Olena Mandrik1, James Fotheringham2, Shijie Ren1, Jeffrey A Tice3, Richard H Chapman4, Matthew D Stevenson1, Steven D Pearson4, Serina Herron-Smith4, Foluso Agboola4, Praveen Thokala1. 1. Health Economic and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, United Kingdom. 2. Health Economic and Decision Science, School of Health and Related Research, The University of Sheffield, Sheffield, United Kingdom j.fotheringham@sheffield.ac.uk. 3. Department of Medicine, University of California, San Francisco, California. 4. Institute for Clinical and Economic Review, Boston, Massachusetts.
Abstract
BACKGROUND AND OBJECTIVES: Despite existing therapies, people with lupus nephritis progress to kidney failure and have reduced life expectancy. Belimumab and voclosporin are two new disease-modifying therapies recently approved for the treatment of lupus nephritis. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: A de novo economic model was developed to estimate the cost-effectiveness of these therapies, including the following health states: "complete response," "partial response," and "active disease" defined by eGFR and proteinuria changes, kidney failure, and death. Short-term data and mean cohort characteristics were sourced from pivotal clinical trials of belimumab (the Belimumab International Study in Lupus Nephritis) and voclosporin (the Aurinia Urinary Protection Reduction Active-Lupus with Voclosporin trial and Aurinia Renal Response in Active Lupus With Voclosporin). Risk of mortality and kidney failure were on the basis of survival modeling using published Kaplan-Meier data. Each drug was compared with the standard of care as represented by the comparator arm in its respective pivotal trial(s) using US health care sector perspective, with a societal perspective also explored. RESULTS: In the health care perspective probabilistic analysis, the incremental cost-effectiveness ratio for belimumab compared with its control arm was estimated to be approximately $95,000 per quality-adjusted life year. The corresponding incremental ratio for voclosporin compared with its control arm was approximately $150,000 per quality-adjusted life year. Compared with their respective standard care arms, the probabilities of belimumab and voclosporin being cost effective at a threshold of $150,000 per quality-adjusted life year were 69% and 49%, respectively. Cost-effectiveness was dependent on assumptions made regarding survival in response states, costs and utilities in active disease, and the utilities in response states. In the analysis from a societal perspective, the incremental ratio for belimumab was estimated to be approximately $66,000 per quality-adjusted life year, and the incremental ratio for voclosporin was estimated to be approximately $133,000 per quality-adjusted life year. CONCLUSIONS: Compared with their respective standard care arms, belimumab but not voclosporin met willingness-to-pay thresholds of $100,000 per quality-adjusted life year. Despite potential clinical superiority in the informing trials, there remains high uncertainty around the cost-effectiveness of voclosporin.
BACKGROUND AND OBJECTIVES: Despite existing therapies, people with lupus nephritis progress to kidney failure and have reduced life expectancy. Belimumab and voclosporin are two new disease-modifying therapies recently approved for the treatment of lupus nephritis. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: A de novo economic model was developed to estimate the cost-effectiveness of these therapies, including the following health states: "complete response," "partial response," and "active disease" defined by eGFR and proteinuria changes, kidney failure, and death. Short-term data and mean cohort characteristics were sourced from pivotal clinical trials of belimumab (the Belimumab International Study in Lupus Nephritis) and voclosporin (the Aurinia Urinary Protection Reduction Active-Lupus with Voclosporin trial and Aurinia Renal Response in Active Lupus With Voclosporin). Risk of mortality and kidney failure were on the basis of survival modeling using published Kaplan-Meier data. Each drug was compared with the standard of care as represented by the comparator arm in its respective pivotal trial(s) using US health care sector perspective, with a societal perspective also explored. RESULTS: In the health care perspective probabilistic analysis, the incremental cost-effectiveness ratio for belimumab compared with its control arm was estimated to be approximately $95,000 per quality-adjusted life year. The corresponding incremental ratio for voclosporin compared with its control arm was approximately $150,000 per quality-adjusted life year. Compared with their respective standard care arms, the probabilities of belimumab and voclosporin being cost effective at a threshold of $150,000 per quality-adjusted life year were 69% and 49%, respectively. Cost-effectiveness was dependent on assumptions made regarding survival in response states, costs and utilities in active disease, and the utilities in response states. In the analysis from a societal perspective, the incremental ratio for belimumab was estimated to be approximately $66,000 per quality-adjusted life year, and the incremental ratio for voclosporin was estimated to be approximately $133,000 per quality-adjusted life year. CONCLUSIONS: Compared with their respective standard care arms, belimumab but not voclosporin met willingness-to-pay thresholds of $100,000 per quality-adjusted life year. Despite potential clinical superiority in the informing trials, there remains high uncertainty around the cost-effectiveness of voclosporin.
Authors: Bevra H Hahn; Maureen A McMahon; Alan Wilkinson; W Dean Wallace; David I Daikh; John D Fitzgerald; George A Karpouzas; Joan T Merrill; Daniel J Wallace; Jinoos Yazdany; Rosalind Ramsey-Goldman; Karandeep Singh; Mazdak Khalighi; Soo-In Choi; Maneesh Gogia; Suzanne Kafaja; Mohammad Kamgar; Christine Lau; William J Martin; Sefali Parikh; Justin Peng; Anjay Rastogi; Weiling Chen; Jennifer M Grossman Journal: Arthritis Care Res (Hoboken) Date: 2012-06 Impact factor: 4.794
Authors: Barry I Freedman; Carl D Langefeld; Kelly K Andringa; Jennifer A Croker; Adrienne H Williams; Neva E Garner; Daniel J Birmingham; Lee A Hebert; Pamela J Hicks; Mark S Segal; Jeffrey C Edberg; Elizabeth E Brown; Graciela S Alarcón; Karen H Costenbader; Mary E Comeau; Lindsey A Criswell; John B Harley; Judith A James; Diane L Kamen; S Sam Lim; Joan T Merrill; Kathy L Sivils; Timothy B Niewold; Neha M Patel; Michelle Petri; Rosalind Ramsey-Goldman; John D Reveille; Jane E Salmon; Betty P Tsao; Keisha L Gibson; Joyce R Byers; Anna K Vinnikova; Janice P Lea; Bruce A Julian; Robert P Kimberly Journal: Arthritis Rheumatol Date: 2014-02 Impact factor: 10.995
Authors: Megan R W Barber; John G Hanly; Li Su; Murray B Urowitz; Yvan St Pierre; Juanita Romero-Diaz; Caroline Gordon; Sang-Cheol Bae; Sasha Bernatsky; Daniel J Wallace; Joan T Merrill; David A Isenberg; Anisur Rahman; Ellen M Ginzler; Michelle Petri; Ian N Bruce; Mary A Dooley; Paul R Fortin; Dafna D Gladman; Jorge Sanchez-Guerrero; Kristjan Steinsson; Rosalind Ramsey-Goldman; Munther A Khamashta; Cynthia Aranow; Meggan Mackay; Graciela S Alarcón; Susan Manzi; Ola Nived; Andreas Jönsen; Asad A Zoma; Ronald F van Vollenhoven; Manuel Ramos-Casals; Guillermo Ruiz-Irastorza; S Sam Lim; Kenneth C Kalunian; Murat Inanc; Diane L Kamen; Christine A Peschken; Søren Jacobsen; Anca Askanase; Vernon Farewell; Thomas Stoll; Jill Buyon; Ann E Clarke Journal: Arthritis Care Res (Hoboken) Date: 2020-12 Impact factor: 4.794
Authors: Julie E Davidson; Qinggong Fu; Beulah Ji; Sapna Rao; David Roth; Laurence S Magder; Michelle Petri Journal: J Rheumatol Date: 2018-03-01 Impact factor: 4.666
Authors: Jacie T Cooper; Andrew Lloyd; Juan Jose Garcia Sanchez; Elisabeth Sörstadius; Andrew Briggs; Phil McFarlane Journal: Health Qual Life Outcomes Date: 2020-09-21 Impact factor: 3.186