Devices

Changes Asked in Real-World Evidence Guidance

David McFarland

— 2 min read
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The Yale Collaboration for Research Integrity & Transparency says it supports appropriate use of high quality real-world evidence in the context of post-market medical device regulation and surveillance. Commenting on an FDA draft guidance, on using real-world evidence to support medical device regulatory decision-making, the group says it opposes allowing real-world evidence as a substitute for traditional clinical trial data to evaluate pre-market applications for Class 3 devices, including applications for expanded indications for use in already approved Class 3 devices. Using such evidence for those devices poses both safety and effectiveness concerns, it says. The group says that some aspects of the draft could be strengthened to ensure that regulatory decisions are based on reliable, high-quality data. Changes it suggests include: 

  •          revise the guidance to state that case reports would not be used in isolation to guide regulatory approval decisions, but only in concert with additional more rigorous studies;
  •          any real-world evidence should use an intervention and a control group;
  •          clarify that use of real-world evidence in place of randomized controlled trials is not acceptable;
  •          state that real-world evidence should include a concurrent control group;
  •          clarify limits on using historical controls as part of real-world evidence;
  •          reject any use of real-world evidence accumulated in a broader patient population or wider set of circumstances than described in the device labeling;
  •          state that if real-world evidence is used to conduct post-approval studies imposed at the time of device approval, such studies must be reported on one-year outcomes within two years of market availability;
  •          state that if real-world evidence is used to conduct post-approval studies imposed at the time of device approval they should enroll representative patient populations to those receiving the device in clinical practice; and
  •          request that investigators for post-approval studies register their trials prior to inception and report their results on ClinicalTrials.gov within 12 months of study completion.

The International Society for Pharmacoeconomics and Outcomes Research says the guidance is a significant step toward clarifying and formally recognizing potential regulatory uses for real-world evidence with the criteria needed to assess the relevance and reliability of real-world data. It suggests including one or two examples to explain some of the terms and definitions in the guidance and also encourages the agency to develop a plan to accrue and release accepted data sources and applications through ClinicalTrials.gov.

The American Association of Pharmaceutical Scientists says that given the growth of combination products, with most of them primarily reviewed by CDER, it would have been very useful for the guidance to be sponsored by CDER. It also asks for the agency’s thinking on getting real-world data and real-world evidence from outside the U.S.

The comment from the Pew Charitable Trusts says the use of real-world evidence requires these two policy changes to ensure utility of the data to better evaluate device safety and performance: first, real-world information should include the devices used in care by adding unique device identifiers to the data sources and second, FDA, manufacturers, healthcare providers, and other stakeholders should establish a national medical device evaluation system to collect better data on device performance.

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