Comparative effectiveness and cost-effectiveness of natalizumab and fingolimod in rapidly evolving severe relapsing-remitting multiple sclerosis in the United Kingdom
Creators: name: Spelman, T.
name: Herring, W. L.
name: Acosta, C.
name: Hyde, R.
name: Jokubaitis, V. G.
name: Pucci, E.
name: Lugaresi, A.
name: Laureys, G.
name: Havrdova, E. K.
name: Horakova, D.
name: Izquierdo, G.
name: Eichau, S.
name: Ozakbas, S.
name: Alroughani, R.
name: Kalincik, T.
name: Duquette, P.
name: Girard, M.
name: Petersen, T.
name: Patti, F.
name: Csepany, T.
name: Granella, F.
name: Grand’Maison, F.
name: Ferraro, D.
name: Karabudak, R.
name: Jose, M.
name: Trojano, M.
name: van Pesch, V.
name: Van Wijmeersch, B.
name: Cartechini, E.
name: McCombe, P.
name: Gerlach, O.
name: Spitaleri, D.
name: Rozsa, C.
name: Hodgkinson, S.
name: Bergamaschi, R.
name: Gouider, R.
name: Soysal, A.
name: Castillo-Triviño
name: Prevost, J.
name: Garber, J.
name: de Gans, K.
name: Ampapa, R.
name: Simo, M.
name: Sanchez-Menoyo, J. L.
name: Iuliano, G.
name: Sas, A.
name: van der Walt, A.
name: John, N.
name: Gray, O.
name: Hughes, S.
name: De Luca, G.
name: Onofrj, M.
name: Buzzard, K.
name: Skibina, O.
name: Terzi, M.
name: Slee, M.
name: Solaro, C.
name: Oreja-Guevara
name: Ramo-Tello, C.
name: Fragoso, Y.
name: Shaygannejad, V.
name: Moore, F.
name: Rajda, C.
name: Morales, E. Aguera
name: Butzkueven, H.
Publisher: name: Taylor & Francis
Year: 2023
Subjects: subject: Medicine
subject: Pharmacology
subject: Immunology
subject: FOS: Clinical medicine
subject: Science Policy

description: To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS). Real-world data from the MSBase Registry were obtained for patients with RES-RRMS who were previously either naive to disease-modifying therapies or had been treated with interferon-based therapies, glatiramer acetate, dimethyl fumarate, or teriflunomide (collectively known as BRACETD). Matched cohorts were selected by 3-way multinomial propensity score matching, and the annualized relapse rate (ARR) and 6-month–confirmed disability worsening (CDW6M) and improvement (CDI6M) were compared between treatment groups. Comparative effectiveness results were used in a cost-effectiveness model comparing natalizumab and fingolimod, using an established Markov structure over a lifetime horizon with health states based on the Expanded Disability Status Scale. Additional model data sources included the UK MS Survey 2015, published literature, and publicly available sources. In the comparative effectiveness analysis, we found a significantly lower ARR for patients starting natalizumab compared with fingolimod (rate ratio [RR] = 0.65; 95% confidence interval [CI], 0.57–0.73) or BRACETD (RR = 0.46; 95% CI, 0.42–0.53). Similarly, CDI6M was higher for patients starting natalizumab compared with fingolimod (hazard ratio [HR] = 1.25; 95% CI, 1.01–1.55) and BRACETD (HR = 1.46; 95% CI, 1.16–1.85). In patients starting fingolimod, we found a lower ARR (RR = 0.72; 95% CI, 0.65–0.80) compared with starting BRACETD, but no difference in CDI6M (HR = 1.17; 95% CI, 0.91–1.50). Differences in CDW6M were not found between the treatment groups. In the base-case cost-effectiveness analysis, natalizumab dominated fingolimod (0.302 higher quality-adjusted life-years [QALYs] and £17,141 lower predicted lifetime costs). Similar cost-effectiveness results were observed across sensitivity analyses. This MSBase Registry analysis suggests that natalizumab improves clinical outcomes when compared with fingolimod, which translates to higher QALYs and lower costs in UK patients with RES-RRMS. There are several medications used to treat people with relapsing remitting multiple sclerosis, such as interferon-based therapies (Betaferon/Betaseron (US), Rebif, Avonex, Extavia), glatiramer acetate (Copaxone), teriflunomide (Aubagio), and dimethyl fumarate (Tecfidera), collectively named BRACETD. Other treatments for multiple sclerosis (MS) have a narrower use, such as natalizumab (Tysabri) or fingolimod (Gilenya), among others. This study objective was to assess how well natalizumab and fingolimod helped treating MS (clinical effectiveness) and subsequently estimate what the cost of these treatments is in comparison to the benefit they bring to people with rapidly evolving severe MS that use them in the United Kingdom (UK) (cost-effectiveness). We used an international disease registry (MSBase), which collects clinical data from people with MS in various centers around the world to compare the effectiveness of natalizumab, fingolimod and BRACETD treatments. We used a technique called propensity score matching to obtain results from comparable patient groups. People treated with natalizumab had better disease control, namely with fewer relapses and higher improvement on their disability level, than patients on fingolimod or BRACETD. Conversely, there were no differences between each group of people on a measure called disability worsening. Based on these clinical results, we built an economic model that simulates the lifetime costs and consequences of treating people with MS with natalizumab in comparison with fingolimod. We found that using natalizumab was less costly and was more effective compared to using fingolimod in UK patients.