Mickaël Hiligsmann

Patients' preferences for osteoporosis drug treatment: a discrete-choice experiment

IntroductionThe patient’s perspective is becoming increasingly important in clinical and policy decisions. In this study, we aimed to evaluate the preferences of patients with, or at risk of, osteoporosis for medication attributes, and to establish how patients trade between these attributes.MethodsA discrete choice experiment survey was designed and patients were asked to choose between two hypothetical unlabelled drug treatments (and an opt-out option) that vary in five attributes: efficacy in reducing the risk of fracture, type of potential common side-effects, mode and frequency of administration and out-of-pocket costs. An efficient experimental design was used to construct the treatment option choice sets and a mixed logit panel data model was used to estimate patients’ preferences and trade-offs between attributes.ResultsA total of 257 patients with, or at risk of, osteoporosis completed the experiment. As expected, patients preferred treatment with higher effectiveness and lower cost. They also preferred either an oral monthly tablet or 6-month subcutaneous injection above weekly oral tablets, 3-month subcutaneous, 3-month intravenous or yearly intravenous injections. Patients disliked being at risk of gastro-intestinal disorders more than being at risk of skin reactions and flu-like symptoms. There was significant variation in preferences across the sample for all attributes except subcutaneous injection.ConclusionsThis study revealed that osteoporotic patients preferred 6-month subcutaneous injection and oral monthly tablet, and disliked gastro-intestinal disorders. Moreover, patients were willing to pay a personal contribution or to trade treatment efficacy for better levels of other attributes. Preferences for treatment attributes varied across patients and this highlights the importance of clinical decision-making taking individual preferences into account to improve osteoporosis care.

Interventions to improve osteoporosis medication adherence and persistence: a systematic review and literature appraisal by the ISPOR Medication Adherence & Persistence Special Interest Group

This study aims to systematically review, critically appraise and identify from the published literature, the most effective interventions to improve medication adherence in osteoporosis. A literature search using Medline, EMBASE, Cochrane library, and Cumulative Index to Nursing and Allied Health Literature was undertaken to identify prospective studies published between January 1, 1999 and June 30, 2012. We included studies on adult users of osteoporosis medications that tested a patient adherence intervention (e.g., patient education, intensified patient care, different dosing regimens) and reported quantitative results of adherence. The Delphi list was modified to assess the quality of studies. Of 113 articles identified, 20 studies fulfilled the inclusion criteria. The most frequent intervention was education (n = 11) followed by monitoring/supervision (n = 4), drug regimens (n = 2), drug regimens and patient support (n = 1), pharmacist intervention (n = 1), and electronic prescription (n = 1). Although patient education improved medication adherence in four studies, two large-scale randomized studies reported no benefits. Simplification of dosing regimens (with and without patient support program) was found to have a significant clinical impact on medication adherence and persistence. Monitoring/supervision showed no impact on medication persistence while electronic prescription and pharmacist intervention increased medication adherence or persistence. In conclusion, this review found that simplification of dosing regimens, decision aids, electronic prescription, or patient education may help to improve adherence or persistence to osteoporosis medications. We identified wide variation of quality of studies in the osteoporosis area. The efficacy of patient education was variable across studies, while monitoring/supervision does not seem an effective way to enhance medication adherence or persistence.

Trends in hip fracture incidence and in the prescription of antiosteoporosis medications during the same time period in Belgium (2000–2007)

To examine the secular trend of hip fracture incidence in Belgium between 2000 and 2007 and the concomitant change in the prescriptions of antiosteoporosis medications.

Standardising the descriptive epidemiology of osteoporosis: Recommendations from the Epidemiology and Quality of Life Working Group of IOF

SummaryThe Committee of Scientific Advisors of International Osteoporosis Foundation (IOF) recommends that papers describing the descriptive epidemiology of osteoporosis using bone mineral density (BMD) at the femoral neck include T-scores derived from an international reference standard.IntroductionThe prevalence of osteoporosis as defined by the T-score is inconsistently reported in the literature which makes comparisons between studies problematic.MethodsThe Epidemiology and Quality of Life Working Group of IOF convened to make its recommendations and endorsement sought thereafter from the Committee of Scientific Advisors of IOF.ResultsThe Committee of Scientific Advisors of IOF recommends that papers describing the descriptive epidemiology of osteoporosis using BMD at the femoral neck include T-scores derived from the National Health and Nutrition Examination Survey III reference database for femoral neck measurements in Caucasian women aged 20–29 years.ConclusionsIt is expected that the use of the reference standard will help resolve difficulties in the comparison of results between studies and the comparative assessment of new technologies.

A scoping review of studies comparing the medication event monitoring system (MEMS) with alternative methods for measuring medication adherence

Different methods are available for measuring medication adherence. In this paper, we conducted a scoping review to identify and summarize evidence of all studies comparing the Medication Event Monitoring System (MEMS) with alternative methods for measuring medication adherence. A literature search was performed using the open database www.iAdherence.org that includes all original studies reporting findings from the MEMS. Papers comparing methods for measuring adherence to solid oral formulations were included. Data was extracted using a standardized extraction table. A total of 117 articles fulfilled the inclusion criteria, including 251 comparisons. Most frequent comparisons were against self-report (n = 119) and pill count (n = 59). Similar outcome measures were used in 210 comparisons (84%), among which 78 used dichotomous variables (adherent or not) and 132 used continuous measures (adherence expressed as percentage). Furthermore, 32% of all comparisons did not estimate adherence over the same coverage period and 44% of all comparisons did not use a statistical method or used a suboptimal one. Only eighty-seven (35%) comparisons had similar coverage periods, similar outcome measures and optimal statistical methods. Compared to MEMS, median adherence was grossly overestimated by 17% using self-report, by 8% using pill count and by 6% using rating. In conclusion, among all comparisons of MEMS versus alternative methods for measuring adherence, only a few used adequate comparisons in terms of outcome measures, coverage periods and statistical method. Researchers should therefore use stronger methodological frameworks when comparing measurement methods and be aware that non-electronic measures could lead to overestimation of medication adherence.

Risk of bias in trial-based economic evaluations: Identification of sources and bias-reducing strategies

Objective: The objectives of this article are first to give an overview of the risks of bias in trial-based economic evaluations and, second, to identify how key sources for bias can be revealed and overcome (i.e. what bias-reducing strategies might be employed) in future trial-based economic evaluations in the field of health psychology. Design: Narrative review discussing sources of bias in trial-based economic evaluations and bias-reducing strategies. Results: We identified 11 biases and assigned them to a particular trial phase. A distinction is made between pre-trial biases, biases during the trial and biases that are relevant after the actual trial. All potential forms of bias are discussed in detail and strategies are shown to detect and overcome these biases. Conclusion: In order to avoid bias in trial-based economic evaluations, one has to be aware of all the possible forms of bias. All stakeholders have to examine trial-based economic evaluations in a rigorous and stringent manner. This article can be helpful in this examination as it gives an overview of the possible biases which researchers should take into account.

Cost-effectiveness of vitamin D and calcium supplementation in the treatment of elderly women and men with osteoporosis

BACKGROUND The supplementation with vitamin D and calcium has been recommended for elderly, specifically those with increased risk of fractures older than 65 years. This study aims to assess the cost-effectiveness of vitamin D and calcium supplementation in elderly women and men with osteoporosis and therefore to assess if this recommendation is justified in terms of cost-effectiveness. METHODS A validated model for economic evaluations in osteoporosis was used to estimate the cost per quality-adjusted life-year (QALY) gained of vitamin D/calcium supplementation compared with no treatment. The model was populated with cost and epidemiological data from a Belgian health-care perspective. Analyses were conducted in women and men with a diagnosis of osteoporosis (i.e. bone mineral density T-score ≤-2.5). A literature search was conducted to describe the efficacy of vitamin D and calcium in terms of fracture risk reduction. RESULTS The cost per QALY gained of vitamin D/calcium supplementation was estimated at €40 578 and €23 477 in women and men aged 60 years, respectively. These values decreased to €7912 and €10 250 at the age of 70 years and vitamin D and calcium supplementation was cost-saving at the age of 80 years, meaning that treatment cost was less than the costs of treating osteoporotic fractures of the no-treatment group. CONCLUSION This study suggests that vitamin D and calcium supplementation is cost-effective for women and men with osteoporosis aged over 60 years. From an economic perspective, vitamin D and calcium should therefore be administrated in these populations including those also taking other osteoporotic treatments.

Understanding the stakeholders' intention to use economic decision-support tools: A cross-sectional study with the tobacco return on investment tool

BACKGROUND Despite an increased number of economic evaluations of tobacco control interventions, the uptake by stakeholders continues to be limited. Understanding the underlying mechanism in adopting such economic decision-support tools by stakeholders is therefore important. By applying the I-Change Model, this study aims to identify which factors determine potential uptake of an economic decision-support tool, i.e., the Return on Investment tool. METHODS Stakeholders (decision-makers, purchasers of services/pharma products, professionals/service providers, evidence generators and advocates of health promotion) were interviewed in five countries, using an I-Change based questionnaire. MANOVAs were conducted to assess differences between intenders and non-intenders regarding beliefs. A multiple regression analysis was conducted to identify the main explanatory variables of intention to use an economic decision-support tool. FINDINGS Ninety-three stakeholders participated. Significant differences in beliefs were found between non-intenders and intenders: risk perception, attitude, social support, and self-efficacy towards using the tool. Regression showed that demographics, pre-motivational, and motivational factors explained 69% of the variation in intention. DISCUSSION This study is the first to provide a theoretical framework to understand differences in beliefs between stakeholders who do or do not intend to use economic decision-support tools, and empirically corroborating the framework. This contributes to our understanding of the facilitators and barriers to the uptake of these studies.

Cost-effectiveness of denosumab in the treatment of postmenopausal osteoporotic women.

Denosumab is a novel biological agent for the treatment of osteoporosis in postmenopausal women with increased risk of fractures. With limited healthcare resources, economic evaluations are increasingly being used by decision-makers to optimize healthcare resource allocation. The cost–effectiveness of denosumab has been evaluated in various studies, and a systematic literature study was conducted up to April 2012 to identify all published research articles and research abstracts presented at various congresses. This article provides a systematic review of four articles and eight abstracts reporting on the cost–effectiveness of denosumab in the treatment of osteoporosis. In most economic evaluations, denosumab has been considered as a cost-effective treatment compared with first-line and second-line options (including generic alendronate) in the treatment of women with high risk of fractures.

Similarities and differences between stakeholders' opinions on using Health Technology Assessment (HTA) information across five European countries: results from the EQUIPT survey.

BackgroundThe European-study on Quantifying Utility of Investment in Protection from Tobacco (EQUIPT) project aimed to study transferability of economic evidence by co-creating the Tobacco Return On Investment (ROI) tool, previously developed in the United Kingdom, for four sample countries (Germany, Hungary, Spain and the Netherlands). The EQUIPT tool provides policymakers and stakeholders with customized information about the economic and wider returns on the investment in evidence-based tobacco control, including smoking cessation interventions. A Stakeholder Interview Survey was developed to engage with the stakeholders in early phases of the development and country adaptation of the ROI tool. The survey assessed stakeholders’ information needs, awareness about underlying principles used in economic analyses, opinion about the importance, effectiveness and cost-effectiveness of tobacco control interventions, and willingness to use a Health Technology Assessment (HTA) tool such as the ROI tool.MethodsA cross sectional study using a mixed method approach was conducted among participating stakeholders in the sample countries and the United Kingdom. The individual questionnaire contained open-ended questions as well as single choice and 7- or 3-point Likert-scale questions. The results corresponding to the priority and needs assessment and to the awareness of stakeholders about underlying principles used in economic analysis are analysed by country and stakeholder categories.ResultsStakeholders considered it important that the decisions on the investments in tobacco control interventions should be supported by scientific evidence, including prevalence of smoking, cost of smoking, quality of life, mortality due to smoking, and effectiveness, cost-effectiveness and budget impact of smoking cessation interventions. The proposed ROI tool was required to provide this granularity of information. The majority of the stakeholders were aware of the general principles of economic analyses used in decision making contexts but they did not appear to have in-depth knowledge about specific technical details. Generally, stakeholders’ answers showed larger variability by country than by stakeholder category.ConclusionsStakeholders across different European countries viewed the use of HTA evidence to be an important factor in their decision-making process. Further, they considered themselves to be capable of interpreting the results from a ROI tool and were highly motivated to use it.