The FDA has confirmed through an announcement that it has removed a limitation on the use of real-world evidence -RWE when it comes to reviews of drug and device applications.
Interestingly, the new guidance from the agency goes on to state that it is going to accept RWE without needing that identifiable individual patient data collected from real-world data sources to always be submitted as a part of the marketing submission. Due to this change in RWE limitation, the FDA looks forward to considering updating its guidance when it comes to drugs and biologics.
As per the FDA, it had in the past promoted RWE limitation as an advance in regulatory policy. But the prior expectations mean that most RWE cannot be considered in product applications. The FDA states that, since 2016, there have been 35 drugs, biologics, or even vaccines that have included RWE within their applications. Over 250 premarket authorizations within the same period included RWE; however, the rate of RWE-based authorizations has, as a matter of fact, plateaued in recent years, confirms the FDA.
The FDA remarked that it historically insisted that RWE submitted to the agency include private and confidential information and that too at the individual patient level. However, this renders most of the large databases having valuable macro-level data impractical, the agency said.
It says that the updated guidance goes on to come in response to the position of sponsors and scientists that meaningful information can indeed come from big data sources without any private, individual information. Due to this, the FDA reviewers now seek to consider the strength of the submitted RWE, and that too on an application-by-application basis.
As per the FDA, the policy change could help with the utilization of de-identified databases, which contain millions of patient records. That includes the national cancer registries, such as the Surveillance, Epidemiology, and End Results; hospital systems databases; insurance claims databases; and electronic health record networks of the National Cancer Institute.
Marty Makary, the FDA Commissioner, says that they are removing unnecessary barriers that have prevented them from making use of real-world evidence so as to get life-changing treatments to patients faster. This kind of common-sense reform is sure to unlock access to massive databases such as the cancer and cystic fibrosis registries, which contain critical insights pertaining to how treatments actually work in the real world.