How is the Rare Disease Clinical Outcome Assessment (COA) Resource different from other searchable COA databases?

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.

Why was the Resource developed?

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.

How do I conduct a search for a COA?

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.

Where can I find a list of all COAs currently included in the Resource?

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.

Why isn’t a copy of each COA provided?

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.

I searched for a COA that is not currently included in the Resource. Why was it not included? How can I recommend a COA for inclusion in the 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 over time. A consensus process between an advisory panel and the Rare Disease COA Resource Development Subcommittee determined the final COAs to include in the Rare Disease COA Resource. Suggestions for COAs to be included in future iterations of the Rare Disease COA Resource may be sent to: coapadmin@c-path.org