Prescription Drug User Fee Act Reauthorization
September 17, 2010
Dr. Theresa Mullin
Associate Director for Planning and Business Informatics
WO51 RM1178 HFP-1
10903 New Hampshire
Dear Dr. Mullin,
The FDA should be commended for their outreach to many third-party groups over the past several months. As a result of these meetings and other conversations, the patient, advocacy and professional organizations listed below have identified the attached topics of mutual interest relating to the upcoming reauthorization of the Prescription Drug User Fee Act.
These topics, relating to FDA user fee programs and other essential functions of the FDA, are areas that we feel are important to be part of future conversations and negotiations with industry.
Please do not hesitate to contact us should you need further clarification or input.
Sincerely,
American Association for Cancer Research
Alliance for Aging Research
Children's Cause for Cancer Advocacy
Friends of Cancer Research
Leukemia & Lymphoma Society
Men's Health Network
National Patient Advocate Foundation
Ovarian Cancer National Alliance
Society for Women's Health Research
Susan G. Komen for the Cure
Current User Fee Associated Programs:
Continue stepwise funding increase for drug safety programs.
Additional user fees authorized through the 2007 PDUFA IV program were specifically allocated to drug safety programs. This was done in a stepwise fashion (+$10M annually) on top of calculated annual user fees. Although direct appropriations would be desired, continuing this program could help make up for shortcomings in public funding that has slowed the implementation of some drug safety programs, such as the Sentinel Network.
Assessment of review performance goals should be based on what is needed to meet existing deadlines, not re-establish timeframes to match the current capacity of the agency.
The PDUFA program establishes performance goal review timeframes in return for user fee resources to support the review functions of the agency. In past reauthorizations, the goal timeframes have gotten shorter. While it is acknowledged that the responsibilities of FDA continue to increase, the focus of PDUFA V reauthorization should be on addressing the needs of the FDA to meet existing performance goals.
Publicize materials that detail additional information beyond if deadlines were met or not, but also include timeframes by which they were met or missed.
Several analyses have shown that the majority of action surrounding the review of a new application occurs immediately around the target PDUFA date. This has been raised as a point of concern and caused some to conclude that final elements of the review process are handled in a last minute fashion. Many performance metrics are set up with the goal of meeting with 90% of the time. Additional information could help alleviate concerns, since there is currently no easy way to know if goals were met (or missed) by 1 week or 10 weeks.
Require electronic submission for initial and supplemental NDAs/BLAs whenever possible.
While FDA has concluded that electronic submission has no significant impact on time of review, requiring (or at least encouraging) all electronic submission may provide benefit to the agency in areas such as saving resources used to convert paper submission to electronic files, use of past applications as a longitudinal research tool for classes of drugs, or facilitate rapid information sharing by storing data on an accessible portal for FDA reviews.
Establish a common template that can be used as a starting point for all REMS that includes processes for obtaining external stakeholder input.
The inclusion of REMS into the regulatory framework has been a topic of interest for many sectors. As additional experience is gained on designing and measuring the impact of REMS a common template could help streamline the process for those that have not had experience with different aspects of REMS. The draft guidance currently under review to do this should include elements such as processes that a sponsor could use to interact with external stakeholders in order to gain perspectives on the implementation and impact of a REMS. There is nothing that precludes this type of interaction, but it is not widely used. Increased input from external representatives may help design, implement, and evaluate the most effective/efficient mitigation strategies.
Other Programs:
Regulatory Science
There has been continued interest to increase the scientific capabilities of the FDA as well as develop innovative approaches to many regulatory activities. Topics such as new methods of clinical trial design, biomarker qualification/development, regulation of tests (genetic, direct to consumer, IVDMIA and laboratory developed) and subpopulation analysis are areas that the biomedical research community has looked toward FDA for leadership. Opportunities to enhance an active scientific role for FDA should be explored.
Patient Input
In 1996, FDA established the Patient Representatives Program allowing for a patient representative to participate as a member of an advisory committee. In order to allow for involvement earlier in the development process, the Patient Consultant Program was implemented in 2001. While these programs have provided important channels to receive patient input, they deserve greater support to allow more patients the opportunity to participate and they should be used with increased frequency.
Advisory Committees and Conflicts of Interest
Advisory committees continue to be a key source of scientific expertise utilized by FDA. Managing potential conflicts is an important part of the process. In order to streamline this task, administrative COI activities should be consolidated into a division in the Office of the Commissioner so that the individual centers and offices can focus on scientific needs and identifying the best committee members. In addition, the role of advisory committees should be examined and potentially be expanded beyond the review of a single drug application and be used as an opportunity for input on broad scientific questions faced by the agency.
Post-market Surveillance
The FDAAA included a number of programs that were designed to create additional active safety surveillance mechanisms. For example, as authorization for appropriation of $25M was provided for activities such as developing or linking data sets with the goal of aggregating data on 100M patients by 2012 (Sec. 905). This authorization expires in 2012 and should be renewed or increased.
Developing Treatments for Small Populations
Despite the success of programs established through the Orphan Drug Act, drug development for relatively small populations continues to be a challenge. In addition to diseases of rare occurrence, increased understanding of biology has led to identification of smaller subpopulations that are most appropriate to receive a drug. Additional incentives and methods for development of drugs intended for rare and refined populations should be explored. This may include programs for expedited review of new products or abbreviated review and patent restoration for repurposed products for which a new use has been demonstrated.
Best Pharmaceuticals for Children Act/ Pediatric Research Equity Act
BPCA and PREA have provided the necessary incentives for research and drug development of medicine designed for young patients. Without these programs critical research on new and existing treatment may not be conducted and used sub optimally in children. As a part of the next reauthorization cycle, these acts should be included and reauthorized.
Overall FDA Funding
The FDA and its advocates must continue to make a strong case for additional appropriated funding for the agency. The FDA should not survive on user fees along and the off-balance ration of user fees to public funding has created public perception problems and potentially ignored overshadowed other functions of the agency that are not supported by the user fee program.