Rare ≠ uncommon: patient advocates aim to increase visibility of orphan diseases
Making the case for policies and investments that could increase the speed and scale of drug development
The burden of rare diseases is hiding in plain sight. A lack of disease codes and imprecise electronic health records create the false impression that conditions that affect small numbers of people are uncommon, and this impression can hurt patients. Illuminating the costs and highlighting the burdens on society, patient advocates hope, will make the case for policies and investments that could increase the speed and especially the scale of drug development.
About one in 10 Americans lives with a rare condition and, according to four recently released studies, the total economic costs rival those of the most common diseases. The studies were conducted or commissioned by the EveryLife Foundation for Rare Diseases; NIH’s National Center for Advancing Translational Sciences (NCATS); Advocate Aurora Health, a not-for-profit healthcare system; and the Government Accountability Office. Authors of the studies summarized their collective findings and recommendations in Health Affairs. ...