FDA and Rare Diseases

FDA is in a unique position to help those who suffer from rare diseases. The agency’s rare disease initiatives include the following:

  • FDA’s Office of Orphan Products Development gives grants to further the development of drugs, biologics (such as vaccines or blood products), medical devices, and medical foods for the treatment of rare diseases. (Medical foods are specially made and processed foods, used under a health care professional’s supervision, to meet a patient’s distinctive nutritional requirements.)
  • FDA plays an important role in granting orphan designations and marketing approvals for drugs, biologics, and medical devices to treat rare diseases. Designating a potential product as “orphan” encourages companies to develop that product by giving them financial and other incentives.
  • FDA’s Office of New Drugs within the Center for Drug Evaluation and Research established the Rare Diseases Program to assist and support the research, development, regulation, and approval of drug and biologic products for the treatment of rare disorders.
  • As countries combine their efforts to spotlight “Rare Diseases and Health Inequalities,” FDA continues to collaborate with international regulatory agencies to address unmet medical needs of patients with rare diseases globally. FDA’s Office of Orphan Products Development and the European Medicines Agency (EMA) work together regularly and have a common application for orphan product designation.
  • FDA advocates for rare disease awareness and treatments through work with patients and patient organizations.