PDUFA Draft Bill Would Broaden FDA’s Mission, Make Guidance Development More Public
This article was originally published in The Pink Sheet Daily
Executive Summary
FDA would be tasked with protecting public health while promoting economic growth and job creation under a discussion draft from the House authorizing committee. Draft Guidances would be voided if not finalized within a year under the proposal.
A draft of the FDA user fee reauthorization legislation circulating in the House includes several modifications to agency operations that would alter the tone of its interaction with industry.
Under the discussion draft, FDA’s mission statement would be changed to focus on a regulatory system that: advances medical innovation; protects public health while promoting economic growth and job creation; promotes predictability and allowing flexibility; identifies and uses the most innovative and least burdensome tools for achieving regulatory ends; and incorporates a "patient-focused benefit-risk framework that accounts for varying degrees of risk tolerance, including for people living with a life-impacting chronic disease or disability.”
The change in mission would be difficult for current agency leadership to accept. Asked about the concept of adding economic goals to FDA’s objectives during a House hearing in January, FDA Commissioner Margaret Hamburg cautioned, “It is going to be very hard for us to factor into this science-based decision-making the question of how does approving or not approving a product impact jobs and how would approving or not approving a product impact [the] jobs of a competitor.” (Also see "PDUFA V: Can FDA Fight Off Mission Creep This Cycle?" - Pink Sheet, 6 Feb, 2012.)
The draft legislation also sets distinct timeframes FDA must follow to issue guidance documents. For instance, three months before issuing a draft guidance, the agency must issue a federal register notice informing the public of its intent to issue the guidance. FDA must finalize all draft guidance documents within a year after their release, or the draft guidance will become null and void.
The section of the draft legislation that addresses drug shortages is illustrative of the tenor of the bill. While FDA is seeking more powers to require manufacturers to notify the agency of potential shortages, the discussion draft addresses the issue primarily through waiving regulatory requirements in order to allow replacement products to reach market faster (see related story, (Also see "Drug Shortage Provisions In Draft PDUFA Bill Would Expedite Major Manufacturing Changes" - Pink Sheet, 14 Mar, 2012.)).
It is not clear when a formal user fee reauthorization bill will be introduced in the House, or in the Senate.
Rep. Joe Pitts, R-Pa., who chairs the House Energy and Commerce health subcommittee, has said he hopes to mark up user fee reauthorization legislation in April and have a final bill, including device, drug, and other FDA user fee programs, signed into law by the end of June.
Fostering Innovation
The committee draft picks up whole previously introduced House bills to incentivize development of antibiotics for drug resistant microbes and to accelerate approval of therapies that have the potential to address unmet medical needs for serious or life-threatening diseases.
The two topics have been the subject of legislative activity on both sides of Capitol Hill, giving the PDUFA reauthorizing panels a starting point for their deliberations.
Including the Generating Antibiotic Incentives Now Act (H.R. 2182 and S. 1734) in the committee draft leaves intact a list of drug-resistant pathogens that legislators believe should be the target of antibiotic development (Also see "Antibiotics Could Get Double Exclusivity Under House Bill; FDA Makes Its Own Moves" - Pink Sheet, 20 Jun, 2011.).
FDA would prefer having autonomy in the matter. Center for Drug Evaluation and Research Director Janet Woodcock asked the committee’s Health Subcommittee to allow FDA to select the targets for antibiotic development during March 8 hearings (Also see "FDA Antibiotic Legislation Should Focus On Criteria, Not Bacteria, Woodcock Says" - Pink Sheet, 8 Mar, 2012.).
The agency also is looking into an approval pathway that would allow antibiotics to be approved for limited indications based on small clinical development programs (Also see "FDA Exploring Limited-Use Approvals For Antibiotics Based On Small Development Programs" - Pink Sheet, 7 Mar, 2012.). The Infectious Diseases Society of America proposed such a pathway in testimony submitted to the committee for the record.
FDA has just begun to explore that option, so the approach may not be sufficiently defined in time for inclusion in a PDUFA reauthorization bill. PDUFA V must be signed into law before Oct. 1 to avoid a lapse in the user fee program. As a practical matter, if the program is not renewed by the end of July, FDA must give notice to staff whose positions are funded by the user fees and could lose their jobs if the program is not reauthorized on-time.
The section on accelerated drug approval is the Faster Access to Specialized Treatments Act introduced by Reps. Edolphus Towns, D-N.Y., and Cliff Stearns, the chairman of the Energy and Commerce Oversight and Investigations Subcommittee (Also see "Accelerated Approval Legislation Picking Up Speed; ULTRA Becoming FAST" - Pink Sheet, 2 Mar, 2012.).
This enunciates the ability of FDA to grant accelerated approval based on a surrogate endpoint that is reasonably likely to predict clinical benefit or a clinical endpoint that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit.
Among the provisions is a directive for FDA to establish a program to encourage companies and disease communities to develop surrogate and clinical endpoints, including biomarkers, that can be used to help demonstrate the efficacy of a drug for accelerated approval.
The goal is to promote use of accelerated approval beyond HIV and cancer drugs, which are most typically the beneficiaries of the pathway. That goal is shared by FDA, which plans to issue guidance to clarify the full scope of the program and to revisit the standards of evidence for the pathway (Also see "FDA Expanding Accelerated Approval Through Guidance, Collaboration" - Pink Sheet, 12 Mar, 2012.).
Other provisions of the bill will renew pediatric pharmaceuticals legislation, eliminate the law’s Section 712 on conflict-of-interest, and set requirements for electronic submission of applications.