Promising Pathway Act

The Promising Pathway Act calls for the U.S. Food and Drug Administration (FDA) to establish a rolling, real-time review pathway to grant or deny conditional approval status for therapies or treatments intended to treat rapidly progressive, terminal diseases or conditions. The granting of conditional approval requires early evidence of safety and effectiveness as established by clinical investigations.

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About the legislation

The Promising Pathway Act calls for the U.S. Food and Drug Administration (FDA) to establish a rolling, real-time review pathway to grant or deny a two-year conditional approval status for therapies or treatments intended to treat rapidly progressive, terminal diseases or conditions. The granting of conditional approval requires early evidence of safety and effectiveness as established by clinical investigations. People who gain access to therapies through this process must participate in an observational registry. The FDA can withdraw conditional approval at any time based on safety or can deny renewal if the requirements for such renewal are not met. Drugs must meet existing FDA standards for full approval.

In sum, the Promising Pathway Act provides a pragmatic pathway for the FDA to conditionally approve drugs for individuals with rapidly progressive, terminal illnesses who will die of their diseases waiting for the usual FDA approval pathways to unfold.