REMS improvements proposed for PDUFA V in 2012

Stakeholders at FDA meeting express appreciation that the agency listened to their input.

The Prescription Drug User Fee Act (PDUFA) is up for reauthorization in September 2012. Originally enacted in 1992 to provide authority for FDA to collect user fees from industry to help fund the agency’s timely review of new drugs and biologics (in addition to congressional appropriations), PDUFA must be reauthorized by Congress every 5 years.

Most recently, under the Food and Drug Administration Amendments Act (FDAAA) of 2007, Congress reauthorized PDUFA IV. PDUFA IV includes authority for FDA to require manufacturers to develop and comply with Risk Evaluation and Mitigation Strategies (REMS) to mitigate safety issues for certain drugs through components including a medication guide and elements to assure safe use.

Now the PDUFA V reauthorization process is under way. In January 2012, FDA will submit recommendations for PDUFA reauthorization to Congress, kicking off congressional activity leading up to the September 2012 deadline. In an example of the Capitol Hill metaphor of a “Christmas-tree effect,” PDUFA V will serve as an FDA legislative vehicle onto which other FDA-related items may be tacked on like ornaments on a Christmas tree. 

As part of the PDUFA V process, FDA held a public meeting October 24 to discuss the agency’s proposed recommendations based on its agreements with manufacturers, including an increase in annual user fee collections to $693.1 million in fiscal year (FY) 2013 from the current baseline of $652.7 million for FY 2012. (Regarding the Christmas-tree effect, FDA and industry said at the meeting that they hope to see a clean bill move through Congress.)

The meeting’s big takeaway was that participants expressed strong appreciation for the efforts of FDA to gather input from provider, patient, and consumer stakeholders through a series of nearly monthly meetings, APhA Senior Director of Government Affairs Marcie Bough, PharmD, told Bough spoke on the panel for health professional perspectives, focusing on REMS.

"APhA commends FDA for its repeated efforts to engage stakeholders and the public in the development of the proposed PDUFA reauthorization agreement,"Bough said at the meeting. "We believe that the dialogue between FDA and stakeholders at the same time FDA was meeting with industry has greatly improved the reauthorization process, is reflected in the proposal, and exceeded the minimum statutory requirements for gathering public input."

Bough said at the meeting that APhA supports the following REMS improvements in PDUFA V:

  • Outlined strategy and time frame for FDA to continue to discuss and gather public input on improving REMS programs
  • Improved REMS standardization and use of existing and evolving medical and pharmacy practice technologies and processes
  • Earlier consideration, communication, and discussion of REMS in the drug review process
  • Development of guidance on assessing REMS program effectiveness, impact on patient access, and burden on the health care system
  • Development of guidance on how to apply statutory criteria for when a REMS is required

Noting that “improving REMS is a key priority for APhA,” Bough said that APhA sponsored two stakeholder meetings on REMS that each generated white papers. APhA’s 2011 REMS White Paper summarizes recommendations from the 2010 stakeholder meeting of 34 participants on improving program design and implementation, and builds onAPhA’s 2009 white paper on REMS. Bough also highlighted that APhA’s advocacy efforts on improving REMS programs is reflected in the REMS provisions outlined in the proposal.

In the context of proposed PDUFA V recommendations related to pharmacogenomics, Bough also mentioned that APhA recently released a white paper on how pharmacists can integrate pharmacogenomics into practice using medication therapy management. The white paper is available online at the Journal of the American Pharmacists Association website.

Bough also encouraged FDA to consider how pharmacists can be involved in the Sentinel Initiative to evaluate drug safety issues.

According to a slide from the FDA Perspective presentation of Theresa Mullin, PhD, Director, Office of Planning and Informatics, FDA Center for Drug Evaluation and Research, the input from health professionals included considering written information for patients that’s more effective than current medication guides; making REMS more standardized and establishing metrics to evaluate the success of REMS; assessing the burden placed by REMS on the health care system; and obtaining pharmacist input on REMS design.