We shouldn’t strong-arm insurers — and all of us who pay premiums and taxes — into paying for unproven treatments. Let research guide our health care.
Late last year, an Oklahoma jury awarded $25.5 million to Orrana Cunningham’s husband. In 2014, she had nasopharyngeal cancer growing behind her nose. Her doctors recommended proton therapy, a type of radiation treatment, but Aetna refused to pay. She created a GoFundMe campaign and mortgaged her home to pay $92,082. Unfortunately, Mrs. Cunningham died within months from complications of her cancer. Jurors condemned Aetna, saying it acted “recklessly” in its review and delay of her care, and that it “should’ve paid for the treatment.”
As oncologists we feel the heartbreak and tragedy of Mrs. Cunningham’s death at age 54. But her case raises an important societal question: Should Americans, through health insurance companies or government programs, pay for costly treatments that have not been shown to be more beneficial or less harmful than standard treatment, just because a physician prescribes them or a patient wants them? At stake are the health of millions of Americans with complex and debilitating illness, billions in health care costs, the affordability of health insurance, and data-driven medical care.
Proton therapy costs about twice as much as state-of-the-art radiation therapy. It has only been considered ‘medically necessary’ and a standard of care for selected cancers, including pediatric and brain and spinal cord tumors, mainly because its potential to reduce side effects, such as cognitive decline in a child’s developing brain.
Today, there is only one published randomized trial of proton therapy — for inoperable lung cancer — showing that proton therapy did not lessen toxicity compared to standard radiation. There are no randomized trials showing that patients with cancer live longer with proton therapy. While some research suggests better outcomes with proton therapy, other research does not. And, just like every medical intervention, proton beam radiation may be harmful. In 2018, the National Cancer Institute published a consensus warning that proton therapy may have unexpectedly caused brainstem necrosis in children, a devastating, but rare, complication of radiation.
Proton therapy’s benefits aren’t established
For the vast majority of adults with cancer, it is still not clear proton therapy provides benefits. Currently, the National Cancer Institute and Patient-Centered Outcomes Research Institute are funding seven randomized trials comparing proton therapy to standard radiation for treating cancers of the lung, breast, prostate, liver, esophagus and brain to answer the question. (We are conducting one of these trials for breast cancer.)
There are 26 proton centers in operation and three under construction or development. Yet, each year only a few thousand Americans could clearly benefit. That’s not enough to support all these facilities, the least expensive of which costs over $40 million. Some proton centers have gone bankrupt. Others use the treatment across a wide swath of adults with cancer, where it may or may not lead to better outcomes.
But prescribing treatments that have not been proven more effective can harm patients, drive up health care costs, and increase insurance premiums and taxes for everyone. Consider what happened with bone marrow transplantation for metastatic breast cancer in the 1990s. With great hope but before any reliable data proving that it prolonged survival, patients with breast cancer, their doctors, their families and advocacy groups demanded insurers cover the $100,000 treatment. They sued insurers, and lobbied legislatures to enact laws requiring coverage. People condemned the insurers as putting profits over patients, just as the lawyer who represented Ms. Cunningham’s family charged.
Fringe treatments are expensive and harmful
Who was right? Ultimately, research showed that bone marrow transplantation did not extend life. The human and economic toll was profound: more than 30,000 women received the toxic treatment, some of whom died prematurely from it. The definitive studies were delayed because patients got transplants “off study” rather than as part of the research trials. And taxpayers and insurance policy holders paid billions for treatments that didn’t work.
The refrain repeats. Last month, a major randomized trial was published showing that robotic surgery, touted as a better but more expensive alternative to open surgery, actually reduced the life of patients with early stage cervical cancer — a highly curable malignancy — by about 10 percent. This research was also delayed because patients received the robotic surgery off study, meaning that thousands of patients with cervical cancer received a harmful treatment.
What should be done? First, as a society, we should stop demanding — and legally requiring — commercial insurers, Medicare, and Medicaid pay for proton beam and other expensive treatments before research shows them to be more effective than the alternative.
Second, industry and government funders, doctors, patients, hospitals and payers must accelerate impactful research. In particular, doctors should strongly endorse enrollment in randomized clinical trials and should counsel patients about the possible benefits and harms of the latest medical technologies.
Unproven treatments can be a waste of money
Third, insurers should pay for unproven treatments only when received through studies. Specifically, while the intervention is being tested, insurers and government programs should set the price of new treatments equal to the current standard of care, a model known as reference pricing. The price should be reset only if research ultimately shows better (or worse) outcomes. This approach promotes both access and innovation. The University of Pennsylvania already does this for proton therapy and has arranged with some insurers to review cases within 24 hours.
Fourth, rather than just arguing that their technology is safe and effective, companies making proton centers, surgical robots and other novel therapies should fund trials to answer the question of whether their products are actually more effective. This is what drug companies do. Funding research is in their interest. When one manufacturer’s heart stent was shown to be better than another’s in a randomized trial published in 2008, there was a $1.7 billion movement in market share in a single year toward the better therapy.
The way to advance medical care for seriously ill patients like Mrs. Cunningham is not to strong-arm insurers — and really all of us who pay premiums — into paying for unproven treatments. It’s to have doctors, researchers and scientists prove as quickly as possible what care is best, and then put it into practice widely once the conclusions are in..
Ezekiel Emanuel is an oncologist, chair of the Department of Medical Ethics and Health Policy at the University of Pennsylvania and most recently author of Prescription for the Future. Justin Bekelman is a radiation oncologist at Penn Medicine and director of the Penn Center for Cancer Care Innovation at the Abramson Cancer Center.