The RARE Act of 2019 would establish the National Rare Disease or Condition Surveillance System to provide valuable information to researchers of rare diseases or conditions.
This bill would allow up to $10 million in expenditures annually by the Center for Disease Control and Prevention from 2020 through 2024 to expand and enhance the existing infrastructure and activities for tracking the epidemiology of up to four rare diseases or conditions, defined in the bill as a disease that affects less than 200,000 people in the US. This expansion would include coordinating with experts who conduct research and share data on rare diseases, maintaining a centralized database with research findings and data on rare diseases, awarding grants to nonprofit entities, publishing research and data in a publicly-accessible manner, and generating reports to track the progress of implementing the Surveillance System and of aggregating information regarding the selected rare diseases.
The criteria for selecting the rare diseases mandates high mortality or morbidity rates and a potential for meaningful research. Once the rare diseases have been selected, the Department of Health and Human Services shall provide for the collection and storage of information regarding the risk factors, demographics, prevention, diagnosis, incidence, prevalence, treatment, and other important factors of the diseases. This Surveillance System does not replace, but may assist in, ongoing research on spina bifida, muscular dystrophy, and fragile X syndrome.
In addition to the Surveillance System, this bill intends for the Agency for Healthcare Research and Quality to increase physicians' awareness of rare diseases or conditions and would allow up to an additional $1 million for the purpose of updating and republishing the National Academies’ 2010 report “Rare Diseases and Orphan Products: Accelerating Research and Development.”