On October 22, 2025, the US Food and Drug Administration (FDA) released a final guidance document on Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments. LUNGevity provided comments when the FDA released the draft guidance on the topic, and we applaud the Agency for finalizing its guidance. We evaluated changes between the draft and final guidance documents and the incorporation of edits reflecting LUNGevity’s comments.
Changes in the Final Guidance Reflecting LUNGevity’s Input
Patient Engagement on Clinical Outcome Assessments (COAs)
In our comments to the Agency, LUNGevity highlighted the importance of consulting and engaging with patients to ensure the relevance of the concept of interest and measurement instrument to the patient. In the final guidance, language was added in multiple sections explicitly regarding the involvement of patients, patient advocacy groups, and/or caregivers in collaboration to identify and develop new COAs.
Timing the Evaluation of New COAs
LUNGevity also requested clarification on the appropriate timing for evaluating new COAs, specifically on the Agency’s use of the term “earlier trials” for evaluation prior to the registrational trial. We suggested the addition of considerations for how evaluation fits into expedited development, dosage optimization, and rare population drug development expectations and timelines. The final guidance featured additional language addressing the use of early-phase trials to evaluate new COAs, although it did not mention considerations for accounting for the additional complexities of expedited development, etc. on the feasibility to evaluate new COAs in early-phase trials.
Mode of Administration of COAs
LUNGevity countered the draft guidance’s statement that the mode of administration would greatly impact the rigor of assessment, noting that flexibility in the use of paper versus electronic adaptation supports patient centricity. We also noted the draft guidance’s brief comments on digital health technologies (DHTs) that needed further clarification to highlight the complexities of their use. The mode of administration section and language around DHTs were removed from the final guidance. Additional language and citations were added supporting the comparability of measurement properties across different modes of administration, reflective of our initial comments.
Additional Notable Changes to the Final Guidance
In addition to changes made in the final guidance reflective of LUNGevity’s comments, there were a few other noteworthy changes reflected in the final guidance.
Use of COA Scores as Supportive Evidence
The draft guidance noted that COA scores could be used to support efficacy in a clinical trial, in addition to effectiveness and safety. However, the final guidance removed efficacy but also added the use of COAs to support dosage optimization and tolerability. This is in line with the Agency’s recent efforts to encourage the use of patient reported outcomes (PROs) and additional PED in dosage optimization.
Meaningful Aspect of Health
While the term “meaningful aspect of health (MAH)” was not used in the draft guidance, this concept was defined and used throughout the final guidance as a way COA scores can be interpreted (as either direct or partial measures of MAH). This concept may be helpful in providing context for the use of COAs but is not ubiquitous in the literature.
Components Comprising an Evidence-Based Rationale
The eight components comprising an evidence-based rationale for proposing a COA as fit-for-purpose were modified significantly. Largely, a new component regarding whether “the COA is administered appropriately” was added. This new section focuses on those administering the COA and resources to ensure proper administration.
Additionally, the component featured in the draft guidance on whether “differences in COA scores can be interpreted and communicated clearly in terms of the expected impact on patients’ experiences” was removed in the final guidance. This component focused on clinically meaningful changes and their justification, which were largely addressed through the MAH-related additions.