On August 31, 2020, the Food and Drug Administration (FDA) issued draft guidance regarding principles for selecting, developing, modifying, and adapting patient-reported outcome instruments for use in medical device evaluation.[1]  Patient-reported outcome (PRO) instruments facilitate the systematic collection of how patients feel and function during a clinical trial.  FDA recognizes this information as important because by integrating patients’ voices throughout the total product lifecycle, concepts important to patients can be considered in the evaluation and surveillance of medical devices.

Goals of the Draft Guidance 

FDA recognizes there are many ways PRO instruments can be used during clinical studies.  As such, FDA issued the draft guidance with the following objectives:

  • Describing principles that may be considered when using PRO instruments in the evaluation of medical devices;
  • Providing recommendations about the importance of ensuring the PRO instruments are fit-for-purpose; and
  • Outlining best practices to help ensure relevant, reliable, and sufficiently robust PRO instruments are developed, modified, or adapted using the least burdensome approach.

Principles to be considered for PRO Instruments

FDA outlines the following principles that should be considered when using PRO instruments in the evaluation of the medical device:

  • Establish and define the concept of interest (COI) the PRO instrument is intended to capture;
  • Clearly identify the role of the PRO in the clinical study protocol and statistical analysis plan;
  • Provide evidence showing that the PRO instrument reliably assesses the concept of interest; and
  • Effectively and appropriately communicate the PRO-related results in the labeling to inform healthcare provider and patient decision making.

PRO Fit-For-Purpose Criteria

As it relates to the fit-for-purpose criteria, FDA outlines three overarching principles to determine if a PRO instrument is fit for its purpose:

  1. Is the concept being measured by the PRO instrument meaningful to patients and would a change in the concept of interest be meaningful to patients?
  2. What role will the PRO instrument serve in the clinical study protocol and statistical analysis plan?
  3. Does the evidence support its use in measuring the concept of interest as specified in the clinical study protocol and statistical analysis plan?

While these points seem straightforward and clear, they are important ones that should be considered when evaluating the PRO instrument will meet FDA’s expectations. Of note, the draft guidance does not provide what level of evidence is required for a PRO to be fit-for-purpose.

PRO Instrument Best Practices

Finally, the draft guidance provides other best practices for PRO development, which include measuring concepts important to patients, ensuring PRO instruments are understandable to patients, and being clear about the role of PRO instrument in the Clinical Study Protocol and Statistical Analysis Plan.

Overall, the draft guidance provides insight into FDA’s current thinking for use of PRO instruments in medical device clinical studies and should be analyzed by sponsors considering PRO instruments. Interested stakeholders can submit comments on this draft guidance for FDA’s consideration until October 30, 2020 to docket FDA-2020-D-1564 available at https://beta.regulations.gov/docket/FDA-2020-D-1564.

[1] FDA Draft Guidance, “Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation” (August 2020) (available at: https://www.fda.gov/media/141565/download).