On Thursday, February 9, 2012, the House Energy and Commerce Subcommittee on Health held the second in a series of hearings regarding the reauthorization of the Prescription Drug User Fee Act, the Medical Device User Fee Act, and the creation of the Generic User Fee and Biosimilar User Fee Acts. The two-panel hearing also covered ongoing issues with drug shortages.
The Generic Drug User Fee Act (GDUFA) would provide approximately $1.5 billion over 5 years to address a backlog of approximately 2,000 generic drug applications and provide timelines and predictability for generic drugs. The FDA would aim to clear the backlog by the end of the first 5 years of the program and level the playing field. After the backlog is cleared, the goal would be to get a response to applicants in 10 months. The Biosimilar User Fee Act (BSUFA) allocates $20 million, $1.8 million through appropriated funds, with the rest of the funds coming through CDER. The agreement is structured similarly to PDUFA in terms o timelines and processes, but also includes a piece on development that would allow a developer to pay a fee and meet with the FDA throughout the development process. It was also announced during the hearing the FDA was releasing 3 guidances on biosimilar pathways the same day.
Dr. Janet Woodcock, FDA
While the user fee agreements were the first components of the hearing, much of the two panels were focused on supply chain safety, domestic versus foreign inspections, and drug shortage issues. These issues are often intertwined or related to generic drug production and manufacturing practices. When asked by Rep. Pallone whether creating parity between domestic and international inspection times would alleviate concerns about drug safety issues, Dr. Woodcock stated that she prefers a risk-based approach that would reward those domestic and international plants that practice good manufacturing practices with more trust. Risk-based surveillance inspections are included as part of GDUFA, but they are also working to achieve parity between inspection times.
During her opening testimony, Dr. Woodcock mentioned that a “perfect storm” had exploded the drug shortage issue. Rep. Latta asked what that perfect storm was – essentially a massive increase in demand on the system for older and newer generic drugs while manufacturers were consolidating aging facilities – and whether anything in GDUFA would help alleviate the shortage issues. Dr. Woodcock stated predictable inspections and a good inspection force will help prevent quality issues from spiraling out of control.
Several Representatives also had questions about the gray market, namely, how those on the gray market are getting their supply and what can be done to deter people from entering the gray market. In response to an anecdote from Rep. Capps about a facility in her district, Dr. Woodcock stated the FDA is now collecting information from those contacted by gray market vendors and forwarding that information to the Department of Justice. Rep. Cassidy asked whether it is possible to predict whether a drug will emerge on the gray market or not. Dr. Woodcock stated the supply volume on the gray market is actually quite small; and they cannot predict which drugs will be in a shortage at any given time. When asked whether an early notification system for possible shortages would cause hording, Dr. Woodcock felt that it is possible, however, the intent of the early notification system would be to prevent shortages in the first place, and the FDA would only go public if a shortage is imminent or in effect.
At the end of the hearing, Rep. Engle asked how cuts to appropriations impact user fees. Dr. Woodcock explained to the Committee that user fees and the programs they fund are established assuming a certain level of appropriated funding. If they do not receive that base level of funding, the programs established under the user fees are compromised, not nearly as effective, and would have an overall negative impact on the agency as a whole.
Heather Bresch, president, Mylan Inc.; David Gaugh, vice president for regulatory sciences, Generic Pharmaceutical Association; Bill Greene, chief pharmaceutical officer, Pharmaceutical Services, and member, Pharmaceutical Sciences, St. Jude Children’s Research Hospital
Panel 2 faced many of the same questions as panel 1, starting with how GDUFA will increase efficiency and predictability. Ms. Bresch stated the funding is critical to address the needs of the agency in a globalizing environment.
When the topic of risk-based inspection of overseas and domestic production plants was brought up, the panel generally agreed that while it was a good idea, it is also very important to establish a level of consistent screening to ensure those facilities are continuing with good manufacturing practices and have not slipped. Ms. Bresch stated in response to a question by Rep. Dingell regarding supply chain safety that the base laws they are working with were established in 1938 and cannot handle the complexities that come along with a global market.
Rep. Shimkus was perplexed by the drug shortage issue and asked why suppliers were not rising to meet an obvious demand. Mr. Gaugh responded by saying it is likely because economics is not necessarily the primary driving factor behind shortages, that production and facility issues are. He noted it takes approximately 7 years to expand a production line capable of producing the drugs in shortage. Asked by Rep. Engle how St. Jude’s handles dug shortages, Dr. Greene responded with simple administrative processes to identify whether they are getting orders or not. Earlier in the hearing, Dr. Greene noted that many physicians only realize a drug is in shortage when they are shorted or do not receive drug shipments at all.