Is Drug Development More Aligned With Burden of Disease in the United States or Globally?

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A study recently published in the British Medical Journal sought to evaluate the correlation between FDA drug approvals from 2010-2019 and the burdens of disease in the United States and globally.¹

“Pharmaceutical innovation can contribute to reducing the burden of disease in human populations. This research asks whether products approved by the FDA from 2010 to 2019 and expedited review programs incentivizing development of products for serious disease were aligned with the US or global burden of disease,” the study authors wrote.

The study utilized 387 FDA approvals from 2010-2019 as data points. Of these, 58.7% were granted at least one designation for expedited review programs, including 12.7% for accelerated approval, 20.2% for breakthrough therapy, 36.7% for fast track designation, and 53.5% for priority review. The study also used burden of disease (disability-adjusted life years [DALYs]), years of life lost (YLL), and years of life lived with disability (YLD) for 122 World Health Organization (WHO) Global Health Estimates (GHE) conditions in the United States and global population to review the approvals’ alignment with their respective burdens of disease.

The authors found that FDA drug approvals during the time period were more strongly aligned with the United States’ burden of disease rather than global. Among the full results, the authors found that the majority of drug approvals were for diseases indicated for conditions in the top quartile of US DALYs. This was not evident for the global burden of disease.

“These analyses demonstrated a strong association between drug approvals and conditions with higher US DALYs. This was evident in the observation that the majority of all approvals had a first indication associated with conditions in the top quartile of US DALYs and that conditions with at least one new drug approval had significantly higher US DALYs than those without new approvals,” the authors wrote. “In contrast, <27% of drug approvals were indicated for conditions in the top quartile of global disease burden, and there was a less pronounced difference in the global burden of disease for conditions with or without new drug approvals.”

The results of this study are consistent with previous research, suggesting that the lack of alignment between US drug approvals and the composite health needs of global human populations has continued to be a challenge, according to the study. Among the limitations of the study, the authors noted that they considered the burden of disease at the level of GHE conditions, which compromises multiple indications. This could lead to an overestimation of the burden of disease when the drug’s usage compromises multiple indications of any particular GHE condition.

“Drug approvals from 2010 to 2019 were more strongly aligned with US disease burden than global disease burden and more strongly associated with YLL than YLD. The likelihood of an approved product receiving one or more designations for expedited review was not positively associated with either the US or global burden of disease and may inadvertently disincentivize product development for diseases that contribute the most to the global disease burden. These results may inform policies to better align pharmaceutical innovation with burdens of disease,”
the authors concluded.

Reference

1. Jackson MJ, Vaughan G, Ledley FD. Association between expedited review designations and the US or global burden of disease for drugs approved by the US Food and Drug Administration, 2010–2019: a cross-sectional analysis. BMJ Open 2024;14:e076542. doi: 10.1136/bmjopen-2023-076542. https://bmjopen.bmj.com/content/14/3/e076542