Chemotherapy and Toxicities of Immune Checkpoint Inhibitors May Be Prohibitively Expensive Interview with:
Neil T. Mason, MBA
Personalized Medicine Strategist
Personalized Cancer Medicine
Division of Population Science
Moffitt Cancer Center

MedicalResearch: What is the background for this study? What are the main findings?

Response: Immune checkpoint inhibitors targeting PD-1 (nivolumab and pembrolizumab) and CTLA-4 (ipilimumab) have revolutionized the treatment of metastatic disease in melanoma and non-small cell lung cancer with additional indications showing positive results. These drugs have elicited profound and durable responses in a significant number of patients, but have been criticized for their high cost. Though the price of the drugs themselves can reach over $100,000 per year, they can also cause severe, life threatening toxicities that are difficult and expensive manage.

This model utilizes patient data from a large, NCI-designated cancer center to estimate the average cost of treatment with immune checkpoint inhibitors based on average duration of treatment and reported incidence of major toxicities. Based on the model, PD-1 inhibitor therapies are less costly than ipilimumab due to the significantly higher cost per dose of ipilimumab and average treatment duration of less than a year for PD-1 inhibitors. Managing drug-related toxicities were estimated to contribute between $8,200 and $9,600 to the cost of therapy with nivolumab adding the most cost.

MedicalResearch: What should readers take away from your report?

Response: Managing immune checkpoint inhibitor-related adverse events can account for almost 20% of the cost of treatment based on the incidence of these toxicities and the average number of doses of each drug received. PD-1 inhibitors have demonstrated superior efficacy to ipilimumab in many cases and are likely to be less costly overall. However, the introduction of clinical trials combining both drug classes has improved efficacy significantly, but yielded a concomitant increase in toxicities. Extrapolating from this model, it is clear that these regimens will be extremely, and possibly, prohibitively expensive.

MedicalResearch: What recommendations do you have for future research as a result of this study?

Response: Now that the PD-1 inhibitors have been on the market for a year or more, empirical cost of care studies need to be performed and integrated into cost-effectiveness analyses based on data from clinical practice rather than estimated from data collected as part of clinical trials. Perhaps more importantly, new biomarkers and diagnostics need to be developed to better predict which patients are most likely to benefit from these expensive drugs as well as to predict which patients are most likely to suffer catastrophic adverse events. This will allow us to make better informed cost/benefit decisions and better target the right drugs to the right patients.

MedicalResearch: Is there anything else you would like to add?

Response: These practical analyses are much needed if patients (and health systems) are going to make objective choices about the management of their disease.


Abstract presented at 2016 ASCO meeting June 2016

J Clin Oncol 34, 2016 (suppl; abstr 6627)

Author(s): Neil Thomas Mason, Nikhil I. Khushalani, Jeffrey S. Weber, Scott Joseph Antonia, Howard L. McLeod; H. Lee Moffitt Cancer Center & Research Institute, Tampa, FL; NYU Langone Medical Center, New York, NY; Moffitt Cancer Center, Tampa, FL

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

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