The Food and Drug Administration (FDA) has released a draft guide to explain how evidence from a single clinical investigation can serve as substantial evidence for the approval of new drugs. The guide is a continuation of previous guidance from 1998 and 2019. It is clarified that the level of confirmatory evidence required will be determined based on the results of the sole clinical investigation. Early communication with sponsors is encouraged by the FDA for this process.
The draft guide further provides details on how data from clinical trials, related indications, approved drugs of the same class, and real-world evidence (RWE) may qualify as confirmatory evidence. The FDA offers advice to sponsors to consult with relevant review divisions about their plans to use RWE.
Whether real-world data (RWD) sources can be used to develop RWE that serves as confirmatory evidence is dependent on various factors, as per the draft. These include the reliability and relevance of the RWD source, the quality of the study design, and the use of appropriate pre-specified statistical methods and analyses.
The draft emphasizes that it would be beneficial for sponsors to discuss with the relevant review divisions any plans to utilize RWE as confirmatory evidence in a drug development program.
The guidance aims to provide a clearer understanding of how evidence from a single clinical investigation can be leveraged for new drug approval. It also underlines the importance of early and frequent communication with sponsors to ensure that the process is efficient and effective.
The draft also underlines the potential role of RWE and RWD in drug development. These sources of data can provide additional insights and context that may be invaluable in the drug approval process.
Overall, the FDA emphasizes the importance of a comprehensive and multi-faceted approach to confirmatory evidence. The guidance recognizes the potential of using varied sources of data, including RWE and RWD, to support the approval of new drugs.
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