We are at a critical inflection point in therapy development, with the opportunity to build on scientific momentum to unlock complex rare diseases and save lives. Our rare disease community is eager to support FDA leaders who respond to unmet patient needs with urgency by empowering review staff to deploy the full range of tools, expertise, and infrastructure at their disposal. At the same time, we are committed to working with the agency to further improve upon these capabilities as science continues to evolve. A regulatory environment that maintains scientific momentum in rare disease is imperative and will best be achieved through the:
- Convening of meaningful patient community engagements and inclusion of patient experience at each stage of therapy development and regulatory review, with clear communication from the FDA on how this information is incorporated into regulatory decision-making;
- Use of congressionally authorized tools that enable tailored regulatory approaches for rare disease therapies, including the accelerated approval pathway;
- Support of innovative trial design approaches, including endpoint selection, choice of control, and evidentiary requirements – and honoring alignment, if it’s been reached;
- Promotion of regulatory approaches that account for the totality of evidence, recognizing that each trial and trial participant contributes critical knowledge in small and heterogeneous patient populations;
- Timely convening of public advisory committees that involve patient representation and experts in rare disease and the science of small studies; and
- Full implementation and resourcing of the FDA Rare Disease Innovation Hub to improve consistency of these approaches in practice.
Our rare disease community is eager to engage and we stand ready to support new leadership to ensure that every potential rare disease innovation has an optimal pathway to becoming an effective, life-altering therapy.
