FDA Dual Track Legislation Advances

S&P Global: Four Republican lawmakers have authored new legislation to permit drugs for critically ill patients to enter the market before completing late-stage trials, saying the bill was necessary because the U.S. Food and Drug Administration’s regulatory process was too slow and burdensome.

The bill would create a time-limited conditional approval pathway in the U.S. similar to a system that has long been used by European regulators.

…The conditional approval would be valid for one year and could be renewed annually for up to five years….Companies would be required to meet certain obligations, like completing clinical investigations to provide full demonstration of safety and effectiveness and other studies.

…Companies could seek full U.S. approval at any time. The FDA would be required to let manufacturers include in their applications the real-world evidence they collected during the conditional approval period.

The lawmakers want the FDA to be able to grant the limited marketing authorization to new drugs that have successfully completed phase 1 and 2 trials, with the idea that companies could generate revenue to help fund their phase 3 studies.

They emphasized their legislation is targeted especially at small biopharmaceutical companies that may struggle to cover the costs of late-stage trials.

Under the dual-track approval system, companies would be able to sell pharmaceuticals earlier but would be required to track outcomes so greater real world information would be developed in the FDA process. The result is a more dynamic approval process better suited to modern medicine. The idea is due to the excellent Bartley J. Madden (note my bias).

Madden and Nobel-prize winner Vernon Smith explained the dual-track idea, noting:

Today’s world of accelerating medical advancements is ushering in an age of personalized medicine in which patients’ unique genetic makeup and biomarkers will increasingly lead to customized therapies in which samples are inherently small. This calls for a fast-learning, adaptable FTCM environment for generating new data. In sharp contrast, the status quo FDA environment provides a yes/no approval decision based on statistical tests for an average patient, i.e., a one-size-fits-all drug approval process.

A similar process has been adopted in Japan for regenerative medicine.