Cost-Effectiveness of IncobotulinumtoxinA in the Treatment of Sialorrhea in Patients with Various Neurological Conditions

Abstract Introduction Sialorrhea is a common and debilitating symptom associated with neurological conditions, which can result in considerable physical and psychosocial complications. In Australia, management options are limited and further impeded by the lack of approved treatments. Whilst there is emerging evidence for the efficacy and tolerability of botulinum toxin (BoNT) for the treatment of sialorrhea in patients with neurological conditions, the cost-effectiveness of the treatment is yet to be established. Objectives To evaluate the cost-effectiveness of incobotulinumtoxinA for the treatment of chronic troublesome sialorrhea caused by various neurological conditions from the Australian healthcare perspective. Methods A Markov state transition model was developed to perform a cost-utility analysis comparing incobotulinumtoxinA with standard of care (SoC). The model consisted of a hypothetical cohort of patients transiting between three severity-based health states, defined according to the Drooling Severity and Frequency Scale (DSFS), in 16-weekly cycles over 5 years. All clinical and utility inputs were sourced from a single placebo-controlled randomised clinical trial. Only direct healthcare costs were considered, and potential indirect costs such as carer’s time and lost productivity were ignored. The primary outcome measure was the incremental cost per quality-adjusted life-year (QALY). Univariate and probabilistic sensitivity analyses were conducted. Results The model demonstrated that proportionally more patients spent time in less severe sialorrhea health states in the incobotulinumtoxinA arm. For example, over the 5-year period, patients receiving incobotulinumtoxinA were estimated to spend 1.6 years with minimal or no sialorrhea, while no patients achieved this level of improvement under SoC. IncobotulinumtoxinA was shown to have an incremental cost per QALY gained of A$23,445 when compared with SoC. Conclusions The quality of life (QoL) of patients with sialorrhea caused by neurological conditions was considerably compromised. IncobotulinumtoxinA was shown to successfully alleviate sialorrhea and it was demonstrated to be a cost-effective intervention when compared with SoC alone

Cost-consequence analysis of daily continuous remote monitoring of implantable cardiac defibrillator and resynchronization devices in the UK.

The need for ongoing and lifelong follow-up (FU) of patients with cardiac implantable electric devices (CIED) requires significant resources. Remote CIED management has been established as a safe alternative to conventional periodical in-office FU (CFU). An economic model compares the long-term cost and consequences of using daily Home Monitoring® (HM) instead of CFU

The Authors' Reply

Cost-effectiveness, Cost-utility, Pioglitazone, Rosiglitazone, Type-2-diabetes-mellitus

A Lifetime Modelled Economic Evaluation Comparing Pioglitazone and Rosiglitazone for the Treatment of Type 2 Diabetes Mellitus in the UK

Introduction: Adding pioglitazone or rosiglitazone to existing therapy are alternative treatment options for patients with type 2 diabetes mellitus who have insufficient glycaemic control while receiving the maximal tolerated dose of metformin monotherapy. Our objective was to develop a lifetime model of type 2 diabetes mellitus and its sequelae in order to compare the costs and benefits of pioglitazone versus rosiglitazone in combination with metformin. Methods: A decision-analytic model employing a first order Monte Carlo simulation of a Markov process was constructed. The model incorporated surrogate outcome measures from a large randomised controlled trial (RCT) [n_=_802] that compared the glycaemic and lipid control of pioglitazone and rosiglitazone monotherapy. These efficacy data were used with a recently validated and peer-reviewed UKPDS (UK Prospective Diabetes Study) algorithm to simulate the progression of these surrogate outcomes to final health outcomes, including quality of life (QOL) and mortality, and to calculate the risks of diabetic complications and death. The model perspective was of the UK NHS and included direct healthcare costs only (Lstg , 2004/5 values). Patient outcomes measured in the model included life-expectancy (LE) and QALYs. The base-case analysis was run for 56-year-old male Caucasions with a haemoglobin A1c (HbA1c) of 7.57% and a body mass index of 33.14 kg/m2. Results: Patients treated with pioglitazone experienced a reduction in the total cholesterol to high-density lipoprotein-cholesterol (TC___HDL-C) ratio of 0.34, whereas the TC___HDL-C ratio increased by 0.65 in those receiving rosiglitazone (p_Cost-effectiveness, Cost-utility, Pioglitazone, Rosiglitazone, Type-2-diabetes-mellitus

Indirect comparison of bronchial thermoplasty versus omalizumab for uncontrolled severe asthma

<p><i>Objective:</i> Bronchial thermoplasty (BT) as an add-on therapy for uncontrolled severe asthma is an alternative to biologic therapies like omalizumab (OM). We conducted an indirect treatment comparison (ITC) to appraise comparative effectiveness of BT and OM. <i>Methods</i>: A systematic literature review identified relevant randomized controlled trials. The ITC followed accepted methodology. <i>Results</i>: The ITC comprised a sham-controlled trial of BT (AIR2) and two placebo-controlled trials of OM (INNOVATE; EXTRA). Comparing the BT post-treatment period to ongoing treatment with OM, showed no significant differences in the rate ratios (RRs) for severe exacerbations (RR of BT versus OM = 0.91 [95% CI: 0.64, 1.30]; <i>p</i> = 0.62) or hospitalizations (RR = 0.57 [95% CI: 0.17, 1.86]; <i>p</i> = 0.53); emergency department visits were significantly reduced by 75% with BT (RR = 0.25 [95% CI: 0.07, 0.91]; <i>p</i> = 0.04); the proportions of patients with clinically meaningful response on the asthma quality-of-life questionnaire were comparable (RR = 1.06 [95% CI: 0.86, 1.34]; <i>p</i> = 0.59). The RR for exacerbations statistically favours OM over the total study period in AIR2 (RR = 1.50 [95% CI: 1.11, 2.02]; <i>p</i> = 0.009) likely reflecting a transient increase in events during the BT peri-treatment period. <i>Conclusions</i>: The ITC should be interpreted cautiously considering the differences between patient populations in the included trials. However, based on the analysis, BT compares well with a potentially more costly pharmacotherapy for asthma. Clinicians evaluating the relative merits of using these treatments should consider the totality of evidence and patient preferences to make an informed decision.</p

Indirect comparison of bronchial thermoplasty versus omalizumab for uncontrolled severe asthma

Objective: Bronchial thermoplasty (BT) as an add-on therapy for uncontrolled severe asthma is an alternative to biologic therapies like omalizumab (OM). We conducted an indirect treatment comparison (ITC) to appraise comparative effectiveness of BT and OM. Methods: A systematic literature review identified relevant randomized controlled trials. The ITC followed accepted methodology. Results: The ITC comprised a sham-controlled trial of BT (AIR2) and two placebo-controlled trials of OM (INNOVATE; EXTRA). Comparing the BT post-treatment period to ongoing treatment with OM, showed no significant differences in the rate ratios (RRs) for severe exacerbations (RR of BT versus OM = 0.91 [95% CI: 0.64, 1.30]; p = 0.62) or hospitalizations (RR = 0.57 [95% CI: 0.17, 1.86]; p = 0.53); emergency department visits were significantly reduced by 75% with BT (RR = 0.25 [95% CI: 0.07, 0.91]; p = 0.04); the proportions of patients with clinically meaningful response on the asthma quality-of-life questionnaire were comparable (RR = 1.06 [95% CI: 0.86, 1.34]; p = 0.59). The RR for exacerbations statistically favours OM over the total study period in AIR2 (RR = 1.50 [95% CI: 1.11, 2.02]; p = 0.009) likely reflecting a transient increase in events during the BT peri-treatment period. Conclusions: The ITC should be interpreted cautiously considering the differences between patient populations in the included trials. However, based on the analysis, BT compares well with a potentially more costly pharmacotherapy for asthma. Clinicians evaluating the relative merits of using these treatments should consider the totality of evidence and patient preferences to make an informed decision.</p