Stakeholders weigh in on FDA’s draft clinical outcome assessment template

Pharmaceutical industry stakeholders suggested some modifications to the US Food and Drug Administration’s (FDA) recently released draft clinical outcome assessments (COA) template for ensuring that treatments are patient-centric and support effective regulatory decision-making.

The comments were submitted in response to a public workshop hosted by the FDA on 18 September 2025. During this virtual workshop, FDA officials discussed the methodological challenges of collecting patient experience data. They also presented a draft version of an updated evidence dossier template designed to help facilitate the submission of evidence to the FDA in support of a COA.

The template would update the appendix in the agency’s 2009 guidance, Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. The template outlines a structure for submitting information to FDA; use of the COA template is optional.

A COA is a measure that describes or reflects how a patient feels, functions or survives and can provide data on patients’ lived experience of a disease or condition.

Companies also supported FDA's proposal to include a new COA that focuses on the meaningful aspects of health (MAH) when evaluating the benefits of medical products. This proposal was discussed during the workshop and subsequently incorporated into the FDA’s finalized guidance on Patient-Focused Drug Development (PFDD). The guidance outlines strategies for sponsors to select, modify, and evaluate COAs to aid in regulatory decisions and the development of medical products. (RELATED: FDA finalizes guidance on patient-focused drug development, Regulatory Focus 18 November 2025)

The template should complement guidances

At least two commenters, the Biotechnology Innovation Organization (BIO) and Johnson & Johnson requested assurance that the COA template would complement, rather than expand, FDA’s existing four PFDD guidances.

“While BIO supports a consistent structure for evidence submission, some elements of the current draft appear to exceed what is essential for sound regulatory decision-making. Certain evidentiary requests, such as extensive psychometric analyses, duplicative data summaries, site-level documentation or transcripts, may introduce unnecessary burden without proportional regulatory value. Clarifying which components are essential versus supportive would help sponsors focus resources on the most scientifically relevant data to aid in regulatory decision-making,” BIO wrote

Johnson & Johnson wrote that the proposed COA dossier template should be consistent with the FDA’s guidances on PFDD.

“We recommend the Agency ensures alignment of the analyses, terms, and figures presented during the workshop and included in the draft COA dossier template with the final PFDD guidance.”

Improvement over 2009 version

Companies submitted a wide range of comments on the proposed template. IQVIA said that “Overall, we believe that the draft template represents a major step forward” compared to the listing of information on a PRO that was included in FDA’s 2009 guidance on patient-reported outcome measures.

Yet the company suggested that the template provide more information on evidence generated through digital health technologies (DHTs). The group states that “as DHTs are increasingly used to collect COA data (e.g., wearable sensors, mobile apps), clarity on how such evidence should be documented within the dossier is critical for consistency and regulatory review.”

The Pharmaceutical Research and Manufacturers of America (PhRMA) suggested that the COA template be revised to allow sponsors to link efficacy information in their submissions rather than summarize the results in the template.

The group requested that FDA “revise the draft dossier template to recommend that sponsors reference (e.g., via a hyperlink) the location of efficacy results within the broader submission, rather than summarizing them in the dossier. This would help to focus the dossier on compiling the development and validation evidence that underpins the reliability and validity of COA-derived data, rather than duplicating information contained in other parts of a submission.”

Meaningful aspect of health supported

PhRMA also supported FDA’s proposal to add a new concept called the meaningful aspect of health, a concept that was incorporated in FDA’s final guidance on PFDD, but requested more examples of how the MAH would work. This concept was discussed at the workshop and also included in FDA’s final guidance on PFDD.

“PhRMA values FDA’s inclusion of these concepts in PFDD Guidance and we would welcome additional examples to further differentiate MAHs from COIs [concepts of interest] and to illustrate how FDA evaluates MAHs and COIs during regulatory decision-making.”

The finalized guidance adds a new COA – the meaningful aspect of health – to evaluate the benefit of a medical product. The guidance states that “the MAH could be a narrow concept such as nausea intensity or a broader aspect such as lower limb-related function. The MAH and COI may be identical or very similar.”

Comments; COA dossier template

Stakeholders weigh in on FDA’s draft clinical outcome assessment template

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