By Judith Garber
This piece was originally published for the “Slow Medicine” segment on the Center for Health Journalism blog.
Health insurers have gotten a lot of bad press recently – and much of it is well deserved. From Blue Cross Blue Shield denying emergency room claims that aren’t “real emergencies” to Aetna’s medical director admitting he did not review patient recordsbefore denying coverage, major insurers have done plenty to keep their PR teams busy.
However, a recent CNN story about an insurer denying coverage uses the narrative of the “heartless insurer” that would not pay for an unproven and expensive treatment. The article tells the story of Kate Weissman, a young woman with cervical cancer. Her doctors recommended proton beam therapy (PBT), but she was denied coverage by United HealthCare. Weissman and her family describe the painful process of unsuccessfully appealing the decision before they eventually decided to cough up a whopping $95,000 of their own money to pay for the treatment.
The story is painted as yet another case in which insurers deny paying for whatever they can, to the detriment of patients’ physical, emotional, and financial health. The difference in this case is the lack of evidence supporting proton beam therapy compared to standard radiation treatment in most cases.
Proton beam therapy is an alternative to standard radiation that uses protons instead of photons to target cancerous tumors, avoiding radiation of surrounding tissue. Because PBT theoretically reduces radiation exposure, it is useful for treating cancers in sensitive areas like the brain stem, eye or spinal cord, and for treating cancer in children without giving them excess radiation.
There is a stunning lack of evidence to support proton beam therapy for common cancers.
However, there is a stunning lack of evidence to support PBT for more common cancers — despite the fact that PBT has been around for more than 30 years. Comprehensive reviews of the evidence by the independent nonprofit Institute for Clinical and Economic Review in 2014 and the American Society of Radiation Oncologyin 2012 found evidence of net benefit from PBT only for ocular tumors, brain and spinal tumors, and pediatric cancers, compared to standard radiation treatment. For hepatocellular carcinoma and liver, lung, and prostate cancer, PBT was “comparable” but not better than standard radiation. For all other cancers, there was not enough evidence to determine a health benefit.
Even more recent research hasn’t helped the case for PBT. A 2016 report from the Canadian Agency for Drugs and Technology found that “overall, none of the clinical evidence was suggestive of a substantial incremental benefit of PBT over photon radiotherapies.” In 2016, the first randomized controlled trial for PBT for lung cancer found it no more effective than conventional radiation therapy and no less toxic. And a 2018 systematic review of quality of life and patient-reported outcomes after PBT found increased benefit only for certain brain, head and neck, lung, and pediatric cancers.
The authors of these systematic reviews make it clear that the quality as well as quantity of evidence for PBT is very low. Most of the studies on PBT are retrospective, which are more likely to have errors of confounding and bias. In many studies, the group receiving PBT and the control group had major demographic and clinical differences, and many trials did not measure important endpoints such as survival, recurrence of cancer and long-term toxicity.
Given the lack of evidence that PBT is more effective for cervical cancer, Weissman’s doctors in the CNN story dismiss these concerns, repeatedly using Weissman’s recovery as “proof” that PBT was the right choice:
“Being cancer-free two years later without major side effects, her doctors say, is proof of why they sought proton beam therapy.”
“Her recovery, Growdon tells her in the examining room, exemplifies why they sought the treatment.”
This narrative is extremely misleading. Weissman is alive and well now after PBT, but she could be doing just as well had she gotten standard radiation — the fact is, we don’t know. It is wrong to call PBT “lifesaving,” as this article does, because her life would likely have been saved by standard radiation as well.
The $95,000 question is not, “Does PBT work?” It’s “Does PBT work better than existing, less expensive treatments.”
This article is just one example of the media and medical institutions promoting tests and treatments that have not yet been proven to benefit patients. From immunotherapy, to at-home genetic testing, to screenings and scans, stories in health news often increase the hype around medical advances and new technology, without fully examining the extent of the evidence or the potential harms.
The $95,000 question is not, “Does PBT work?” It’s “Does PBT work better than existing, less expensive treatments.” The burden of proof is on advocates of the therapy to show that it’s superior; until then, we shouldn’t be paying for it.