Men hoping to avoid some side effects of prostate cancer treatment are shelling out tens of thousands of dollars for a procedure whose long-term effects are unknown and insurers, including Medicare, won't pay for.
Proponents say high-intensity focused ultrasound (HIFU) can have fewer negative side effects than surgery or radiation, while giving some patients another option between actively watching their cancer and those more aggressive steps. Critics, however, say the procedure is being oversold, leading some patients to get a treatment they don't need.
Device makers are busy selling the $500,000-and-up machines to doctors around the country and offering training courses. Billboards advertising this "new non-invasive treatment for prostate cancer" are springing up, while treatment center websites promise "a safer method" with benefits such as "no erectile dysfunction and no incontinence," although studies show those side effects can occur, but less often than with other types of more aggressive treatments. The treatment can range in cost from $15,000 to $25,000.
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HIFU is the latest treatment to prompt concerns over whether there should be limits -- such as requiring tracking of results -- placed on expensive new technology while additional data is gathered.
"This is going to join the group of uncertain-yet-available therapies that physicians can use, yet we have no clear understanding of who will benefit in a real world population," said Art Sedrakyan, a professor of health care policy and research at Weill Cornell Medicine in New York. The treatment of prostate cancer has been a particularly controversial -- and lucrative -- niche, since the disease for some men can be slow growing and their tumors wouldn't be fatal. A host of new "non-surgical' treatments are now also on offer using sophisticated machines to destroy cancer cells with proton beams or other types of high dose radiation.
Using HIFU, devices direct ultrasound waves to heat prostate tissue to about 195 degrees, ablating all or just portions of the gland. Focusing on just a portion of the gland is a newer trend in prostate cancer treatment. Anesthesia is used.
HIFU machines have been used in Europe longer than in the U.S, although national health programs in the United Kingdom and elsewhere limit coverage to patients enrolled in clinical trials or other research programs. While the devices are approved in Canada, the national health program does not pay for it. Until recently, some U.S. men traveled to have the procedure done by U.S. doctors who set up shop in Mexico, the Bahamas or Bermuda.
In the U.S., advisory committees to the Food and Drug Administration twice turned down applications from manufacturers to market HIFU devices as a treatment for prostate cancer, citing not enough long-term evidence.
But in October 2015, the FDA approved Charlotte, N.C.-based SonaCare Medical's device for the ablation of prostate tissue. Data submitted by the company included an analysis of 116 men who had their entire prostrate treated and were followed for 12 months. "While the oncological outcomes form this study are inconclusive, the results provide reasonable assurance of safety and effectiveness of the device in the context of prostate tissue ablation," the FDA said in its review.
A device by Lyon, France-based EDAP gained a similar approval shortly thereafter.
Researchers say it's too soon to state conclusively that partially treating the disease works as well as totally removing it. There is also debate over the type of patients best suited for the treatment: low risk, intermediate risk or those who have failed other types of prostate cancer treatment.
Ongoing and previous studies from abroad are available, but have limitations, including fairly short follow-up periods.
"The biggest studies in the world are only four or five years into it," said Michael Koch, chairman of the urology department at Indiana University School of Medicine, a proponent of HIFU for some patients. "We don't have survival data to see if [it] does better than surgery or radiation."
To get more complete answers, some physicians say it's critical to track outcomes with de-identified patient information in a nationwide registry.
It's not a new idea. Indeed, some technologies have been granted approval by the FDA or coverage by Medicare with a condition that patients must be enrolled in clinical trials or registries.
"Short of the FDA saying to device makers, 'You need to do this,'" establishing a comprehensive tracking method is challenging, said Jim Hu, a urologist and a robotic surgery specialist at Weill Cornell.
Hu co-authored a paper in the Journal of the American Medical Association in July with Sedrakyan and UCLA urology resident Aaron Laviana calling for a registry. Meetings between registry proponents, the FDA and the device makers are ongoing, but challenges remain, Hu said.
A registry is currently operating in England that will soon open to U.S. users of SonaCare Medical's HIFU devices. While SonaCare funds the registry, it does not have control of the data, said SonaCare CEO Mark Carol. His firm will also contribute funding to a broader U.S. registry that Hu and his co-authors support, which would incorporate results from other HIFU devices as well, calling the plan an "ambitious and worthwhile undertaking."
The evidence gathered could convince Medicare and other insurers that a treatment is valuable -- and worth covering. But some practitioners may not want insurers to cover the treatment because when a treatment isn't covered, cash payments by patients can often exceed what practitioners would be paid by insurers. Currently, men usually pay for it themselves.
"The financial piece of this is somewhat perverse," said Hu. "Men are being charged $25,000 for this, yet no one feels pressure to demonstrate the efficacy of the treatment."
Adding A New Alternative To Treatment Debate
Meanwhile, the continuing debate regarding patients' treatment options has created an opportunity for HIFU.
Surgery and radiation can pose problems such as incontinence or impotence, while hormonal treatments also cause impotence and can also result in hot flashes, muscle weakness and other problems.
When the cancer is aggressive, the benefit of these approaches outweigh such side effects. But for men with lower-risk profiles, based on factors such as age, and results of tests and biopsies, the choice is more difficult. Some health care experts encourage these patients to opt instead for "active surveillance," which means keeping an eye on the cancer through regular testing.
But some men are uncomfortable just watching -- and that's where HIFU could play a role.
Patients who have an area in the prostate with a higher grade tumor could choose to treat just that portion with HIFU, said Indiana University's Koch, who was part of the clinical trials of SonaCare's device in the U.S. and has accepted funding from the company.
"If we can treat [that area] with therapy, we can get them back on active surveillance," said Koch.
Others say more study is needed.
With prostate cancer, there may be a dominant tumor, but small cancer cells elsewhere in the gland, said Justin Bekelman, associate professor of radiation oncology at the University of Pennsylvania.
Still others note that patients who choose HIFU need to select physicians with lots of experience and training because the procedure is complex.
"HIFU is a steep learning curve. Some of the doctors buying these machines are not ready for it," said Jim Wickstrom, who had the procedure four years ago in Bermuda and is a big proponent. He says patients should do their research and choose only very experienced physicians who are willing to show their outcome data.
Registries could also help provide more information on outcomes, but one big challenge is who would pay for it.
"The main reason those (registry efforts) fail is the industry doesn't want to fund those studies, even if they think the treatment is better," said Sean Tunis, president and CEO of the Center for Medical Technology Policy in Baltimore and a former medical director at the Center for Medicare & Medicaid Services.
Mainly, that's because they don't need to unless required by the FDA, said Tunis. They already have marketing approval based on a limited number of studies demonstrating safety and efficacy.
Also, while registries are useful they also have limitations.
For one thing, because they are not randomly controlled trials, registries aren't the best way to compare treatment A to treatment B, said Fred Masoudi, a professor at the University of Colorado's medical school. But they can show how treatments, drugs or devices perform in common use.
"It's not a foregone conclusion that results will be the same [as in clinical trials], which is why registry programs are so important," said Masoudi, who has been involved with other such registries.
Where The Market Stands
SonaCare said it has sold more than 30 of the machines in the U.S., with medical centers in California, Florida, New York, North Carolina and Texas already using them. Competitor EDAP reported late in August that earnings from its HIFU division rose 68 percent in the first six months of 2016.
Patient advocates caution men to do a lot of research before choosing any type of treatment.
"With any procedure, there is a risk of temporary or ongoing issues with ED or incontinence," said Chuck Strand, CEO of Us Too, a prostate cancer patient group that is partially funded by industry. "Anytime anyone is looking at any therapy, we urge them to do their homework, ask lots of questions and get a second or even third opinion."