On 13 December 2016, the 21st Century Cures Act was signed into law by Congress. This requires the US Food and Drug Administration (FDA) to produce a framework and guidance for evaluating real world evidence (RWE) in the context of drug approvals.

However, critics have taken issue with other parts of the act, claiming these will ultimately serve to erode the drug approval process, potentially leading to increased risks for patients taking FDA-approved drugs. Unfortunately, this causes difficulties regarding the usefulness of RWE in supporting drug approval decisions. It also highlights the issues surrounding the inclusion of multiple provisions within single legislative acts.

The FDA previously issued draft guidance on the inclusion of RWE for medical devices on 27 July, 2016, but did not follow with similar guidance for drugs. Now the agency is required to do so. 

According to the Cures Act, RWE is defined as 'data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials (RCT)'. This can be taken to mean any data that can be collected from real life medical practice settings, including electronic medical records and insurance claims, as well as from patient registries. Drug developers and health regulatory agencies have traditionally relied exclusively upon RCT to support drug development and approval decisions, and those seeking approval in the US will now be encouraged to include RWE in submissions.

RWE is defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomised clinical trials (RCT)”

RWE has already been in use in assessing drug reimbursement decisions by health technology agencies (HTA), and an analysis published at PharmaForum on 27 January, 2015, evaluating nearly 1,840 decisions globally found that just 6% of these decisions included observational RWE data. Of these, 77% resulted in a positive reimbursement decision, compared to 67% resulting in positive decisions for those cases not incorporating RWE.

The number of those decisions including RWE was rather small, making it difficult to draw conclusions on the significance of the correlation. Individual case studies also reveal instances where developers used RWE to show increased durability of response, increased survival, quality of life, or economic value for their drugs. 

With the FDA moving ahead to provide a framework for inclusion of RWE in approval decisions, there will be robust opportunities to demonstrate increased efficacy, safety, and economic value in settings beyond RCTs. The stage is set for a paradigm shift in the drug approval process based on the inclusion of RWE that should more substantially prove or disprove a drug’s safety and effectiveness. The usefulness of its inclusion should not be diminished by criticism of other provisions of the recently enacted 21st Century Cures Act.