PRO progress

FDA hopes a planned compendium of patient-reported outcomes instruments will provide patient groups and industry a jumping-off point to develop new PROs and improve upon existing ones. But attendees at an agency workshop said what's really needed to expedite the development of patient-centric PROs is a public-private consortium focused on specific disease areas or symptoms, along with FDA guidance.

PROs are the most direct way to measure how patients experience the benefit or harm of a therapy. However, FDA has rarely been willing to approve drugs based on PRO endpoints, or even to include PRO data in labels, except for a handful of indications that lack hard endpoints or specific diagnostic tests, such as pain and fatigue. ...