The Prescription Drug User Fee Act (PDUFA), the law that allows the FDA to collect fees from drug manufacturers to fund the new drug approval process, is set to expire in September 2017. As part of the process to reauthorize this law, the FDA must hold monthly meetings with a stakeholders, patient groups, health care professionals, and academic experts to discuss ways to improve the drug review process.
These meetings, required to be held during the time that FDA is negotiating with industry, began in September 2015.
To date, Cure SMA has actively participated in 5 meetings with FDA where we have highlighted the importance of patient centered drug development. The primary issues we are advocating for include:
- Strengthen and incorporate the patient voice throughout all stages of the drug development and approval process.
- Assessment and inclusion of patient benefit/risk by reviewers early in the process.
- Maintain strong incentives for orphan drugs.
- The criteria the FDA would allow for accelerated approval.
- Adequate funding for the FDA appropriately to ensure safe and expedient review of drugs and biologics and in order to have specific expertise in orphan disease areas.
- Membership on FDA advisory panels – should include rare disease patients.
- Continue priority review voucher for pediatrics.
At the December meeting, Cure SMA testified before FDA. Below is a portion of that testimony, which also included introductory remarks on SMA and Cure SMA.
Cure SMA will continue to actively participate in these PDUFA meetings and advocate for the issues of importance to our community.
Cure SMA Testimony Before the FDA
We strongly urge the FDA to ensure that user fee agreement funds are utilized to strengthen and incorporate the patient voice throughout all stages of the drug development and approval processes, including increased representation on FDA Advisory Committees. The FDA’s inclusion of patients with rare diseases on Advisory Committees and Panels will ensure that the interests of patients with rare diseases are adequately represented.
Additionally, we support the inclusion of the patient’s viewpoint throughout the drug development process. There must be an increased emphasis to incorporate the patient voice earlier in the development process. The FDA also must actively strive to understand the needs of SMA patients across types and the disease’s impact on parents and caregivers.
We also are hopeful that strong incentives are maintained for orphan drugs. We support the Orphan Products Grants Program and the expansion of the program to allow for additional funding for clinical research which tests the safety and efficacy of drugs in rare diseases or conditions.
We must also ensure that the user fee agreement provides adequate funding for the FDA. Ample funding helps to facilitate the drug review process. Moreover, sufficient funding will allow for expedient review of drugs and biologics.
We also strongly support the FDA’s hiring of internal experts on orphan diseases. The Food and Drug Administration Safety and Innovation Act, signed into law in July of 2012 added a provision that included consultation with external experts on rare diseases, and the 21st Century Cures Act would lift certain salary caps at the FDA, which would encourage the hiring of experts in rare disease areas. However, should this legislation fail to come to fruition, we urge this issue be addressed in PDUFA reauthorization legislation.
In conclusion, on behalf of Cure SMA, thank you for allowing us to present our views on the reauthorization of the Prescription Drug User Fee program. We look forward to continued discussions as the process moves forward.