Changes in mortality associated with cancer drug approvals in the United States from 2000 to 2016

Aims: To estimate the extent to which the approvals of new pharmacological therapies were associated with cancer mortality in the USA between 2000 and 2016.

Materials and methods: The analysis quantified cancer drug approvals across the 15 tumor types with the highest incidence. Number of approvals in a given time period for each tumor was translated into a treatment stock measure, defined as a weighted sum of new indication approvals since 1976. The primary outcome was the annual tumor-specific cancer mortality, defined as the number of deaths per 100,000 U.S. population. The analysis used a multivariable ordinary least squares and a fixed effects model, controlling for incidence (new cases per 100,000 U.S. population) and the primary exposure, the treatment stock measure by year.

Results: Between 2000 and 2016, deaths per 100,000 population across the 15 most common tumor types declined by 24%. Additionally, 10.2 new indications were approved per year across the 15 most common tumor types. Cancer drug approvals were associated with statistically significant deaths averted in 2016 for colorectal cancer (4,991, p = 0.004), lung cancer (33,825, p < 0.001), breast cancer (11,502, p < 0.001), non-Hodgkin's lymphoma (6,636, p < 0.001), leukemia (4,011, p < 0.001), melanoma (1,714, p < 0.001), gastric cancer (758, p = 0.019), and renal cancer (739, p < 0.001). Between 2000 and 2016, new cancer treatments were correlated with 1,291,769 (p < 0.001) total deaths prevented across the 15 most common tumor types.

Limitations and conclusions: Cancer drug approvals between 2000 and 2016 were associated with significant reduction in deaths from the most common cancers in the USA. Mortality changes were largest in prevalent tumor types with relatively more approvals, i.e. lung cancer, breast cancer, melanoma, lymphoma and leukemia. Future research evaluating the relationship between drug approvals and cancer mortality post 2016 is needed.