Sep 23, 2020

The US Food and Drug Administration released a draft guidance document on August 31 that aims to help medical device manufacturers, FDA staff, and other stakeholders utilize patient-reported outcome (PRO) instruments for medical device evaluation. PRO instruments (alternatively called patient-reported outcome measures or PROMs) are questionnaires designed to record PROs in the form of data that can be utilized for clinical assessment of a device’s safety and effectiveness. According to the guidance, PRO instruments “facilitate the systematic collection of how patients feel, function, and survive as valid scientific evidence to support the regulatory and healthcare decision-making process.”

FDA efforts to give greater weight to PROs in its regulatory and clinical activities date back to at least 2015, when the Agency created the Patient Engagement Advisory Committee to further the cause of patient-centered healthcare. The FDA’s Center for Devices and Radiological Health (CDRH) also announced in 2016 that more effectively generating data from PRO instruments would be one of its key priorities.

Draft guidance outlines best practices for PRO instrument selection, development, and refinement

The draft guidance, which was jointly issued by the CDRH and the Center for Biologics Evaluation and Research (CBER), offers principles, recommendations, and an outline of best practices for choosing, creating, and altering PRO instruments to inform medical device evaluation. The document begins by providing a summary of previously published FDA resources dealing with PRO instruments, including a 2009 guidance on their use to support labeling claims and a list of case studies. The best practices presented in the guidance can be summarized as follows:

  • Develop PRO instruments based on concepts that patients identify as important;
  • Make sure that PRO instruments are easy for patients to understand;
  • Clearly identify the PRO instrument’s context of use (COU) in your clinical study protocol and statistical analysis plan;
  • Use existing PRO instruments as templates or jumping-off points and review the validity evidence associated with them;
  • Investigate sources of real-world evidence outside the clinical setting that could provide validity evidence for PRO instruments; and
  • Work with other stakeholders to develop and refine PRO instruments in the pre-competitive space.

The FDA is accepting comments on this draft guidance online or by mail until October 30, 2020.

Related US FDA medical device and IVD regulatory resources:


  • Timothy Herr