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Original Investigation
December 29, 2016

Assessment of Overall Survival, Quality of Life, and Safety Benefits Associated With New Cancer Medicines

Author Affiliations
  • 1London School of Economics and Political Science, London, England
  • 2Harvard Medical School, Boston, Massachusetts
  • 3Division of Hematology-Oncology, Department of Medicine, Massachusetts General Hospital, Boston
  • 4Institute of Global Health Innovation, Imperial College London, South Kensington Campus, London, England
JAMA Oncol. Published online December 29, 2016. doi:10.1001/jamaoncol.2016.4166
Key Points

Question  What are the overall survival, quality of life, and safety benefits of recently licensed cancer medicines?

Findings  An analysis of health technology assessment reports found that new cancer drugs were associated with increased overall survival by an average of 3.43 months between 2003 and 2013, with 43% increasing overall survival by 3 months or longer, 11% by less than 3 months and 30% were not associated with an increase in overall survival. Most new cancer drugs improved quality of life, and were associated with reduced patient safety.

Meaning  The added benefits of new cancer medicines vary widely across and within therapeutic indications and may be based on modeled data, indirect or nonactive comparisons, or nonvalidated evidence.


Importance  There is a dearth of evidence examining the impact of newly licensed cancer medicines on therapy. This information could otherwise support clinical practice, and promote value-based decision-making in the cancer drug market.

Objective  To evaluate the comparative therapeutic value of all new cancer medicines approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) between 2003 and 2013.

Design, Setting, and Participants  We used a narrative synthesis approach to systematically synthesize and analyze English, French, and Australian health technology assessments (HTAs) of all new cancer medicines licensed in the United States and Europe between 2003 and 2013.

Interventions  Sixty-two new molecular entities with a primary oncology indication.

Main Outcomes and Measures  Overall survival (OS), quality of life (QoL), and safety.

Results  Of the 62 new active cancer molecules approved by the FDA and EMA between 2003 and 2013, 53 were appraised by English, French, or Australian HTA agencies through May 2015. Of these 53 drugs, 23 (43%) increased OS by 3 months or longer, 6 (11%) by less than 3 months, and 8 (15%) by an unknown magnitude; there was no evidence to suggest that the remaining 16 (30%) increased OS over best alternative treatments. Where overall survival gains could be quantified, all new cancer drugs were associated with a mean (SE) total increase in OS of 3.43 (0.63) months over the treatments that were available in 2003. Drug-related improvements in OS were, however, widely distributed across therapeutic targets—ranging between 0 (thyroid, ascites) and 8.48 months (breast cancers)—and were sometimes based on modeled data, indirect or nonactive comparisons, or nonvalidated evidence. Although 22 (42%) of 53 new medicines were associated with an increase in QoL, 24 (45%) were also associated with reduced patient safety. Of the 53 new cancer drugs, 42 (79%) were associated with at least some improvement in OS, QoL, or safety.

Conclusions and Relevance  Although innovation in the oncology drug market has contributed to improvements in therapy, the magnitude and dimension of clinical benefits vary widely, and there may be reasons to doubt that claims of efficacy reflect real-world effectiveness exactly. These findings raise important questions for clinical decision-making and value-based policy.