This month, the National Organization for Rare Disorders (NORD) is joining the larger United States’ rare disease community in celebrating the 40th anniversary of the Orphan Drug Act (ODA). Signed into law in 1983, the ODA marked one of the first major victories for rare disease advocates. It was developed in response to the “orphan drug problem”; namely, that drugs were not being developed for small patient populations. In the decades since, over 1,100 orphan indications for treatments have received FDA approval.
In addition to introducing incentives for the development and distribution of therapeutics for people with rare diseases, the ODA inspired the creation of NORD, and with it the rare disease advocacy community as it exists in the United States today. Over the past four decades, they have worked to amplify the voices of patients and caregivers. NORD has also contributed to a range of policies, projects, and studies which have helped improve the accessibility of care and promote research in the rare disease field.
Despite the progress brought about by the ODA, there is still a long way to go. Fewer than 10 percent of known rare diseases have a treatment, and cures are even more scarce. However, the advancements of the past 40 years demonstrate that the rare disease community is powerful, and will continue working to drive innovation and bring about change.
(source: OrphaNews 18 January 2023)