The FDA's Rare Diseases Program: Driving the development of orphan drugs

Rare diseases are characterized by the US Food and Drug Administration (FDA) as those conditions that affect less than 200,000 people in the American population. While 200,000 may seem a large number, it is obviously characteristic of rare diseases to be…well, rare, says Dr Nicola Davies in her regular exclusive contribution to The Pharma Letter.

For example, Cushing syndrome - an endocrine disorder that affects adults 20-50 years of age, has a prevalence rate of 5.9 for every 100,000 people. This small statistic of incidence is just one of the hurdles that pharma companies must face in the development of orphan drugs. Orphan drugs are expensive to develop, design, and market - while providing a low rate of return - making these drugs expensive to produce and distribute. This not only means that the development of orphan drugs can be unattractive to companies, but it also makes access to orphan drugs difficult and expensive for patients who need them the most.

To combat the unavailability of drugs for rare diseases and to encourage the development of orphan drugs, the Center for Drug Evaluation and Research (CDER) - a branch of the FDA - initiated the Rare Diseases Program to work with industry partners to develop orphan drugs.