Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance
guidance describes fda evaluates patientreported outcome pro instruments used effectiveness endpoints clinical trials also describes current thinking sponsors develop use study results measured pro instruments support claims approved product labeling see appendix point 1 address use pro instruments purposes beyond evaluation claims made drug medical product labeling explicitly addressing review issues identified guidance sponsors increase efficiency endpoint discussions fda product development process streamline fdas review pro endpoint adequacy provide optimal information patients perspective treatment benefit time product approval pro measurement aspect patients health status comes directly patient ie without interpretation patients responses physician anyone else clinical trials pro instrument used measure impact intervention one aspects patients health status hereafter referred pro concepts ranging purely symptomatic response headache complex concepts eg ability carry activities daily living extremely complex concepts quality life widely understood multidomain concept physical psychological social components data generated pro instrument provide evidence treatment benefit patient perspective data meaningful however evidence pro instrument effectively measures particular concept studied generally findings measured pro instruments may used support claims approved product labeling claims derived adequate wellcontrolled investigations use pro instruments reliably validly measure specific concepts issue glossary defines many terms used guidance particular term instrument refers actual questions items contained questionnaire interview schedule along additional information documentation supports use items producing pro measure eg interviewer training instructions scoring interpretation manual term conceptual framework refers items grouped according subconcepts domains eg item walking without help may grouped another item walking difficulty within domain ambulation ambulation may grouped concept physical ability fdas guidance documents including guidance establish legally enforceable responsibilities instead guidance documents describe agencys current thinking topic viewed recommendations unless specific regulatory statutory requirements cited use word agency guidance documents means something suggested recommended required first publication draft guidance food drug administrationfebruary 2006