Helping the FDA to Understand and Support Geroscience and Longevity Medicine Advancements
In 2016, Congress passed the 21st Century Cures Act, intended to accelerate medical product development and commercialization. The Cures Act established a new expedited product development program, the Regenerative Medicine Advanced Therapy (RMAT) Designation.
This designation, intended to expedite the approval of cell, gene, and tissue-based products, can be granted for products that are intended to treat a serious disease and have shown preliminary clinical evidence indicating the drug has the potential to address unmet medical needs. Benefits of the designation include increased FDA interaction and eligibility to apply for priority review for approval of the new therapy. To date, 68 therapies have received the FDA’s RMAT Designation, and three have been approved: one therapy to treat a rare, fatal birth defect, one therapy to treat a serious form of lymphoma, and one therapy to treat severe burns. Other therapies that have received the designation target sickle cell disease, fatal genetic disorders, and cancers that no longer respond to chemotherapy.
While there are other FDA Designations that are technology agnostic – the Breakthrough and Fast Track Designations – the RMAT Designation has some advantages over those pathways. For example, while the benefits of the Breakthrough Designation and RMAT Designation are very similar, the RMAT Designation has slightly less strenuous requirements for receiving the designation. This allows the FDA to better incentivize development for this novel class of therapeutics.
The RMAT Designation has catapulted regenerative medicine into the mainstream. A Longevity Medicine (LM) Designation could do the same for longevity therapies. While some aging therapies fall under the category of regenerative medicines, some would not. They will need to meet the stricter requirements of a Breakthrough Designation. Additionally, because aging is not currently recognized as a disease by the FDA, and because many of these therapies would be preventative in nature, it is possible that they may not qualify as intending to treat, modify, or reverse a serious or life-threatening disease or condition. Instead, sponsors would need to resort to showing that the therapy could treat one specific age-related disease, and then conduct additional trials to expand into other indications.
A4LI calls for the creation of a specialized Longevity Medicine Designation that could be tailored to reflect the unique potential of this class of therapies. This designation could help to create regulatory clarity for aging therapies and accelerate their development.
Support Foundational Drug Discovery Advancements
Our government must invest in cutting-edge, paradigm-shifting drug discovery methods and techniques.
The Alliance for Longevity Initiatives would like policymakers to encourage the advancement and adoption of better drug discovery technologies and methodologies, to increase the speed and accuracy at which we can create and test lifesaving drugs. This may include anything from passing legislation to advance organ-on-a-chip technologies to appropriating more funds to the FDA so the agency can hire more staff, to increase the speed and efficiency at which drugs are reviewed.