Cancer Treatment Cost-effectiveness: Difficult To Define Due to Perception

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If value is hard to define because it is perceived differently by different people, how can oncologists and policymakers accurately gauge cost-effectiveness?
If value is hard to define because it is perceived differently by different people, how can oncologists and policymakers accurately gauge cost-effectiveness?

Value-based care, an increasingly important concept in oncology, is grounded on the relationship between the efficacy and cost of an anticancer drug. Patients with cancer, the reasoning goes, ought to receive the best value of care, accounting for efficacy, toxicity, comorbidities, price, and so forth.

The authors of an article recently published in The New Bioethics note, however, that the notion of value requires that an arbitrary price be put on life.1 This refers to the often-small improvements in overall survival seen with some newly approved cancer treatments. Some of these benefits improve the average patient's life expectancy by as little as 10 days.

Yet these survival benefits, no matter how marginal, are often the basis for drug approvals and consequent marketing campaigns. Richard Sullivan, MD, PhD, founding director of the Institute of Cancer Policy in London, England, and co-author of this essay, noted that “the bar for marketing authorisation is set low, and is getting lower. This means greater and greater uncertainty as to the efficacy, toxicity, tolerability, and overall value.

“All the evidence from current studies shows that more cancer medicines are being approved which do not deliver clinically meaningful benefit and this is in the context of clinical trials. We know in the real world most cancer medicines' efficacy drops some 20 to 40% because we start treating real patients with a variety of comorbidities.”

Patients in the “real world” do not, according to recent research, resemble patients who participate in clinical trials. This sometimes negates whatever improvements justify drug approvals in the United States and Europe.2,3

Doubts about the true efficacy of recently approved drugs combined with effective marketing campaigns make “value” a difficult term to quantify. If the efficacy for the average patient outside of a clinical trial cannot be guaranteed by the results seen in a clinical trial, the notion of reproducibility — or rather the purpose of reproducibility — is undermined, and a drug's value determined by a clinical trial is not the value seen in the clinic.

Bishal Gyawali, MD, a medical oncologist working in Japan who is affiliated with the Institute of Cancer Policy and the Anticancer Fund, Belgium, and co-author of the recent essay, noted to me that this issue is particular to cancer treatments, because “cancer medicines usually target metastatic disease where pushing away death is the main focus…paradoxically [these drugs] receive special treatment by the oncology community compared with surgery or radiotherapy services that aim at ‘curing' early stages of cancer.”

Dr Gyawali noted a recent article published in the Annals of Oncology that questioned the value added by England's National Health Service (NHS) Cancer Drug Fund, which was established to “improve access” to cancer medications not necessarily approved by the National Institute for Health and Care Excellence (NICE).4

Of 47 indications approved by the Cancer Drug Fund, only 18 “reported a statistically significant OS [overall survival] benefit, with an overall median survival of 3.1 months.” The authors concluded that the Fund did not meaningfully benefit patients or society.

This problem of marginal benefits is not, Dr Gyawali noted, a consequence only of cancer medicines having a special status in society, but “that the industry can easily hijack the tools of evidence-based medicine to prove small marginal gains as statistically significant while they have no clinical meaning to patients.

“A few such strategies are: overpowered trials that can show even a week of OS gain as significant, using non-inferiority trial design to show that the new drug is not inferior rather than superior to a previous drug, using inferior controls to show the intervention arm as doing better, and using excellent patients in trials to show gains that get lost when used in real world patients.”

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