Chuck Mansky, an economist at Northwestern University, studies decision making under uncertainty. This prepared him better than many other cancer patients to evaluate whether to continue immunotherapy that was causing him severe illness.
Over a period of 6 months in 2022, Mansky received monthly doses of the drug nivolumab to combat advanced melanoma.
He said that the treatment caused him damage to the thyroid gland and severe dryness in the eyes, lips and mouth, which led him to question the feasibility of continuing it for a year, as recommended by the US Food and Drug Administration.
At that point, Mansky had no signs or symptoms of cancer.
After reviewing several medical studies, he concluded that the severity of the side effects likely indicated that the treatment had achieved the maximum possible benefit.
He said in an interview in June in Spain, where he was receiving an award for his economic research: “The oncologist could not assure me that a full year was the ideal duration of treatment, and no one could confirm that... so I made the decision myself and stopped my treatment.”
This experience prompted Mansky to join a group of researchers, doctors and patients, seeking to determine the appropriate doses and optimal treatment duration for cancer drugs.
Members of the group believe that reducing doses or duration of treatment could save billions of dollars and reduce severe side effects.
A recent survey conducted by the KFF Foundation showed that 43% of adults in the United States did not adhere to taking their medications during the past year due to their high cost.
A study conducted by Vanderbilt University, published in 2022, also found that 30% of cancer drug prescriptions for Medicare beneficiaries were not filled by pharmacies.
However, it is rare to conduct studies to determine the appropriate doses after putting drugs on the market, while pharmaceutical companies have no interest in reducing doses, because that may reduce sales.
A study estimated that using lower doses of 29 cancer drugs could have saved about $31 billion in 2024.
“Decisions are not always made in the best interest of patients; there are also financial considerations,” said Matthew Goetz, MD, a breast cancer researcher at the Mayo Clinic Comprehensive Cancer Center.
Although some oncologists treat their patients with low doses of some cancer drugs, they fear that they may be exposed to lawsuits from patients or their families for prescribing lower than recommended doses.
In doing so, Julie Gralow, executive vice president and chief medical officer of the American Society of Clinical Oncology (ASCO), has made studying dosing a priority.
In this context, it is working with scientists in India on an ambitious clinical trial that compares the usual dose of “nivolumab” with four lower doses. It is also leading an $11 million trial to investigate the effectiveness of lowering the doses of some breast cancer drugs.
“We want to maintain the effectiveness of the treatment, but we also want patients to have a good quality of life,” she said, especially patients with advanced cancer, in whom a complete cure is unlikely.
In 2021, Richard Puzder, who headed the FDA's cancer drugs division for years, launched "Project Optimus" to encourage companies to determine optimal doses before approving drugs.
Emily Hilliard, a spokeswoman for the US Department of Health and Human Services, said that the administration has issued non-binding guidance on studying doses in 2024, and has also incorporated the principles of “Project Optimus” into the approval process for new cancer drugs.
Jessica Bowman, from the Fox Chase Cancer Center in Philadelphia, points out that these efforts may provide more accurate information about appropriate doses, but they do not necessarily mean a lower cost of treatment.
“New drugs are likely to remain expensive, even at lower doses,” she said.
