At the FDA's Center for Devices and Radiological Health (CDRH), we strive to ensure patients and their care partners remain the focus of our regulatory decision-making process. One way we do that is by encouraging the inclusion of clinical outcome assessments (COAs) in the evaluation of medical devices.
June 29, 2022 update: The Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, and the Center for Devices and Radiological Health are announcing the availability of the draft guidance Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments, which is available for comment. Read the draft guidance. Visit the Federal Register for information on how to comment on the guidance.
On this page:
- What Are Clinical Outcome Assessments?
- How CDRH Uses COAs in Regulatory Decision Making
- COA Case Studies
- CDRH Collaborations, Articles, and Workshops on PROs
- Planning to Incorporate COAs in a Regulatory Submission?
- Contact Us
A clinical outcome assessment (COA) describes or reflects how a person feels, functions, or survives and can be reported by a health care provider, a patient, a non-clinical observer (such as a parent), or through performance of an activity or task. There are four types of COAs:
- Patient-reported outcomes (PROs),
- Clinician-reported outcomes (ClinROs),
- Observer-reported outcomes (ObsROs), and
- Performance outcomes (PerfOs).
While each type of COA focuses on the patient, they provide a different perspective on a patient's health status.
PROs provide information on the patient's health condition as directly reported by the patient, without outside interpretation from anyone. These outcomes are assessed using PRO instruments such as questionnaires, numeric rating scales, or diaries.
ClinROs are reports coming from a trained health-care professional regarding their interpretation of signs or behaviors that can be observed related to a patient's disease or condition.
ObsROs are assessments of observable signs, events, or behaviors related to a patient's health condition as reported by individuals who observe the patient in daily life, like parents or caregivers.
PerfOs are measurements collected when a patient is asked to complete a well-defined, repeatable, and standardized task, such as reading an eye chart.
For regulatory purposes, high-quality information from COAs can provide valuable evidence for benefit-risk assessments. They can also be used in medical device labeling to communicate the effect of a treatment on patient symptoms and functioning. COAs may be used to determine who is eligible for a clinical study and measure how well the device performs in treating or diagnosing the condition. COAs may also be used to help measure the safety of the device. Evidence from COAs may also be useful to payors and healthcare providers.
These case studies highlight the roles COAs can play in clinical investigations supporting medical device submissions. They are not intended to be a comprehensive review of the clinical studies associated with each submission.
- Patient-Reported Outcomes in Intraocular Lens Labeling
- Observer and Patient-Reported Outcomes for Ear Tube Delivery System
- Clinical Outcome Assessments Inform Indications for Use in Breakthrough Heart Failure Symptoms Device
- Clinical Outcome Assessments Complement Clinical Evidence in Label Expansion for Transcatheter Aortic Valve Replacement
- Patient-Reported Outcomes in Endpoints for Devices Treating Benign Prostate Hyperplasia Symptoms
- Clinical Outcome Assessments Measure Aesthetic Procedure Success
- Clinical Outcome Assessments in Composite Endpoint for Upper Extremity Prosthetics
- Clinician-Reported Outcomes in Co-Primary Endpoint for Stroke Treatment Device
- Clinical Outcome Assessments in Composite Endpoints for Orthopedics
CDRH regularly collaborates with professional societies, academic researchers, patient groups, public-private partnerships, and other stakeholders to advance the development and validation of COAs for regulatory use. For additional information about CDRH's collaborative work and articles related to COAs, see the Collaborations and Articles sections below. For additional information about our workshop on COAs, see Medical Devices Virtual Public Meeting - Patient-Reported Outcomes (PROs) and Medical Device Evaluation: From Conception to Implementation.
- 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-Stanford CERSI)
- Adapting a Measure of Heart Failure to an Adolescent Population (UCSF-Stanford CERSI)
- Qualitative analysis of gender differences in heart failure PROs (Duke University, Yale-Mayo CERSI)
- Patient-Reported Outcomes for Dialysis Vascular Access (Kidney Health Initiative)
- Patient-Reported Outcomes for Muscle Cramping in Patients on Dialysis (Kidney Health Initiative)
- Patient-Reported Outcome Measure Development for Intraocular Lens Symptoms (American Academy of Ophthalmology Task Force & Industry Collaboration)
- Patient Reported Outcomes for Minimally Invasive Glaucoma Surgery (MIGS) (UCSF-Stanford CERSI, Johns Hopkins University 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
- Flythe, J. E., et al. (2020). "Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices." CJASN 15(10): 1522-1530
- Chen, C., Ridgeway, J.L., Bocell, F.D., Tanenbaum, M.L., Hood, K.K., Behnken, E., Schmidt, J., Hanes, S.J., Saha, A., Caldwell, B., Tarver, M., Peiris, V., Almond, C.S., Johnson, J.N. (2020). Qualitative Exploration of the Pediatric Heart Failure Experience for Development of a Patient-Reported Outcome Measure. The Journal of Heart and Lung Transplantation, 39(4), S452. https://doi.org/10.1016/j.healun.2020.01.280
- Hejjaji, V., Tang, Y., Coles, T., Jones, P. G., Reeve, B. B., Mentz, R. J., . . . Spertus, J. A. (2021). Psychometric Evaluation of the Kansas City Cardiomyopathy Questionnaire in Men and Women With Heart Failure. Circulation: Heart Failure, 14(9),doi:10.1161/CIRCHEARTFAILURE.120.008284
- Cui, Q. N., Hays, R. D., Tarver, M. E., Spaeth, G. L., Paz, S. H., Weidmer, B., . . . Singh, K. (2021). Vision-Targeted Health-Related Quality-of-Life Survey for Evaluating Minimally Invasive Glaucoma Surgery. American Journal of Ophthalmology, 229, 145-151. doi:https://doi.org/10.1016/j.ajo.2021.03.064
- Davis J, Olazo K, Sierra M, Tarver ME, Caldwell B, Saha A, Lisker S, Lyles C, Sarkar U. Do patient-reported outcome measures measure up? A qualitative study to examine perceptions and experiences with heart failure proms among diverse, low-income patients. J Patient Rep Outcomes. 2022 Jan 15;6(1):6. doi: 10.1186/s41687-022-00410-9
- Matts, S.T., Webber, C.M., Bocell, F.D. et al. Inclusion of patient-reported outcome instruments in US FDA medical device marketing authorizations. J Patient Rep Outcomes 6, 38 (2022). https://doi.org/10.1186/s41687-022-00444-z
If you are interested in incorporating COAs in your regulatory submissions, CDRH has multiple resources to help with selecting, using, developing and modifying appropriate COAs. The FDA has issued guidance documents that may help inform your approach to including COAs in the evaluation of medical devices.
- Final Guidance: Principles for Selecting, Developing, Modifying, and Adapting Patient-Reported Outcome Instruments for Use in Medical Device Evaluation (January 2022)
- Final Guidance: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims (December 2009)
Q-submission: We invite sponsors to discuss their plan to use COAs, including adapting or developing PRO instruments, with CDRH through the Q-submission program. A pre-submission can initiate early discussions with regulatory staff, as described in the guidance document Requests for Feedback and Meetings for Medical Device Submissions: The Q-Submission Program.
Medical Device Development Tools: COAs are one type of tool that can be qualified under the Medical Device Development Tools (MDDT) program. This program is intended to facilitate device development and timely evaluation of medical devices, and promote innovation, by providing a more efficient and predictable means for collecting the necessary information to support regulatory submissions and associated decision making. The FDA qualifies 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. See Qualified Medical Device Development Tools (MDDTs) for a list of qualified tools.
PRO Report: This document provides more detailed information on PROs. It discusses the value of using PROs in regulatory submissions, reimbursement decisions, and clinical practice. Additionally, CDRH efforts and accomplishments to date relating to PROs are highlighted.
PRO Compendium: The PRO Compendium (XLS) lists some, but not all, PRO instruments that have been used and reported in medical device premarket clinical investigations submitted to CDRH. We encourage sponsors interested in using a PRO instrument in a clinical investigation to schedule a pre-submission meeting to discuss their plans.
If you have questions about clinical outcome assessments, email CDRH-PRO@fda.hhs.gov.