The FDA recognizes the important role that Real World Evidence (RWE) can play in the drug review process and has been making efforts to encourage its use in certain submissions made to the agency.
RWE is generated from Real World Data (RWD), i.e. data relating to the safety and efficacy of an investigational drug that is collected outside of the strict confines of the traditional clinical trial setting.
Earlier this year, the FDA released a draft guidance document for industry defining what the agency constitutes as RWD and RWE and explaining how companies should submit this to the FDA for review.
According to the document, the kinds of sources that companies can utilize include: “Data derived from EHRs; Medical claims and/or billing data; Product and/or disease registry data; Other data sources that can inform on health status (e.g., data collected from mobile technologies, patient-generated data).”
The document also explains that, when including RWE as part of a submission to the FDA, companies should notify the agency of the purpose of using RWE, identify the study design using RWE, and highlight the RWD sources used to generate the RWE.
However, while this guidance document does remove some of the ambiguity around the agency’s treatment of RWE, certain key industry players are calling for the FDA to develop the document further, before releasing the final version.
Leading biopharma companies Novartis and Gilead, for example, have asked for the FDA to provide a more comprehensive list of accepted RWD sources. The drug manufacturers believe that lab data and data from (a) expanded access protocols, (b) the delivery of health care and (c) pharmacy claims should also all be accepted by the agency as suitable tools for generating RWE. Gilead goes further and suggests the FDA should also include an additional section explaining which data sources the agency will not consider appropriate.
Industry groups BIO and PhRMA also had comments on how the agency can improve the guidance document. Both groups call on the FDA to make efforts to further harmonize the submission process for RWD and RWE across different agency centers, including the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research and the Center for Devices and Radiological Health. BIO also suggested the FDA should publish aggregate, deidentified data on the number and context of RWE submissions made to the agency as well as information on the therapeutic areas and the study designs and data types used in these submissions.