Available data and models for the health-economic evaluation of treatment in Alzheimer's disease (AD) have limitations causing uncertainty to decision makers. Forthcoming treatment strategies in preclinical or early AD warrant an update on the challenges associated with their economic evaluation. The perspectives of the co-authors were complemented with a targeted review of literature discussing methodological issues and data gaps in AD health-economic modelling. The methods and data available to translate treatment efficacy in early disease into long-term outcomes of relevance to policy makers and payers are limited. Current long-term large-scale data accurately representing the continuous, multifaceted, and heterogeneous disease process are missing. The potential effect of disease-modifying treatment on key long-term outcomes such as institutionalization and death is uncertain but may have great effect on cost-effectiveness. Future research should give priority to collaborative efforts to access better data on the natural progression of AD and its association with key long-term outcomes.

The purpose of this study was to review and summarise the literature on appropriateness criteria for treatment of osteoporotic vertebral compression fractures (OVCF), with appropriateness defined as a treatment where the expected benefits outweigh the expected harms, confirmed by available evidence and expert opinion. A comprehensive search of peer-reviewed publications (PubMed, EMBASE) and grey literature was performed. To be included for analysis, documents had to be a review article (e.g. clinical guideline or meta-analysis), focus on OVCF and make a statement on treatment appropriateness. Eleven publications fulfilled the eligibility criteria. Among the five publications that made recommendations about non-surgical management (NSM), there is agreement that conservative methods are appropriate in OVCF patients who have low level of pain, and that the majority of patients should be treated with conservative methods before other treatments are initiated. All publications made recommendations about vertebral augmentation procedures (VAP), i.e. vertebroplasty (VP) and/or balloon kyphoplasty (BKP). VAP are mostly considered appropriate in patients with high level of pain who do not respond to NSM. However, results cannot be generalised due to heterogeneity of treatment recommendations and patient selection. Although there is a consensus that NSM should be considered as the first-line treatment, there is more heterogeneity in treatment recommendations for VAP. This could most likely be explained by an insufficient clinical evidence base for VAP and heterogeneity of OVCF patients, leading to greater reliance on expert opinion affecting the quality of evidence in the primary sources.

Objectives: As the pharmaceutical industry increases its focus on treating chronic diseases, it is vitally important to obtain accurate estimates of future costs and benefits. To date, the standard discounted utility model (DU) has almost exclusively been used, even though many studies showed that time-inconsistent, hyperbolic discounting models better describe human behaviour. Such models are not currently being employed, mainly due to confusion and uncertainty about their underlying theory and applicability. The aim of this study is to highlight the differences between these two models when assessing health benefits over long time horizons. MethOds: The difference in discounting was demonstrated using test data over a 15-year time horizon. The DU model used a constant discount rate over time, whereas the hyperbolic model contained two parameters, one for perception of time and one for the departure from the traditional model (Loewenstein and Prelec,1992). The parameters used to calculate the discount functions were those proposed by Cairns and van der Pol (2000). Results: Not only did the QALY within each treatment group drop significantly but the total QALY gain under the hyperbolic model was 61% lower compared with the DU model. As a result, the DU model overestimated the added value of the intervention, whereas the hyperbolic model, keeping all other variables constant, led to a lower ICER. cOnclusiOns: As average life expectancy increases so does the financial burden of long-term interventions for chronic diseases. This necessitates a more refined assessment for reimbursement that more accurately reflects people's behaviour. We have demonstrated that, depending on the values of the parameters, using a more valid discounting approach can dramatically affect the estimate of ICER. We conclude that researchers should not rely on the DU model for treatments that achieve long-term health effects as it fails to account for people's changing behaviour as they get older.