10/27/2020

The Food and Drug Administration (FDA) recently released a draft guidance entitled “Principles for Selecting, Developing, Modifying and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Clinical Evaluation.” This draft guidance follows a guidance from 2019, “Patient-Reported Outcome Measures: Use in Medical Product Development to support Labeling Claims”, and a push starting in 2016 by the Center for Devices and Radiological Health (CDRH) to incorporate the use of patient-reported outcomes (PROs) in evaluating medical devices.

This draft guidance aims to:

Outline principles to consider when using PRO instruments
Give recommendations of how to assure instruments are fit-for-purpose
Describe best practices to establish PRO instruments as impactful, dependable and user friendly

Background:

What is a Patient-reported outcome (PRO)?

A PRO is a type of clinical outcome assessment based on a report by the patient directly, with no involvement of a patient’s clinician.

What is a Patient-reported outcome instrument?

A measure or tool used to collect the PRO such as patient diaries and questionnaires to measure symptoms.

What does fit-for-purpose mean?

This describes a validated conclusion that a tool is deemed acceptable to be used in its context in measuring outcomes of a clinical trial.

Summary of the Guidance:

PRO instruments are able to measure the impact of a medical device on a patient’s well-being. According to the guidance, PRO tools are “qualified under the Medical Device Development Tools (MDDT) program as tools that medical device sponsors can use in the development and evaluation of medical devices.”
PRO instruments should measure concepts that are important to patients and be easily understood by patients.
PRO instrument’s context of use (COU) should be established in the trial protocol and statistical analysis plan.
Sponsors and other stakeholders are encouraged to use existing instruments but also to collaborate with others in the pre-competitive space to modify and improve existing PRO tools.
Alternative Platforms should be explored for incorporating PRO instruments into real-world data sources.

If using electronic patient-reported outcome (ePRO) formats, bear in mind the most recent EMA guidance on quality management of electronic systems as well as the relevant sections of ICH E6 (R2) that pertain to qualification and validation of computerized systems, quality control and recording, handling and storage of clinical data. Utilizing innovative technologies in trial management lends to patient ease of use and more easily accessible data for stakeholders. The clinical systems must be validated to ensure patient confidentiality and safety as well as data integrity.

The FDA believes the information obtained from the patient’s perspective, if included on medical device labeling, could help patients and physicians make more informed decisions on possible treatment and/or management options.

How do you feel about the use of PRO instruments supporting regulatory submissions? You may comment on the draft guidance here through October 30, 2020.

-The Clinical Pathways Team

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