Costs and Number Needed to Treat (NNT) to Achieve Improvements With C5 Inhibitors in Generalized Myasthenia Gravis From the Japanese Healthcare Perspective: A Network Meta-Analysis
Author(s)
Koichi Shiraishi, PhD1, Mieko Ogino, MD2, Akiyuki Uzawa, MD3, Eiko Yamamura, MA1, Kazuhiko Tawara, PhD1, Hicham Benhaddi, PhD4, Kentaro Taki, PhD1, Abhiroop Chakravarty, PhD5, Vikalp Kumar Maheshwari, MBA, B.Tech5, Yohei Ohashi, PhD1.
1UCB, Tokyo, Japan, 2International University of Health and Welfare School of Medicine Center for Medical Education, Chiba, Japan, 3Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan, 4UCB, Colombes, France, 5Parexel, Hyderabad, India.
1UCB, Tokyo, Japan, 2International University of Health and Welfare School of Medicine Center for Medical Education, Chiba, Japan, 3Department of Neurology, Graduate School of Medicine, Chiba University, Chiba, Japan, 4UCB, Colombes, France, 5Parexel, Hyderabad, India.
OBJECTIVES: Zilucoplan, ravulizumab and eculizumab are complement C5 inhibitors approved for the treatment of generalized myasthenia gravis (gMG). This research aims to estimate the number needed to treat (NNT) and cost per improved outcome (CPIO) with C5 inhibitors in gMG in Japan.
METHODS: A Bayesian network meta-analysis was conducted using results from Phase III studies with zilucoplan (RAISE; NCT04115293), ravulizumab (CHAMPION MG; NCT03920293) and eculizumab (REGAIN; NCT01997229). NNTs and CPIO were estimated for one-point improvements in change from baseline (CFB) for myasthenia gravis (MG) activities of daily living profile (MG-ADL), Quantitative MG (QMG), MG composite (MGC), and MG Quality-of-Life (MG-QoL-15r) or one additional responder based on MG-ADL (≥3-point improvement) and QMG (≥5-point improvement), where available. All assessments were at primary efficacy endpoints; 12-weeks (RAISE) and 26-weeks (REGAIN and CHAMPION MG). Median values were reported for outcomes and associated costs in million (M) ¥.
RESULTS: For MG-ADL CFB, NNT (CPIO) was 0.48 (M¥16.41), 0.59 (M¥27.78), and 0.52 (M¥33.68) for zilucoplan, ravulizumab and eculizumab, respectively. For QMG CFB, NNT (CPIO) was 0.34 (M¥11.66), 0.50 (M¥23.61), and 0.33 (M¥21.38) for zilucoplan, ravulizumab and eculizumab, respectively. For MGC CFB, NNT (CPIO) was 0.31 (M¥10.68), and 0.30 (M¥19.32) for zilucoplan and eculizumab, respectively. For MG-QoL-15r CFB, NNT (CPIO) was 0.40 (M¥13.75), and 0.59 (M¥27.83) for zilucoplan and ravulizumab, respectively. For MG-ADL responder, NNT (CPIO) was 4.08 (M¥139.60), 4.75 (M¥224.50), and 5.48 (M¥351.90) for zilucoplan, ravulizumab and eculizumab, respectively. For QMG responders, the NNT (CPIO) was 5.94 (M¥203.50), 4.59 (M¥217.10), and 4.51 (M¥289.60) for zilucoplan, ravulizumab, and eculizumab, respectively.
CONCLUSIONS: Zilucoplan showed similar or lower NNTs (0.31-0.48) and CPIO (M¥10.68-M¥16.41) compared to ravulizumab (NNT: 0.50-0.59; CPIO: M¥23.61-M¥27.83) and eculizumab (NNT: 0.30-0.52; CPIO: M¥19.32-M¥33.68) across all CFB endpoints. Furthermore, zilucoplan demonstrated superior cost-benefit, requiring lower treatment costs to achieve each improved outcome.
METHODS: A Bayesian network meta-analysis was conducted using results from Phase III studies with zilucoplan (RAISE; NCT04115293), ravulizumab (CHAMPION MG; NCT03920293) and eculizumab (REGAIN; NCT01997229). NNTs and CPIO were estimated for one-point improvements in change from baseline (CFB) for myasthenia gravis (MG) activities of daily living profile (MG-ADL), Quantitative MG (QMG), MG composite (MGC), and MG Quality-of-Life (MG-QoL-15r) or one additional responder based on MG-ADL (≥3-point improvement) and QMG (≥5-point improvement), where available. All assessments were at primary efficacy endpoints; 12-weeks (RAISE) and 26-weeks (REGAIN and CHAMPION MG). Median values were reported for outcomes and associated costs in million (M) ¥.
RESULTS: For MG-ADL CFB, NNT (CPIO) was 0.48 (M¥16.41), 0.59 (M¥27.78), and 0.52 (M¥33.68) for zilucoplan, ravulizumab and eculizumab, respectively. For QMG CFB, NNT (CPIO) was 0.34 (M¥11.66), 0.50 (M¥23.61), and 0.33 (M¥21.38) for zilucoplan, ravulizumab and eculizumab, respectively. For MGC CFB, NNT (CPIO) was 0.31 (M¥10.68), and 0.30 (M¥19.32) for zilucoplan and eculizumab, respectively. For MG-QoL-15r CFB, NNT (CPIO) was 0.40 (M¥13.75), and 0.59 (M¥27.83) for zilucoplan and ravulizumab, respectively. For MG-ADL responder, NNT (CPIO) was 4.08 (M¥139.60), 4.75 (M¥224.50), and 5.48 (M¥351.90) for zilucoplan, ravulizumab and eculizumab, respectively. For QMG responders, the NNT (CPIO) was 5.94 (M¥203.50), 4.59 (M¥217.10), and 4.51 (M¥289.60) for zilucoplan, ravulizumab, and eculizumab, respectively.
CONCLUSIONS: Zilucoplan showed similar or lower NNTs (0.31-0.48) and CPIO (M¥10.68-M¥16.41) compared to ravulizumab (NNT: 0.50-0.59; CPIO: M¥23.61-M¥27.83) and eculizumab (NNT: 0.30-0.52; CPIO: M¥19.32-M¥33.68) across all CFB endpoints. Furthermore, zilucoplan demonstrated superior cost-benefit, requiring lower treatment costs to achieve each improved outcome.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE287
Topic
Clinical Outcomes, Economic Evaluation, Real World Data & Information Systems
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Neurological Disorders, No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases