PRO progress

How FDA, industry and patient community can increase number and quality of PROs

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.

Many of the PROs in use today measure symptoms or benefits that were not selected by patients and are not important to them. Even where PROs have been developed with patient input and used in clinical trials to support labeling claims, most companies end up developing their own from scratch, because the PRO itself is not clearly identified on the label.

And homegrown PROs frequently lack the validation needed to inform regulatory decision-making.

Janet Woodcock, director of the Center for Drug Evaluation and Research, kicked off the April 1 workshop by noting that even in cases where homegrown PROs have been robust enough to support labeling claims or to provide supportive evidence of the drug's treatment benefit, the PROs were developed using physician input, not patient input.

"It turns out that what is really bothering the patient and what is really bothering the doctor can be radically different things," said Woodcock. "We have to start understanding the impact of the disease on the people who have it and what they value most as you set up a measurement and go forward with truly patient-focused drug development."

FDA unveiled its plans to create a

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