Association of immunotherapy with durable survival as defined by value frameworks for cancer care

Omer Ben-Aharon, Racheli Magnezi, Moshe Leshno, Daniel A. Goldstein*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

44 Scopus citations


IMPORTANCE Modern immuno-oncology agents have generated great excitement because of their potential to provide durable survival for some patients. However, there is concern regarding the cost of cancer care, and multiple frameworks have been developed to assess value. The American Society of Clinical Oncology (ASCO) framework awards bonus points if substantial durable survival is demonstrated. OBJECTIVE To assess whether modern immuno-oncology agents reach defined efficacy thresholds in value frameworks. DESIGN, SETTING, AND PARTICIPANTS In this analysis, all US Food and Drug Administration (FDA) approvals for immuno-oncology agents between March 2011 and August 2017 were reviewed. Data required for the ASCO framework were collected, specifically improvement in proportion of patients alive with the test regimen and survival rate with standard treatment. MAIN OUTCOMES AND MEASURES Awarding of bonus points for durable survival based on the ASCO criteria. RESULTS Twenty-three metastatic indications for 6 immuno-oncology agents (ipilimumab, pembrolizumab, nivolumab, atezolizumab, avelumab, and durvalumab) were approved by the FDA from March 2011 to August 2017. Ten (43%) of the approvals were based on survival end points, while 13 (57%) were based on response rates. Only 3 drug indications fulfilled the threshold defined for the survival rate of patients receiving standard care (minimum 20%). Nine indications achieved the required level of improvement in proportion to patients alive in the test regimen compared with the standard (above 50%). There was overlap between these 2 criteria for 3 drug indications, allowing them to gain the durable survival bonus points awarded by the ASCO framework. CONCLUSIONS AND RELEVANCE Durable survival and response rates of modern immuno-oncology agents are rarely recognized as significant by current oncology value frameworks. This may be due to insufficient demonstration of efficacy of such agents or inappropriately calibrated value frameworks.

Original languageEnglish
Pages (from-to)326-332
Number of pages7
JournalJAMA Oncology
Issue number3
StatePublished - Mar 2018


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