QALYs and ambulatory status: societal preferences for healthcare decision making.

BACKGROUND: This research aimed to review the theoretical and methodological aspects of the quality-adjusted life year (QALY) which give rise to potential for bias against certain patient populations, including those with problems with walking or an inability to walk (ambulatory disabilities), when health technology assessment decisions rely on QALY gain to show cost-effectiveness. Societal preferences for treating ambulatory versus non-ambulatory patients were also investigated. METHODS: We reviewed published literature to identify information on theoretical underpinnings of the QALY, measurement of utilities for QALY assessment, and empirical evidence of societal preferences for the treatment of ambulatory and non-ambulatory patients. RESULTS AND DISCUSSION: Health states which represent mobility impairment and the inability to walk receive low valuation from general public preferences. Non-ambulatory patients, for example those with advanced neuromuscular disease, have lower utilities determined by standardized preference-based measurement (PBM) tools. Any treatment that increases survival but could not restore ambulation would result in lower lifetime QALY gains for non-ambulatory versus ambulatory patients. Treatments could therefore potentially be deemed less cost-effective, or not cost-effective at all for this patient population.Empirical research indicates a societal preference for equal treatment of patients regardless of ambulatory status. The main limitation of our review was the non-systematic approach to evidence search and review, however, given the broad scope of content required to meet the aims of the review, we believe that the targeted approach was appropriate. The evidence presented in this article highlights the need for alternatives to strict QALY-based approaches to prevent avoidable health inequities when determining cost-effectiveness of healthcare interventions for non-ambulatory populations against fixed cost-effectiveness thresholds. An alternative metric, the Equal Value of Life Years Gained (evLYG), has been proposed as a supplementary measure for use alongside the QALY for its potential to alleviate bias against disabled patient populations during the assessment of healthcare treatments.

Updated cost-effectiveness analysis of onabotulinumtoxinA for the prevention of headache in adults with chronic migraine who have previously received three or more preventive treatments in the UK.

Aims: OnabotulinumtoxinA is recommended by NICE for the treatment of chronic migraine. This economic evaluation provides updated estimates of the cost-effectiveness of onabotulinumtoxinA for chronic migraine using new utility estimates in an existing model structure.Methods: A previously published model was revised to include EQ-5D utility estimates from a large observational study (REPOSE; n = 633). Efficacy data were taken from the pooled phase III PREEMPT clinical trial program, while resource utilization estimates were obtained from the International Burden of Migraine Study (IBMS). The model estimated costs and quality-adjusted life years (QALYs) gained over 2 years from the UK NHS perspective.Results: OnabotulinumtoxinA treatment resulted in total discounted incremental costs of £1,204 and an incremental discounted QALY gain of 0.07 compared with placebo in patients with chronic migraine who have previously failed three or more preventive treatments, corresponding to an incremental cost-effectiveness ratio (ICER) of £16,306 per QALY gained. Scenario analysis showed that the administration of onabotulinumtoxinA by a specialist nurse rather than a neurology consultant reduced the ICER from £16,306 to £13,832 per QALY gained. Removal of the positive stopping rule recommended in current NICE guidance increased the ICER to £20,768 per QALY for onabotulinumtoxinA vs. placebo. Combining these two scenarios produced an ICER of £17,686 per QALY gained.Conclusion: NICE recommended onabotulinumtoxinA for the prevention of chronic migraine in 2012 amid concerns about the uncertainty of ICER estimates, with a positive stopping rule used to manage some of these uncertainties. Since the publication of the NICE guidance, the REPOSE study provides a more recent source of utility data based on real-world evidence. The results of analyses including these utilities suggest that the application of the positive stopping rule may not be necessary to ensure cost-effectiveness and that this aspect of the current NICE guidance for onabotulinumtoxinA may merit reconsideration.

Early introduction of a semi-elemental formula may be cost saving compared to a polymeric formula among critically ill patients requiring enteral nutrition: a cohort cost-consequence model.

OBJECTIVES: Gastrointestinal (GI) intolerance is associated with adverse outcomes in critically ill patients receiving enteral nutrition (EN). The objective of this analysis is to quantify the cost of GI intolerance and the cost implications of starting with semi-elemental EN in intensive care units (ICUs). STUDY DESIGN: A US-based cost-consequence model was developed to compare the costs for patients with and without GI intolerance and the costs with semi-elemental or standard EN while varying the proportion of GI intolerance cases avoided. MATERIALS AND METHODS: ICU data on GI intolerance prevalence and outcomes in patients receiving EN were derived from an observational study. ICU stay costs were obtained from literature and the costs of EN from US customers' price lists. The model was used to conduct a threshold analysis, which calculated the minimum number of cases of GI intolerance that would have to be avoided to make the initial use of semi-elemental formula cost saving for the cohort. RESULTS: Out of 100 patients receiving EN, 31 had GI intolerance requiring a median ICU stay of 14.4 days versus 11.3 days for each patient without GI intolerance. The model calculated that semi-elemental formula was cost saving versus standard formula when only three cases of GI intolerance were prevented per 100 patients (7% of GI intolerance cases avoided). CONCLUSION: In the US setting, the model predicts that initial use of semi-elemental instead of standard EN can result in cost savings through the reduction in length of ICU stay if >7% of GI intolerance cases are avoided.

Quantifying the economic burden of productivity loss in rheumatoid arthritis.

OBJECTIVE: In light of the large number of recent studies and systematic reviews investigating the cost of RA, this article examines the methods used to assess the impact of RA on employment and work productivity, and provides an overview of the issues surrounding work productivity loss in the RA population. METHODS: A review of the published literature was conducted in order to identify relevant articles. These articles were then reviewed and their methodologies compared. The various methods used to calculate economic loss were then explained and discussed. RESULTS: We found that although methods of lost productivity and associated costs varied between studies, all suggest that RA is associated with significant burden of illness. Economic analyses that exclude indirect costs will therefore underestimate the full economic impact of RA. However, the methods used to calculate productivity loss have a significant impact on the results of indirect cost analyses, and should be selected carefully when designing such studies. Several factors relating to the disease, the job and socio-demographics have been found to predict work disability. CONCLUSIONS: Consideration of these factors is vital when measuring the extent of both absenteeism and presenteeism, and will allow for more accurate estimation of the impact of RA on work productivity. This information may also guide interventions aiming to prevent or postpone work disability and job loss.

Cost Effectiveness of Paricalcitol versus a non-selective vitamin D receptor activator for secondary hyperparathyroidism in the UK: a chronic kidney disease markov model.

BACKGROUND: secondary hyperparathyroidism (SHPT) is a common complication of chronic kidney disease (CKD) and a frequent cause of clinically significant bone disease. Non-selective vitamin D receptor (VDR) activator treatment has been used to treat the condition but is ineffective for many patients with hypercalcaemia and hyperphosphataemia and may precipitate worsening of their condition. Compared with non-selective VDR activator treatment, use of the VDR ligand paricalcitol may increase survival and reduce the risk of morbidities in patients with SHPT, which may have health economic consequences. OBJECTIVE: the objective of this study was to determine the cost effectiveness of paricalcitol versus a non-selective VDR activator for the treatment of SHPT in patients with CKD in the UK setting. METHODS: A Markov process model was developed employing data sources from the published literature, paricalcitol clinical trials and observational studies, official UK price/tariff lists and national population statistics. The comparator was alfacalcidol, a non-selective VDR activator medication. The primary perspective of the study was that of the UK National Health Service (NHS). The efficacy outcomes (reductions in SHPT, proteinuria, complications and mortality) were extrapolated to: number of life-years gained (LYG) and number of quality-adjusted life-years (QALYs). Clinical and economic outcomes were discounted at 3.5%. The year of costing for costs determined in the study was 2006. RESULTS: the reference case analysis was a 10-year time horizon, based on a comparison of paricalcitol with a non-selective VDR activator, which is started in CKD stage 3 (moderate reduction in glomerular filtration rate [GFR] with kidney damage) and continued in CKD stage 4 (severe reduction in GFR) and CKD stage 5 (established kidney failure). The use of paricalcitol leads to an additional medical cost of pound3224 ($US5970). The health benefits of paricalcitol lead to an increase in LYG of 0.52 and a gain in QALYs of 0.465. Therefore the use of paricalcitol results in an incremental cost-effectiveness ratio of pound6933/QALY ($US12 840/QALY) from the primary perspective of the NHS. One-way sensitivity analyses and probabilistic sensitivity analyses confirmed the robustness of the model. CONCLUSION: this model showed that the favourable clinical benefit of paricalcitol results in positive short- and long-term health economic benefits. This study suggests that the use of paricalcitol in patients with early CKD may be cost effective from the UK NHS perspective versus non-selective VDR activator medication.

A multicentre, retrospective study of resource utilization and costs associated with glaucoma management in France and Sweden.

PURPOSE: To assess resource utilization and costs associated with glaucoma management in France and Sweden. METHODS: A total of 267 patient records (121 in France, 146 in Sweden) with diagnoses of primary open-angle glaucoma (POAG) and ocular hypertension (OH), treated medically, were reviewed for a 2-year period (beginning during 1997-99) for relevant clinical and resource utilization data. Economic data were applied to estimate treatment costs. RESULTS: The annual cost of treating glaucoma was estimated at SEK5305 (531 euro )/patient in Sweden and 390 euro/patient in France. In both countries, medication costs comprised about half of the total costs. Surgical procedures and hospitalizations represented greater proportions of total cost in France (7.0% and 9.6%, respectively) than in Sweden (3.7% and 0.6%, respectively). CONCLUSION: Medication costs represent a high proportion of total treatment costs. These findings highlight the relative importance of medical therapy and of assessing the cost-effectiveness of medications in glaucoma.