmap describes a recommended path sponsors can take to arrive at a fit-for-purpose COA and, ultimately, an endpoint constructed from scores on the COA. Throughout this path, it would be helpful to communicate with FDA by describing the COA-based endpoint approach.”
The changes from the draft to the final guidance included a clarification of the relationships between relevant terminology; a reformulation of the conceptual framework into a description of the COA-based endpoint approach; and the addition of clarifying language on the importance of qualitative data and patient input, according to a Federal Register notice.
Other changes include updates to the evidence-based rationale, updated references that reflect the state of science, and relocating content that was more relevant to COA-based endpoints to PFDD Guidance 4: “Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making.” In addition, editorial changes were made to improve flow and clarity.
Additionally, a new COA – the meaningful aspect of health – was introduced to evaluate the benefit of a medical product. A new section called “The COA is Administered Appropriately” helps to support the conclusion that a COA is appropriate in a particular context of use.
The section on conceptualizing clinical benefits and risks provides a new example. For instance, when evaluating a treatment for managing moderate to severe endometriosis-associated pain, it is crucial to assess dyspareunia (pain during intercourse) and evaluate how the severity of how moderate to severe endometriosis-associated pain affects daily activities.
Federal Register notice; Final guidance
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