On September 28th, during the FDA’s Public Meeting on the Recommendations for Prescription Drug User Fee Act (PDUFA) Reauthorization, Annie Kennedy, Chief of Policy and Advocacy for the EveryLife Foundation for Rare Diseases, provided testimony on behalf of the Foundation based on engagements with a coalition of community stakeholders.
Kennedy began by thanking the FDA’s personnel for their leadership and tireless work to protect the nation this past year and for their continued commitment to the public health crisis of rare disease.
In addition, she recognized the tremendous evolution of patient engagement since PDUFA V and most importantly highlighted the lives changed and saved through products approved to treat rare conditions, especially those that have benefited from the inclusion of more robust patient insights and patient experience data as part of the review process.
She noted that these approvals have not come because FDA lowered the evidentiary bar for rare disease products. Rather, they came because FDA and Congress have recognized the need for a nuanced approach to review of such products given the unique nature of rare diseases, including patient populations, trial sizes and other critical data points.
Kennedy applauded the FDA for the commitments included in the PDUFA VII Commitment letter released last month and encouraged the FDA to go above and beyond the commitments in the letter. By doing so, Kennedy, stated the FDA will continue to ensure that the promise of today’s pipelines will change health outcomes for THIS generation of patients.
To view the public meeting recording visit: https://bit.ly/3ikF2uG
