Thanks to an unprecedented and highly contentious clinical trial that it sponsored, the CMS now has proof in hand that a costly and difficult lung operation offers a modest measure of improvement for a small portion of the 2 million Americans who suffer from emphysema.
As a result, Medicare announced in August that it will reimburse for the operation for some beneficiaries, but that still hasn't healed the hard feelings or quieted the national debate over who, if anyone, should finance large-scale clinical studies testing new surgical procedures.
"Given how much abuse we took for insisting on the trial and refusing to pay for (the procedure) outside the trial, I must say I feel somewhat vindicated by the results," says Bruce Vladeck, who launched the study in 1997 when he was administrator at HCFA, predecessor to the CMS. Vladeck is now professor of health policy and geriatrics at Mount Sinai School of Medicine in New York. "I think the results are very clear for some patients but for others, the risks outweigh any benefits. Now we have a rational basis for figuring out for which patients under which circumstances to cover it."
As Medicare wraps up its years-long deliberation as to whether it should pay for the controversial procedure known as lung volume reduction surgery, nearly everyone concerned agrees that the right thing was done, although some question whether it was done for the right reasons.
"It's a complex problem. On the one hand Medicare has a long history of being slow or recalcitrant in providing services that have been accepted by the medical community as appropriate and that were paid for years by private insurers. If you want to be cynical you could say this was just another delaying tactic," says Joel Cooper, chief of cardiac and thoracic surgery at Washington University School of Medicine and Barnes-Jewish Hospital, both in St. Louis, and the pioneer of the revival of the procedure in the early 1990s. "On the other hand to be fair to Medicare, after we (revived) this operation, there was a lot of abuse."
Like it or not, a safety net called the Food and Drug Administration ensures, at least to some degree, that innovative drugs and medical devices won't reach the market until it is proven that, at the minimum, the medical advance in question won't unnecessarily kill anyone. But when it comes to new surgical procedures, which are not subjected to the same kind of regulatory oversight, physicians and hospitals tend to put the cart before the horse, pioneering techniques on patients before clinical trials can verify the results. Most of the time, however, because of the difficulty in designing and financing them, far-sweeping clinical trials testing new surgical procedures are never conducted at all.
Perhaps nothing in medicine illustrates this conundrum better than lung volume reduction surgery. And with the lukewarm positive results now in, it still remains to be seen if hospitals will rush to be accredited to perform the surgery, let alone if it will be financially worth the effort.
"I think this is an area where patient safety and medical economics go hand in hand," says John Reilly, associate professor of medicine at Harvard Medical School and clinical director of the division of pulmonary and critical-care medicine at Brigham and Women's Hospital in Boston. Brigham and Women's was one of 17 clinical sites nationwide that participated in the study.
Emphysema is a chronic lung condition in which breathing becomes difficult because of damage to the walls between the lung's tiny air sacs. The condition eventually leads to reduced elasticity, making the lungs floppy and overexpanded like a spent balloon. Cigarette smokers are at greatest risk for the disease, which predominantly affects people over age 50. In the reduction surgery, as much as 35% of the lung is surgically removed based on the theory that the smaller lung will pull open the airways and allow the breathing muscles to return to a more comfortable position so that they can work more efficiently.
A surgical resurgence
The surgical procedure was pioneered in the late 1950s and early 1960s but it soon fell out of favor because of alarmingly high mortality rates. Then in the mid-1990s, the surgery experienced a short-lived renaissance when Cooper revived the technique and reported much better results thanks at least in part to his expertise as a lung transplant surgeon. Impressed by Cooper's published data, lung surgeons at hospitals nationwide readily began adopting it. "People thought it was the lung equivalent of coronary artery bypass surgery," Reilly says. In truth, the procedure is as complex and as fraught with risk as lung transplantation, he says.
All along Medicare had-unwittingly, some say-paid for the procedure. It was the federal government's abrupt decision in December 1995 to discontinue paying for the surgery that precipitated the first-of-its-kind Medicare-sponsored clinical trial designed to determine the procedure's safety and effectiveness.
The decision to discontinue payment and then, later, proceed with the clinical trial was based "on the lack of medical evidence and potential for extensive morbidity and mortality among Medicare beneficiaries," Vladeck later testified before Congress.
Analyzing its data after it made the decision to stop covering the procedure, HCFA identified 711 Medicare claims for the surgery from October 1995 to January 1996. Of those patients, 185 people-26%-had died by January 1997. The alarmingly high death rate and a correspondingly high risk of complications-40% of the patients were hospitalized within about 12 months of surgery-provided a compelling rationale for a controlled study, Vladeck said at the time.
But the most vocal opponent of the clinical trial says that clinically there was nothing to prove. "The trial had little to do with the operation," Cooper of Washington University says. "What it had to do with was how you control and introduce in a responsible fashion new procedures and often unproven procedures. How do you introduce them into the medical system without bankrupting the system and without denying patients access to a promising procedure which may be their only hope?"
Cooper says he strongly objected to the randomized nature of the trial, which by a roll of the dice blocked half of the volunteers from getting the surgery. He also objected to Medicare's stipulation that it would pay for the surgery only for beneficiaries participating in the study. Though selected to participate in the trial, Cooper and his team at Barnes-Jewish withdrew. "We felt that (methodology) was very coercive," he says.
Before the trial, Cooper says his center performed about 150 of the operations, perhaps two-thirds of those on Medicare patients. He and his team have operated on another 200 patients since the trial began though not with Medicare's blessing. He says he sued Medicare on behalf of his patients 28 times and won every case.
Eventually, 1,218 emphysema patients were selected for the trial, all of whom underwent six to 10 weeks of medical therapy also known as pulmonary rehabilitation. Of those, 608 patients were randomly assigned to also undergo surgery. In May 2001, trial investigators stopped enrolling a subgroup-140 patients out of the 1,218-who were determined to be at a very high risk of dying from the operation with very little benefit. "These were the worst of a bad lot, you would say," Reilly says.
Results of the completed study were published in May in the New England Journal of Medicine along with another study on its cost-effectiveness. The study found that the procedure improves the quality of life for certain patients, but it does not necessarily improve chances of survival except for a small group of patients whose emphysema is predominantly in the upper reaches of the lungs and who also have a low exercise capacity. Two subgroups out of five, including the group that was withdrawn early, were found to get no benefit. In terms of cost-effectiveness, the operation was found to be less beneficial in the short term compared with medical therapy because of the cost of the surgery, bad outcomes, lengthy hospitalizations and a greater number of nursing home admissions the first few months after the surgery.
But no one is sure why some patients do well with the surgery and others do not. "It's like the stock market," Reilly says. "After the fact people are happy to tell you why it did what it did but not before."
Based on the results of the National Emphysema Treatment Trial, the CMS announced in August that it intends to pay for the procedure for three subsets of Medicare patients but only at hospitals specifically accredited by Medicare for the procedure, including those hospitals that participated in the trial. The CMS, which sponsored the study along with the National Heart, Lung and Blood Institute and the Agency for Healthcare Research and Quality, is developing accreditation standards and hasn't yet determined how much it will reimburse. The best guess is that the national coverage decision will be completed in six to nine months, says Frank Sciurba, associate professor of medicine in the division of pulmonary and critical medicine and director of the emphysema research center at the University of Pittsburgh Medical School.
"We now have better, not perfect, but better guidelines as to who will do well and, most importantly, who we would hurt," says Sciurba, the principal investigator for the study at the Pittsburgh site, which enrolled 65 patients with half that number getting surgery. From 1994 to 1997 when the trial got under way, Pittsburgh surgeons performed 200 procedures, he says. For the trial, the surgeons operated using a less invasive video-assisted technique where instead of one large incision, doctors make three small cuts. Data from a study comparing the two techniques is still being analyzed, he says.
The AHRQ estimated Medicare's cost per patient in the trial was about $61,000 for the surgery and the first year of hospitalization, according to a Medicare spokesman. With the results now in, Cooper estimates that one in 15 emphysema patients may be appropriate candidates for the procedure, perhaps up to 10,000 U.S. patients per year. Reilly, who oversaw about 70 patients in the trial at Brigham and Women's, estimates the operation outside the Medicare trial would cost from $20,000 to $45,000, with an average length of stay of eight to 10 days, "with a proportion (staying) in longer."
Reilly says he will be interested to see what happens now that the CMS has opened it up again, but he isn't expecting a flood of patients. "It's a big operation and people are understandably nervous about going for an operation where you might have a 5% chance that you are not going to survive," he says. "These patients are sick. The best operation economically is one that doesn't have complications and has a reasonable reimbursement rate, so if a hospital is going to do it, they will need to set up a program with really good results. If a patient is in the ICU a couple of weeks, your profit margin is gone."
Though the number of centers that eventually perform the surgery may be limited, the procedure could generate business for hospitals that perform the medical testing, rehabilitation and diagnostics for evaluating patients for the surgery, Sciurba says. Cooper says evaluations would likely include chest X-rays, CT scans and sometimes even cardiac catheterizations. "I think most of the profit lies in evaluations," he says, adding that charges could add up to as much as $10,000 per patient.
"I think it will be a profitable item for hospitals, but the profits are not in the patients who have the operation but in the evaluation," Cooper says.