The US Food and Drug Administration (FDA) has finalized a slightly tweaked guidance on medical device companies’ use of real-world data (RWD) and real-world evidence (RWE) in winning approvals for their products.
FDA says the guidance is meant to explain the "characteristics and sources of RWD and characteristics of RWE that may be sufficient for use in making various regulatory decisions."
In addition to the draft guidance, the final guidance includes further clarity on what the document means for companies moving forward, noting: "While FDA encourages the use of relevant and reliable RWD, this guidance neither mandates its use nor restricts other means of providing evidence to support regulatory decision-making."
The final version also notes that, "Because of the rapidly advancing methodology for generating and interpreting RWD, this guidance will not elaborate on the methodological approaches that can be used."
And FDA has not offered any additional examples beyond what was offered in the draft (as some companies requested) though the agency has re-written the relevance factors that it will assess to determine if the RWD are suitable for regulatory use.
"FDA recognizes that a wealth of data covering medical device experience exists and is routinely collected in the course of treatment and management of patients," the agency said. Under certain circumstances RWD may constitute RWE, FDA says, "that may be of sufficient quality to help inform or augment FDA's understanding of the benefit-risk profile of devices at various points in their life cycle, and could potentially be used to aid FDA in regulatory decision-making."
The agency also noted that the guidance is a "cornerstone" of its strategic priority to build a national evaluation system for health technology.
Background and Comments
In July 2016, FDA released the draft version of the guidance and in June the agency points to an example of RWE that was used in its decision to expand the use of a transcatheter aortic valve replacement.
Companies and industry groups commenting on the draft largely complimented FDA for starting the conversation on the use of RWD for devices, though some sought further expansions of the draft and additional examples.
For instance, industry group AdvaMed said the draft was "heavily focused on device registry data" and might also incorporate other sources, like insurance claims databases or electronic health records. The group further sought clarity and examples for how RWE can support regulatory decision-making for Class II devices in the pre-market phase of development.
UK-based manufacturer Smith & Nephew added: "While this draft guidance mentions other data sources, 510(k)s and IDEs, it is largely focused on registries and implants, resulting in a document that is very narrowly focused on Class Ill devices, with no real content or examples related to the broader landscape and opportunities. FDA should clarify the application of the Draft Guidance to Class II devices that are not subject to a registry and provide examples from both the premarket and post market perspectives."
Several commenters, including Abbott Laboratories, also sought increased discussion of the use of RWE for in vitro diagnostics (IVDs).