The Senate Committee on Health, Education, Labor, and Pensions convened a hearing Thursday, March 29, 2012, to discuss the various user fee agreements up for reauthorization or approval. The hearing featured two panels: the first panel with government officials and the second with members of industry and trade organizations. The hearing called attention to the many problems that would arise if Congress does not reauthorize PDUFA or MDUFA prior to September 2012 when the previous agreements sunset.
Dr. Janet Woodcock, Director, Center for Drug Evaluation and Research, FDA; Dr. Jeffrey Shuren, Director, Center for Devices and Radiological Health, FDA
When both panelists were asked by Chairman Harkin what the effect on patients would be if the agreements were to sunset, Dr. Shuren responded first claiming CDRH could experience significant employee layoffs and resulting in eventual outsourcing of most department functions overseas if agreements are not reached after long periods of time. Dr. Woodcock stated they would have to lay off most of their reviewers and post-market surveillance staff, and a continuous lack of user fees would mean patients in other countries would get new drugs first, setting the agency back decades. Harkin also asked how the new PDUFA agreement enhances orphan drug development. Dr. Woodcock stated the additional staff increased PDUFA funds would encourage development, as would a provision in the new bill to help small or new companies submitting orphan drugs for review. Sen. Mikulski also inquired about the consequences of not passing user fee agreements in a timely manner. Currently, Rep. Pitts has indicated he would like the user fee agreements to be approved and to the President by June 2012. Dr. Shuren said if Congress does not abide by that schedule, reduction in force (RIF) notices would be issued as early as July 2012. He is worried staff will begin to leave for more stable positions, and cautioned that once one person leaves, more are willing to follow.
Sen. Enzi brought up the patient perspective, asking Dr. Woodcock how the new agreement takes into account the risk patients are willing to take when faced with life-threatening diseases. The new PDUFA as a whole, she said, moves toward patient centered drug development by taking steps to understand and measure what risks patients will take and looking at ways to incorporate patient reported outcomes into clinical trials. A pilot of these patient centered programs is happening right now with obesity drugs. Dr. Shuren was asked what he is doing to improve medical device approval as a whole. He said they are starting with some of the most basic things, like implementing a standardized reviewer certification program all reviewers must complete when they are hired so all staff are trained in appropriate procedures. Additionally, they are developing core curriculums for all levels of staff so again, everyone is on the same page and has an appropriate level of information related to their position.
Sen. Bennet brought up the breakthrough therapies bill introduced by himself, Sen. Hatch, and Sen. Burr. He asked Dr. Woodcock to describe the bill in general, and what it would mean to the FDA. Dr. Woodcock responded with a similar description to that of what was talked about in last week’s Friends briefing.
Sen. Roberts, who is an opponent of user fees but understands there is no alternative, questioned the panel about meeting deadlines set forth in PDUFA and what the FDA is doing to deter what he feels is a shift toward a primarily regulatory culture at the FDA that excludes industry input. Dr. Woodcock responded by saying CDER has been addressing those cultural issues over the past 4 years, but will not change their attitude and approach to safety and efficacy. She also stated that the heavy workload at the FDA is a factor preventing FDA from having interactions with members of industry.
Dr. Shuren was also asked several questions about various topics related and unrelated to MDUFA including what specific metrics are being used to measure the success or failure of FDA in meeting its MDUFA goals and the unique device identifier rule. Dr. Shuren said there are 2 pages of the commitment letter dedicated to metrics to measure FDA’s device performance goals which would increase the number of data points ten times what they currently measure to 3 million data points, more than any other country. He added that they also included a metric for total time. Speaking of the unique device identifier rule, Dr. Shuren called it “a game changer” and called on the members of the committee to continue to express its importance and put pressure on OMB, where the rule is stalled awaiting consideration.
Dr. David Wheaton, SVP Scientific and Regulatory Affairs, PhRMA; Sara Radcliffe, Executive Vice President, Health, BIO; David Gaugh, VP for Regulatory Affairs, GPhA; Dr. David Nexon, Senior Executive Vice President, AvaMed; Allan Coukell, Director of Medical Program, The Pew Charitable Trust
Panel 2 opened with all groups expressing their support for the user fee agreements, while voicing the need for continued oversight of FDA. Dr. Wheaton of PhRMA voiced concern that the PDUFA agreement is a bit bloated and any additions to the agreement should be kept to a minimum. Chairman Harkin asked the group if they will help in keeping the FDA responsible for the goals outlined in the user fee proposals, and the groups responded with a resounding yes.
Sen. Bennet then asked the group about the importance of a uniform drug distribution system, pointing out that we know more about a gallon of milk on the shelves of the store than the drugs we take. Sara Radcliffe of BIO said a uniform distribution system is very important, and that they are looking at proposals sufficient enough for all stakeholders to get behind. David Gaugh of GPhA said his group supports the serialization of drugs, but we need to work on a national framework to avoid patchwork regulations that could ultimately do more harm than good.
The group was also asked by Sen. Blumenthal about the development of new antibiotics as well as the FDA’s efforts to curb drug shortages. During panel one, Sen. Blumenthal expressed his view that the FDA response to drug shortages has been “inadequate” and that they could be doing more to alleviate current shortages and prevent future shortages. Mr. Gaugh said he felt there has been a high level of collaboration between FDA and the generic industry where the bulk of shortages occur.