The National Center for Advancing Translational Sciences (NCATS) released a study on the impact of rare diseases on patients and healthcare systems. The study found that health care costs for rare disease patients are underestimated and that they are three to five times higher than costs for individuals without rare diseases. The study used patient medical records to estimate average medical costs for rare disease patients and to track the diagnostic journeys of four patients. A link to the study can be found here.
Last week, the Government Accountability Office (GAO) released its study on the cost of undiagnosed and untreated rare diseases. The EveryLife Foundation for Rare Diseases’ Community Congress led the policy efforts in support of this study to be included in the Further Consolidated Appropriations Act, 2020 (HR 1865) which was signed into law in December 2019.
Both the NCATS and the GAO studies build on the National Economic Burden of Rare Disease Study released by the EveryLife Foundation earlier this year. The Foundation’s study was the first of its kind, providing the most comprehensive assessment of the total cost of rare diseases in the U.S. to date. The study estimated the economic cost of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019. To generate the data for this study, the Foundation identified both direct medical costs, via an analysis of claims data, and indirect and non-medical costs, via a national survey (The Rare Disease Impact Survey) that reflected data from 1,399 members of the rare disease community.
These reports will help ensure that the experience of the rare disease community is reflected accurately in policy discussions and serve as powerful tools that underscore the public urgency and awareness of the public health crisis of rare disease. The reports would not have been possible without the continued and tireless engagement and commitment of the rare disease community.
