The National Health Council (NHC) and the National Organization for Rare Disorders (NORD) are circulating the below sign-on letter in support of timely reauthorization of the Food and Drug Administration’s (FDA) user fee agreements.
People with chronic and rare diseases and disabilities rely on FDA to access innovative, safe, and effective treatments. Not only do user fees provide the funding for FDA to quickly review products, but the current user fee agreements also provide significant improvements and advancements to the health care community that simply cannot wait.
Deadline: We ask that all signatures be submitted by April 20.
April XX, 2017
Dear Majority Leader McConnell, Speaker Ryan, and Minority Leaders Schumer and Pelosi,
On behalf of the millions of men, women, and children in the United States living with chronic or rare diseases and disabilities, the undersigned organizations urge you to quickly reauthorize the Food and Drug Administration (FDA) user fee agreements.
The current FDA user fee agreements are the culmination of months of negotiation between FDA and the medical product industry, with significant input from the patient advocacy community. We stand behind these agreements and ask that they are reauthorized as quickly as possible.
The FDA largely relies on user fees authorized by Congress to operate. Without the user fees, a majority of drug, biologic, and device reviewers would be laid off, and the necessary review of innovative therapies would be substantially impaired, if not halted all together. Further delay, such as a one year extension of the current agreement, will impact FDA’s ability to carry out its vital mission and delay many of the much welcomed improvements included in the Commitment Goals Letters.
The Prescription Drug User Fee Act (PDUFA) Commitment Goals Letter includes many important improvements and advancements to FDA processes and initiatives. These include the continuance of the Patient-Focused Drug Development program, requiring guidance for industry; the strengthening of the Breakthrough expedited review pathway; and the expansion of the Rare Diseases program, among others.
Other provisions included in the agreement, such as increased emphasis on biomarkers and surrogate endpoints, streamlining combination product reviews, studying the feasibility of incorporating real-world evidence (RWE) provided from clinical settings, and improving FDA hiring and retention practices, will lead to a more efficient FDA that is better equipped to review 21st Century treatments in a timely fashion.
The Medical Device User Fee Amendments (MDUFA) Commitment Goals letter also contains several crucial reforms including the further inclusion of patient preference information and patient-reported outcomes in device development and review, as well as the creation of the National Evaluation System for health Technology (NEST).
Additionally, the Generic Drug User Fee Amendments (GDUFA) and Biosimilars User Fee Act (BsUFA) are designed to bring lower-cost alternative prescription medicines to market as quickly as possible. These alternatives represent one of the best ways to lower the cost of care.
We request the Committees of jurisdiction and Congress as a whole keep the user fee reauthorization process timely, bipartisan, uncontentious, and focused on the patients FDA serves every day.
The chronic and rare disease and disability patient communities rely on FDA to ensure that innovative, safe, and effective treatments reach those in need. We thank Congress for moving forward with these critical funding mechanisms, and look forward to their swift and unimpeded passage.
American Autoimmune Related Diseases Association (AARDA)
American Cancer Society Cancer Action Network
Amyloidosis Support Groups
APBD Research Foundation
Bridge The Gap - SYNGAP Education and Research Foundation
Caregiver Action Network
Central Pain Nerve Center
Children's Cardiomyopathy Foundation
Children's PKU Network
Colon Cancer Alliance
Congenital Adrenal hyperplasia Research, Education & Support Foundation (CARES Foundation, Inc.)
Consortium of Multiple Sclerosis Centers
National Health Council
U.S. Pain Foundation