NEW YORK (Reuters Health) - Drugs used in the hope of slowing multiple sclerosis progression may help some patients, but at a very high cost, according to a study out Wednesday.
The medications in question are the so-called disease-modifying drugs (DMDs) that have been available since the 1990s to treat multiple sclerosis.
The drugs include beta interferons (brand-names like Avonex, Refib and Betaseron), glatiramer (Copaxone) and natalizumab (Tysabri). They are given by injection or infusion and can help prevent MS symptom flare-ups and delay long-term disability from the disease.
But the price tag is large, with each drug now costing upwards of $3,000 a month in the U.S.
The new study, published in the journal Neurology, estimates that people who use the medications for a decade would get a modest health benefit for the money.
They would gain an extra two months or less of good health over 10 years, the researchers say, compared with using only therapies that help ease MS symptoms -- like medications for pain, fatigue and muscle spasms.
Overall, the study estimated, DMDs cost close to $1 million for each year of relatively healthy life a person with MS could expect to gain with 10 years of use.
In contrast, common thresholds used to define a "cost-effective" treatment range between $50,000 and $150,000 for each good-quality year of life gained.
The figure for DMDs is an "order of magnitude higher," said Katia Noyes, a researcher at the University of Rochester in New York who led the study.
However, she stressed that the findings do not mean that people with MS shouldn't take the drugs.
"This study was not designed to try to deprive people with MS of any therapy," Noyes said in an interview. It was not intended, she said, to tell doctors what to prescribe or insurance companies what to pay for.
But, Noyes added, in the age of healthcare reform, studies need to look at the value of different medical therapies, and what factors seem to affect their cost-effectiveness for patients overall.
In the case of DMDs for MS, she said, starting earlier in the course of the disease appears to be better.
The researchers estimate that starting the drugs before any noticeable disability makes the medications more cost-effective -- though they still hover above $700,000 for each good-quality year of life gained.
Part of the issue is the nature of MS and how DMDs work. In MS, the immune system mistakenly attacks nerve fibers in the body, causing symptoms like muscle weakness, numbness, vision problems and difficulty with coordination and balance. In most cases, those symptoms wax and wane -- worsening for a period of time, followed by a period of milder symptoms or none at all. Eventually, the cycle often leads to physical disability.
Early on, DMDs can reduce the number of symptom flare-ups. But their central effect on quality of life would be in stalling disability, Noyes noted.
"The main benefit of these drugs is in the long-term," she said. "That may be 10 or 20 years down the road."
Noyes also pointed out that MS is "not unique" in having therapies with a high price for the benefit.
Other examples include implanted defibrillators for people at high risk of cardiac arrest, many cancer treatments and some arthritis therapies. And there are many medical interventions, Noyes said, whose cost-effectiveness has never been studied.
"The good news from this study is that these drugs do seem to be effective, on average," said Kathleen A. Smyth, a researcher at the Neurological Outcomes Center at Case Western Reserve University in Cleveland.
"The bad news is, these positive outcomes come at a very high price," said Smyth, who wrote an editorial published with the study.
People already on the MS drugs know well the high costs, she noted in an interview, but the price tag might come as a shock to new patients.
However, Smyth said, people with MS need to make treatment decisions on an individual basis, after discussions with their doctor -- and not based on cost alone.
Like Noyes, she pointed out that earlier treatment appeared more effective in this study. So while it may ultimately cost more, starting a DMD earlier may benefit patients more.
"I don't think there's anything in this study that should stop people from getting an early diagnosis or starting treatment," Smyth said.
She also noted that the picture may change in the future. When individual DMDs' patents eventually expire, for example, the cost of some could well come down. And then certain medications may become preferred by insurance companies.
Both Noyes and Smyth said the findings highlight a wider issue: the high price Americans pay for prescription drugs.
Avonex, for example, cost Americans with MS about $34,000 for the year in 2010. The price in the UK was equivalent to about $12,000 -- because that's all the National Health Service will pay for the drug.
And costs are only going up. Makers of the DMDs have raised their prices since last year, in response to the approval of the first oral MS drug -- Gilenya, made by Swiss drugmaker Novartis AG.
The annual cost of that medication in the U.S. will be about $48,000. But since it's a pill, the medication is expected to cut significantly into sales of the other DMDs.
As it stands, Smyth said, no particular DMD stands out as a "best buy" for Americans.
Manufacturers of the DMD drugs did not respond in time to Reuters Health requests for comment on the new study, however a spokesman for Biogen Idec, maker of Avonex, did offer a written statement on the question of drug costs:
"The overall cost of disease management for a chronic disease like MS is expensive and includes much more than medication," wrote Jeff Boyle. "Avonex is approved for the treatment of relapsing remitting multiple sclerosis (RRMS) and we believe the pricing is consistent with the real world value it delivers to patients with MS. Avonex remains the least expensive option for those looking to slow the progression of their MS and is the only once-a-week treatment for RRMS that is proven to reduce relapses, start working after the first attack and delay physical disability progression, helping patients stay active longer."
SOURCE: bit.ly/Q5TNl Neurology, online July 20, 2011.
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