RARE Daily

FDA Issues Grant to C-Path Institute to Establish Rare Disease Clinical Outcome Assessment Consortium

March 20, 2020

Rare Daily Staff

The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research has awarded a grant to the Critical Path Institute to establish a Rare Disease Clinical Outcome Assessment Consortium.

The National Organization for Rare Disorders was named a sub-awardee on the grant.

Once established, the Rare Disease Clinical Outcome Assessment Consortium’s activities will be aimed at accelerating the development of new medical products to treat people with rare diseases by creating and curating a resource of information on publicly available clinical outcome assessments identified as potential fit-for-purpose endpoint measures in treatment trials for rare diseases.

The expectation is that existing clinical outcome assessments may be able to be used or modified for use across multiple diseases sharing common characteristics.

As a first step toward establishing the new consortium, C-Path has created the Rare Disease Subcommittee within its Patient-Reported Outcome Consortium. The Patient-Reported Outcome Consortium will serve as an incubator for a pre-competitive, multi-stakeholder consortium within C-Path’s Clinical Outcome Assessment program.

The Patient-Reported Outcome Consortium’s Rare Disease Subcommittee includes representatives from C-Path, NORD, FDA, the Patient-Centered Outcomes Research Institute, the National Center for Advancing Translational Sciences, and biopharmaceutical firms within the Patient-Reported Outcome Consortium that are developing treatments for rare diseases.

In addition, plans are underway to enable rare disease-focused biotech firms not currently members of the Patient-Reported Outcome Consortium to be included in the strategic planning for the new consortium.

As the Rare Disease Subcommittee lays the groundwork for the consortium, it has launched two initiatives. The first involves a multi-pronged effort aimed at tackling challenges in the assessment of clinical benefit in rare disease treatment trials. The first pilot project under this initiative is to identify clinical outcome assessments that can be used in children to assess activities of daily living, which is a meaningful aspect of life impacted by many rare diseases.

The second initiative involves a literature review to explore ways in which researchers have handled heterogeneity in clinical trials including an examination of the advantages and disadvantages of the approaches to personalizing endpoints. Development of best practice recommendations for assessing clinical benefit in rare disease trials will be explored.

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