Background The pathogenetic pathways and clinical presentation of systemic juvenile idiopathic arthritis (sJIA) differs from other forms of juvenile idiopathic arthritis (JIA). As a result the common JIA treatments are less effective and cost-effective for patients with sJIA.
Objectives To assess the cost-effectiveness of tocilizumab (TCZ) in Finnish sJIA patients with inadequate response to previous therapy with NSAIDs and systemic corticosteroids.
Methods The cost-effectiveness of TCZ was analysed in a cohort of two year old patients using a spreadsheet based probabilistic Markov state transition model with three month model cycles and 16 year duration. In the first scenario (that matches treatment of sJIA with prominent joint symptoms) TCZ was compared to methotrexate (MTX) in a treatment sequence where both treatment comparators were followed by etanercept, adalimumab and abatacept. In the second scenario (that matches treatment of sJIA with prominent systemic symptoms) MTX was replaced with anakinra as a treatment comparator. In the model the patients switch on to the next treatment line when they do not get adequate treatment response (ACR response less than 30%). TENDER trial based efficacy estimates were used for TCZ whereas the efficacy of other treatments was based on adjusted indirect comparison of clinical trials selected based on systematic literature review . The sJIA associated and response related resource use was based on Finnish expert opinion and valued with year 2010 Finnish unit costs. The analyses were performed from health care payer perspective. Results were presented as cost per quality adjusted life-year (QALY) gained using annual 3% discount rate.
Results Treatment sequences initiated with TCZ, MTX and anakinra produced 4.47, 3.41 and 2.83 QALYs, respectively. An additional QALY gained on TCZ cost 15 181 euros when compared with MTX and 14 496 euros when compared with anakinra. Based on cost-effectiveness acceptability frontiers, TCZ had 93% and 88% probability for being cost-effective at the willingness to pay (WTP) level of 20 000 euros/QALY gained when compared with MTX and anakinra, respectively. At the WTP threshold of 27 000 euros/QALY gained in the TCZ vs. MTX comparison and 37 000 euros/QALY gained in the TCZ vs. anakinra comparison, the probability of TCZ being cost-effective reached 100%. The performed sensitivity analyses suggest that the results are most sensitive to changes in the assumed health care resource use. Due to conservative modelling assumptions (e.g. 16 year timeframe, stable TCZ efficacy in responders over time and low drug costs after loss of treatment response) the obtained results underestimate TCZ’s true cost-effectiveness.
Conclusions TCZ is an effective and potentially cost-effective sJIA treatment in Finland.
Diamantopoulos A, LeReun C, Westhovens R, Dejonckheere F. Indirect comparison of ACR response of biologic treatments in active sJIA. Submitted to EULAR 2012 (EULAR12-2334).
Disclosure of Interest T. Hallinen Shareholder of: ESiOR Oy, Consultant for: Several pharmaceutical (including Roche Oy), food industry, diagnostics and device companies, hospitals and academic institutions, Employee of: ESiOR Oy, E. Soini Shareholder of: ESiOR Oy, Consultant for: Several pharmaceutical (including Roche Oy), food industry, diagnostics and device companies, hospitals and academic institutions, Employee of: ESiOR Oy, A. Diamantopoulos Consultant for: F. Hoffman-La Roche Ltd., F. Dejonckheere Employee of: F. Hoffman-La Roche Ltd., V. Vihervaara Employee of: Roche Oy, A. Hautala Employee of: Roche Oy, K. Aalto Consultant for: Pfizer, Roche Oy, Speakers Bureau: Abbot, Roche, Sobi, and Pfizer
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