FDA guidance on patient reported outcomes

The value gained from understanding health outcomes from the patient’s perspective has been acknowledged increasingly in recent years.1 2 National surveys of patient experience are now a feature of NHS regulation,3 and the drugs industry now recognises the use of such outcomes in labelling (efficacy) claims to promote market access.4 5 Release of the long awaited definitive guidance from the US Food and Drug Administration (FDA) on the use of patient reported outcome (PRO) measures to support labelling claims6 marks a coming of age for PRO measurement.

After publication of the draft guidance in 2006,5 there was much speculation about the meaning and implications of the FDA’s stance on the development and use of PRO measures. The draft offered guiding principles to those conducting and supporting industry sponsored clinical trials, and it was largely welcomed as encouraging the adoption of scientific standards. However, several aspects of the draft guidance were seen as contestable (for example, the use of ”minimally important differences”) or lacking in detail (for example, recommendations for use of ”conceptual frameworks” and “endpoint models”). Consequently, the draft guidance generated more questions than answers about the nature of PRO research and the level of evidence needed to demonstrate good scientific practice. Indeed, …