The development of orphan drugs, those used for the safe and effective treatment, diagnosis or prevention of genetic and rare diseases or disorders, is on the upswing.
In the past five years, 61 orphan drugs were approved. This is the largest for any five-year period since 1983, when Congress passed the Orphan Drug Act, a measure that gives financial incentives to encourage companies to develop drugs and biologics for rare diseases. The legislation includes tax credits to defray the costs of clinical trials and provides for fast-track FDA approval and market exclusivity. It's important because, before its passage, drug companies were weighing the financial viability of researching and developing these drugs. The Orphan Drug Act led to the development of hundreds of new drugs to treat a variety of conditions.
With orphan therapies on the rise, more people with rare diseases and genetic disorders have more treatment options designed to help them lead healthier, more productive lives.