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Posted 06 October 2017 | By Michael Mezher
The US Food and Drug Administration (FDA) on Friday awarded $32 million in research grants to fund 15 clinical studies and six natural history studies for rare diseases.
The grants are being funded through FDA's orphan products grants program, with $22 million going to the clinical studies and $9.8 million in combined funding from FDA and the National Center for Advancing Translational Sciences' (NCATS) Therapeutics for Rare and Neglected Diseases program going to fund the natural history studies.
About a third of the clinical trials being funded by the grants are aimed at rare forms of cancer, particularly cancers that affect the brain and peripheral nervous system. Other studies set to receive funding include trials to address unmet needs for patients with diseases such as sickle cell disease, Prader-Willi syndrome and cystine nephrolithiasis.
In total, FDA says it received 76 grant applications for clinical trials in FY 2017 and 80 grant applications for natural history studies under the grants program.
According to FDA, the orphan products grants program has paid out more than $390 million to more than 600 clinical studies since its creation under the 1983 Orphan Drug Act. The agency also says that studies funded by the program have been used to support the approval of 55 orphan drugs.
This marks the first time FDA is funding natural history studies through its grants program after the agency announced the availability of the grants in February 2016.
Natural history studies are used to gain a greater understanding of a disease and its progression. For rare diseases, natural history studies can be an important tool for establishing baseline information about a disease and identifying potential outcomes or biomarkers to use in clinical trials.
In some cases, natural history data can be used as a historical control in settings where patient recruitment is especially challenging or where a placebo arm would be unethical.
"One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials," said Nora Yang, director of portfolio management and strategic operations in NCATS' Division of Pre-Clinical Innovation.
Speaking at RAPS' 2017 Convergence in September, FDA Commissioner Scott Gottlieb said the agency is working with researchers to develop natural history models to simulate placebo arms for studies in diseases such as Alzheimer's and muscular dystrophy.
"If we're able to make better use of rigorous, reliable natural history models, especially for rare diseases, it can help us make the clinical trial process more efficient," Gottlieb said.
FDA: Clinical Trials, Natural History Studies
Tags: Natural History Studies, Rare Diseases, Orphan Drugs, Grants
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