Promising Pathway Act

The Promising Pathway Act (PPA) H.R. 4408/S.1906 calls for the U.S. Food and Drug Administration (FDA) to establish a rolling real-time, priority review pathway to grant or deny provisional approval status for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions.

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About the legislation

The Promising Pathway Act (PPA) calls for the U.S. Food and Drug Administration (FDA) to establish a rolling, real-time, priority review pathway to grant or deny provisional approval status for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions. The granting of provisional approval requires demonstration of substantial evidence of safety (the same level as currently required for standard FDA approval) and relevant early evidence of positive therapeutic outcomes based on adequate and well controlled investigations. The PPA also allows for the use of real world evidence to support an application. The PPA provides for provisional approval only, for two year periods up to eight years. Patients must participate in an observational registry. The FDA must review the registry annually and shall withdraw provisional approval if a drug has serious side effects or is ineffective. Drugs must meet existing standards for full approval. In sum, the PPA provides a pragmatic pathway for the FDA to provisionally approve drugs for individuals with rapidly progressive terminal illnesses who will die of their diseases waiting for the usual FDA approval pathways to unfold. Provisional approval would be subject to fulsome FDA guardrails and oversight.