The Biotechnology Industry Organization is seeking to use PDUFA reform to write a requirement into FDA's legislative mandate for more binding consultations between BLA sponsors and FDA staff before the actual application review begins.

BIO wants to ensure that agreements reached between sponsors and reviewers during end-of-Phase II conferences do not change by the time the final review occurs.

The BIO board approved a position on binding end-of-Phase II conferences on Feb. 19 as part of its legislative wish list for the ongoing discussions of the reauthorization of the Prescription Drug User Fee Act (PDUFA III).

BIO believes that required meetings with binding agreements prior to the start of key Phase III trials could add predictability to the application process.

BIO also believes that the rules for the end-of-Phase II meetings should permit independent consultants to sit in on protocol deliberations. BIO would also have the meetings occur at the sponsor's discretion.

BIO will circulate its PDUFA proposal on Capitol Hill in the coming weeks and also intends to discuss it with FDA. The draft from the board has to go to an association steering committee before becoming officially public. Genzyme CEO Henri Termeer chairs the PDUFA steering committee.

Repeating a longtime complaint by application sponsors about the review process, BIO believes that the agency review staff often changes its view of clinical trial programs after the programs have begun.

In its budget request for 2003, FDA has proposed an increase in user fees for prescription drugs of 61% over 2002, taking the overall figure to $272 mil. The agency's initial target for completing negotiations with industry on user fees is May (¹ [A#00640060005], p. 7).

BIO estimates that, according to FDA's request, the increased user fees would more than double the cost of BLA applications for biotech companies. The current amount per BLA is approximately $330,000; the proposed fee could climb to $700,000 per application.

BIO objects to the size of the increase and feels that FDA has not adequately justified an application fee of that amount.

BIO also expects updated, clear performance goals and measures from FDA in upcoming PDUFA legislation. BIO is suggesting, for example, regular checkpoints during the five-year user fee legislative cycles.

Industry should meet with FDA on either a semi-annual or annual basis to evaluate the agency's performance, BIO said.

In addition, BIO wants user fees to be applied towards improving FDA's ability to evaluate newer technologies.

The organization identified two areas where user fees should be used to bolster FDA review skills: gene therapy and combination therapies, such as treatments involving a medical device and a biologic.

Another issue that the BIO steering committee will review is FDA's request to use user fee resources to improve risk management efforts.

The Pharmaceutical Research & Manufacturers of America appears to be open to discussion of postmarketing surveillance fees as part of PDUFA III. However, the association wants to make a clear distinction between approval-related functions and FDA's enforcement activities.

User fees are not appropriate as a funding source for enforcement actions, according to PhRMA's position (² Also see "PhRMA Sees Path To Rx Benefit In 2002; Wants “Clean” PDUFA Renewal" - Pink Sheet, 28 January, 2002., p. 26).

BIO supports retaining the 50% threshold for user fees as a proportion of total FDA funding. Under the original user fee law enacted in 1992, fees are not to exceed 50% of FDA's total budget for new drug review operations.

BIO noted that as user fees become more important as a funding source, they can undermine the credibility of the system. The association cites concerns by consumer advocacy groups on the independence of a user fee-supported review process as an argument for upper limits on the contribution by user fees.

BIO Wants More Binding FDA/Sponsor Meetings As Part Of PDUFA III

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