Sep 2, 2016

Recent guidance from US medical device market regulators explains when and how real-world data and evidence obtained via methods other than clinical trials are used to make market authorization and related decisions pertaining to the products they oversee.

The Food and Drug Administration guidance lists various methods whereby real-world data may be collected, such as simple or pragmatic clinical trials, observation, retrospective database studies, case reports and electronic health records. Aggregation and analysis of real-world data, in turn, results in real-world evidence.

According to the agency, US market registrants may need to utilize real-world data and evidence in instances where standard clinical evidence proves too difficult to obtain because of a device’s development cycle or innovative design.

“In some cases, a traditional clinical trial may be impractical or challenging to conduct, given the realities of medical device innovation and development cycles, ethical issues that may arise with treatment assignment, and other similar challenges in executing traditional trials with high quality,” states the guidance.

Although real-world evidence can serve as an acceptable alternative to clinical evidence to support some devices’ safety and effectiveness claims, use of such evidence must prove relevant and reliable when assessed by FDA reviewers; furthermore, manufacturers intending to submit real-world evidence to support their US market applications should notify the FDA via the agency’s pre-submission process.

(For more information on the FDA’s pre-submission process and related communications issues, download our whitepaper.)

The guidance provides examples of when real-world evidence can be effective:

  • Generating hypotheses for prospective clinical studies
  • For expanding a device’s labeling to include additional indications for use
  • Ongoing public health surveillance projects
  • Post-approval studies of devices
  • Replacing individual Medical Device Reports (MDR)
  • Providing postmarket data under the Expedited Access Pathway(EAP) program

Thus, provided that US market applicants’ real-world evidence can meet relevance and reliability requirements, such material can in certain instances help meet FDA standards for device safety and effectiveness without incurring additional time and costs of conducting full clinical trials.


  • Stewart Eisenhart