On Wednesday, February 1, 2012, the House Energy and Commerce Subcommittee on Health held a hearing to discuss the upcoming reauthorization of the Prescription Drug User Fee Act, among other things. FDA Commissioner Dr. Margaret Hamburg served as the witness to the first panel. A second panel consisted of Geno Germano, president and general manager for specialty care and oncology, Pfizer Inc.; David Gollaher, president and CEO, CHI-California Healthcare Institute; Richard Pops, chairman and CEO, Alkermes, representing BIO; Allan Coukell, director, Medical Programs, Pew Health Group, Pew Charitable Trusts; Diane Dorman, vice president of public policy, National Organization of Rare Disorders (NORD); David Wheadon, senior vice president of scientific and regulatory affairs, PhRMA; and Daniel Frattarelli, chairman, Committee on Drugs, American Academy of Pediatrics.
Dr. Hamburg pointed out early on in the hearing PDUFA negotiations are moving along ahead of schedule. In addition, she said the enhanced communication during drug development is one of the biggest improvements over the previous PDUFA. The hearing covered a multitude of subjects including use of toxic agents in cosmetics, over the counter inhalers, and antibiotic resistance. However, the most relevant issues addressed were conflict of interest rules and waivers, the benefit of regulatory science moving forward, how PDUFA V expands on pilot programs in PDUFA IV, and FDA oversight of international activities.
During questioning from Rep. Phil Gingrey (GA), the Congressman raised concerns that if conflict of interest rules were not changed, the FDA would be phased out of their advisory role since there would be no one left who qualifies for an advisory committee. Dr. Hamburg responded by stating advisory committees are not the only way to bring in expertise, and cited a partnership with George Washington University regarding obesity drugs and the use of regulatory science to bring the right people in on a regulatory capacity. Later in the hearing on Panel 2, Diane Dorman of NORD would state that conflict of interest rules are can in fact be detrimental to the review of products.
Dr. Hamburg continually brought up the use of regulatory science initiatives as a way to deal with some of the problems the Subcommittee brought up during the hearing. In addition to citing regulatory science as a new way to work through conflict of interest issues, she emphasized its importance in getting new drugs to market. When asked by Rep. Leonard Lance (NJ) about what the FDA is doing to encourage the advancement of personalized medicine and the use of biomarkers, Dr. Hamburg noted that regulatory science will further allow them to adapt to getting new therapies that do not fit the previous model through and to patients. She said in addition to the Biomarkers Consortium, the inclusion of biomarkers in PDUFA V and the enthusiasm shared by the industry shows they are serious about the use of biomarkers.
Rep. Lois Capps (CA) brought up the Sentinel Initiative for post-market surveillance, and asked how the FDA will use pilot programs from PDUFA IV, like Sentinel, moving forward. In response, Dr. Hamburg said they intend on using the data available and analytics to be able to quickly ask or answer questions about safety. There are up to 100 million patient lives in the database, and they are working on strengthening the use of more detailed electronic submissions to deal with data in a more targeted way, i.e., gathering data on patients based on sex or ethnicity.
The FDA’s involvement in the regulation and oversight of foreign activities, particularly clinical trials and inspections, were brought up several times during the hearing. According to Dr. Hamburg, the FDA has little oversight over clinical trials performed overseas, although they are working with regulatory authorities in other countries to bolster their presence. She agreed the agency needs additional resources and authority to provide an appropriate level of oversight on international clinical trials. Rep. Bill Cassidy (LA) brought up the contrast of inspection times between domestic and international production plants. He questioned whether unions are preventing the FDA from properly conducing inspections overseas. Dr. Hamburg stated unions are not central to the difference in inspections, but rather the issues are increased cost and complications of inspecting plants. She also noted the FDA is working to increase their presence globally, and the Generic Drug User Fee Act will go a long way in gathering the resources needed to create parity between domestic and international inspection times.
At the end of the first panel, Rep. Frank Pallone (NJ) asked a question about a proposal to revise the FDA mission to include innovation and job creation as tenets of the agency. Dr. Hamburg responded by saying that the core of the agency is science-based, and it would not be in the best interest of the agency for patients if they would have to include job creation assessments into whether they approve a drug or not. They would then have to think how approving one drug would impact a competitor, and it would be inappropriate to bring that into the debate of a drug that could potentially save patients’ lives.
Panel 2 members agreed PDUFA has been beneficial, and the need to pass a strong piece of legislation is crucial to continuing the benefits seen since the first PDUFA passed in 1992 and reiterated many of the points made about PDUFA’s importance in panel 1. “The reauthorization of PDUFA offers hope that we may build on previous successes by strengthening the review process still further and by creating an environment that encourages innovation and investment,” stated Ms. Dorman in her testimony. While each panelist had a different perspective on why PDUFA is important, such as keeping review times down as many drugs must obtain a patent on a compound sometimes ten years prior to FDA review, they all felt as though the investments made in PDUFA V are critical to bringing new, effective therapies to patients and should be passed quickly.
Panel 2 also focused more on the economics implications of PDUFA, with panelists stating that the guidelines in PDUFA have direct impacts not only on patients and their ability to get the medications they need, but the ability of the biopharma industry to create jobs. The panel discussed a variety of other issues, such as encouraging more pediatric clinical trial testing and opportunities for the FDA to learn from their own best practices. David Gollaher of California Healthcare Institute noted the area of cancer drug approvals have been very successful, and offered up the suggestion of comparing that model to their standard drug approval model to see what works and what does not.