The study seems likely to reignite the national debate about how best to rein in runaway health care costs. It found that adding an injectable disease-modifying drug to the treatment of MS patients roughly doubles the cost of care, while only providing small population-level improvements in survival and quality of life.
Doctors who were not involved in the study were alarmed by its findings.
"We know, unequivocally, that these drugs slow the progression of the disease and slow the course of the disease," says Karen Blitz-Shabbir, MD, director of multiple sclerosis care center at North Shore-LIJ Glen Cove Hospital in New York.
"When I first started training, people were hospitalized all the time and did much worse than they do now," she says, "So we know for sure these drugs are good."
But the cost of the drugs continues to soar.
Even in the face of new competition from a new pill, pharmaceutical companies recently announced increases of nearly 40% over 2010 prices. Disease-modifying drugs for MS carry wholesale prices of as much as $48,000 a year.
"Will the health care companies, the health care industry, prevent patients from accessing the drugs because of a study like this?" Blitz-Shabbir says. "The real problem is the pharmaceutical cost is out of control."
The study is published in Neurology.
Calculating Cost-Effectiveness of MS Drugs
Using mathematical models developed using data from a large, national patient survey, researchers found that the cost-effectiveness for the disease-modifying drugs Avonex, Betaseron, Copaxone, and Rebif exceeded $800,000 per quality-adjusted life year.
A quality-adjusted life year is a measure that tries to account for both the length of time a person could expect a treatment to extend life and how good he or she feels during that time.
For example, the study found that on average, patients taking Copaxone for 10 years could expect the drug to add less than one quality-adjusted month to their lives compared to patients who were only getting supportive therapy to control symptoms.
Patients taking Avonex, Betaseron, and Rebif gained about two quality-adjusted months compared to those getting supportive care.
The study also looked at how likely the drugs were to prevent relapses.
Those taking disease-modifying drugs spent on average nearly six out of 10 years with no relapses, compared to five years with no relapses for those who did not take disease-modifying drugs.
However, the effectiveness varied significantly among individuals, with some experiencing double the improvements and being able to live independently and prolong their employment and others not getting any meaningful effect.
Why Cost-Effectiveness Matters
"We were surprised how non-cost-effective they are," says study researcher Katia Noyes, PhD, MPH, assistant professor of health services research at the University of Rochester, N.Y.
Cost-effectiveness is a metric used by economists to try to gauge the size of the benefit patients appear to get for their health care dollar.
It's become a buzzword as the government and health insurers try to figure out which treatments are worth covering and how to control skyrocketing health care costs.
In the U.S., there is no set threshold that determines cost-effectiveness, but other countries and some health care agencies use a figure around $50,000 per quality-adjusted life year as one cutoff.
The study found disease-modifying drugs for multiple sclerosis are about 16 times more expensive than that.
"When we talk about effectiveness, we're talking about average effectiveness. If we look at the individual person, many of them will have great benefits, and there will be some who have no benefit at all or even negative effects," Noyes says. "So here is a trade-off between national policy or population-based policy and individual patients."
For the study, researchers extracted data on 844 people with MS who were part of a large national patient database. They collected information on things like health care costs, lost work time, disease severity, disease progression, and medications, and they used that information to develop mathematical models.
Over 10 years, the models estimated that the average cost to treat one patient was $267,710 with supportive care, but nearly doubled when researchers added the costs of disease-modifying drugs to the equation.
Overall, health outcomes for patients on the disease-modifying drugs appeared to be only slightly better than for those on supportive care.
Making Improvements in Cost-Effectiveness
Other countries pay far less for the same medications.
Patients in the U.K., for example, get the drug Betaseron for about $12,000 a year, while it costs about $34,000 in the U.S.
Researchers say that if the U.S. were to see prices for these drugs like those paid by patients in the U.K., the treatments could achieve more accepted levels of cost-effectiveness.
The cost-effectiveness also improved when people were started on the drugs early in the course of their disease, when the medications could still prevent permanent nerve damage, perhaps keeping them healthier, longer.
The impact of the study will be "a good deal of discussion concerning where we are in the treatment of MS in terms of cost, obviously, and in terms of where we would like to go in the future," says Nicholas LaRocca, PhD, vice president of health care delivery and policy research at the National Multiple Sclerosis Society. The organization helped to fund the study.
"I would imagine that some people will look at this study and will feel that it will discourage the availability of treatment, but I don't really see that happening," he says. "We need to do more studies like this."
Other experts pointed out that the high costs are already hitting patients hard.
"Some patients will get a new prescription, but they don't get the prescription filled because it's so costly," says Kathleen A. Smyth, PhD, co-director of the Neurological Outcomes Center and associate professor of medicine at Case Western Reserve University.
Symth, who wrote an editorial that accompanied the study, says she thinks the message of the study isn't that patients shouldn't be treated with the drugs. "It's that we have to get drug prices under control in this country."