Manufacturers and FDA Gear Up for User-Fee Action - PDUFA renewal legislation sets stage for new policies affecting revenue, research, and oversight. - BioPharm International


Manufacturers and FDA Gear Up for User-Fee Action
PDUFA renewal legislation sets stage for new policies affecting revenue, research, and oversight.

BioPharm International
Volume 24, Issue 9, pp. 20-24


FDA laid out its goals for user-fee renewal legislation at House and Senate hearings in July 2011. FDA Commissioner Margaret Hamburg outlined for the Senate Health, Education, Labor, and Pensions (HELP) Committee how user fees support patient access to new drugs and medical products. Panel leaders expressed strong support for renewing user fees for drugs and devices, hopefully by next spring. But Sen. Richard Burr (R-NC) threatened to block PDUFA reauthorization if FDA doesn't change its approval process for medical devices. Hamburg acknowledged that devices raise problems, but blamed approval delays on poor quality applications that require reviewers to ask manufacturers for additional data not found in the original filing.

Woodcock provided the House Energy and Commerce Health subcommittee with a fairly positive report on FDA's drug approval process, describing how proposed PDUFA V enhancements will further address drug access and safety issues. Most Democrats and Republicans praised the fee program, as did industry and patient representatives.

Energy and Commerce Committee Chairman Fred Upton (R-MI) laid out top Republican concerns: REMS requirements may be thwarting new drug development, and "rigid and unrealistic" conflict-of-interest policies keep top scientists off advisory committees, thus preventing these panels from vetting new drug candidates in a timely manner. Marc Boutin, executive vice-president of the National Health Council, backed conflict-of-interest policy changes, noting that the rules have created "alarming" committee vacancy rates.

Woodcock credited the 20-year-old user-fee program with establishing a more efficient, predictable drug approval process that has sped up the review process for new drugs and biologics "without compromising the agency's high standards for demonstration of safety, efficacy, and quality of new drugs." FDA has provided patients with access to more than 1500 new drugs during the past two decades, she commented, including important cancer therapies that a recent study shows are available to US patients before reaching the market in Europe.

Woodcock also reported that FDA is on track to approve more new medicines in 2011 than in recent years, with 20 new drugs already okayed at the mid-year point; the number rose to 21—the 2010 total—soon after when FDA added AstraZeneca's Brilinta to the list. Woodcock also noted that CDER's rate of first-cycle approvals for priority new molecular entities (NMEs) is going up, citing recent action on landmark treatments for hepatitis C and for late-stage melanoma. And she noted how the REMS program can facilitate access to high-risk products, such as a new thyroid cancer treatment that FDA approved with a REMS to limit product use to truly needy patients.

The real problem, she said, is not slow approvals, but that too many experimental drugs continue to fail during development. PDUFA support for more FDA–sponsor meetings will help, along with added resources to develop further guidance and new methods for testing and evaluating experimental drugs.

Many of these undertakings are included in a CDER report identifying science and research needs that can enhance the development of new drugs and biologics. This July 2011 publication from CDER's Science Prioritization and Review Committee builds on the Critical Path Opportunities reports of 2004 and 2006 in citing research projects that FDA would like to pursue, ideally in partnership with industry, academia, or other government agencies (3). The topics include ways to improve analysis of postmarket data, product-quality assessment, clinical trial design, and individualized treatments.

Jill Wechsler is BioPharm International's Washington editor, Chevy Chase, MD, 301.656.4634,


1. K. Frazier, Wall Street Jrnl., July 13, 2011.

2. J. McWillians et al., JAMA 306 (4), 402–409 (2011).

3. CDER Science Prioritization andReview Committee, "Identifying CDER's Science and Research Needs Report," (FDA, Rockville, MD, July 2011). ?

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