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HTA Study Raises Questions on Real-World Benefits of Newly Approved Cancer Drugs


While rapid innovations in the field of oncology have improved treatments, the magnitude and dimension of clinical benefits vary widely.

What is the impact of newly approved cancer drugs on patient outcomes? Do they increase survival to the same extent? Is the patient’s quality of life (QOL) better with one over the other? These were the questions queried by a retrospective study that analyzed health technology assessment reports for 62 new anticancer drugs approved in Europe and the United States between 2003 and 2013.

Fifty-three of 62 new active cancer molecules that were approved by the FDA and the European Medicines Agency were appraised by health technology assessment (HTA) agencies in England, France, and Australia through May 2015. Twenty-three (43%) drugs from this pool increased overall survival (OS) by at least 3 months and 6 (11%) increased OS by less than 3 months. The effect of 8 (15%) drugs on OS was of an unknown magnitude, while evidence for the remaining 16 (30%) drugs was not favorable in terms of OS. Overall, the new anticancer agents were responsible for a mean increase in OS of 3.43 months over treatments that were available back in 2003.

The authors note that the type of disease was a significant determinant of patient response and outcome, ranging from no increase in OS for ascites and thyroid cancers, to 8.48 months for breast cancers. Twenty-two (42%) of the 53 drugs improved patient QOL while 24 (45%) reduced patient safety. A majority (79%) of drugs had some form of positive outcome on the patient—either improved OS, QOL, or safety.

This real-world evidence holds equal, if not greater, importance to results from clinical trials, which are conducted in a more controlled environment and in a select population of patients. Data such as the one presented here can present a big advantage to all stakeholders—physicians, pharmacy benefit managers, health plans, as well as patients and their caregivers—to make more informed decisions when developing treatment plans.

In the United States, work by groups such as the Institute for Clinical and Economic Review or ICER has been encouraging conversations around HTA. Speaking with Evidence-Based Oncology™, Steven D. Pearson, MD, MSc, FRCP, founder and president of ICER said that payers are also thinking along the lines of indication-specific pricing. “I think they are interested in it, along with outcomes-based contracting and some other approaches, as tools that are certainly worth considering in specific circumstances,” he added.

The authors of the study write that while rapid innovations in the field of oncology have improved treatments, the magnitude and dimension of clinical benefits vary widely, meaning drug efficacy documented in clinical trials may not necessarily reflect the drug’s performance in the real-world setting.

“These findings raise important questions for clinical decision-making and value-based policy,” they concluded.


Salas-Vega S, Iliopoulos O, Mossialos E. Assessment of overall survival, quality of life, and safety benefits associated with new cancer medicines [published online December 29, 2016]. JAMA Oncol. doi:10.1001/jamaoncol.2016.4166.

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