The rare disease community has reason to cheer a continuing resolution (CR) signed today by President Biden, which includes reauthorization of the FDA user fees (known as PDUFA VII) and urgently needed enhancements to rare disease programs. The CR, signed into law this afternoon, reauthorizes FDA user fees for five years and provides two-and-a-half-months of funding for the federal government that will enable the FDA to continue the lifesaving momentum of its work.
“The EveryLife Foundation is grateful to all the community members who have worked so hard to ensure that the needs, priorities, opportunities, and urgency of our rare disease community is so strongly reflected in the user fee package,” said Julia Jenkins, EveryLife Foundation for Rare Diseases Executive Director. “We are pleased to see many of the priorities we’ve articulated throughout this process reflected in these commitments.”
The CR honors several improvements to rare disease programs, which were first laid out in the August 2021 FDA Commitment letter’s Performance Goals . These enhanced program commitments are the culmination of many years of formal engagement with the patient community, which informed the negotiations between the FDA with biopharmaceutical industry leaders.
The FDA user fee reauthorization is considered a ‘clean reauthorization’. This means that it provides five-years of authorization for the FDA user fee program but does not contain any new policy provisions that were included in the bill passed by the House in June or the bill passed by the Senate Health, Education, Labor and Pensions (HELP) Committee. However, thanks to the robust engagement between patient advocates and those who negotiated the FDA Commitments, PDUFA VII represents a continuation of the FDA’s commitment to advancing the fields of rare disease therapy development and patient engagement. These include:
- The establishment of Rare Disease Endpoint Advancement (RARE) pilot;
- The establishment of the Split Real Time Application Review (STAR) pilot;
- Advancing patient-focused drug development within CBER; and
- Refining and advancing policies to support use of Real-World Evidence (RWE) and innovative trial designs
These are all examples of the commitments that will further support patient engagement and patients with rare conditions over the course of the performance period.
“Given the promise and anticipated innovations in rare disease therapy development, the EveryLife Foundation is especially gratified to see the emphasis on regulatory process enhancements through resources, public workshops, pilots, and guidances,” said Annie Kennedy, EveryLife Foundation for Rare Diseases Chief of Policy, Advocacy and Patient Engagement. “These commitments will address emerging issues such as Real-World Evidence, Bayesian Modeling, and enhanced formats for engaging with the agency.”
The CR also includes two-and-a-half-month reauthorizations for eight FDA programs often included in user fee packages, including the orphan drugs grants and the humanitarian use device programs.
The CR funds the government through December 16th, ensuring that federal agencies will not run out of funding on October 1st. This provides an additional window of opportunity for advocates to ensure that appropriation provisions they are advocating for are included in the final omnibus bill expected this December. In addition, the omnibus negotiations also provide another opportunity for rare disease advocates to seek the inclusion of the many important policy provisions that were originally included in the user fee negotiations such as elements of the STAT Act, clinical trial diversity, and enhancements to the accelerated approval pathway.
We will continue to advocate to ensure that the promise of today’s pipelines will change health outcomes for THIS generation of patients.
