Many Oncology RCTs Do Not Adequately Capture Financial Toxicity


Financial toxicity is defined as financial hardship related to cancer and can have major effects on patients’ quality of life

Many oncology randomized clinical trials (RCTs) do not capture financial toxicity experienced by patients in real-life settings.

That’s according to findings of a cross-sectional analysis of articles published in Journal of Cancer Policy. Financial toxicity is defined as financial hardship related to cancer and can have major effects on patients’ quality of life (QOL).

In addition to measuring how financial toxicity was captured in RCTs, researchers also assessed how often the study drug or other expenses were covered by sponsors. The high cost of cancer treatment has garnered national attention in recent years.

“Novel anticancer agents often cost a patient more than $100,000 a year,” the authors explained. “In the United States, the national patient economic burden associated with cancer care was estimated to be $21.1 billion in, comprised of $16.22 billion out-of-pocket costs and $4.87 billion patient time cost.”

Moreover, it’s estimated 32% of US cancer survivors experienced financial difficulties related to the disease.

High costs of anticancer drugs and medical services can result in a range of negative consequences, including delayed medical care or decreased adherence to treatment.

To better understand how and if financial toxicity is measured in cancer trials, researchers searched for relevant articles published in 6 high-impact journals: The New England Journal of Medicine, The Lancet, JAMA, The Lancet Oncology, Journal of Clinical Oncology, and JAMA Oncology.

All included articles must have reported on an RCT between January 2018 and December 2019, studied an anticancer drug, and included QOL results.

Analyses revealed:

  • For all 73 studies that met inclusion criteria, 34 studies (47%) utilized QOL questionnaires without direct assessment of financial difficulties
  • Among trials directly assessing financial difficulties through QOL questionnaires, the financial component was not specifically reported in 29 reports (out of 39, 74%), reported with no difference in 7 trials (out of 39, 18%), or reported differences favoring the control arm (n = 1), the experimental arm (n = 1), or with mixed results (n = 1)
  • The study drug was provided by the sponsor in at least 51 trials (70%), provided according to local rules in 3 trials (4%), and undetermined in the remaining 19 trials (26%)
  • Two trials (3%) included payments or compensation to enrolled patients

These limitations suggest QOL assessments included in the RCTs are not generalizable to real-world settings. If drug expenses are covered in trials, enrolled patients’ QOL is not affected by out-of-pocket expenses, co-pays, or other treatment-related costs found in real-world settings.

Regulatory bodies often rely on QOL results from RCTs to inform their decisions, the authors noted. For example, “The FDA values health-related QOL when assessing the marketing authorization eligibility of novel anticancer therapies.”

Researchers note most QOL questionnaires included in the analysis could have been developed before financial toxicity was identified as a specific QOL component to be included in questionnaires.

“Our work highlights a tension: Clinical trials must protect patients from additional financial difficulties, and at the same time, financial toxicity and QOL data from these trials do not reflect the financial hardship experienced by patients in real-life settings,” the authors wrote.

Collecting real-world data may better characterize the financial toxicity patients face when receiving cancer care and treatment, while regulators could demand cohort studies and real-world evidence as a postmarketing requirement to ensure QOL results from RCTs reproduce outside trials, the researchers suggested.

The short period for included articles marks a limitation to the current study. In several instances, researchers were also unable to retrieve data about who supplied the study drug.


Olivier T, Haslam A, and Prasad V. Is financial toxicity captured in quality of life assessments in oncology randomized clinical trials? J Cancer Policy. Published online April 17, 2023. doi:10.1016/j.jcpo.2023.100423

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