Cure SMA Argues for Greater Patient Input in Drug Development Process at FDA Meeting

Cure SMA has urged the U.S. Food and Drug Administration (FDA) to ensure that user fee agreement funds are used to incorporate and strengthen patient opinions in the drug development and approval processes.

The plea came in Cure SMA testimony at a December meeting, one of several monthly meetings the FDA is required to hold while it works with industry representatives as part of reauthorization process for the Prescription Drug User Fee Act (PDUFA). Specifically, the law requires the FDA to also meet with stakeholders, patient groups, healthcare professionals, and academic experts, and consider their opinions in the reauthorized law. PDUFA allows the FDA to collect fees from drug manufacturers to fund the new drug approval process, and will expire in September 2017 if not reauthorized.

Cure SMA supports the PDUFA as a means of ensuring adequate FDA funding. “Ample funding helps to facilitate the drug review process. Moreover, sufficient funding will allow for expedient review of drugs and biologics,” Cure SMA said in a press release.

The December meeting was one of five reauthorization meetings that Cure SMA has participated in, advocating for the importance of patient perspectives through all stages of the drug development and approval processes.

According to Cure SMA, its goals include better representation of SMA and all patients on FDA advisory committees and panels, and the inclusion of patients with rare diseases.  The organization also supports the incorporation of patient input earlier in the drug development process, earlier review and inclusion of patient benefit/risk assessment, and continued priority review vouches for potential pediatric disease treatments.

Cure SMA also supported continued strong incentives for orphan drugs, saying in its December testimony, “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.”

And it favored the continued hiring by the FDA of 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,” Cure SMA reported testifying in the release.

Cure SMA will continue to actively participate in the PDUFA meetings and advocate for the issues of importance to the community.