The Economic Burden of Rare Disease — The 2023 NEXT Report

June 28, 2023

Separate studies conducted by EveryLife Foundation and Chiesi Global Rare Diseases examined the overall cost of rare disease, as well as the cost per patient, using a number of factors, including treatments and therapies, decrease in quality of life, and mortality costs.

Read an excerpt from the 2023 NEXT Report to learn more the economic burden of rare disease and the Chiesi study, and continue reading by clicking the link at the bottom:

“In 2021, the EveryLife Foundation for Rare Diseases issued a study on the economic burden of rare disease that found the direct and indirect cost of these conditions in the United States totaled nearly $1 trillion a year. Now, a separate, 2022 study from Chiesi Global Rare Diseases, a business unit of the international pharmaceutical and healthcare focused Chiesi Group, with support from the health information technology company IQVIA, suggests that the costs could be as high at $8.6 trillion.

The Chiesi study found that the per patient, per year costs of rare diseases are more than 10 times higher than for more common diseases. The study found that the direct, indirect, and mortality costs associated with rare diseases are all higher in cases where a patient is without a treatment than in cases where a treatment exists.

“These results highlight the need for policymakers to nurture and sustain innovation based on the positive economic return from rare disease therapies and justify an increased governmental investment in diagnosis and newborn screening to ensure wider patient access to therapies,” the authors wrote. “Incentives for drug development, particularly restoring
the Orphan Drug Tax Credit to 50 percent and maintaining its current applicability to multiindications, encourage investment and have led to progress in rare disease drug approvals.”

The Chiesi study differed in a number of ways from the EveryLife study, but most notably in its inclusion of mortality costs—the value of lost years of life—in its calculations. It used the U.S. Department of Transportation’s value of a year of life at $130,000 and a life expectancy of 79 years to determine mortality costs.”

NEXT report provides an overview of developments across the rare disease landscape and highlights trends in research, diagnosis, development, and treatment, as well as the changing regulatory and financial environment. Read about:

  • How new genome sequencing technologies are cutting the cost and accelerating the speed of making a diagnosis
  • How patients are taking a hands-on approach to research to change the fundamental understanding of some rare diseases
  • What advances were made on the policy front in rare disease and the unfinished work that will be addressed in the coming year

Read the report

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