Over the next four years, more than $22 million in grants have been awarded for clinical trials and research into treatment for patients with rare diseases, the US Food and Drug Administration (FDA) has announced. In a statement released by the regulatory body, FDA Commissioner Scott Gottlieb, MD, suggested the grants could help to reduce the price of medication for many patients with certain rare conditions.
The FDA received 76 applications for the 2017 fiscal year, of which the funding rate was 20 percent. The grants are awarded through the Office of Orphan Products Development's (OOPD) Orphan Products Clinical Trials Grants Program, which encourages clinical development of drugs and other products used to treat rare conditions. Since it was established in 1983, the OOPD has funded more than 590 studies and resulted in over 60 new products gaining market approval.
Orphan Drug Designation is granted to products developed to treat rare diseases. Pharmaceutical companies receive a number of benefits designed to incentivize the development of crucial medicines that may not otherwise be profitable to develop. Among the top-selling orphan drugs in the US last year were Copaxone (glatiramer acetate), Rituxan (rituximab), and Sensipar (cinacalcet hydrochloride).
A total of 15 new grants were announced by the FDA, with around one-third of the funds allocated to cancer research, particularly cancers impacting the brain and peripheral nervous system, such as glioblastoma and anaplastic astrocytoma. Two of the largest grants were awarded to a Phase 2 Study of ABI-009 for the Treatment of Advanced Perivascular Epithelioid Cell Tumors and a Phase 2 Study of Topical Sodium Nitrite for the Treatment of patients with Sickle Cell Disease & Leg Ulcers. The clinical trials, conducted by AADi, LLC and the Albert Einstein College of Medicine respectively, will receive $2 million over four years.
Other diseases with unmet medical needs and treatments to benefit from the latest FDA grants include idiopathic osteoporosis in premenopausal women, Stargardt disease, and treatment of hyperphagia in Prader-Willi syndrome. A grant was also allocated to a study into a new combination of existing antibiotics for treating pulmonary tuberculosis and multidrug-resistant tuberculosis.
"By helping to support the cost of development of these potential new drugs, and reduce some of the financial risks, we also hope that these grants will lower the cost of the capital needed to develop these products, boost competition and translate into lower prices for successful medicines," Gottlieb stated. He added this could further improve access to new therapies for patients with rare diseases.
The National Organization for Rare Disorders estimates 30 million Americans suffer from 7,000 rare diseases. According to the EvaluatePharma Orphan Drug Report 2017 orphan drugs are set to account for 21.4 percent of branded prescriptions worldwide by 2022.