The EveryLife Foundation for Rare Diseases kicked off Rare Disease Week on Capitol Hill 2022 by joining rare disease leaders in urging Congress to protect and strengthen the FDA’s accelerated approval (AA) pathway. The group of experts made the case during a virtual Rare Disease Congressional Caucus Briefing attended by nearly 400 rare disease stakeholders.
The AA pathway allows the U.S. Food & Drug Administration (FDA) to use a surrogate endpoint (also called a biomarker) to evaluate the safety and efficacy of therapies for serious conditions with unmet needs. By evaluating a surrogate endpoint that is reasonably likely to predict clinical benefit, the pathway allows patients to access treatments at the earliest possible moment.
Affirming the urgent and unmet need of the rare disease community, leaders attested to the scientific rigor and safety of the AA pathway and its proven benefit to patients with life-threatening conditions. Among the facts presented were the following:
- Accelerated approval is not a lower standard. Treatments approved via the AA pathway are subject to the same statutory standards for proving safety and efficacy as drugs approved via traditional pathways,
- More than 75% of all products approved via the AA pathway have been converted to traditional approvals, with many more still in the process of collecting confirmatory data, and
- Spending on drugs approved through the AA pathway accounted for less than one percent of annual Medicaid spending between 2007 and 2018.
Presenters called on Congress to support policies that would protect and strengthen the accelerated approval pathway for use in rare disease therapy development, enable more product innovation incentives, and facilitate access to products approved by the pathway, including the following:
- Support the prioritization of resources and collaboration necessary to advance knowledge on rare disease biomarkers and intermediate clinical outcomes,
- Protect and strengthen the development of incentives including the Orphan Drug Tax Credit,
- While recognizing the regulatory authority of the FDA, facilitate earlier payer engagement prior to regulatory approval around the selection and use of endpoints and surrogate biomarkers in clinical studies and regulatory review,
- Identify opportunities for confirmatory trial process improvements through more structured and earlier stakeholder engagement in the pre-market setting, and
- Enable broader use of real-world evidence to satisfy post-approval confirmatory requirements.
The briefing was moderated by Frank Sasinowski, MS, MPH, JD, Hyman, Phelps & McNamara Director and EveryLife Foundation for Rare Diseases Board of Directors Vice Chair, and featured rare disease community leaders including: Pat Furlong, Parent Project Muscular Dystrophy Founding President and CEO, Annie Kennedy, EveryLife Foundation for Rare Diseases Chief of Policy, Advocacy, and Patient Engagement, GK Raju, PhD (MIT), Light Pharma Inc, Ellis Unger, MD, former Director of the Office of Drug Evaluation-I in FDA’s Office of New Drugs in the Center for Drug Evaluation and Research, and Teonna Woolford, Sickle Cell Reproductive Health Education Directive CEO.
The briefing also included early results from a study as to the utilization of the accelerated pathway conducted by researchers at LightPharma, sponsored by the EveryLife Foundation. While full results will be made available later this spring, high-level findings were also published in new online resource materials designed to educate stakeholders about the AA pathway and how they can help ensure it remains a vital tool for rare disease drug development. Advocates interested in joining the Foundation in its efforts to strengthen and support the accelerated approval pathway are encouraged to sign-up for action alerts and event invitations at everylifefoundation.org. View the Caucus Briefing recording.
The EveryLife was founded to improve access to the accelerated approval pathway to bring lifesaving treatments to patients with rare diseases. It has been working in collaboration with the community over the past 13 years to help advance the science for the use of biomarkers for rare diseases and to build congressional and public awareness about the challenges of rare disease drug development.
