Discussions Produce Set Of Recommendations For PDUFA Renewal

Washington, D.C. (March 13, 2002)-The Biotechnology Industry Organization (BIO) announced today that discussions with the Food and Drug Administration and the Pharmaceutical Research and Manufacturers of America have produced a set of recommendations for a second renewal of the Prescription Drug User Fee Act (PDUFA).

“The measures described in these recommendations would benefit all concerned-the FDA, the companies developing new therapeutics, and, most importantly, the patients waiting for those new products,” said BIO President Carl B. Feldbaum. “They will provide the FDA, particularly the Center for Biologics Evaluation and Research (CBER), with tools to safely speed review of desperately needed new therapies.

“CBER clearly has been underfunded,” said Feldbaum. “Indeed, the FDA as a whole is underfunded relative to the demands being made upon the agency. We are concerned that congressional appropriations for the agency have been flat in the PDUFA era, even as spending at the National Institutes of Health has more than doubled. We need to have a 21st century regulatory system in place that can handle the wave of new products emerging from the biotechnology pipeline. We urge Congress to step up to the plate, as the regulated industries have. Given the positive impact of new biotech products on human health, this should be considered a societal imperative.”

The FDA’s new performance commitments under recommendations for PDUFA renewal are included in a draft goals letter to Congress.

First passed in 1992 and renewed (with revisions) in 1997, PDUFA is a program under which biotechnology and pharmaceutical companies pay user fees to the FDA for the review of new drug applications and biologics licensing applications (NDAs/BLAs), as well as annual fees for manufacture and sale of products.

“PDUFA helped slash average review times for drugs and biologics by more than half from its inception through 1999,” said Feldbaum, “but in 2000 and 2001 overall review times began to creep back up, and review times for biologics actually began to climb in 1998.” The FDA has cited insufficient resources as a contributing factor in the rise. Fee revenues were lower than expected in 2000 and 2001, while the FDA faced a growing volume of non-fee-generating services, such as the review of investigational new drug applications and fee-exempted NDAs and BLAs.

Under the joint recommendations for a second renewal (PDUFA III), projected fees would rise from approximately $160 million in 2001 to $223 million in 2003, with the amount expected to near $260 million by 2007.

In 2003, some of the funding increase would be directed toward invigorating the review process at CBER, which handles most recombinant biotechnology products (with a few exceptions, such as hormones). CBER review periods have lagged behind those recorded at the Center for Drug Evaluation and Research (CDER).

The FDA, in its draft goals letter, has pledged to develop improved review practices, allow sponsors to request independent consultants to participate in discussions on Phase III trial design, and earmark a percentage of PDUFA funds for improvements to the drug review process.

Review practices. The recommendations include an FDA commitment to developing a “good review practices” guidance that stresses early and ongoing communication with applicants, identification of deficiencies early in the review process, and improvements to the review of applications during the first cycle.

Independent consultants. Product developers will have the option of requesting that an independent consultant be involved in discussions on the design of pivotal Phase III trials. Consultants could be used for products that represent significant advances or have potential to meet unmet medical needs. The FDA would select and screen the consultant and would retain final decision-making authority.

“The involvement of independent scientific and medical consultants would increase the confidence that a protocol is appropriate and will meet relevant approval requirements,” said Feldbaum. “Both sides will benefit from the exposure to outside expertise, particularly when it comes to first-in-class products.”

Performance review. Under the joint recommendations, a portion of the user fees would be available through the FDA commissioner’s office for targeted initiatives to improve the drug review process. “We hope this program will both improve performance across the board and bring CDER and CBER into greater alignment,” said Feldbaum.

“We commend the FDA and all the parties involved up to this point and look forward to working with Congress to renew this historic legislation,” said Feldbaum. “I want to thank, in particular, Henri Termeer, president and CEO of Genzyme, who led BIO’s team as chairman of our PDUFA Committee. I’d also like to thank the more than 20 regulatory and legislative experts from BIO member companies who made enormous contributions to this landmark effort.”

BIO represents more than 1,000 biotechnology companies, academic institutions, state biotechnology centers and related organizations in all 50 U.S. states and 33 other nations. BIO members are involved in the research and development of health-care, agricultural, industrial and environmental biotechnology products. For more information, please visit our Web site, .

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