麻豆传媒

LAS VEGAS -- Riluzole (Rilutek) should remain on the Veterans Administration formulary as a treatment for patients with amyotrophic lateral sclerosis (ALS), despite having only modest benefits and considerable cost, a researcher said here.

The published literature on riluzole suggests that the drug -- the only FDA-approved treatment for ALS -- can extend survival for up to 3 months, but provides no actual symptomatic improvement, according to Andrea Susi, PharmD, of Northeastern University in Boston.

The annual increase in cost of treatment for the more than 1,000 patients in the VA health system with ALS was estimated to be $10,648,818, but that is only 0.019% of the VA's budget, Susi reported in a poster session at the midyear clinical symposium of the American Society of Health-System Pharmacists.

"In evaluating this drug, we felt it was important to look at it from a humanistic point of view. It's the only drug out there for ALS," she told 麻豆传媒.

"This is particularly important for the VA, because higher-than-expected rates of ALS are being seen among vets who served in the Gulf wars," she added.

The pharmacologic properties of riluzole include inhibition of glutamate release, inactivation of voltage-dependent sodium channels, and blocking intracellular events at amino acid receptors.

Adverse events include weakness, nausea, and liver enzyme abnormalities.

A systematic review of the literature identified nine efficacy studies and overall survival or time until the need for tracheostomy was 2 to 3 months.

What these studies did not clarify, however, was whether the mortality benefits occur early in the course of the disease, when quality of life is still preserved, Susi noted.

But in four additional studies that assessed the economic impact of treatment, it did appear that the survival impact was early, when symptoms are mild and quality of life remains good.

For instance, while the costs were higher than with supportive care alone, increased productivity and less use of other resources were found to offset the cost, she explained.

A budget impact analysis using an estimated 1,010 patients then determined that with monthly costs of $674 for riluzole and $2,660 for supportive care, the total cost per year with a 3-month increase in survival would be $40.4 million.

For supportive care alone with no survival increase, the annual cost would be $29.7 million.

Because the estimates of the number of ALS patients in the VA system and actual life expectancy have varied, the researchers also conducted sensitivity analyses adjusting for these factors.

They found that if the prevalence reached 1,233 patients and life expectancy was 36 months, the total annual cost with riluzole would be $19.5 million, and if the prevalence was lower, with 987 patients, the annual cost fell to 9.7 million.

If life expectancy was 48 months, the additional annual cost of the drug would be 13.1 million, but if life expectancy was only 24 months, the price would be $8.1 million each year.

"We concluded that the drug should remain on the VA formulary because of patient need and the minimal impact on the overall VA budget. However, further research should explore the economic impact of initiating treatment in different stages of the disease," Susi concluded.

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