The role of budget impact and its relationship with cost-effectiveness in reimbursement decisions on health technologies in the Netherlands.

Health authorities using cost-effectiveness analysis (CEA) for informing reimbursement decisions on health technologies increasingly require economic evaluations encompassing both CEA and budget impact analysis (BIA). Good Research Practices advocate that the economic and clinical assumptions underlying these analyses are aligned and consistently applied. Nonetheless, CEAs and BIAs often are stand-alone analyses used in different stages of the decision-making process. This article used policy reports and Ministerial correspondence to discuss and elucidate the role of budget impact and its relationship with cost-effectiveness in reimbursement decisions in the Netherlands. The results indicate that CEAs and BIAs are both considered important for informing these decisions. While the requirements regarding CEAs-and application of the associated decision rule-are consistent across the different stages, the same does not hold for BIAs. Importantly, the definition of and evidence on budget impact differs between stages. Some important aspects (e.g. substitution and saving effects) typically are considered in the assessment and appraisal stages but are seemingly not considered in price negotiations and the final reimbursement decision. Further research is warranted to better understand why BIAs are not aligned with CEAs (e.g. in terms of underlying assumptions), vary in form and importance between stages, and do not have a clear relationship with the results of CEAs in the decision-making framework. Improving the understanding of the circumstances under which decision-makers attach a relatively larger or smaller weight to (different aspects of) budget impact may contribute to increasing the transparency, consistency, and optimality of reimbursement decisions in the Netherlands.

Psychometric evaluation of the Mental Health Quality of Life (MHQoL) instrument in seven European countries.

INTRODUCTION: To make efficient use of available resources, decision-makers in healthcare may assess the costs and (health) benefits of health interventions. For interventions aimed at improving mental health capturing the full health benefits is an important challenge. The Mental Health Quality of Life (MHQoL) instrument was recently developed to meet this challenge. Evaluating the pyschometric properties of this instrument in different contexts remains important. METHODS: A psychometric evaluation of the MHQoL was performed using existing international, cross-sectional data with 7155 respondents from seven European countries (Denmark, France, Germany, Italy, Portugal, The Netherlands, Portugal and the United Kingdom). Reliability was examined by calculating Cronbach's alpha, a measure of internal consistency of the seven MHQoL dimensions, and by examining the association of the MHQoL sum scores with the MHQoL-VAS scores. Construct validity was examined by calculating Spearman's rank correlation coefficients between the MHQoL sum scores and EQ-5D index scores, EQ-VAS scores, EQ-5D anxiety/depression dimension scores, ICECAP-A index scores and PHQ-4 sum scores. RESULTS: The MHQoL was found to have good internal consistency for all seven countries. The MHQoL sum score and the MHQoL-VAS had a high correlation. Spearman's rank correlation coefficients were moderate to very high for all outcomes. CONCLUSION: Our results, based on data gathered in seven European countries, suggest that the MHQoL shows favourable psychometrical characteristics. While further validation remains important, the MHQoL may be a useful instrument in measuring mental health-related quality of life in the Western European context.

Do Health Technology Assessment organisations consider manufacturers' costs in relation to drug price? A study of reimbursement reports.

INTRODUCTION: Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers' costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. METHOD: An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. RESULTS: A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer's costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers' costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer's costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. CONCLUSION: Despite the attention given to manufacturers' costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs.

Do Profit Margins of Pharmaceuticals Influence Reimbursement Decisions? A Discrete Choice Experiment Among Dutch Healthcare Decision Makers.

OBJECTIVES: This study aimed to investigate whether the profit margins of pharmaceuticals would influence the outcome of reimbursement decisions within the Dutch policy context. METHODS: We conducted a discrete choice experiment among 58 Dutch decision makers. In 20 choice sets, we asked respondents to indicate which of 2 pharmaceutical treatment options they would select for reimbursement. Options were described using 5 attributes (disease severity, incremental costs per quality-adjusted life-year, health gain, budget impact, and profit margin) with 3 levels each. Additionally, cognitive debriefing questions were presented, and for validation debriefing, interviews were conducted. Choice data were analyzed using mixed logit models, also to calculate marginal effects and choice probabilities. RESULTS: Results indicated that the specified levels of profit margins significantly influenced choices made. Decision makers were less likely to reimburse a product with a higher profit margin. The relative importance of profit margins was lower than that of the included traditional health technology assessment criteria, but not negligible. When asked directly, 61% of respondents indicated that profit margin should play a role in reimbursement decision making, although concerns about feasibility and the connection to price negotiations were voiced. CONCLUSIONS: Our results suggest that if available to decision makers the profit margin of pharmaceutical products would influence reimbursement decisions within the Dutch policy context. Higher profit margins would reduce the likelihood of reimbursement. Whether adding profit margin as an additional, explicit criterion to the health technology assessment decision framework would be feasible and desirable is open to further exploration.

Broadening the application of health technology assessment in the Netherlands: a worthwhile destination but not an easy ride?

Currently, reimbursement decisions based on health technology assessments (HTA) in the Netherlands mostly concern outpatient pharmaceuticals. The Dutch government aspires to broaden the systematic application of full HTA towards other types of health care in order to optimise the content of the basic benefit package. This paper identifies important challenges for broadening the scope of full HTA to other types of health care. Based on a description of the Dutch reimbursement decision-making process, five important characteristics of outpatient pharmaceuticals were identified, which are all relevant to the successful application of HTA: (i) closed reimbursement system, (ii) absence of alternative policy measures, (iii) existence of marketing authorisation, (iv) identifiable and accountable counterparty, and (v) product characteristics. For a selection of other types of health care, which may be subject to HTA more frequently in the future, deviations from these characteristics of outpatient pharmaceuticals are discussed. The implications of such deviations for performing HTA and the decision-making process are highlighted. It is concluded that broadening the application of HTA will require policy makers to meet both important policy-related and methodological challenges. These challenges differ per health care domain, which may inform policy makers which expansions of the current use of HTA are most feasible.

[Back pain: patients versus research]. / Rugpijn: patiënten versus onderzoek.

In 2008, approximately 1,559,000 Dutch patients consulted their GP because of back pain. One out of 20 back pain patients is ultimately referred to a specialist, most often to a neurologist. A smaller number (also) go to a neurosurgeon or orthopaedist. Once referred, patients often receive treatments such as pain killers or discectomy. Scientific evidence in systematic reviews is scarce for the treatments we investigated because of small patient groups and a lack of randomised research. We made an infographic to show the numbers of patients with back pain who visit their GP or a specialist.