12:00 AM
May 06, 2013
 |  BioCentury  |  Regulation

FDA's starting lineup

Parsing the first set of diseases in FDA's patient-focused initiative

Some of the 16 diseases FDA picked for its first round of public meetings under its Patient-Focused Drug Development initiative are obvious choices where it is not hard to imagine why the agency would need patient feedback. Others are genuine head-scratchers, and at least one surprised its own patient advocates with its inclusion.

FDA declined to discuss its rationale for the specific diseases it chose for the first round of meetings, which will be held over fiscal 2013-15. A second round of four disease-focused meetings is planned for FY16-FY17, bringing the total to 20, and the agency has said it has additional ways of addressing patient concerns in diseases not picked for the meetings.

The first meeting was held last month and focused on chronic fatigue syndrome (CFS) (see Cover Story).

The agency started with a list of 39 proposed diseases. According to a notice published in the Federal Register in April, FDA's criteria for picking the first 16 diseases included the severity and chronic nature of a condition, a lack of approved therapies, uncertainty about clinical endpoints, the total size of the patient population and the number of patient subpopulations affected by the disease.

The extent of public comments submitted to the docket and testimony at a public meeting also informed FDA's selection (see "Patient Focus, Round One," A7).

CFS, irritable bowel syndrome (IBS), fibromyalgia syndrome and female sexual dysfunction appear to be logical choices because they lack a standard set of diagnostic criteria and are characterized by a wide variety of physical and psychological symptoms, as well as uncertainty about their most clinically relevant outcome measures.

Other diseases on the list have been the subject of difficult reviews, which could provide clues about what the agency hopes to learn from patients.

Breast cancer is one example. In its review of Avastin bevacizumab to treat metastatic breast cancer, FDA had to confront the question of how much risk is acceptable for a given increment of progression-free survival (PFS).

Roche's Genentech Inc. unit received accelerated approval of Avastin in 2008 based on a 5.5-month improvement in PFS in a Phase III trial to treat metastatic breast cancer in combination with paclitaxel. However, after confirmatory trials showed...

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