Rare Daily Staff

The U.S. Food and Drug Administration said it has awarded two new research grants totaling more than $4.1 million over the next four years to fund natural history studies in medullary thyroid carcinoma and cardiac disease in patients with Duchenne muscular dystrophy.

Natural history studies closely look at how specific diseases progress over time. The natural history of a disease is the course a disease takes from its onset, through the presymptomatic and clinical stages, to an outcome in the absence of treatment. Information from natural history studies can facilitate design of efficient clinical trials to test future treatments.

“These studies provide important roadmaps for how to conduct subsequent studies,” said FDA Principal Deputy Commissioner Amy Abernethy. “With the natural history of many rare diseases remaining relatively unknown, efficiently developing diagnostics and therapies for patients poses unique challenges. Promoting and conducting work in this area is critical.”

The agency awarded approximately $1.7 million to the University of Texas MD Anderson Cancer Center for a prospective study in medullary thyroid carcinoma. The study seeks to leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions regarding initiation of and adherence to chemotherapy in medullary thyroid cancer. The hope is to incorporate the information into the design of clinical trials of emerging therapies for this disease.

The FDA also awarded approximately $2.4 million to Vanderbilt University Medical Center for a prospective study in cardiac disease in Duchenne muscular dystrophy. The study aims to focus on heart muscle disease, which is the leading cause of death in Duchenne muscular dystrophy. The study will combine genetic differences with imaging and blood biomarkers to identify surrogate biomarkers that predict the risk of cardiac dysfunction in Duchenne muscular dystrophy and other related diseases. The information has the potential to improve future clinical trial efficiency in these diseases by decreasing their size and cost.

The FDA received 31 grant applications that were reviewed and evaluated for scientific and technical merit by more than 45 rare disease, natural history, regulatory, and statistical experts that included representatives from academia, patient groups, the National Institutes of Health, and the FDA.

Photo: FDA Principal Deputy Commissioner Amy Abernethy

X