Establishment of a Rare Disease Clinical Outcome Assessment (COA) Consortium
Over 350 million people worldwide are impacted by one of more than 7,000 recognized rare diseases. These conditions result in substantial burden on patients, families, communities, and healthcare systems. Most of these conditions are serious and life-altering, frequently requiring lifetime treatment. Approximately 50% of rare diseases impact children. Current estimates are that less than 5% of rare diseases have approved treatments. Appropriate clinical outcome assessments (COAs) to measure clinical benefit of treatment do not exist or remain unidentified for far too many rare diseases. With no approved therapies for most rare diseases and considerable uncertainty around appropriate treatment trial endpoints, a significant unmet public health need remains unfulfilled.
The Rare Disease COA Consortium seeks to meet this need with the creation of the Rare Disease COA Resource describing potentially fit-for-purpose publicly available COAs for use across rare disease treatment programs.
To enable precompetitive, multi-stakeholder collaboration aimed at identifying scientifically sound tools and methodologies for collecting clinically meaningful outcomes data in treatment trials for rare diseases.
To be an essential catalyst in medical product development for measuring what matters to people with rare diseases.
Rare Disease COA Resource
The Rare Disease Clinical Outcomes Assessment (COA) Consortium aims to accelerate the development of new medical products intended to safely and effectively treat people with rare diseases with the creation and curation of a Rare Disease COA Resource. This resource will provide information on publicly available COAs identified as potentially fit-for-purpose endpoint measures in treatment trials for rare diseases. The premise is that existing COAs may be used or modified for use across multiple diseases sharing common characteristics.
A domain-level approach was selected to begin populating the Rare Disease COA Resource. The first iteration of the Rare Disease COA Resource focuses on the assessment of daily function in pediatric, non-oncologic populations. This overarching domain was broken down into the daily function subdomains of self-care, gross motor function, fine motor function, and communication. Other subdomains will be added subsequently.
Access the Rare Disease COA Resource
For questions or additional information about membership in the Rare Disease COA Consortium, please contact Lindsey Murray at email@example.com.
Specific funding for the establishment of the Rare Disease COA Consortium was provided by FDA grant U01FD006882.