A quartet of recent studies and reports from a variety of authors are shedding new light on the collective economic cost impacts posed by rare diseases, disorders and conditions that combined affect between 25 to 30 million people in the United States. These studies’ findings are summarized in an article co-authored by the EveryLife Foundation for Rare Diseases and published in Health Affairs entitled, “The Economic Burden of Rare Diseases: Quantifying the Sizeable Collective Burden and Offering Solutions.” In the piece, the authors propose several recommendations that, if taken, will address the gaps and challenges they identify.
While all four studies were conducted independently and constructed differently, they reach consensus on key points in terms of certain data sources, approaches, and conclusions. The authors synthesize these similarities and offer five recommendations to enhance rare disease data collection and analysis.
One of the studies, called the National Economic Burden of Rare Disease Study, was commissioned by the EveryLife Foundation. The study included one of the largest surveys thus far of multiple rare disease communities, generating 1,399 fully completed responses from individuals representing about 400 rare disease communities.
“Thanks to the engagement of the community, we now have a growing set of data that show what we have long known: rare diseases present a public health crisis and demand additional research funding, enhanced awareness, and improved access to diagnosis, care, and treatment,” said Annie Kennedy, EveryLife Foundation for Rare Diseases Chief of Policy, Advocacy, and Patient Engagement. “The EveryLife Foundation remains committed to working alongside the community to advance policies that will address this urgent need.”
The Health Affairs article was authored by: Sheldon Garrison, Ph.D., Research Scientist with Rogers Behavioral Health; Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement of the EveryLife Foundation for Rare Diseases; Nick Manetto, Principal with Faegre Drinker Consulting and advisor with the EveryLife Foundation for Rare Diseases; Anne R. Pariser, MD, Director of the Office of Rare Diseases Research at NIH National Center for Advancing Translational Sciences (NCATS); Joni L. Rutter, Ph.D., acting director of NCATS; and, Grace Yang, Vice President of the Lewin Group.
