February 10, 2006

Data generated by a patient-reported outcome (PRO) instrument can provide evidence of a treatment benefit from the patient perspective, according to an FDA draft guidance that explains how drug sponsors may use PRO generated data to support Rx labeling claims.

For this data to be meaningful, however, there should be evidence that the PRO instrument effectively measures the particular concept that is studied, said the guidance published in the Federal Register.

"Generally, findings measured by PRO instruments may be used to support claims in approved product labeling if the claims are derived from adequate and well-controlled investigations that use PRO instruments that reliably and validly measure the specific concepts at issue," the FDA added.

A PRO instrument measures the effect of an intervention on one or more aspects of a patient's health status -- which the FDA describes as "concepts" -- including purely symptomatic concepts, such as a headache, to more complex concepts, such as the ability to carry out activities of daily living. PRO instruments also can be used to measure "extremely complex" concepts such as quality of life, which may have physical, psychological and social components.

The detailed FDA guidance describes the agency's current thinking on how sponsors can develop and use study results measured by PRO instruments to support claims in approved product labeling. The guidance also explains how the FDA evaluates such instruments for their usefulness in measuring and characterizing the benefit of medical product treatment. Topics covered in the guidance include creation of the PRO instrument, study designs and data analysis.

The guidance, "Patient-reported Outcome Measures: Use in Medical product Development to Support Labeling Claims," is online at http://www.fda.gov/OHRMS/DOCKETS/98fr/06d-0044-gdl0001.pdf (http://www.fda.gov/OHRMS/DOCKETS/98fr/06d-0044-gdl0001.pdf).