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. ### Competing Interest Statement Dr. Ledley has received from National Biomedical Research Foundation and the Institute for New Economic Thinking for this work. Dr. Ledley has also received funding for unrelated work from National Pharmaceutical Council. Both Dr. Ledley and Dr. Vaughan have received funding for unrelated work from West Health Policy Center. ### Funding Statement This study was funded by the National Biomedical Research Foundation and the Institute for New Economic Thinking for this work. ### Author Declarations I confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained. Yes I confirm that all necessary patient/participant consent has been obtained and the appropriate institutional forms have been archived, and that any patient/participant/sample identifiers included were not known to anyone (e.g., hospital staff, patients or participants themselves) outside the research group so cannot be used to identify individuals. Yes I understand that all clinical trials and any other prospective interventional studies must be registered with an ICMJE-approved registry, such as ClinicalTrials.gov. I confirm that any such study reported in the manuscript has been registered and the trial registration ID is provided (note: if posting a prospective study registered retrospectively, please provide a statement in the trial ID field explaining why the study was not registered in advance). Yes I have followed all appropriate research reporting guidelines, such as any relevant EQUATOR Network research reporting checklist(s) and other pertinent material, if applicable. Yes All databases used in writing this article are publicly available and cited in the text. All extracted data is provided in supplemental table 1 or supplemental table 2. Results from statistical outputs are provided in supplemental table 4-6.