For the United States, the total economic burden of rare disease is estimated to be $2.2 trillion per year.
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A diagnosis of disease creates a significant amount of pressure not just on the individual patient but their families as well. But, when it comes to rare diseases, the burden is even higher due to the highly specialized nature of care when it’s available.
A new report from Chiesi Global Rare Diseases, a division of Chiesi Farmaceutici S.p.A., highlights the significant burdens associated with a rare disease diagnosis. The white paper, “The Burden of Rare Diseases: An Economic Evaluation,” which was co-authored by IQVIA, was released on Feb. 28, a day set aside to highlight rare diseases. As could be expected, it revealed a high financial burden tied to rare disease diagnosis. In the United States, the analysis examined 8.4 million people who are impacted by 373 different rare diseases. Globally though, the number of people affected by rare disease is even higher. There are an estimated 300 million people impacted by rare diseases.
The study was launched in order to explore the direct, indirect and mortality-related costs for a sample of patients across five therapeutic areas. The goal was to evaluate and compare the burden of care for rare diseases that have treatments versus those that do not. The costs are high for both groups but for those that do not have available treatments, they are significantly higher. If treatment options become available, the economic burden can be reduced for that segment of patients, the report shows.
The study revealed that the burden of a rare disease is approximately 10 times higher than that of “mass market diseases,” such as some cancers or some autoimmune diseases on a per patient per year basis. For the United States, the total economic burden of rare disease is estimated to be $2.2 trillion per year. Globally, that number is even higher. The economic burden for rare diseases across the world ranges from $7.2 trillion to $8.6 trillion per year. Without available treatments for these diseases, the costs are expected to increase even more. The paper suggests that a lack of treatment for these rare diseases is associated with a 21.2% increase in total costs, per patient per year.
Giacomo Chiesi, head of Chiesi Global Rare Diseases and one of the study co-authors, told BioSpace in an email that the burdens of a rare disease are difficult not only for the patient but their families as well. For those folks who are not wealthy, the financial burdens of rare disease are even weightier. Chiesi said the goal of the report is to help people gain a better understanding of the “cost drivers and economic impact” from a lack of available treatments for those living with rare diseases. He said the paper provides a critical benchmark that highlights the unmet needs of the rare disease community.
“The economic burden is further exacerbated by a lack of treatment options, which is the case for many rare diseases. But there is some hope as we found that on average access to rare disease treatments can reduce costs by about 21% compared to having no treatments available,” Chiesi said in the email.
Chiesi stressed the importance of the involvement of policymakers to assist those impacted by rare diseases. With support from lawmakers, the economic burdens shouldered by approximately 95% of families struck by rare disease can find financial support from public and private partners.
“We urge policymakers to consider the tremendous need for equity among the rare disease population by initiatives that foster innovation and the development of new safe and effective therapies, which are shown to generate substantial value for society,” Chiesi said.
