At the Center for Devices and Radiological Health (CDRH), we strive to ensure patients and their care-partners stay at the center of our regulatory decision-making process. One way we incorporate the patient perspective as evidence in our decisions, is by encouraging the inclusion of patient-reported outcomes measures (PROMs) in the evaluation of medical devices.
This web page focuses on the use of patient-reported outcomes (PRO) as one type of patient input.
According to the FDA-NIH Biomarkers, EndpointS and other Tools (BEST) Working Group, a patient-reported outcome (PRO) is “a measurement based on a report that comes directly from the patient (i.e., study subject) about the status of a patient’s health condition without amendment or interpretation of the patient’s response by a clinician or anyone else.” PROs may capture symptoms such as itching as well as functioning in daily life such as the ability to carry groceries. A patient-reported outcome measure (PROM) is the instrument or tool, typically a questionnaire or diary, used to gather the health status of the patient.
Examples of PROMs include:
- Numeric rating scale of pain intensity
- Patient-completed diary of seizure episodes
- Verbal rating of severity of visual symptoms
PROMs can be used to capture a patient’s everyday experience with a medical device, including experience outside of the clinician’s office, and the effects of a treatment on a patient’s activities of daily living. In some cases, PROMs enable us to measure important health status information that cannot yet be detected by other measures, such as pain.
For regulatory purposes, high-quality information from PROMs can provide valuable evidence for benefit-risk assessments and can be used in medical device labeling to communicate the effect of a treatment on patient symptoms and functioning. PROMs can be used to determine who should be included in a clinical study, measure effectiveness of a device in treating or diagnosing the condition, and determine the safety of the device in terms of symptom and functional impacts.
Beyond regulatory uses, evidence from PROMs may be useful for “downstream” decision-makers in the pathway to market, such as payers, health care systems, and providers.
CDRH regularly collaborates with professional societies, academic researchers, patient groups, public-private partnerships, and other stakeholders to advance the development and validation of PROs for regulatory use.
- LASIK Quality of Life Collaboration Project (National Eye Institute and Department of Defense)
- The Impact of Race and Ethnicity on Responses to Heart Failure Patient-Reported Outcome Measures (UCSF CERSI)
- Adapting a Measure of Heart Failure to an Adolescent Population (UCSF CERSI)
- Analysis of Sex Specific Differences in Quality of Life Measures for Heart Failure (OWH Grant, (Yale-Mayo CERSI)
- Comparing Qualitative and Quantitative Approaches to Eliciting Patient Preferences: A Case Study on Innovative Upper Limb Prosthesis (Hopkins CERSI)
- Patient-Reported Outcome Measures for Novel Renal Devices (Kidney Health Initiative)
- Patient-Reported Outcome Measure Development for Intraocular Lens Symptoms (American Academy of Ophthalmology Task Force & Industry Collaboration)
- Patient-Reported Outcome Measure Development for Mild to Moderate Glaucoma (UCSF-CERSI)
- Post-market surveillance with a novel mHealth platform (Yale-Mayo CERSI)
- Value and Use of Patient-Reported Outcomes (PROs) in Assessing Effects of Medical Devices (CDRH Strategic Priorities 2016-2017)
- FDA issues first report on patient reported outcomes in medical device premarket submissions and postmarket studies (FDA In Brief)
- Articles from the LASIK Quality of Life Collaboration Project
If you are interested in incorporating PROMs in your regulatory submissions, CDRH has produced several resources to assist in selecting or developing an appropriate PROM. We invite sponsors to discuss their plan to use, adapt, or develop PROMs with CDRH through the Q-submission program by submitting a pre-submission as described in the guidance document, Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program.
PRO Compendium: CDRH sees many examples of PROMs used in clinical investigations every year, but individual sponsors may not be aware of the utility of PROs in a particular device area. PRO data have been used to inform regulatory decisions across a wide variety of devices and indications at CDRH. The PRO Compendium (XLS) lists some, but not all, of the PROMs that can be used and reported in medical device pre-market clinical investigations submitted to CDRH. We encourage sponsors interested in using a PROM in a clinical investigation to schedule a pre-submission meeting to discuss their plans.
PRO Case Studies: To clarify the potential application of PROMs in clinical investigations, the PRO Case Studies (PDF) include detailed and self-contained examples of PROs used in CDRH regulatory submissions. The PROs highlighted in these examples do not encompass all potential uses of PROMs in device evaluation, but instead highlight various contexts in which PROMs have been used in regulatory device submissions, such as effectiveness and safety endpoints.
Medical Device Development Tools: The Medical Device Development Tools (MDDT) program enables the FDA to qualify tools that medical device sponsors can use in the development and evaluation of medical devices. Qualification means CDRH has evaluated the tool and concurs with available supporting evidence that the tool produces scientifically-plausible measurements and works as intended within the specified context of use. PROMs are one type of tool that can be qualified under the MDDT program.
CDRH continues to work with the medical device ecosystem to include the patient perspective in regulatory submissions, where appropriate. We invite sponsors to discuss the incorporation of PROMs in regulatory submissions through the Q-Submission Program.
If you have questions about patient-reported outcomes, email CDRH-PRO@fda.hhs.gov