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The study estimated the extent to which drug innovations over the past 30 years may have improved outcomes for six diseases. We analyzed six diseases (ischemic heart disease, lung cancer, breast cancer, human immunodeficiency virus [HIV] infection, type 2 diabetes mellitus, and rheumatoid arthritis [RA]) with significant mortality or morbidity for which there have been major drug innovations over the past 30 years. We used U.S. data from the Global Burden of Disease (GBD) database and a patient registry to perform counterfactual time-series analyses predicting the improved health outcomes that may have been associated with major drug innovations. For 5 conditions using data from the GBD study, years of life lost per individual with the condition could have been higher by 17.1% (breast cancer) to 660.6% (HIV infection) in 2017 had the major drug innovations not been introduced. For RA, using patient registry data, patients’ functional status could have been 11.5% worse had biological therapies not been introduced. Policies targeting drug prices should be broadened to consider the price and value of all health-care services. The societal importance of the pharmaceutical industry’s ability to respond rapidly to emerging diseases should be recognized.

What might have happened: the impact of interrupting entry of innovative drugs on disease outcomes in the United States