Individuals suffering with rare diseases had very few therapeutic options 40 years ago. But that began to change with the stroke of a pen by then-President Ronald Reagan in January 1983 when he signed the bipartisan Orphan Drug Act into law. On Jan. 4, the rare disease community will celebrate the 40th anniversary of the Orphan Drug Act (ODA) — legislation that incentivized drug companies to put more resources toward the research, development, and distribution of therapeutics for people with rare diseases, who until then had been ‘orphaned’ by the medical and scientific community.
The creation of the ODA and the founding of the National Organization for Rare Disorders (NORD) are forever intertwined. The rare patients and caregivers who started the rare advocacy movement — which convinced lawmakers to introduce orphan drug legislation — knew their work was not done after the bill became law. They formed NORD to unify the voices of the unheard within the rare disease community under the premise that a rising tide could lift all boats.