The Burrill Report


During the first U.S. Congressional hearing on reauthorizing a critical law meant to expedite reviews of human drug applications, biopharmaceutical industry representatives and investors aired concerns about a lack of predictability and certainty at the U.S. Food and Drug Administration, while affirming support for an outline hammered out between the parties in June.

The hearing on the pending reauthorization the Prescription Drug User Fee Act (PDUFA), which establishes industry fees and agency commitments during the drug review process, featured a lineup of testimony in which the FDA defended its track record on timely drug reviews and industry representatives took government policies to task for restraining the biotechnology industry from reaching its full potential in the United States.

“Given certainty, predictability and transparency in the approval process, venture capitalists will continue to fund new research, and companies will continue to develop new and innovative drugs,” said Rep. Joseph R. Pitts, chairman of the House’s Energy and Commerce Subcommittee on Health, in an opening statement.

The current PDUFA authorization expires September 30, 2012. Pitts says he intends to have the reauthorization legislation signed into law by June 30 of next year.

Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, highlighted both her agency’s past accomplishments in expediting drug reviews and plans for the latest revision of the law during the hearing.

As part of her testimony, Woodcock responded in part to claims that the United States approves new drugs less quickly than its foreign counterparts, particularly the European Medicines Agency, citing a recent study by the cancer research think tank and advocacy organization Friends of Cancer Research published in Health Affairs that found the FDA is quicker at approving cancer drugs than its European counterpart [See story].

She also outlined highlights of enhancements for PDUFA V under consideration, including:

• A 6 percent increase in the amount of PDUFA fees paid by industry, adding $40.4 million to the estimated PDUFA user fee revenue during fiscal 2012, as well as provisions for further workload and inflation-related adjustments to fees over time

• A review program for new drug applications, new molecular entities, and original biologics license applications that would increase communication between the agency and sponsors during the application review

• A series of initiatives to improve the development and application of new tools, standards and approaches to assess the safety, effectiveness, quality and performance of FDA- regulated products

• A more transparent approach to making benefit-risk assessments in the agency’s drug regulatory decision-making

• The establishment of two post-market, safety-focused initiatives

• A requirement for standard electronic submissions and standardization of drug review applications

Paul Hastings, president and CEO of OncoMed Pharmaceuticals, testified on behalf of the Biotechnology Industry Organization, saying that “Regulatory uncertainty, longer drug development timelines, and an increasing regulatory and Congressional focus on risk instead of reward in pharmaceutical innovation deters limited partners from investing in biotech venture capital firms and subsequently deters venture capitalists from investing in biotechnology discovery companies.”

Jonathan Leff, a managing director at Warburg Pincus, also expressed concerns about the funding issues facing the industry and the risks if investors are unable to earn returns investing in the development of innovative new therapies. “The U.S. medical innovation ecosystem is in jeopardy,” he said.

Marc Boutin, Executive Vice President and Chief Operating Officer of the National Health Council, testified about the need for better incorporation of patient input into the benefit-risk assessment for new drugs and a reevaluation of conflict of interest policies related to the selection of panelists for FDA advisory committees.

As with any opportunity to impact the shape of PDUFA, a law with profound influence on the FDA’s operations, the parties testifying took full advantage of the opportunity highlight the agency’s shortcomings and areas for improvement. What imprint their arguments have will come clear September 1, when the FDA publishes details of the agreement and recommendations on its website. A public meeting on the recommendations is expected to follow in October. By January 15, 2012, FDA will send its final recommendations to Congress and, if Pitts succeeds, all will be done by July.


July 07, 2011
http://www.burrillreport.com/article-nearing_the_home_stretch.html