The cost-effectiveness of oral contraceptives compared to ‘no hormonal treatment’ for endometriosis-related pain: An economic evaluation

<div><p>Objective</p><p>To develop a preliminary cost-effectiveness model that compares oral contraceptives and ‘no hormonal treatment’ for the treatment of endometriosis-related pain.</p><p>Methods</p><p>A <i>de novo</i> preliminary state transition (Markov) model was developed. The model was informed by systematic literature review and expert opinion. The uncertainty around the results was assessed both by deterministic and probabilistic sensitivity analyses. The economic evaluation was conducted from National Health Service (NHS) England perspective. The main outcome measure was incremental cost per quality-adjusted life year (QALY), with cost-effectiveness plane and cost-effectiveness acceptability curves presented for alternative willingness-to-pay thresholds.</p><p>Results</p><p>Oral contraceptives dominated ‘no hormonal treatment’ and provided more QALYs at a lower cost than ‘no hormonal treatment’, with a cost-effectiveness probability of 98%. A one-way sensitivity analysis excluding general practitioner consultations showed that oral contraceptives were still cost-effective.</p><p>Conclusions</p><p>The analyses showed that oral contraceptives could be an effective option for the treatment of endometriosis, as this treatment was shown to provide a higher level of QALYs at a lower cost, compared to ‘no hormonal treatment’. The results are subject to considerable parameter uncertainty as a range of assumptions were required as part of the modelling process.</p></div

Cost-effectiveness of structured education in children with type-1 diabetes mellitus

Objectives: Kids in Control OF Food (KICk-OFF) is a 5-day structured education program for 11- to 16-year-olds with type 1 diabetes mellitus (T1DM) who are using multiple daily insulin injections. This study evaluates the cost-effectiveness of the KICk-OFF education program compared with the usual care using data from the KICk-OFF trial. Methods: The short-term within-trial analysis covers the 2-year postintervention period. Data on glycated hemoglobin (HbA1c), severe hypoglycemia, and diabetic ketoacidosis (DKA) were collected over a 2-year follow-up period. Sub-group analyses have been defined on the basis of baseline HbA1c being below 7.5 percent (58.5 mmol/mol) (low group), between 7.5 percent and 9.5 percent (80.3 mmol/mol) (medium group), and over 9.5 percent (high group). The long-term cost-effectiveness evaluation has been conducted by using The Sheffield Type 1 Diabetes Policy Model, which is a patient-level simulation model on T1DM. It includes long-term microvascular (retinopathy, neuropathy, and nephropathy) and macrovascular (myocardial infarction, stroke, revascularization, and angina) diabetes-related complications and acute adverse events (severe hypoglycemia and DKA). Results: The most favorable within-trial scenario for the KICk-OFF arm led to an incremental cost-effectiveness ratio (ICER) of £23,688 (base year 2009) with a cost-effectiveness probability of 41.3 percent. Simulating the long-term complications using the full cohort data, the mean ICER for the base case was £28,813 (base year 2011) and the probability of the KICk-OFF intervention being cost-effective at £20,000/QALY threshold was 42.6 percent, with considerable variation due to treatment effect duration. For the high HbA1c sub-group, the KICk-OFF arm was “dominant” (meaning it provided better health gains at lower costs than usual care) over the usual care arm in each scenario considered. Conclusions: For the whole study population, the cost-effectiveness of KICk-OFF depends on the assumption for treatment effect duration. For the high baseline HbA1c sub-group, KICk-OFF arm was estimated to be dominant over the usual care arm regardless of the assumption on the treatment effect duration

Prevalence, diagnostic delay and economic burden of endometriosis and its impact on quality of life: results from an Eastern Mediterranean population

BACKGROUND: There are limited data on endometriosis from the Eastern Mediterranean region. This study for the first time estimates the prevalence and impact of endometriosis on women in Northern Cyprus, an under-represented region in Europe. METHODS: Cyprus Women's Health Research Initiative, a cross-sectional study recruited 7646 women aged 18-55 in Northern Cyprus between January 2018 and February 2020. Cases were identified using self-reported and ultrasound data and two control groups were defined, with (n = 2922) and without (n = 4314) pain. Standardized tools, including the 11-point Numerical Rating Scale and the Short Form 36 Health Survey version 2, were used to assess pain and quality of life, respectively. RESULTS: Prevalence and median diagnostic delay of endometriosis were 5.4% [95% confidence interval (CI): 4.9-5.9%, n = 410] and 7 (interquartile range 15.5) years. Endometriosis cases experienced a higher prevalence of bladder pain compared with asymptomatic pain controls (6.3% vs. 1.0%, P < 0.001) and irritable bowel syndrome relating to pelvic pain compared with symptomatic (4.6% vs. 2.6%, P = 0.027) and asymptomatic (0.3%, P < 0.001) controls. The odds of endometriosis cases reporting an anxiety diagnosis was 1.56 (95% CI: 1.03-2.38) higher than the symptomatic and 1.95 (95% CI: 1.30-2.92) times higher than the asymptomatic controls. The physical component score of the health-related quality-of-life instrument suggested a significant difference between the endometriosis cases and the symptomatic controls (46.8 vs. 48.5, P = 0.034). Average annual economic cost of endometriosis cases was Int$9864 (95$8811-$10 917) including healthcare, costs relating to absence and loss of productivity at work. CONCLUSION: Prevalence was lower than the global 10% estimate, and substantial proportion of women without endometriosis reported moderate/severe pelvic pain hinting at many undiagnosed cases within this population. Coupled with lower quality of life, significant economic burden and underutilized pain management options, the study highlights multiple opportunities to improve care for endometriosis patients and women with pelvic pain

The impact of diabetes-related complications on preference-based measures of health-related quality of life in adults with Type I diabetes

Introduction: This study estimates health-related quality of life (HRQoL) or utility decrements associated with type 1 diabetes mellitus (T1DM) using data from a UK research programme on the Dose Adjustment For Normal Eating (DAFNE) education programme. Methods: A wide range of data was collected from 2,341 individuals who undertook a DAFNE course in 2009-12, at baseline and for two subsequent years. We use fixed and random effects linear models to generate utility estimates for T1DM using different instruments: EQ-5D, SF-6D and EQ-VAS. We show models with and without controls for HbA1c and depression, which may be endogenous (if, for example, there is reverse causality in operation). Results: We find strong evidence of an unobserved individual effect, suggesting the superiority of the fixed effects model. Depression shows the greatest decrement across all the models in the preferred fixed effects model. The fixed effects EQ-5D model also finds a significant decrement from retinopathy, BMI and HbA1c(%). Estimating a decrement using the fixed effects model is not possible for some conditions where there are few new cases. In the random effects model diabetic foot disease shows substantial utility decrements, yet these are not significant in the fixed effects models. Conclusion: Utility decrements have been calculated for a wide variety of health states in T1DM which can be used in economic analyses. However, despite the large dataset, the low incidence of several complications leads to uncertainty in calculating the utility weights. Depression and diabetic foot disease result in a substantial loss in HRQoL for patients with T1DM. HbA1c(%) appears to have an independent negative impact upon HRQoL, although concerns remain regarding the potential endogeneity of this variable

Cost-effectiveness of insulin pumps compared with multiple daily injections, both provided with structured education, for adults with type 1 diabetes:a health economic analysis of the Relative Effectiveness of Pumps over Structured Education (REPOSE) randomised controlled trial

Objectives To assess the long-term cost-effectiveness of insulin pumps and Dose Adjustment for Normal Eating (pumps+DAFNE) compared with multiple daily insulin injections and DAFNE (MDI+DAFNE) for adults with type 1 diabetes mellitus (T1DM) in the UK. Methods We undertook a cost–utility analysis using the Sheffield Type 1 Diabetes Policy Model and data from the Relative Effectiveness of Pumps over Structured Education (REPOSE) trial to estimate the lifetime incidence of diabetic complications, intervention-based resource use and associated effects on costs and quality-adjusted life years (QALYs). All economic analyses took a National Health Service and personal social services perspective and discounted costs and QALYs at 3.5% per annum. A probabilistic sensitivity analysis was performed on the base case. Further uncertainties in the cost of pumps and the evidence used to inform the model were explored using scenario analyses. Setting Eight diabetes centres in England and Scotland. Participants Adults with T1DM who were eligible to receive a structured education course and did not have a strong clinical indication or a preference for a pump. Intervention Pumps+DAFNE. Comparator MDI+DAFNE. Main outcome measures Incremental costs, incremental QALYs gained and incremental cost-effectiveness ratios (ICERs). Results Compared with MDI+DAFNE, pumps+DAFNE was associated with an incremental discounted lifetime cost of +£18 853 (95% CI £6175 to £31 645) and a gain in discounted lifetime QALYs of +0.13 (95% CI -0.70 to +0.96). The base case mean ICER was £142 195 per QALY gained. The probability of pump+DAFNE being cost-effective using a cost-effectiveness threshold of £20 000 per QALY gained was 14.0%. All scenario and subgroup analyses examined indicated that the ICER was unlikely to fall below £30 000 per QALY gained. Conclusions Our analysis of the REPOSE data suggests that routine use of pumps in adults without an immediate clinical need for a pump, as identified by National Institute for Health and Care Excellence, would not be cost-effective. Trial registration number ISRCTN61215213

Estimating a preference-based single index measuring the quality of life impact of self-management for diabetes

Objective. Self-management is becoming increasingly important in diabetes but is neglected in conventional preference-based measures. The objective of this paper was to generate health state utility values for a novel classification system measuring the quality-of-life impact of self-management for diabetes, which can be used to generate quality-adjusted life years (QALYs). Methods. A large online survey was conducted using a discrete choice experiment (DCE), with duration as an additional attribute, on members of the UK general population (n = 1,493) to elicit values for health (social limitations, mood, vitality, hypoglycaemia) and non-health (stress, hassle, control, support) aspects of self-management in diabetes. The data were modelled using a conditional fixed-effects logit model and utility estimates were anchored on the one to zero (full health to dead) scale. Results. The model produced significant and consistent coefficients, with one logical inconsistency and 3 insignificant coefficients for the milder levels of some attributes. The anchored utilities ranged from 1 for the best state to −0.029 for the worst state (meaning worse than dead) defined by the classification system. Conclusion. The results presented here can potentially be used to generate utility values capturing the day to day impact of interventions in diabetes on both health and self-management. These utility values can potentially be used to generate QALYs for economic models of the cost-effectiveness of interventions in diabetes

Using a discrete choice experiment involving cost to value a classification system measuring the quality of life impact of self-management for diabetes

Objective: This paper describes the use of a novel approach in health valuation of a discrete choice experiment (DCE) including a cost attribute to value a recently developed classification system for measuring the quality of life impact (both health and treatment experience) of self-management for diabetes. Methods: A large online survey was conducted using DCE with cost on UK respondents from the general population (n=1,497) and individuals with diabetes (n=405). The data was modelled using a conditional logit model with robust standard errors. The marginal rate of substitution (MRS) was used to generate willingness to pay estimates for every state defined by the classification system. Robustness of results was assessed by including interaction effects for household income. Results: There were some logical inconsistencies and insignificant coefficients for the milder levels of some attributes. There were some differences in the rank ordering of different attributes for the general population and diabetes patients. The willingness to pay to avoid the most severe state was £1,118.53 per month for the general population and £2,356.02 per month for the diabetes patient population. The results were largely robust. Conclusion: Health and self-management can be valued in a single classification system using DCE with cost. The MRS for key attributes can be used to inform cost-benefit analysis of self-management interventions in diabetes using results from clinical studies where this new classification system has been applied. The method shows promise, but found large willingness to pay estimates exceeding the cost levels used in the survey