Cost-utility analysis of biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) combined with methotrexate for Thai rheumatoid arthritis patients with high disease activity.

BACKGROUND: New biologic disease-modifying antirheumatic drugs (bDMARDs), targeted synthetic DMARDs (tsDMARDs) and biosimilar DMARDs (bsDMARDs) all showed greater clinical benefits in the treatment of patients with rheumatoid arthritis (RA) with high disease activity, but imposed higher costs than standard treatment. This study evaluated the cost-effectiveness of 11 alternative treatment strategies for RA patients with high disease activity whose treatment with three conventional synthetic DMARDs (csDMARDs) failed. METHODS: A Markov model was constructed using a societal perspective to estimate relevant costs and health outcomes in terms of quality-adjusted life years (QALYs) for a lifetime horizon (100 years), given a 3% annual discount. Alternative treatment strategies including five bDMARDs, two tsDMARDs, and four bsDMARDs in combination with methotrexate (MTX) were compared with the standard of care (SoC), i.e., cyclosporine and azathioprine. Direct and non-medical care costs were estimated by identifying the resources used, then multiplied by the standard costing menu in the year 2022. Utility and transitional probabilities were collected in three advanced tertiary hospitals. A network meta-analysis was used to estimate the efficacy of each treatment. Lifetime cost, QALYs and an incremental cost-effectiveness ratio were calculated and compared to the cost-effectiveness threshold of 160,000 THB per QALY gained (US $4,634, where 1 USD = 34.53 THB in 2022). Probabilistic and one-way sensitivity analyses were performed to estimate parameter uncertainties. RESULTS: The bDMARDs, tsDMARDs or bsDMARDs combined with MTX provided 0.09 to 0.33 QALYs gained with additional costs of 550,986 to 2,096,744 THB (US $15,957 to $60,722) compared to the SoC. The ICER ranged from 2.3 to 8.1 million THB per QALY (US $65,935 to $234,996) compared to the SoC. None of these combinations was cost-effective in the Thai context. The results were sensitive to the mortality hazard ratio of patients with high disease activity. CONCLUSIONS: Combinations of MTX with either bDMARDs, tsDMARDs or bsDMARDs were not economically attractive compared to the standard practice. However, they reduced disease activity and improved patient quality of life. The price negotiation process for these treatments must be conducted to ensure their financial value and affordability before they are included in the pharmaceutical reimbursement list.

Cost-utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand.

OBJECTIVES: This study aimed to analyse the cost-utility and budget impact of adding tocilizumab to the standard treatment for patients with refractory systemic juvenile idiopathic arthritis (sJIA) in Thailand. DESIGN: Economic evaluation using a decision-analytical model. SETTING: Thailand. PARTICIPANTS: Patients with refractory sJIA who were ≥2 years old. METHODS: The use of tocilizumab as an add-on therapy to standard treatment was compared with standard treatment alone. A simulated health state transition model was used to estimate the lifetime costs and health outcomes from a societal perspective. Direct medical costs were collected from tertiary hospital databases while direct non-medical costs were derived from interviews. Health-related quality of life (QoL) was measured using the proxy version of three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). Future costs and outcomes were discounted at an annual rate of 3%. The base case population was patients aged 9.41 years old at refractory disease onset. The results were reported as incremental cost-effectiveness ratios (ICER) in US dollar (USD). One-way and probabilistic sensitivity analysis were conducted to investigate parameter uncertainty. The 5-year budget impact was estimated from a governmental perspective. RESULTS: The ICER of standard treatment plus tocilizumab was US$35 799 per quality-adjusted life-year (QALY) gained compared with standard treatment alone, which was not cost-effective at the threshold of US$5128 per QALY gained. The estimated 5 years budget impact was approximately US$4.8 million. CONCLUSIONS: The use of standard treatment plus tocilizumab was not cost-effective in the Thai context, which has limited data. However, there is currently no second-line treatment for refractory sJIA in the Thai National List of Essential Medicines; thus, patients must receive higher doses of standard treatment which can cause many side effects. In contrast, tocilizumab showed obvious efficacy in clinical trials in improving treatment response and QoL. Therefore, the price of tocilizumab should be negotiated to reduce the financial impact on the healthcare system.