Dyfrig A Hughes1, Giovanna Culeddu2, Catrin O Plumpton2, Eifiona Wood2, Andrew D Dick3, Ashley P Jones4, Andrew McKay4, Paula R Williamson4, Sandrine Compeyrot Lacassagne5, Ben Hardwick4, Helen Hickey4, Patricia Woo5, Michael W Beresford6, Athimalaipet V Ramanan7. 1. Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, United Kingdom. Electronic address: d.a.hughes@bangor.ac.uk. 2. Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, United Kingdom. 3. Bristol Eye Hospital, Bristol, United Kingdom; Bristol Medical School, University of Bristol, Bristol, United Kingdom. 4. Institute of Translational Medicine, University of Liverpool, Liverpool, United Kingdom. 5. UCL Institute of Ophthalmology and National Institute for Health Research (NIHR) Biomedical Research Centre at Moorfields Eye Hospital and University College London Institute of Ophthalmology, London, United Kingdom. 6. Institute of Translational Medicine, University of Liverpool, Liverpool, United Kingdom; Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom. 7. Bristol Medical School, University of Bristol, Bristol, United Kingdom; University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom.
Abstract
PURPOSE: To investigate the cost effectiveness of adalimumab in combination with methotrexate, compared with methotrexate alone, for the management of uveitis associated with juvenile idiopathic arthritis (JIA). DESIGN: A cost-utility analysis based on a clinical trial and decision analytic model. PARTICIPANTS: Children and adolescents 2 to 18 years of age with persistently active uveitis associated with JIA, despite optimized methotrexate treatment for at least 12 weeks. METHODS: The SYCAMORE (Randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis) trial (identifier, ISRCTN10065623) of methotrexate (up to 25 mg weekly) with or without fortnightly administered adalimumab (20 or 40 mg, according to body weight) provided data on resource use (based on patient self-report and electronic records) and health utilities (from the Health Utilities Index questionnaire). Surgical event rates and long-term outcomes were based on data from a 10-year longitudinal cohort. A Markov model was used to extrapolate the effects of treatment based on visual impairment. MAIN OUTCOME MEASURES: Medical costs to the National Health Service in the United Kingdom, utility of defined health states, quality-adjusted life-years (QALYs), and incremental cost per QALY. RESULTS:Adalimumab in combination with methotrexate resulted in additional costs of £39 316, with a 0.30 QALY gain compared with methotrexate alone, resulting in an incremental cost-effectiveness ratio of £129 025 per QALY gained. The probability of cost effectiveness at a threshold of £30 000 per QALY was less than 1%. Based on a threshold analysis, a price reduction of 84% would be necessary for adalimumab to be cost effective. CONCLUSIONS:Adalimumab is clinically effective in uveitis associated with JIA; however, its cost effectiveness is not demonstrated compared with methotrexate alone in the United Kingdom setting.
RCT Entities:
PURPOSE: To investigate the cost effectiveness of adalimumab in combination with methotrexate, compared with methotrexate alone, for the management of uveitis associated with juvenile idiopathic arthritis (JIA). DESIGN: A cost-utility analysis based on a clinical trial and decision analytic model. PARTICIPANTS: Children and adolescents 2 to 18 years of age with persistently active uveitis associated with JIA, despite optimized methotrexate treatment for at least 12 weeks. METHODS: The SYCAMORE (Randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis) trial (identifier, ISRCTN10065623) of methotrexate (up to 25 mg weekly) with or without fortnightly administered adalimumab (20 or 40 mg, according to body weight) provided data on resource use (based on patient self-report and electronic records) and health utilities (from the Health Utilities Index questionnaire). Surgical event rates and long-term outcomes were based on data from a 10-year longitudinal cohort. A Markov model was used to extrapolate the effects of treatment based on visual impairment. MAIN OUTCOME MEASURES: Medical costs to the National Health Service in the United Kingdom, utility of defined health states, quality-adjusted life-years (QALYs), and incremental cost per QALY. RESULTS:Adalimumab in combination with methotrexate resulted in additional costs of £39 316, with a 0.30 QALY gain compared with methotrexate alone, resulting in an incremental cost-effectiveness ratio of £129 025 per QALY gained. The probability of cost effectiveness at a threshold of £30 000 per QALY was less than 1%. Based on a threshold analysis, a price reduction of 84% would be necessary for adalimumab to be cost effective. CONCLUSIONS:Adalimumab is clinically effective in uveitis associated with JIA; however, its cost effectiveness is not demonstrated compared with methotrexate alone in the United Kingdom setting.
Authors: Fernando García-Rodríguez; Augusto Gamboa-Alonso; Sol Jiménez-Hernández; Lucero Ochoa-Alderete; Valeria Alejandra Barrientos-Martínez; Neri Alejandro Alvarez-Villalobos; Gabriela Andrea Luna-Ruíz; Ingris Peláez-Ballestas; Ana Victoria Villarreal-Treviño; Manuel Enrique de la O-Cavazos; Nadina Rubio-Pérez Journal: Pediatr Rheumatol Online J Date: 2021-10-09 Impact factor: 3.054