FDA Tuesday published the first of four planned guidance documents that aim to move patient-focused drug development from listening sessions to a set of scientifically robust methods that can be integrated into product development.
The guidance documents build on “learnings from the disease-specific PFDD meetings that FDA conducted under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) as an enhancement of the Agency’s implementation of a more structured approach to benefit-risk assessment,” according to a Federal Register notice. The agency added that the meetings “have given FDA a deeper appreciation for the expertise that patients and caregivers can bring to the process and the value of incorporating their voice.”
The four guidance documents will help patients and advocates, manufacturers and FDA develop methods to “bridge from important early-stage efforts to gain patients’ narrative perspectives on the clinical context (e.g., meetings with patients), to development and use of methodologically-sound data collection tools in clinical trials,” FDA stated. They “will also address Agency expectations regarding what sort of analyses might be conducted as part of this work and what sort of documents might be produced, and when appropriate, submitted to FDA.”
The first draft guidance document covers collecting comprehensive and representative input from patients. It describes sampling methods for collecting patient input, and provides guidance on topics such as defining target populations and sampling strategies.
The second document “will discuss methods for eliciting information from individuals identified” in the first guidance, as well as methods for collecting information about the aspects of their disease that are important to patients. It will discuss "best practices in how to do qualitative research including conducting interviews, development of interview guides, selection of types of survey questions, and considerations for collecting demographics and survey information.” One of its goals will be to “help avoid misleading results such as inadvertently priming patients in ways that can lead to results that poorly represent what is important to patients.”
The third guidance document will provide advice on “refining the list of important impacts and concepts from patients to develop potential study instruments,” FDA said.
The final guidance in the set will cover the development of appropriate patient-focused clinical trial endpoints.
FDA did not provide a timeline for publication of the next three guidance documents.