12:00 AM
 | 
Apr 13, 2015
 |  BioCentury  |  Regulation

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 compendium of PROs at the workshop, which was held as part of FDA's mandate under PDUFA V to incorporate patient perspectives into the review process and was the third in a series of meetings the agency has held or participated in over the past year to discuss PROs.

The compendium would include PROs that have been used to support label claims, or that have gone through FDA's new PRO qualification process.

Attendees welcomed the idea of a compendium, but noted it would not remove two roadblocks to increasing the number of patient-centric PROs: a lack of capacity on the part of patient groups to help develop PROs for every company in a given disease space, and legal uncertainty about the level of interaction companies and patients are allowed to have about an unapproved product.

A new consortium focused exclusively on integrating patients into the drug development process that is expected to be announced this month could address the capacity issue by pooling company requests and spreading the load across participating patient groups. It could also sidestep some of the uncertainties about interaction between companies and patients by focusing the discussion on specific symptoms or a disease area rather than a specific drug development program.

Attendees also asked for guidance from FDA on discussing PROs with patient groups. FDA didn't comment on whether it would issue guidance on the topic.

PRO-blems

PROs are measures that are reported directly by patients in clinical trials to communicate how a drug candidate is affecting either a specific symptom like pain, or overall quality of life.

PROs can be complementary to endpoints that directly assess effects on biological and chemical disease processes by allowing companies and patients to assess the effects of a therapy on how a patient feels, functions or performs - elements of the disease which are often difficult or impossible to ascertain...

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