Cost-effectiveness modeling for neuropathic pain treatments: investigating the relative importance of parameters using an open-source model.

AIMS: The study objective was to develop an open-source replicate of a cost-effectiveness model developed by National Institute for Health and Care (NICE), in order to explore uncertainties in health economic modeling of novel pharmacological neuropathic pain treatments. MATERIALS AND METHODS: The NICE model, consisting of a decision tree with branches for discrete levels of pain relief and adverse event (AE) severities, was replicated using R, and used to compare a hypothetical neuropathic pain drug to pregabalin. Model parameters were sourced from NICE's clinical guidelines and associated with probability distributions to account for underlying uncertainty. A simulation-based scenario analysis was conducted to assess how uncertainty in efficacy and AEs affected the net monetary benefit (NMB) for the hypothetical treatment at a cost-effectiveness threshold of £20,000 per QALY. RESULTS: Relative to pregabalin, an increase in efficacy was associated with greater NMB than an improvement in tolerability. A greater NMB was observed when efficacy was marginally higher than that of pregabalin, while maintaining the same level of AEs than when efficacy was equivalent to pregabalin, but with a more substantial reduction in AEs. In the latter scenario, the NMB was only positive at a low cost-effectiveness threshold. LIMITATIONS: The replicate model shares the limitations described in the NICE guidelines. There is a lack of support in scientific literature for the assumption that increased efficacy is associated with a greater reduction in tolerability. The replicate model also included a single comparator, unlike the NICE model. CONCLUSIONS: Pain relief is a stronger driver of NMB than tolerability, at a cost-effectiveness threshold of £20,000 per QALY. Health technology assessment decisions which are influenced by NICE's model may reward efficacy gains, even if they are associated with more severe AEs. This contrasts with recommendations from clinical guidelines for neuropathic pain, which place more equal weighting on improvements in efficacy and tolerability as value drivers.

Should we invest in environmental interventions to encourage physical activity in England? An economic appraisal.

BACKGROUND: The Department of Health in England asked the National Institute for Health and Clinical Excellence (NICE) to develop guidance on environmental interventions that promote physical activity. The economic appraisals summarized in this study informed the development of that guidance. In view of the difficulties inherent in applying conventional health economic evaluation techniques to public health interventions, the economic appraisal employed a multi-faceted approach. METHODS: The analyses comprised of three components. Two cost-utility analyses; the first used a life-time disease progression model which sought to take into account the long-term benefits of physical activity on health outcomes, whereas the second used data from a regression analysis which captured some of the short-term, process benefits of physical activity which might manifest themselves in terms of improved mental health and wellbeing. The third approach was a cost-benefit analysis that took into account benefits beyond healthcare. RESULTS: The cost-utility approaches generated cost-effectiveness estimates ranging between £100 and £10 000 per QALY depending on the level of effectiveness of the intervention and the proportion of the intervention cost that was deemed to be attributable to health. The standardized cost-benefit ratio was 11:1. CONCLUSION: The findings present a consistent case to support environmental interventions that promote increased physical activity in the sedentary adult population. However, some degree of caution should be taken in interpreting the findings due to the limitations of the evidence upon which they are based. Further consideration should also be given to the relative merits of alternative approaches to assessing the value of changes to the built environment that might also benefit health as a positive externality.

A comparison of pharmaceutical reimbursement agencies' processes and methods in France and Scotland.

OBJECTIVES: Pharmaceutical reimbursement agencies' processes and methods of appraisal vary across countries. The objective of this study was to examine the contribution of formal health economic analysis in a process using such analysis in Scotland in comparison to a process not routinely using such analysis in France. METHODS: A framework for classifying reimbursement systems was used to analyze the two systems. A typology of recommendation was defined and a qualitative analysis of decisions on a sample of medicines appraised by both reimbursement agencies was conducted. Reasons for differences in recommendations were analyzed and case studies selected to illustrate the common reasons. RESULTS: Thirty-nine common medicines appraised by both agencies were identified between 2005 and 2010, treating a variety of diseases for which the Scottish Medicines Consortium tended to provide more restrictive, or did not recommend, listing. Similarities in clinical evidence submitted to the respective reimbursement committees were observed. Differences in recommendation can be explained by a combination of the manufacturer's freedom to set price and the incentives provided by the consideration of health economic analysis and quality of life, alongside differences in relevant comparators, relevant outcomes, treatment guidelines, and the propensity to use network meta-analysis, in decision making. CONCLUSIONS: This study provides some explanations and hypotheses for the differences observed in recommendations for a selected sample of medicines with regards to differences in appraisal processes and methods adopted. Further research using larger datasets may allow stakeholders to assess the impact of such differences on the efficient use of health resources.

Estimating the direct costs of bowel cancer services provided by the National Health Service in England.

OBJECTIVES: Bowel cancer is the second most common cancer in England and Wales, accounting for approximately 13,000 deaths per year. Economic evaluations and national guidance have been produced for individual treatments for bowel cancer. However, it has been suggested that Primary Care Trusts develop program budgeting or equivalent methodology demonstrating a whole system approach to investment and disinvestment. The objective of this study was to provide a baseline framework for considering a whole system approach to estimate the direct costs of bowel cancer services provided by the National Health Service (NHS) in England. METHODS: A treatment pathway, developed in 2005, was used to construct a service pathway model to estimate the direct cost of bowel cancer services in England. RESULTS: The service pathway model estimated the direct cost of bowel cancer services to the NHS to be in excess of £1 billion in 2005. Thirty-five percent of the cost is attributable to the screening and testing of patients with suspected bowel cancer, subsequently diagnosed as cancer-free. CONCLUSIONS: This study is believed to be the most comprehensive attempt to identify the direct cost of managing bowel cancer services in England. The approach adopted could be useful to assist local decision makers in identifying those aspects of the pathway that are most uncertain in terms of their cost-effectiveness and as a basis to explore the implications of re-allocated resources. Research recommendations include the need for detailed costs on surgical procedures, high-risk patients and the utilization of the methods used in this study across other cancers.

The feasibility of harmonizing health technology assessments across jurisdictions: a case study of drug eluting stents.

OBJECTIVES: There is considerable interest in the potential for harmonizing health technology assessments (HTA) across jurisdictions. This study aims to consider four HTAs of drug eluting stents to determine the degree to which the methods adopted, evidence considered, and resulting recommendations diverge. METHODS: Four HTAs of drug eluting stents were selected for inclusion and evaluated using a framework developed to systematically capture information on the process adopted, the evidence considered and the recommendations of each HTA. RESULTS AND CONCLUSIONS: The findings suggest that, although there is a common core data set considered by most of the agencies, differences in the approach to HTA, heterogeneity of studies, and the limited relevance of research findings to local practice meant that the core data set had only limited influence on the resulting recommendations. Of the HTA agencies considered in the analysis, many sought to generate additional primary research from local settings to help inform the development of recommendations that were relevant to local practice. This raises questions about the extent to which HTA methods can be harmonized across jurisdictions.