Last week, FDA issued a guidance document for industry finalizing its 2006 draft guidance on the use of patient-reported outcome (PRO) measures and instruments (i.e., a patient questionnaire plus documentation to support its use) in product development and to support labeling claims. FDA explains that PRO instruments are useful in clinical trials and investigations in gathering data for a measure or concept best known by the patient or measured from the patient's perspective. PRO instruments most frequently measure how patients experience disease signs, symptoms and effects on health.

According to the final guidance, companies employing a PRO instrument must be able to demonstrate to FDA that the instrument measures the concept or principle it is intended to measure (i.e., companies must show this through an evaluation of the study population enrolled, clinical trial objectives and design, the PRO instrument's conceptual framework and the instrument's measurement properties). FDA recommends the use of well-established measurement properties before beginning enrollment for confirmatory clinical trials. Moreover, the agency states that sponsors should begin instrument development early in product development and recommends that sponsors engage the appropriate FDA review division in a discussion about the instrument and study protocols before finalization (including labeling goals, conceptual framework and the relationship of PRO endpoints to clinical study endpoints).

FDA states that, for PRO-based claims, companies should provide the agency with the same kind of evidence that is provided for any other labeling claim, and that the final guidance document should include an outline and framework of PRO-based information that companies can provide to FDA for review. PRO-based claims may appear in any section of the product labeling or advertising for prescription drugs or medical devices. This final, detailed guidance document sets a high bar for sponsors in the collection and use of PRO-based data in medical product labeling and advertising.