The U.S. Food and Drug Administration (FDA) last week announced that it had completed its recommendations for three user fee programs that will help speed safe and effective drugs and lower-cost generic drug and biosimilar biological products to patients:
Prescription Drug User Fee Act (PDUFA)
Generic Drug User Fee Act of 2012 (GDUFA)
Biosimilar User Fee Act of 2012
The recommendations were transmitted to Congress today by Health and Human Services Secretary Kathleen Sebelius. The programs include the fifth authorization of the Prescription Drug User Fee Act (PDUFA), and new user fee programs for human generic drugs and biosimilar biological products. Work on the proposals was concluded before the agency’s mid-January deadline.
Consequently, House Republicans last week announced a slew of hearings on health industry fees aimed at speeding up federal regulators’ review of new medicines before they can go to market. The Energy and Commerce Health Subcommittee will kick things off on Feb. 1 with a hearing on PDUFA. FDA Commissioner Margaret Hamburg is scheduled to testify at the hearing.
On Feb. 7 the panel has scheduled a hearing on a new proposed user fee on generic drugs and biosimilars, which aims to bring in more funds to help alleviate the FDA’s backlog of generic drug applications. Janet Woodcock, director of the FDA Center for Drug Evaluation and Research, is scheduled to testify at the hearing.
And on Feb. 15, the subcommittee has scheduled a hearing on the reauthorization of the Medical Device User Fee Act, which also expires Sept. 30. FDA Center for Devices and Radiological Health Director Jeffrey Shuren is due to testify at the hearing. The medical device fee is shaping up to be a tougher sell. Device makers want a revamp of the expedited approval process for low-risk devices, but patient advocacy groups say the current system is already too lax and allows dangerous products to get to market with little to no oversight.
FDA User Fee Programs
Under a user fee program, industry agrees to pay fees to help fund a portion of the FDA’s drug review activities while the FDA agrees to overall performance goals such as reviewing a certain percentage of applications within a particular time frame.
“These final recommendations offer a great example of what can be achieved when the FDA, industry and other stakeholders work together on the same goal,” Hamburg said. “At a time of greater budgetary constraint, user fees provide a critical way for leveraging appropriated dollars, ensuring that FDA has the resources needed to conduct reviews in a timely fashion.”
Said Hamburg: “Human drug user fees have revolutionized the drug review process in the United States since they were adopted 20 years ago, allowing the FDA to speed the application review process without compromising the agency’s high standards.”
The proposed user fee programs for generic drugs and biosimilars are modeled on the successful PDUFA program “which has ensured a predictable, consistent, and streamlined premarket program for prescription drugs,” Hamburg said. As a result of the continued investment of PDUFA resources, the United States now leads the world in first introduction of novel drugs.
PDUFA was created by Congress in 1992 and must be reauthorized every five years. The current program, known as PDUFA IV, will expire on Sept. 30, 2012, unless reauthorized by Congress. FDA’s recommendations for PDUFA V were developed in consultation both with drug industry representatives and with patient and consumer advocates, and are expected to raise more than $712.8 million in fees annually over five years.
Under the recommendations, fees paid by industry would support continued timely review of critical prescription drugs, as well as advance the development of drugs for rare diseases, provide for enhanced communication with small or emerging companies, increase the use of standardized electronic data to improve quality and efficiency, and foster the use of new clinical endpoints that improve drug development times and help address unmet medical needs.
The proposed new Generic Drug User Fee program would provide the FDA with needed funding at a time when generic drug applications are on the rise. Whereas most new drugs are reviewed in 10 months, the typical review time for a generic drug is 30 months. The FDA has a backlog of more than 2,000 generic drug applications awaiting review, according to the Generic Pharmaceutical Association. Generic drug user fees would help ensure consumers timely access to safe, high-quality and effective generic drugs, which account for two-thirds of all prescriptions dispensed in the U.S.
The FDA proposes collecting $299 million in fees annually to hire additional generic drug reviewers starting in fiscal year 2013. That figure would come from an estimated 750 generic drug applications per year as well as other fees, including the inspection of foreign drug manufacturing sites. In return for these fees, the FDA will aim to review 90 percent of generic drug applications within 10 months.
The FDA receives 800 to 900 new generic-drug-related applications annually. These applications are increasingly complex and frequently involve products manufactured outside of the U.S. In exchange for fees on facilities and product applications, the proposal includes performance metrics such as review timeframes and a commitment to achieve parity between surveillance inspections of foreign and domestic establishments by the 2017 fiscal year. As a result, FDA expects that the proposal would effectively eliminate the review backlog and significantly reduce review times.
A biosimilar is a biological product that is highly similar to a U.S.-licensed reference biological product notwithstanding minor differences in clinically inactive components, and for which there are no clinically meaningful differences between the biological product and the reference product in terms of the safety, purity, and potency of the product. The proposed Biosimilar and Interchangeable Products User Fee program is intended for products approved under a new abbreviated approval pathway for biological products shown to be biosimilar to or interchangeable with an FDA-licensed biological product. The Affordable Care Act of 2010 contains a subtitle called the Biologics Price Competition and Innovation Act (BPCI) of 2009, which established this pathway.
Prior to it becoming law, competition in the biologic drug market was stifled. Enactment of BPCI will spark the development of a new segment of the industry, where companies will be able to develop alternative products. This will help spur innovation, improve consumer choice and drive down costs.
The recommended user fee program for biosimilars includes fees for products in development to generate revenue in the near-term and to provide FDA with the resources needed to support development-phase meetings with sponsors of biosimilar biological product candidates
The US regulator noted that it annually receives from 800 to 900 “increasingly complex” generic-drug-related applications that “frequently involve products manufactured outside of the US.” According to the Generic Pharmaceutical Association, the review period for a generic drug typically takes about 30 months and the FDA has a backlog of more than 2000 generic drug applications. The proposal includes “performance metrics, including review timeframes and a commitment to achieve parity between surveillance inspections of foreign and domestic establishments by the 2017 fiscal year,” the agency said, adding that as a result, it expects to “effectively eliminate the review backlog and significantly reduce review times.”