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

Introduction

Pharmaceutical innovation can contribute to reducing the burden of disease in human populations. This research considers whether products approved by the US Food and Drug Administration (FDA) 2010–2019 and policies for expedited review of products for serious disease were aligned with the US or global burden of disease.

Methods

Cross-sectional study of products approved 2010–2019, their first approved indications, designations for expedited review, the burden of disease (DALYs), years of life lost (YLL), and years of life lived with disability (YLD) for 122 WHO Global Health Estimates (GHE) conditions. Statistical analyses of associations between drug approvals, disease burden of conditions comprising first approved indications, and designations for expedited review.

Results

The FDA approved 387 drugs 2010–2019 with lead indications for 59/122 GHE conditions. Conditions with at least one new drug had greater US DALYs (U=1193, p=0.001), US YLL (U=1144, p<0.001), global DALYs (U=1436, p=0.030), and global YLL (U=1304, p=0.004) but not US YLD (U=1583, p=0.158) or global YLD (U=1777, p=0.676). Most approvals were for conditions in the top quartiles of US DALYs or YLL, but <27% were for conditions in the top quartile of global DALYs or YLL. The likelihood of a drug having one or more expedited review designations was negatively associated (odds ratio <1) with US DALYs, US YLD, and global YLD. There was a weak negative association with global DALYs and a weak positive association (odds ratio >1) with US and global YLL.

Conclusions

Drug approvals 2010–2019 were more strongly aligned with US than global disease burden and more strongly associated with YLL than YLD. Expedited review pathways were not aligned with the US or global burdens of disease and prioritize YLL over YLD. These results may inform policies to incentivize pharmaceutical innovation better aligned with global burden of disease.

KEY QUESTIONS

What is already known on this topic

Pharmaceutical innovation is strongly influenced by (US) market opportunities and poorly aligned with the global burden of disease. Previous studies have suggested that regulatory policies designed to expedite development of products for serious disease could promote better alignment between pharmaceutical innovation and global disease burdens.

What this study adds

Drug approvals by the US Food and Drug Administration 2010–2019 were more strongly associated with the US than global burden of disease and were disproportionately focused on disorders contributing to premature death as opposed to disability. The odds of a product being designated for expedited review was negatively associated with the burden of disease and measures of disability but positively associated with years of life lost to disease.

How this study might affect research, practice, or policy

This work demonstrates a persistent failure of drug development for conditions that contribute the most to the global burden of disease and disabilities that is not addressed with policies for expedited review. This analysis may inform new policy explicitly designed to promote innovation for indications associated with the greatest disease burden and, specifically, the burden associated with disabilities.