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FDA's Final PRO Guidance Grounds Policy With More Details

This article was originally published in The Pink Sheet Daily

Executive Summary

Issued in December, the final guidance explains "content validity," and advises sponsors to use target product profiles and to document that patient input informed PRO instrument development.

FDA's final guidance on patient-reported outcomes measurements adds more detail and information on practical application to the draft guidance issued several years ago - in keeping with the comments made on the draft.

The final guidance comes almost four years after the draft guidance was published (Also see "Patient-Reported Outcome Claims In Labeling Should Be Specific To Evidence" - Pink Sheet, 13 Feb, 2006.). Comments submitted on the draft guidance sought greater flexibility and detail regarding FDA's expectations (Also see "Patient-Reported Outcomes Guidance Needs Flexibility, Industry Tells FDA" - Pink Sheet, 15 May, 2006.).

Though the final guidance is almost entirely reworded, the concepts in the draft have not been so much scrapped as crystallized - evidence that the field has gained much ground in the interim.

FDA Director of Study Endpoints and Labeling Development Laurie Beth Burke made clear at a recent PRO workshop that this guidance (which was 12 years in the making) will be the agency's final official word on the topic for a while - though the discussion among the measurement experts at the conference made clear that there is still a substantial amount of work to further define and clarify PRO endpoints (Also see "It's Up To Stakeholders To Further The PRO Measurement Field, FDA Said" - Pink Sheet, 9 Nov, 2009.).

A PRO is any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else. One area where the final guidance provides greater specificity than the draft is in how those PRO instruments are created and what data needs to be submitted for validation.

Some Points Of Clarification

In keeping with the idea that a PRO is a report of the patient's health condition that comes directly from the patient, FDA says it will only consider credible those PRO measurement instruments that were developed according to patient input, and states that sponsors should provide documented evidence of that input.

FDA defines a PRO instrument as "a means to capture data plus all the information and documentation that supports its use" - including the concepts being measured, number of items, conceptual framework of the instrument, medical condition, patient population, data collection method, administration mode, etc.

The final guidance states that sponsors should clearly define the role a PRO endpoint is intended to play in the trial (i.e., primary, secondary, or exploratory), and urges companies to use Target Product Profiles (development program summaries with a focus on labeling goals) to specify labeling claims and facilitate communication with FDA about the trial.

Also new to the final guidance is a section devoted to content validity, which refers to evidence that the instrument measures what it is intended to measure.

The new guidance also comes with an expanded glossary - 31 definitions, compared to the original 16 - to define the PRO field. It now includes definitions for terms such as "sign" (as opposed to "symptom"), "recall period" (the time patients are asked to consider in responding to a PRO item or question), and "saturation" (the point when a PRO has been sufficiently informed and reported).

-Jamie Hammon ([email protected])

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