The FDA has Awarded Over $18 Million for Rare Disease Research

The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news, you can read the source press release at the FDA’s website by clicking here.

The Challenges of Rare Disease Research

Developing treatments for rare conditions can often be particularly challenging. As the FDA article acknowledges, organisations may be less likely to invest in researching treatments for rare diseases due to their small patient populations. While certain regulations, such as Orphan Drug Designation, exist to encourage developers to work on treatments for rare diseases, continued investment in research is needed.

The Orphan Products Clinical Trials Grants Program

Created in 1983, this program, which awards grants, aims to support the clinical development of drugs and other medical products that may benefit rare disease patients. So far it has awarded over $400 million in funding to more than 600 clinical studies. The grants support early-stage studies and can help to encourage more funders to invest. So far, sixty of the products this program has invested in have gone on to receive marketing approval.

Funding for Studies

The twelve grants are going towards research into a range of rare diseases. One-third of the awards will be dedicated to rare cancer research, and one quarter will be studies into rare endocrine disorders. The research projects will also involve participants of different ages, with just under half (42%) of the studies enrolling children and adolescents.

The conditions that will be researched using these grants are: Stargardt disease, primary sclerosing cholangitis, Cushing disease, advanced pancreatic cancer, head and neck squamous cell carcinoma, dystrophic epidermolysis bullosa, myelodysplastic syndrome or acute myeloid leukaemia, graft versus host disease, congenital ichthyosis, dopamine agonist intolerant hyperprolactinemia, congenital adrenal hyperplasia, bronchopulmonary dysplasia.

About Orphan Drugs

Rare conditions are often considered to be ‘Orphan diseases’ by the FDA. The Orphan Drug Act grants special status to drugs or products that are intended to benefit patients who have Orphan conditions. Orphan products may be eligible for various benefits, such as tax credits for clinical trials, and waived prescription drug user fees, amongst others. This system is designed to help get drugs to patients with rare conditions more quickly. To find out more about Orphan drugs, click here.


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