FDA Updates the Clinical Outcome Assessment Compendium
(Thursday, December 9, 2021)
Clinical Outcome Assessments (COAs) are the measures of the clinical response to a treatment as observed by a patient, their physician, an independent observer, or an objective measure of the patient’s performance following treatment with a drug or biologic. COAs are closely related to patient focused drug development where clinically relevant outcomes can and have been used to evaluate an investigational treatment in support of its market approval. The most popular COAs are the Patient Reported Outcomes (PROs) but FDA has also accepted Physician/Clinician Reported Outcomes (ClinRO), Observer Reported Outcomes (ObsRO) and Performance Outcome (PerfO) in support of market approvals of drugs and biologics. Once used in an NDA or BLA application, the COAs can be theoretically used by other developers of treatments for the same indication. Hence, FDA started publishing a list of COAs used in the past as the COA Compendium in 2016. The 2021 version is an update of this compendium. This list is the best place to get started with a patient focused drug development program. Although COAs are useful for all indications, they are particularly valuable for indications that are hard to evaluate based on biochemical endpoints. In such cases, the COAs are the true measure of the benefits of a given treatment. FDA does not guarantee that the a given COA listed in the compendium will be automatically allowed for future indications, but the list is the best place to start for new developers. You still are expected to consider disease specific guidance documents, and FDA feedback in meetings. COAs are different from biomarkers and surrogate markers but could be primary endpoints in clinical trials. Use of COAs has become increasing prominent in NDA and BLA applications over the last 5 years. The COA compendium list more than 230 COAs that have been used for market approval decisions by 7 Offices and 20 Divisions in CDER. This list does not include the COAs that are in development, those currently being used in ongoing clinical trials, or those used for biologics reviewed by CBER. So, there are a lot more COAs being used currently than are evident from the compendium. It should be heartening for developers that FDA is open to considering non-conventional clinical development programs using COAs and one should discuss all options with FDA, even if no one has done it before per their knowledge.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC