The Food and Drug Administration (FDA) have released a new framework which discusses how the agency will use real world evidence (RWE) and real-world data (RWD) to assist companies win new indications for approved drugs and biologics, expand labels or satisfy post-approval study requirements.
The FDA states that the RWE program will evaluate the potential use of RWE to support changes to labelling about drug product effectiveness, including adding or modifying an indication, for example, a change in dose, dose regimen or route of administration, new populations or the addition of comparative effectiveness or safety information. Furthermore, the “RWE framework will also consider the evaluation of observational clinical studies using RWD to support product effectiveness determinations”.
When considering whether the data collected through observational study designs are appropriate to generate RWE for the purpose of supporting effectiveness determinations, the FDA plans to evaluate multiple questions of interest that could affect the ability to draw a reliable causal inference, including, for example, the role of existing evidence (e.g., the natural history of the disease) and how the inclusion of a more diverse population can result in a heterogeneity of treatment effects making it difficult to detect smaller effect sizes”.
The FDA have stated that they will consider the following on a case-by-case basis, for determinations on how to use RWE:
- Whether the RWD are fit for use.
- Whether the trial or study design used to generate RWE can provide adequate scientific evidence to answer or help answer the regulatory question.
- Whether the study conduct meets FDA regulatory requirements (for example, for study monitoring and data collection).
The agency aims to use these three factors to evaluate individual supplemental applications, when appropriate and generally to guide FDA’s RWE program.
Regarding the potential for study designs using RWD to support effectiveness, the framework discusses randomised designs using RWD, non-randomised trials, single-arm trials with RWD control and observational studies.
To read more about FDA’s new strategic framework to advance the use of RWE to support the development of drug and biologics, please click here.