Jackson Matthew J, Vaughan Gregory, Ledley Fred D
Bentley University, Waltham, Massachusetts, USA.
Center for Integration of Science and Industry, Bentley University, Waltham, Massachusetts, USA.
BMJ Open. 2024 Mar 12;14(3):e076542. doi: 10.1136/bmjopen-2023-076542.
Pharmaceutical innovation can contribute to reducing the burden of disease in human populations. This research asks whether products approved by the US Food and Drug Administration (FDA) from 2010 to 2019 and expedited review programmes incentivising development of products for serious disease were aligned with the US or global burden of disease.
Cross-sectional study.
Association of FDA product approvals (2010-2019), first approved indications, designations for expedited review with the burden of disease (disability-adjusted life years (DALYs)), years of life lost (YLL) and years of life lived with disability (YLD) for 122 WHO Global Health Estimates (GHE) conditions in US and global (ex-US) populations.
The FDA approved 387 drugs in 2010-2019 with lead indications associated with 59/122 GHE conditions. Conditions with at least one new drug had greater US DALYs (p=0.001), US YLL (p<0.001), global DALYs (p=0.030) and global YLL (p=0.004) but not US YLD (p=0.158) or global YLD (p=0.676). Most approvals were for conditions in the top quartile 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 designations for expedited review programmes was negatively associated (OR<1) with US DALYs, US YLD and global YLD. There was a weak negative association with global DALYs and a weak positive association (OR>1) with US and global YLL.
FDA drug approvals from 2010 to 2019 were more strongly aligned with US than global disease burden. Designations for expedited review were not aligned with either the US or global burdens of disease and may inadvertently disincentivise development of products addressing global disease burdens. These results may inform policies to better align pharmaceutical innovation with the burdens of disease.
药物创新有助于减轻人群的疾病负担。本研究探讨2010年至2019年美国食品药品监督管理局（FDA）批准的产品以及激励严重疾病产品开发的加速审查计划是否与美国或全球的疾病负担相一致。
横断面研究。
FDA产品批准情况（2010 - 2019年）、首次批准的适应症、加速审查指定与美国和全球（美国以外）人群中122种世界卫生组织全球卫生估计（GHE）疾病的疾病负担（伤残调整生命年（DALYs））、寿命损失年数（YLL）和伤残生命年数（YLD）之间的关联。
2010年至2019年，FDA批准了387种药物，其主要适应症与59/122种GHE疾病相关。至少有一种新药的疾病在美国的DALYs（p = 0.001）、美国的YLL（p < 0.001）、全球的DALYs（p = 0.030）和全球的YLL（p = 0.004）方面更高，但在美国的YLD（p = 0.158）或全球的YLD（p = 0.676）方面并非如此。大多数批准是针对美国DALYs或YLL处于前四分位数的疾病，但针对全球DALYs或YLL处于前四分位数的疾病的批准不到27%。一种药物获得一项或多项加速审查计划指定的可能性与美国的DALYs、美国的YLD和全球的YLD呈负相关（OR < 1）。与全球的DALYs呈弱负相关，与美国和全球的YLL呈弱正相关（OR > 1）。
2010年至2019年FDA批准的药物与美国疾病负担的一致性比与全球疾病负担的一致性更强。加速审查指定与美国或全球的疾病负担均不一致，可能会无意中抑制针对全球疾病负担的产品开发。这些结果可为使药物创新更好地与疾病负担相匹配的政策提供参考。
