New Cancer Drugs May Have Saved More Than 1.2 Million Americans

Mark L. Fuerst

November 12, 2020

Cancer drug approvals between 2000 and 2016 were associated with a significant reduction in deaths from the most common cancers in the United States, according to a new study.

Reductions in mortality were most notable for tumor types with relatively more approvals, including lung and breast cancer, melanoma, lymphoma, and leukemia.

A report from the American Cancer Society (ACS) estimated that, from 1991 to 2017, there were 2,902,200 total cancer deaths avoided from improvements in mortality from all potential sources.

The new findings, reported in the Journal of Medical Economics, suggest that drugs approved between 2000 and 2016 to treat the 15 most common cancer types helped to reduce mortality by 24% per 100,000 people.

"This study provides evidence that a significant share of that reduction from 2000 to 2016 was associated with the introduction of new therapies. The ACS report and other studies demonstrate that the improvements in lung cancer specifically are likely due to new treatments," said lead study author Joanna P. MacEwan, MD, of PRECISIONheor in Los Angeles.

The findings contribute to a better understanding of whether increased spending on cancer drugs are worth the investment, according to the study authors.

"We provide evidence that the gains in survival measured in clinical trials are translating into health benefits for patients in the real world and confirm previous research that has also shown that new pharmaceutical treatments are associated with improved real-world survival outcomes for patients," MacEwan said.

Full Effect Not Yet Observed

The researchers used a series of national data sets from sources including the Centers for Disease Control and Prevention; the U.S. Mortality Files by the National Center of Health Statistics; Survival, Epidemiology and End Results program; and United States Cancer Statistics.

The team calculated age-adjusted cancer mortality rates per year for the 15 most common tumor types and also looked at incident cases of cancer by tumor type, represented as per 100,000 people, for all ages, races, and genders.

The researchers then translated the change in cancer mortality in the U.S. from 2000 to 2016 associated with treatment stocks in each year into deaths averted per year.

Across the 16 years, mortality was down by 1,291,769 deaths. The following cancers had significant reductions in mortality: breast (n = 127,874), colorectal (n = 46,705), lung (n = 375,256), prostate (n = 476,210), gastric (n = 758), and renal (n = 739) cancers, as well as non-Hodgkin lymphoma (n = 48,836) and leukemia (n = 4,011).

Estimated mortality increased by 825 deaths in patients with thyroid cancer and 7,768 deaths for those with bladder cancer. These rises are likely due to the result of sparse drug approvals during this period – five for thyroid cancer and three for bladder cancer – MacEwan said. There were no approvals in liver or uterine cancer and few approvals in pancreatic and oral cancer.

The full effect of new drug introductions may not have been observed yet, MacEwan noted.

"There are fewer patients using the treatments for drugs approved in the later years of our study and less follow-up time to measure outcomes," she said. "Over time, utilization of the newer therapies will likely increase and the full effect on mortality will be observed."

Other Factors at Play

Multiple factors have led to the declines in mortality, said William G. Cance, MD, chief medical and scientific officer for the ACS, who was not involved in this study. "We are slowly sorting out the explanations in greater granularity."

MacEwan said improved cancer screening may partially explain the decline in mortality in some tumor types.

"If screening in a particular tumor type improved during the study period and tumors were diagnosed earlier, then mortality for that tumor type may decline," she said. "However, we did not find strong evidence to suggest that there were significant changes in screening during our study period. Breast cancer screening rates, for example, were stable over our study period."

Cancer screening is not as strong an influence as it should be, Cance said.

"The lung cancer screening rate is low. In breast and colorectal cancers, we need to double down on earlier screening," he said, noting that less than one-quarter of adults between ages 45 and 50 years are currently screened for colorectal cancer. The ACS recommends that people at average risk of colorectal cancer start regular screening at age 45.

More research is necessary to evaluate the relationship between drug approvals and cancer mortality, MacEwan said.

"Research directly linking utilization of new therapies to improved survival or reduced mortality in the real-world setting would more definitively demonstrate the impact of new treatments," she said. "New therapies have improved outcomes for many patients and should continue to be considered as key elements of cancer treatment."

"We need to continue to reduce tobacco smoking and improve on modifiable behaviors at the same time as we work on getting new drugs to cancer patients," Cance said. "We are coming into an era of multiple new therapeutics, including targeted therapies, immunotherapies, and cellular therapies. Clinicians need to look closely at the trial data of new drugs and pay close attention to those that have the most mortality impact."

"We also need equitable distribution of newer drugs," Cance added. "They should be distributed to everybody who deserves them. Mortality is often impacted by social determinants of health."

Funding for this research was provided by Pfizer. Study authors disclosed relationships, including employment, with Pfizer. Cance had no disclosures.

SOURCE: MacEwan JP et al. J Med Econ. 2020 Nov 9;1-12.

This article originally appeared on, part of the Medscape Professional Network.


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