VBCN - July 2016 Volume 3, No 2 - Value in Neurology
Corbin Davis

A cost-effectiveness analysis of relapsing-remitting multiple sclerosis (MS) therapies has shown the importance of using first-line treatments first to treat the disease. In terms of achieving no evidence of disease activity (NEDA), the most effective treatment strategy was natalizumab in the first-line setting, followed by alemtuzumab as first-line treatment, according to a presentation at the 2016 American Academy of Neurology annual meeting.

Based on the researchers' model, however, alemtuzumab was the most cost-effective treatment strategy, yielding a cost-effectiveness of $79,086 per quality-adjusted life-year (QALY).

“Despite a slightly higher 3-year utility, natalizumab's costs far exceeded even the most liberal willingness-to-pay thresholds. Alemtuzumab was the most cost-effective treatment option,” said Daniel Ontaneda, MD, Mellen Center for Multiple Sclerosis, Cleveland Clinic, OH.

Although NEDA has become an attainable goal using currently approved MS therapies, the cost of disease-modifying therapies (DMTs) has risen dramatically in recent years. In addition, although the optimal sequence of therapy remains unknown, a clinical trial to answer this question would be difficult to conduct, Dr Ontaneda reported.

“The comparison of different treatment sequences in terms of both efficacy and cost is of interest to physicians who must make decisions regarding DMT selection in the clinic, and also to health agencies interested in using the optimal cost-effective medication sequence,” he said.

Study Details

The researchers grouped treatment strategies in relation to the route of administration and overall efficacy during 3 years. Yearly utilities were discounted for disease activity (NEDA rates), complications, and death. The 3-year cost of each intervention was calculated as the sum of 3 annual costs.

The researchers modeled “only the most significant and life-changing risks associated with MS disease therapies,” such as progressive multifocal leukoencephalopathy, idiopathic thrombocytopenic purpura, and Berger's disease, Dr Ontaneda noted.

The willingness-to-pay threshold was set at $50,000 per QALY, although $150,000 per QALY may be considered appropriate as well.

Cost Outcomes

The drugs with the highest NEDA values in clinical trials were the most cost-effective.

Despite being the most effective treatment strategy, natalizumab as first-line therapy had a 3-year cost of medication of $192,699. Compared with alemtuzumab, natalizumab's incremental cost-effectiveness ratio (ICER) was $2,834,955, putting it significantly above the willingness-to-pay range of $50,000 to $150,000 per QALY.

This was attributed to the relatively small difference in efficacy (0.0122), with a large cost differential of $34,699, said Dr Ontaneda, adding that the efficacy of the remaining treatment strategies was similar, with all strategies being more expensive and less effective than alemtuzumab.

“With a 3-year cost of $158,000 and an effectiveness of 1.998, the most cost-effective treatment strategy was alemtuzumab, yielding a cost-effectiveness of $79,086 per QALY. Given the extremely high ICER, natalizumab continues to be less cost-effective compared with alemtuzumab,” said Dr Ontaneda.

The results were robust, and the cost-effectiveness did not change with sensitivity analyses, although the researchers noted limitations in modeling a complex disease state.

“Data are limited by the adequacy of the model, which is unable to capture the entire complexity of DMT efficacy and risk,” Dr Ontaneda concluded.

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