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

ABSTRACT: 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.

SUBMITTER: Kittiratchakool N

PROVIDER: S-EPMC7493110 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature

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

Kittiratchakool Nitichen N Kulpokin Disorn D Chanjam Chonticha C Vilaiyuk Soamarat S Charuvanij Sirirat S Phongsamart Gun G Khaosut Parichat P Tanya Manasita M Nanagara Ratanavadee R Nantapaisarn Sira S Leelahavarong Pattara P

BMJ open 20200915 9

<h4>Objectives</h4>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.<h4>Design</h4>Economic evaluation using a decision-analytical model.<h4>Setting</h4>Thailand.<h4>Participants</h4>Patients with refractory sJIA who were ≥2 years old.<h4>Methods</h4>The use of tocilizumab as an add-on therapy to standard treatment was compared with standard treatme ...[more]

PMID: 32933962

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Project description:Background: Systemic-Onset Juvenile Idiopathic Arthritis (SJIA) is a rare disease associated with dysregulated interleukin (IL)-1 activity. Objectives: To assess the efficacy and safety of Anakinra, an IL-1 receptor antagonist in SJIA and its effects on gene expression profiling. Methods: We conducted a multicenter, randomized, double-blind placebo-controlled trial. The primary objective was to compare the efficacy of a one-month treatment with anakinra (2 mg/kg subcutaneoulsy daily, maximum 100 mg) to a placebo between 2 groups of 12 SJIA patients each. Response was defined by a 30% improvement of the pediatric American College of Rheumatology criteria for JIA, resolution of systemic symptoms and a decrease of at least 50% of both C-reactive protein and erythrocyte sedimentation rate compared to baseline. An intention-to-treat analyze was performed. After Month 1 (M1), patients taking placebo were switched to Anakinra. Secondary objectives included tolerance and efficacy assessment for 12 months. Results: At M1, concluding the randomized trial, there was a significant difference in the response rate between patients treated with Anakinra (8/12 responders) and placebo (1/12) (p=0.003). The number of adverse events, mainly pain to injections, was similar between both groups. Ten patients from the placebo group switched to Anakinra at M1; nine were responders at M2. Between M1 and M12, six patients stopped treatment for an adverse event (Crohn’s disease, hepatitis), a lack of efficacy or a disease flare (2 cases each). Blood gene expression profiling at enrollment and upon follow-up allowed us to identify one set of dysregulated genes that reverted to normal values in the clinical responders and a second set, including interferon-inducible genes, that was induced by Anakinra. Conclusion: Anakinra is an effective treatment of SJIA. Its effect is associated with normalization of blood gene expression profiles in clinical responders and de novo induction of an interferon signature. (Clinical trials registration number: NCT00339157)

Project description:BackgroundUveitis associated with juvenile idiopathic arthritis is a cause of major ocular morbidity. A substantial proportion of children are refractory to systemic methotrexate and TNF inhibitors. Our aim was to study the safety and efficacy of tocilizumab in children with juvenile idiopathic arthritis-associated uveitis refractory to both methotrexate and TNF inhibitors.MethodsThis multicentre, single-arm, phase 2 trial was done following a Simon's two-stage design at seven tertiary hospital sites in the UK. Patients aged 2-18 years with active juvenile idiopathic arthritis-associated uveitis were eligible. All patients had been on a stable dose of methotrexate for at least 12 weeks and had not responded to treatment with a TNF inhibitor. Patients weighing 30 kg or more were treated with 162 mg subcutaneous tocilizumab every 2 weeks for 24 weeks, and participants weighing less than 30 kg were treated with 162 mg every 3 weeks for 24 weeks. The primary outcome was treatment response defined as a two-step decrease, or decrease to zero, from baseline in the level of inflammation (anterior chamber cells) at week 12, per the standardisation of uveitis nomenclature criteria. A phase 3 trial would be justified if more than seven patients responded to treatment. An interim analysis was planned to assess whether the trial would be stopped for futility, with futility defined as two or fewer treatment responses among ten participants. Adverse events were collected up to 30 calendar days after treatment cessation. The primary analysis was done in the intention-to-treat population and the safety analysis was done in all patients who started the treatment. This trial is registered with the International Standard Randomised Controlled Trial Number registry (ISRCTN95363507) and EU Clinical Trials Register (EudraCT 2015-001323-23).Findings22 participants were enrolled to the trial between Dec 3, 2015, and March 9, 2018, and 21 participants received treatment. One participant was found to be ineligible immediately after enrolment and was therefore withdrawn. Seven of 21 (median unbiased estimate of proportion 34% [95% CI 25-57]) responded to treatment (p=0·11). Safety results were consistent with the known safety profile of tocilizumab.InterpretationThe primary endpoint was not met, and thus the results do not support a phase 3 trial of tocilizumab in patients with juvenile idiopathic arthritis-associated uveitis. Importantly, data on the use of tocilizumab in clinical practice is now captured in national registries. Despite this trial not meeting the threshold required to justify a larger phase 3 trial, several patients responded to treatment; as such, tocilzumab might still be a therapeutic option in some children with uveitis refractory to anti-TNF drugs, given the absence of other treatment options.FundingVersus Arthritis and the National Institute for Health Research Clinical Research Network: Children.

Dataset Information

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

Publications

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

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