There has been much written about the cost of cancer chemotherapy. In the US alone
over 2.5 trillion dollars are spent on healthcare each year. Among the most expensive drugs are cancer therapies. This came to light in an analysis in JAMA Oncology.
What Are We Getting for Our Money?
The article examined the impact of modern cancer therapy on quality of life and survival (Assessment of Overall Survival, Quality of Life, and Safety Benefits Associated with New Cancer Medicines. Salas-Vega, S: JAMA Oncology, December 29, 2016). Using the years 2003 to 2013 they found that fewer than half of new treatments improved overall survival by three months or more. This varied by disease from breast at 8.48 months to thyroid with no improvement whatsoever. The overall average was only 3.43 months. Once again the high cost of cancer therapy did not seem to correlate with significant improvements in survival.
This was the subject of a previous paper in the Journal of Clinical Oncology (Cancer Drugs in the United States; Justum Pretium – the just price, Kantarjian H. M. et al, J Clin Oncol 2013). In this analysis the authors noted that of the 12 anticancer drugs approved by the U.S. FDA in 2012 only three prolonged survival and 2 of those 3 by less than 2 months. More concerning was the average drug price at greater than $10,000 per month.
Is the cost of cancer chemotherapy too high? Should we ration agents to prevent over-expenditure? Should Medicare's solvency be placed above the needs of individual patients?
Is Molecular Profiling the Answer?
Most institutions in the United States have responded to this dilemma by expanding molecular profiling programs. Using DNA, it is reasoned, they will identify candidates for the newest treatments and limit drug use to only those most likely to respond. This lofty ideal however has not been met.
For one reason a relative minority of patients actually carry identifiable targets.
More troubling however is that fact that even those patients who are identified as candidates for novel therapies by gene profile often fail to respond.
This was made abundantly clear by a study that examined the outcome of patients who carried a mutation in the gene BRAF V600, the specific target for the drug Vemurafenib (Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations. Hyman, DM et al NEJM August 20, 2015). Of the 27 colon cancer patients identified as candidates for Vemurafenib by gene profile, only 1 of 27 actually responded, providing an overall objective response rate of 4%. If we were to add the cost of screening thousands of patients for actionable mutations to the actual cost of treatment, this patient could prove to be the single most expensive clinical response in history.
Functional Platforms Already A Proven Approach
One approach that has been grossly under-appreciated is the application of three-dimensional organotypic analyses like those conducted in our laboratory. These functional platforms gauge the biological responsiveness of human tumors to drugs regardless of the tumor’s genomic profiles.
With a two-fold improvement in a response and a 44% improvement in survival these functional analyses offer a unique opportunity to address the growing cost and toxicity of therapy, provide better outcomes and lower costs. As the newest classes of drugs cost over $10,000 per month, saving patients just two weeks of ineffective therapy more than pays for the entire cost of the analysis.
With 2017 upon us and a new administration about to take the reins in Washington, is it now time for us to examine better approaches to cancer therapy and to use validated technologies like functional profiling for drug selection. We certainly hope so.
As always, I appreciate your thoughts and comments.