Proton beam machines have become increasingly popular investments for health systems, despite the lack of evidence to support the use of proton beam therapy (PBT) for all but a few types of cancer. Comprehensive reviews of the evidence by the independent nonprofit Institute for Clinical and Economic Review in 2014 and the American Society of Radiation Oncology in 2012 found evidence of net benefit from PBT only for ocular tumors, brain and spinal tumors, and pediatric cancers, compared to standard radiation treatment.

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.

As Emanuel points out, there aren’t enough patients with ocular, brain, or pediatric cancers to warrant so many proton beam machines in the region. So why do hospitals keep building them? Because hospitals would rather refer patients in-house than lose cancer patients to other health centers, explained Brownlee. Unfortunately, as we’ve seen with complications at some pediatric surgery centers, having more centers that conduct fewer procedures each actually increases the chance of complications.

“The reality is, if you are one of the rare adult cancers that can be treated more effectively with this machine, it’s a lot better to send you to a place that does it all the time and has a lot of experience with it,” Brownlee said.

Increasing investments in unproven therapies like PBT is not just a financial risk for the hospital–it increases health care costs for everyone. If insurers pay for PBT (which they are being increasingly pressured to do), insurance premiums will go up. If they don’t pay for PBT, the hospital may cut into other areas to cover their costs. And perhaps most importantly, we have to take into account the enormous opportunity cost of investing in PBT rather than more impactful community health initiatives. 

“There’s real harm in spending money on something that’s not better and is more expensive,” Brownlee said, “and that harm is that hospital is not investing in something else that could improve its community or improve the health of more patients.”

The post Is high-tech always worth the high cost? Experts question Kansas City’s new proton beam machine appeared first on Lown Institute.

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.

The post The narrative of “bad insurer” hides bad evidence appeared first on Lown Institute.

But what happens when the insurance company denies payment for a treatment that hasn’t proven to be more effective than existing treatments, and is twice as expensive? In the case of proton beam therapy, you have a battle of wills, with an industry-backed patient advocacy group fighting against insurers for coverage of dubious treatment, to fund a medical arms race. 

The proton beam explosion

Proton beam therapy (PBT) is a relatively new cancer treatment that uses protons instead of photons to target cancerous tumors, avoiding radiation of surrounding tissue. The popularity of PBT to treat certain cancers has grown substantially in the past decade, as more and more health systems are building proton beam machines and centers for treatment. In 2009 there were six proton beam units; now there are 27, with at least 20 more in the works. 

With the explosion in proton beam centers — which cost at least $200 million to build — this therapy must be revolutionary! Well, not exactly. Proton beam therapy is useful for treating cancers in sensitive areas like the brain stem, eye, or spinal cord, and for treating pediatric cancer patients without exposing children to unnecessary radiation. However, there is little evidence that PBT is more effective than controlling conventional radiation treatment for more common cancers like lung, breast, or prostate cancer. 

Show me the evidence…

In a 2014 comprehensive summary of evidence on proton beam therapy, the independent non-profit Institute for Clinical and Economic Review evaluated the effectiveness of PBT on patient outcomes including tumor recurrence, quality of life, and mortality. Researchers found that PBT offers a superior net health benefit for eye tumors and an incremental benefit for pediatric cancers and tumors in the brain and spine. For liver, lung, and prostate cancer, PBT is comparable to conventional radiology; for all other cancers, there was insufficient evidence to determine net health benefit. 

The ICER report highlighted the overall low strength of evidence for PBT. Five of the six randomized controlled trials did not compare PBT to an alternative treatment. In the non-randomized studies, researchers found “major differences in patient demographics and baseline clinical characteristics” between patient groups. Out of 42 studies, only two were categorized as “good quality” evidence.

Even leaders of proton beam centers have acknowledged that the evidence isn’t really there. “Part of the challenge is that there hasn’t been a lot of clinical evidence to promote proton therapy,” said Stuart Klein, executive director of the University of Florida Proton Therapy Institute in Jacksonville, in Modern Healthcare. However, Klein is convinced that “as time goes on, the level of clinical evidence is going to increase.”

Most of the hospitals and academic centers that have been creating multi-million PBT machines are non-profit, which means that taxpayer dollars are funding this expensive, ineffective treatment that the builders know isn’t proven to work. 

Industry-funded advocacy

Given the lack of evidence for proton beam therapy compared with existing therapies, it’s not surprising that insurers have balked at paying for PBT for all but a few types of cancer. This phenomenon, coupled with advances in traditional radiation therapy over the past few years, means that proton therapy centers are having trouble finding patients, and are subsequently hemorrhaging money. 

Proton beam manufacturers and centers aren’t letting that stand. They are putting pressure on insurers to cover proton therapy treatment through their advocacy group, the Alliance for Proton Therapy Access. APTA recently put out a report chronicling the stories of patients whose proton beam therapy was denied, blaming the insurance industry, and listing the “patients’ bill of rights” for timely proton beam treatment.

The APTA report focuses on the stories of rejected cancer patients, asserting that patients have a right to the treatment their doctor recommends without dealing with delays and denials from insurance companies – and who can argue with that? Indeed, insurance companies often deny claims without adequate review or information. But APTA is deliberately blurring the lines between treatment that is necessary and recommended, and treatment that is not proven to be more effective. Their report doesn’t just include stories of patients with eye and brain cancer, or children with cancer – it includes patients with common cancers, for whom there is no evidence that PBT is more effective. In fact, the report states that “cancer patients of all ages” need access to PBT (emphasis mine). 

Just like with the controversial FDA approval of Sarepta’s drug for Duchenne Muscular Dystrophy, industry leaders with a stake in PBT are hiding behind patient stories to promote their own agenda. Just take a look at the APTA board members: Four industry leaders, one head of a PBT center, and two patients. Those who stand to gain financially from PBT coverage are taking a page right out of Sarepta’s handbook, creating “slickly packaged testimony” from cancer patients and encouraging patient advocates to take up the fight for their rights.

Don’t fall for this trick. Patients have the right to the medical treatment they need to get better, without a fight from insurance. They do not deserve to have their stories used to further industry gains based on unproven, expensive treatments.

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