A clinical outcome assessment (COA) describes or reflects how a person feels, functions, or survives and can be reported by a health care provider or a non-clinical observer (such as a parent), through performance of an activity or task, or by the patient. There are four types of COAs:

• Patient-reported outcomes (PROs),
• Clinician-reported outcomes (ClinROs),
• Observer-reported outcomes (ObsROs), and
• Performance outcomes (PerfOs).

While each 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.

How CDRH Uses COAs in Regulatory Decision Making

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.
 

Incorporation of Clinical Outcome Assessments (COAs) in a Regulatory Submission

CDRH has multiple resources to help with selecting, using, developing and modifying appropriate COAs. The FDA has issued guidance documents and discussion guides that may help inform the approach to include COAs in the evaluation of medical devices.

Q-submission: FDA invites 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

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. COAs are one type of tool that can be qualified under the MDDT program.

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.