Some cancer medicines can save lives, but they can also cause serious side effects and cost enormous amounts of money.
A growing group of doctors, researchers, and patients is asking a simple question: do some people receive more cancer medicine than they actually need?
That question became personal for Northwestern University economist Chuck Manski after he was treated for advanced melanoma. In 2022, he received monthly infusions of nivolumab, an immunotherapy drug that helps the immune system attack cancer.
The treatment caused major problems for Manski, including permanent damage to his thyroid and severe dryness in his eyes, lips, and mouth. After six months, he had no signs or symptoms of cancer, but the standard U.S. treatment plan called for him to continue for a full year.
Manski said his doctor could not tell him whether one year had been proven to be the best treatment length. After reading medical research himself and weighing the possible benefits against the side effects, he decided to stop treatment early.
His experience reflects a wider debate in cancer medicine. Some researchers believe that the doses used for certain modern cancer drugs may be higher than necessary for at least some patients.
This does not mean patients should reduce or stop cancer treatment on their own. The central issue is that more carefully designed studies are needed to identify the lowest dose and shortest treatment period that still controls cancer safely.
Doctors in several countries have already explored different approaches. In Canada, Israel, Sweden, and elsewhere, some specialists have used lower doses, longer gaps between treatments, or shorter courses of drugs such as nivolumab and pembrolizumab.
Researchers in India have gone even further because full-dose immunotherapy is unaffordable for many patients. Some Indian studies have tested nivolumab at only a fraction of the standard amount and found meaningful benefits compared with older chemotherapy.
However, those results have an important limitation. The very low doses were generally compared with chemotherapy rather than directly with the standard full dose of nivolumab, so researchers still cannot say that the tiny doses work equally well.
That is why many cancer specialists want randomized trials that directly compare different dose levels. Such studies could show whether lower doses preserve survival while reducing side effects, treatment visits, and costs.
The financial stakes are huge. The source article reports that one analysis of 29 expensive cancer medicines estimated that using the minimum necessary doses could have saved the U.S. health system about $31 billion in 2024.
Modern immunotherapy drugs are particularly valuable products. Pembrolizumab, sold as Keytruda, and nivolumab, sold as Opdivo, generate billions of dollars in annual sales and are used for many different cancers.
This creates a difficult economic problem. Drug companies have little financial reason to prove that patients can safely use less medicine, while hospitals and other parts of the health system can also receive revenue linked to expensive infused drugs.
Drugmakers argue that changing treatment without strong evidence could put patients at risk. Merck said recommended doses are based on extensive testing and warned that untested reductions in dose or treatment length could reduce a medicine’s ability to fight cancer.
That concern is important because cancer treatment is not an area where doctors can simply assume that less is equally effective. A lower dose that reduces side effects but also allows a cancer to grow would clearly be a poor trade-off.
Still, some doctors already begin certain treatments below the labeled dose when they believe toxicity is a major concern. Breast cancer specialists, for example, are studying whether medicines such as Kisqali and Ibrance can be started at lower doses while maintaining their benefits.
Researchers are also studying treatment length. A U.S. trial is examining whether patients who respond well to pembrolizumab after about 27 weeks can stop instead of continuing for the longer period commonly recommended.
Another approach is to give treatment less often. A pilot program at three Veterans Affairs hospitals reduced the frequency of pembrolizumab treatment and reportedly saved about $1.5 million over two years while also reducing travel and freeing infusion appointments for other patients.
The U.S. Food and Drug Administration has also recognized the problem. Its Project Optimus program encourages drug companies to study dosing more carefully before large cancer trials are completed and before a new medicine reaches widespread use.
Recent drug approvals show why this can matter. According to the source article, several newer lung cancer drugs were tested at two dose levels, and the lower dose was approved when it offered fewer harmful effects.
The larger lesson is that finding a cancer drug that works is only part of the job. Doctors also need to know how much medicine is truly necessary, how often it should be given, and how long treatment should continue.
The evidence described here is promising but incomplete. Lower dosing may reduce side effects, costs, and treatment burden, but ultralow-dose immunotherapy has not yet been proven equivalent to standard treatment in the strongest direct trials.
For patients, this means dose decisions should remain a discussion with an oncology team rather than a do-it-yourself experiment. For researchers and health systems, the study of dosing deserves far more attention because the best cancer treatment is not necessarily the largest dose, but the dose that gives patients the greatest benefit with the least unnecessary harm.
Source: KFF Health News.
