H-SPAN longevity biotech summit panel discusses how the FDA could evaluate preventive therapies targeting multiple age-related diseases
H-SPAN longevity biotech summit panel discusses how the FDA could evaluate preventive therapies targeting multiple age-related diseases
On September 9 at the H-SPAN longevity biotech summit, FDA and biotech representatives discussed how to test therapies intended to delay several age-related diseases. Drug development usually starts with a single diagnosis; prevention before diagnosis requires a way to measure benefit in advance.
The July report on the H-SPAN summit had already raised questions about which disease to bring to the FDA first and which measure to use to demonstrate benefit. At the September panel, these questions became a debate about how to design such a trial.
Robert Kalesnik-Orszulak, head of regulatory affairs at the biotech company Retro Biosciences, described the path for companies seeking to target shared mechanisms of aging: start with one disease and expand the indications after the initial results.
David Fox, an FDA regulatory attorney and coauthor of the Thrive Act, addressed a different task: testing a therapy intended to delay several chronic diseases in people who have yet to receive a serious diagnosis. This would require large groups of participants, long follow-up, and particularly rigorous safety assessment. The trial must specify its endpoint in advance: the outcome used to judge whether the intervention worked.
Kalesnik-Orszulak suggested considering this task under the FDA’s accelerated approval framework. Under that framework, a surrogate endpoint, an early measurement the regulator can accept as predictive of future health benefit, can form the basis of an FDA decision. Fox considered this pathway appropriate for serious diseases, where a different level of uncertainty is acceptable. His Thrive Act describes a separate, staged framework for establishing evidence for prevention in people without a serious diagnosis.
Lowell Zeta, whom the moderator introduced as FDA Deputy Commissioner for Strategic Initiatives and Acting Chief of Staff, invited industry to submit data and proposals. He mentioned Operation Trailblazer for early human studies, a request for proposals on new uses for drugs that have long been on the market, and the program for qualifying biomarkers, which are measurable indicators of the body’s condition.
The panel discussion centered on two related tasks: identifying an early measurement that the FDA can accept as predictive of whether a therapy will reduce the risk of future diseases, and designing a trial capable of demonstrating that reduction.