Canadian Agency Suggests Ruzurgi Be Reimbursed With Conditions

The Canadian Agency for Drugs and Technologies in Health (CADTH) has recommended that Ruzurgi (amifampridine) be reimbursed for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in individuals 6 and older, but only if certain conditions are met.

These include patients for whom the medication has been prescribed by a neurologist with experience caring for LEMS patients, and if Ruzurgi’s cost is reduced to a level that would be more acceptable to public payers.

It is still unclear if the medication is cost-effective, according to CADTH. Ruzurgi’s price would need to be reduced by at least 76% for it to be so.

Health Canada approved Ruzurgi to treat LEMS patients, 6 and older, last year, and the medication was made available to the Canadian public shortly thereafter. It has also been approved for the same indication in the U.S.

Ruzurgi works by binding and blocking certain proteins on nerve cells, helping them send chemical signals to muscles more effectively. Its approvals were supported by data from the Phase 2 DAPPER trial (NCT01511978), which showed the medication was able to ease muscle weakness in people with LEMS.

CADTH is an independent, not-for-profit organization that provides healthcare decision-makers with evidence to help make informed decisions about medications and other health technologies.

The organization’s assessment of Ruzurgi broadly found evidence that the treatment is safe and effective. However, concerns were raised regarding its cost-effectiveness.

Ruzurgi is commercialized in Canada by Médunik Canada, based on an agreement with the medicine’s original developer, Jacobus Pharmaceuticals. The medication is available in 10 mg tablets, and Médunik proposed a price of $27.40 per tablet.

Since Ruzurgi is dosed according to a person’s body weight, a given individual would be expected to pay from $109.59 to $273.97 per day for the medication, which corresponds to an annual cost of $40,000 to $100,000 per patient.

Médunik had submitted an economic analysis that compared Ruzurgi plus best supportive care (BSC) to BSC alone. However, CADTH’s report found several problems with Médunik’s analysis.

First, the Médunik analysis was based on Quantitative Myasthenia Gravis (QMG) scores to assess the severity of LEMS. QMG is designed to assess disease severity in people with another autoimmune condition called myasthenia gravis.

“CADTH’s clinical review found that the QMG score is not considered to be an appropriate or relevant assessment tool for LEMS,” the report states, noting that the test mainly assesses symptoms that affect the upper body, whereas people with LEMS typically have weakness in the arms and legs.

“Consequently, the sponsor’s model does not reflect the true impact of treatment with amifampridine [Ruzurgi] plus BSC on quality-adjusted survival and cost-effectiveness,” the report states.

In addition, the Médunik analysis assumed the disease would continually and substantially worsen with BSC alone, which is not typically the case for people with LEMS, as pointed out in CADTH’s report. Based on the Médunik analysis, all individuals on BSC would be in worse health after five years.

“This finding raises serious concerns about the model’s face validity,” the report states, noting that “collectively, these limitations severely limit the extent to which the sponsor’s results can be interpreted.”

Based on the Médunik model, there was 0% probability that the medication was cost-effective compared to BSC. Notably, this value was calculated based on the assumption that people would be willing to pay $50,000 for one year of good health.

The CADTH report estimated that the price of Ruzurgi would need to drop by at least 76% for the medication to be cost-effective and “more affordable for public payers.”

However, because of the problems with Médunik’s economic analysis, “the cost-effectiveness of amifampridine is unknown and the necessary price reduction is likely much greater than the sponsor’s base-case estimate,” the report concludes.