FDA awards two COA grants to study neurodevelopment disorders and nephrotic syndrome

The US Food and Drug Administration (FDA) announced the awarding of two grants under its Clinical Outcome Assessments (COAs) program to inform the selection of clinical trial endpoints for drugs to treat neurodevelopmental disorders (NDDs) and nephrotic syndrome.
 
The grants “provide avenues to advance the use of patient input as an important part of drug development that can foster innovation and the availability of safe and effective drugs,” said the agency’s 4 May announcement. They have been awarded to researchers at the University of Michigan, Northwestern University and Duke University.
 
These grants are being made under the 21^st Century Cures Act and the sixth authorization for the Prescription Drug User Fee Act, which call for FDA to publish a series of patient-focused guidance documents intended to gain patient’ perspectives on the use of “sound data collection instruments in clinical trials,” according to the funding notice published on 9 July 2020.
 
The end goal is to develop assessments that allow patient input to be used as “a direct source of evidence” when making benefit-risk assessments about drugs, according to FDA’s announcement of the awards.
 
The 2021 awards are for the following:
 
A project to develop and establish a core set of COAs for nephrotic syndrome, with a primary focus on fluid overload. The FDA said that “the investigators expect their work to result in a core set of highly relevant COAs that will advance patient-focused drug development in nephrotic syndrome.” The grant will go to a team of researchers at the University of Michigan and Northwestern University.
 
A project to expand the observer reported communication ability (ORCA) measure for evaluating communication abilities of individuals with NDDs. This project will expand on the existing measurement tool created by Duke University to assess caregiver observations of expressive communication abilities in children with Angelman Syndrome, a severe neurodevelopmental disorder. FDA said that “investigators expect their work to result in an expanded use of the ORCA measure in the research field that can capture communication ability across a diverse range of NDDs and provide an endpoint framework for researchers to evaluate interventions using an outcome that is important to families of children and adolescents with NDDs.”
 
Although FDA did not include award amounts in its announcement, the request for applications (RFA) noted that FDA's Center for Drug Evaluation and Research (CDER) planned to commit up to $4.2 million for FY21 to fund up to three awards.
 
In 2019, the FDA awarded three grants in the areas of migraine, acute pain in infants and young children and physical function severity across a range of chronic conditions. No COA grants were awarded in FY20, an FDA spokesperson confirmed to Focus. (RELATED: FDA awards $4 million in grants to support development of COAs, Regulatory Focus,  16 September 2019)
 
FDA’s COA announcement