FDA's continuous marketing application pilot programs may not be worth continuing, the Biotechnology Industry Organization suggested.

"Without compelling data regarding their success, they may not be worth continuing, especially if they have a significant impact on FDA resources," BIO Managing Director for Science & Regulatory Affairs Alison Lawton said at a Nov. 14 public meeting on reauthorization of the Prescription Drug User Fee Act.

Lawton commented that the programs, implemented under PDUFA III, "were worth evaluating on a pilot basis." However, final agency and industry assessments may show that the programs did not achieve the intended goals of better communication and decreased review times.

"Our preliminary assessments appear to indicate that the programs were not used as often as might have been anticipated," Lawton said.

As of Sept. 30, 12 products were involved in the Pilot 1 CMA program and nine products in the Pilot 2 program (¹ Also see "Second Continuous Marketing Application Pilot Program Is 44% Enrolled" - Pink Sheet, 15 March, 2004., p. 9).

The biotech group is steering clear of proposals for significant changes in the user fee program as the public assessment begins for reauthorization of PDUFA in 2007.

BIO suggested that only "modest programmatic changes" may be necessary upon evaluation of current PDUFA programs and most will not require changes in the law.

"The program should be reauthorized in a timely manner and not redesigned with reforms unrelated to PDUFA goals," which BIO sees as reducing drug development time and improving access to new drugs.

The organization downplayed suggestions that greater resources and authority may be needed for FDA to ensure the safety of new and marketed drugs.

"BIO does not agree with suggestions that PDUFA has contributed to a lowering of FDA's safety review standards or reduction in product safety, or that safety has taken a back seat to speed," citing a Tufts University study that showed no evidence of a correlation between length of review and product withdrawal (² Also see "Drug Withdrawals Due To Safety Unrelated To Approval Speed – Tufts Study" - Pink Sheet, 12 September, 2005. p. 8).

Lawton said that BIO will "focus on safety-related areas to determine if and how improvements can be made."

BIO suggested that more efficient evaluation of trade names by FDA could improve drug safety. "Trade name evaluation is an important aspect of safety in that it helps minimize medication errors," Lawton said.

"BIO would like to see some emphasis placed on greater efficiency, consistency and predictability in the process of evaluating trade names," she said.

"We believe trade name review is not conducted in a timely manner and consistent procedures do not seem to be in place for this aspect of application review," Lawton added.

"BIO looks forward to working with FDA to expeditiously improve the evaluation of trade names."

BIO may get some help from the FDA Chief Counsel's Office with regard to trade name review. FDA Chief Counsel Sheldon Bradshaw spoke on the issue at an American Bar Association Washington Healthcare Summit on Oct. 20 in Washington, D.C.

"I admit, for economic reasons, perhaps it would be helpful to let people know a little earlier if their trade name will be approved," Bradshaw said.

Pharmaceutical Research & Manufacturers of America representative and Wyeth Exec VP-Regulatory Affairs & Compliance Bruce Burlington noted that PDUFA has sped up drug reviews during a time when "expectations on the extent of safety data to support an approval has continued to grow."

Burlington suggested FDA continue work initiated in the last round of PDUFA on the usefulness of risk management plans.

"FDA should facilitate the appropriate use of company-submitted risk management plans so that the approval of critical new medicines is not unduly delayed," Burlington said. Prior to joining Wyeth, Burlington held senior positions in the Center for Drug Evaluation & Research and headed FDA's device center.

PhRMA, like BIO, did not suggest substantive changes to the PDUFA program. However, both expressed interest in seeing continued improvements in review consistency and approval times.

BIO's Lawton suggested that, while progress is being made in the area of Good Review Management Principles, "two areas represent opportunities for further examination and possible enhancements - labeling and post-market commitment negotiations."

PhRMA's Burlington noted that FDA's study of first-cycle reviews will shed light on agency actions in the approval process. "A complete analysis of first-cycle review by FDA will provide important lessons to both the agency and pharmaceutical companies about those issues that contributed to NDAs not receiving approval on the first cycle and multi-cycle reviews."

Lawton added that the first-cycle review study will be helpful in understanding the "difference between product approval times between biological products and drug products, among review divisions and between [CDER] and the Center for Biologics Evaluation & Research."

BIO also wants FDA to provide additional details on why "median approval times are not changing, notwithstanding the fact that overall PDUFA spending on application review has continued to increase annually."

The additional information from FDA would help "develop a clearer understanding of whether and how use of PDUFA funds are contributing to this apparent slippage," Lawton said.

BIO and PhRMA both stressed the need for adequate congressional appropriations to fund non-PDUFA related activities, with BIO also suggesting that lack of adequate funding is affecting review activities as well (see ³ Also see "PDUFA IV: FDA Stresses Burden Of Industry Meetings In User Fee Discussions" - Pink Sheet, 21 November, 2005. ).

BIO indicated, however, that it is not interested in seeing an increase in PDUFA overall fee collection, saying that "maintaining level funding will allow the successful continuation of the program."

PhRMA stressed the need for FDA to become more efficient in its reviews and its assessment of these reviews. "There are still efficiencies to be gained from enhancements and refinements of the existing systems, especially the use of the electronic environment," Burlington said.

BIO agreed with the thrust of PhRMA's argument in encouraging "the agency [to] better consolidate and coordinate IT activities related to electronic submissions."

BIO Suggests Re-Evaluation Of Continuous Marketing Application Programs

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