Incorporating real-world clinical practice in multiple sclerosis economic evaluations

Abstract

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

Acknowledgements

The authors would like to acknowledge RR Allen, Peak Statistical Service, for statistical programming.

Financial & competing interests disclosure

RB McQueen was awarded a PhRMA Foundation Post-Doctoral Fellowship that in part funded this work. TL Vollmer has received consulting fees from separate unrelated work from Consortium of MS Centers, Teva, Novartis, Genentech, Acorda and Biogen. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

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