Using evidence from short-term randomized controlled trials, decision-analytic models project costs, risks and benefits of disease-modifying therapies (DMTs) for multiple sclerosis (MS). Such trial-informed models lack the breadth needed to generalize to clinical practice or policy due to limitations: lack of DMT switching/discontinuation, limited head-to-head DMT comparisons and efficacy, not effectiveness, designs. We present an illustrative example that incorporates treatment switching and discontinuation by estimating the cost-effectiveness (value) of first-line natalizumab versus second-line natalizumab treatment for relapsing-remitting MS patients negative for anti-JC virus antibodies. Treating JC virus-negative relapsing-remitting MS patients with natalizumab as first-line provided better value compared with second-line. Decision-makers should consider this evidence for treatment step-edit policies through modeling scenarios closer to clinical practice.
Keywords: clinical practice; cost–effectiveness; disease-modifying therapy; economic evaluation; multiple sclerosis.