The Relationship Between Pharmaceutical Innovation and Cancer Mortality in Spain, From 1999 to 2016

To investigate the relationship across cancer sites between pharmaceutical innovation and changes in cancer mortality in Spain during the period between 1999 and 2016. We investigated whether the cancer sites for which more new drugs were authorized had larger reductions in mortality from 1999 to 2016 in Spain, controlling for the lagged change in cancer incidence. The principal measure of pharmaceutical innovation is the long-run change in the mean vintage (year of initial authorization in Spain) of the drugs for the treatment of a cancer previously authorized in Spain. The 1999 to 2016 increase in mean age at death tended to be larger, and the 1999 to 2016 increase in the number of deaths and life-years lost before the ages 65 years, 75 years, and 85 years tended to be smaller for cancer sites that had larger current or lagged increases in drug vintage. Pharmaceutical innovation was associated with a 2.77-year increase in mean age at death from cancer from 1999 to 2016—96% of the observed increase. New drug authorization during the previous 17 years were associated with a reduction in the number of life-years lost before the age of 75 years in 2016 of 333 000. Under the assumption that the association between pharmaceutical innovation and mortality reduction is causal, estimated drug expenditure per life year before the age of 75 years gained in 2016 from new cancer drugs that were authorized between 2000 and 2016 was €3269. The cancer sites for which there were more pharmaceutical innovation—more new drugs authorized—had larger 1999 to 2016 reductions in mortality in Spain, controlling for the lagged change in cancer incidence. •Some previous studies have shown that there is a relationship between pharmaceutical innovation and changes in cancer mortality, but the methods used in those studies can be tested and improved upon in several ways.•In this study, which analyzes the relationship between pharmaceutical innovation and cancer mortality in Spain during the period 1999 to 2016, we examine and compare 2 alternative measures of pharmaceutical innovation, analyze 5 measures of mortality, control for other (nonpharmaceutical) innovation, perform falsification tests using data on pharmaceutical innovation in 3 other countries, address the issue of potential violation of parallel trends, and assess the sensitivity of weighted least-squares estimates to the choice of weights.•This study describes improvements in methods for using real-world evidence to asse