Doctors around the world are taking risky gambles with cancer patients’ lives – as part of a well-intentioned, but misguided, attempt to reduce the cost of treatment.
Hospitals in India, Israel, the Netherlands, and elsewhere have recently experimented with reducing the dose size for blockbuster cancer drugs – and some patients have reportedly responded well. Based on that anecdotal evidence, some activists want more doctors to stretch patients’ doses. They claim that this doctor-directed rationing could save billions of dollars in health-care costs without undermining patient outcomes.
But deviating from dosing regimens approved by regulatory agencies like the FDA and the European Medicines Agency is not a risk-free tweak. Before approving a drug, regulators demand that pharmaceutical companies exhaustively test various doses of experimental treatments to determine which ones are safest and most effective for patients.
Encouraging doctors to change that dosing – without rock-solid evidence from legitimate clinical trials – effectively turns patients into unwitting guinea pigs and exposes them to serious harm.
The people advocating for reduced dosing generally mean well. If stretching a monthly dose genuinely had no impact on patients’ health, it’d seem like an easy way to save money, since cancer medicines often cost thousands, or even tens of thousands, of dollars per month.
But a drug’s efficacy at its standard dose does not guarantee that lower doses will work equally well. The FDA approves specific doses based on extensive evidence from both the initial clinical trials, as well as follow-on trials conducted after the initial regulatory approval.
For instance, Keytruda – an immunotherapy that’s approved for more than 40 different uses – was initially approved with weight-based dosing. But the FDA ultimately approved the current fixed dose because later evidence indicated that it was the most convenient and reliable approach for broad patient populations.
Reversing course – without equally strong evidence – would undermine the safeguards meant to protect patients.
If researchers believe an immunotherapy works just as well at a lower dose, there is a safe way to prove it. A company or investigator can study that exact regimen in a series of clinical trials. If the evidence shows strongly that the new regimen is safe and effective, FDA reviewers can decide to add it to the drug’s official label.
That pathway is not an unreasonable burden. Many drugs have gone through additional clinical testing as their approved uses expanded.
There is no good reason to accept a lower standard now. If anything, the plight of patients in developing countries like India is a reason to demand more scientific rigor, not less. Those patients are depending on scientists and regulators in wealthy countries like the United States to develop and test experimental medicines and determine which doses and delivery mechanisms work best.
That’s precisely what Americans have done. When doctors disregard that work, and instead start freelancing, it puts patients’ lives at risk.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest. This column originally appeared at DCJournal.com.
