Frequently Asked Questions

The Resource can assist investigators with selection of publicly available COAs that have the potential to be used to support efficacy endpoints in treatment trials for rare diseases. While the Resource is free to use and does not require a subscription to access, there may be a cost associated with the use of a selected COA measure.

The Resource is an initiative of the Critical Path Institute’s Rare Disease Clinical Outcome Assessment (COA) Consortium, whose mission is 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.

More than 350 million people worldwide are impacted by one of more than 7,000 rare diseases. These conditions result in substantial burden on patients, families, communities and global health care systems. Most of these conditions are serious and life-altering, frequently requiring lifetime treatment. Nearly 50% of rare diseases impact children, of whom 30% will die by age 5. It is currently estimated that less than 10% of rare diseases have approved treatments. COAs to measure clinical benefit of treatment do not exist or remain unidentified for the majority of 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 address this need with the creation of the Rare Disease COA Resource, a searchable database that aims to simplify COA selection for use in rare disease medical product development by providing information on published COAs that have the potential to be used to support efficacy endpoints in treatment trials for rare diseases.

To conduct a COA search, users can navigate to any page on the Resource website and enter the desired search terms within the “COA Search” field, and press “Enter” on their keyboard. If the search yields more than one COA, the user can select multiple measures for comparison.

A complete list of all COAs currently found in the Resource can be accessed here: https://rdcoas.c-path.org/resource-coas/ Users may filter the list by name, acronym, COA type, and subdomain. The list can also be exported and downloaded in .csv format.

Copies of COAs must be obtained in accordance with developer requirements and copyright law. Information on how to obtain copies of COAs included in the Rare Disease COA Resource is available within the search results for each COA.

COAs included in the Rare Disease COA Resource were identified through an extensive landscape review, followed by an in-depth gap analysis evaluating each COA against regulatory expectations. Final selection of COAs for inclusion in the Rare Disease COA Resource was determined through a consensus process involving both the Advisory Panel and the Rare Disease COA Resource Development Subcommittee.

Importantly, only COAs supported by published manuscripts, user manuals, and/or other publicly available documentation demonstrating robust development and methodological rigor were included in the Resource.

The Rare Disease COA Resource will be updated periodically; however, it is possible that additional COAs have been developed since completion of a given domain review. Suggestions for COAs to be considered for inclusion in future iterations of the Rare Disease COA Resource may be submitted to: coapadmin@c-path.org.

We welcome suggestions from users to help improve the Resource. These can be sent to: coapadmin@c-path.org