FDA Policy/General

Comments on Fit-For-Purpose Draft Guidance

Five stakeholders have raised concerns with FDA about the agency’s draft guidance, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. Pfizer says areas for which further clarification would be beneficial are (1) defining new concepts and terms and providing additional context and examples to assist in implementation and (2) furthering early FDA-sponsor interactions with meaningful outcomes.

In its comment, the Biotechnology Innovation Organization raises areas that it says need more explanation, examples, and references: (1) enhanced validity framework and concepts/terminology; (2) FDA-sponsor interactions on clinical outcome assessment (COA) development and implementation; (3) COAs in registration trials; (4) communicating COA data; (5) advancing digital health technology tools and clinical outcomes assessments; and (6) leveraging existing COA instruments.

Janssen says the applicability of the guidance to all COAs, including patient-reported outcomes, observer-reported outcomes, clinician-reported outcomes, and performance-based outcomes “is welcome and explicitly conveys that the methodological standards are relevant across all COAs and not just patient-reported outcomes.”

In its letter, AdvaMed says it has a number of concerns about the draft and its applicability to medical devices and thus recommends that the guidance not apply to devices. “It is unclear why the third guidance in a series of guidance documents required by statute for drugs applies to medical devices,” it says. “We are also concerned that the recommendations provided in this draft guidance are inconsistent with the least burdensome provisions of the Federal Food, Drug, and Cosmetic Act for medical devices.”

Finally, Genentech says it supports the draft’s recognition that some health concepts are complex and that more than one type of COA may be used to assess aspects of a concept of interest. “However,” it continues, “we found the draft guidance to be inconsistent on this point…. We believe that strategies whereby different measurement types are paired or combined will be increasingly important as sponsors aim to address unmet measurement needs and gain a comprehensive understanding of the patient experience with disease and treatment. Therefore, clarity on this topic is critical.”

Points Genentech raises for further clarification are (1) use and communication of COA data; (2) FDA-sponsor interactions on the development and implementation of COAs; (3) leveraging existing COAs; and (4) use of digital health technology tools.

All comment letters also provided specific line-by-line comments.

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