Economic evaluation of osteoporosis screening strategy conducted in the Province of Liège with the cooperation of Liège Province Santé

peer reviewedThe Province of Liege has conducted an osteoporosis screening strategy for women aged 50 to 69 years. The objective of this study is to investigate the economic characteristics of the screening strategy and to assess its cost-effectiveness, using a Markov microsimulation model. Our analyses suggest that the osteoporosis screening strategy is efficient if the medical community and the patients fulfill the recommendations of the Province of Liege health authorities and if persistence is optimized. Therefore, bone mineral density (BMD) measurement should be performed in all individuals with positive ultrasound screening; individuals having a positive BMD diagnosis should be treated and adherence to therapy should be increased. Furthermore, to improve the efficiency of the screening strategy, we suggest to target screening on women with one or more clinical risk factors, or on women aged 65 years and older

Anti-fracture efficacy of intravenous ibandronate: how to translate epidemiological studies into daily clinical practice

peer reviewedNumerous epidemiological approaches are used to demonstrate the efficacy of a new chemical entity. In postmenopausal osteoporosis, anti-fracture efficacy can be assessed through prospective, randomized controlled trials, meta-analyses or real-life setting studies. Intravenous ibandronate was recently marketed, with the aim of optimizing drug absorption and adherence to treatment. Furthermore, this new formulation avoids gastrointestinal side effects and constrains linked to the oral intake of the medication. Spinal anti-fracture efficacy of IV ibandronate derives from a non-inferiority bridging study, using surrogate endpoints, i.e., bone mineral density and biochemical markers of bone turnover, compared to the oral daily formulation, previously registered for the treatment of osteoporosis in Europe. Coherent results from two separate meta-analyses have suggested that the non-vertebral anti-fracture efficacy of IV ibandronate is similar to that observed with oral, daily and weekly bisphosphonates. Similarly, a recent real-life setting study, based on claims from an US database, suggests that hip fractures are reduced, with IV ibandronate, to the same extend as they are with oral bisphosphonates. Notwithstanding, those results should probably be confirmed in an European setting, before being extrapolated, in daily practice, to the Belgian population

Quality of life and societal costs in hypertrophic cardiomyopathy:protocol of the AFFECT-HCM study

Background: Ever since the first description of hypertrophic cardiomyopathy (HCM), the most common genetic cardiac disease, tremendous progress has been made in the evaluation and management of HCM patients, but little attention has been focused on the impact of HCM on societal costs and quality of life (QoL). Aims: This paper describes the study protocol for the AFFECT-HCM study into burden of disease (BoD), which aims to estimate health-related QoL and societal costs in HCM patients and genotype-positive phenotype-negative (G+/P−) relatives during a one-year follow-up study, and relate this to the phenotypical HCM expression. Methods: A total of 400 Dutch HCM patients and 100 G+/P− subjects will be followed for one year in a prospective, multi-centre, prevalence-based BoD study. Societal costs will be measured via a bottom-up approach using the cost questionnaires iMCQ and iPCQ. For QoL, the generic EQ-5D-5L and disease-specific Kansas City Cardiomyopathy Questionnaire will be used. QoL and societal costs will be compared with phenotype-specific HCM characteristics and other determinants to identify factors that influence BoD. Accelerometry will test the correlation between BoD and physical activity. Conclusion: The AFFECT-HCM study will evaluate the BoD in HCM patients and G+/P− subjects to improve the understanding of the societal and economic impact of HCM.</p

Estimating loss in capability wellbeing in the first year of the COVID-19 pandemic:a cross-sectional study of the general adult population in the UK, Australia and the Netherlands

Objectives To estimate capability wellbeing lost from the general adult populations in the UK, Australia and the Netherlands in the first year of the COVID-19 pandemic and the associated social restrictions, including lockdowns. Design Cross-sectional with recalled timepoints. Setting Online panels in the UK, Australia and the Netherlands conducted in February 2021 (data collected 26 January-2 March 2021). Participants Representative general adult (>= 18 years old) population samples in the UK (n = 1,017), Australia (n = 1,011) and the Netherlands (n = 1,017) Main outcome measure Participants completed the ICECAP-A capability wellbeing measure in February 2021, and for two recalled timepoints during the initial lockdowns in April 2020 and in February 2020 (prior to COVID-19 restrictions in all three countries). ICECAP-A scores on a 0-1 no capability-full capability scale were calculated for each timepoint. Societal willingness to pay estimates for a year of full capability (YFC) was used to place a monetary value associated with change in capability per person and per country. Paired t tests were used to compare changes in ICECAP-A and YFC from pre- to post-COVID-19-related restrictions in each country. Results Mean (standard deviation) loss of capability wellbeing during the initial lockdown was 0.100 (0.17) in the UK, 0.074 (0.17) in Australia and 0.049 (0.12) in the Netherlands. In February 2021, losses compared to pre-lockdown were 0.043 (0.14) in the UK, 0.022 (0.13) in Australia and 0.006 (0.11) in the Netherlands. In monetary terms, these losses were equivalent to 14.8 pound billion, AUD$8.6 billion and euro2.1 billion lost per month in April 2020 and 6.4 pound billion, A$2.6 billion and euro260 million per month in February 2021 for the UK, Australia and the Netherlands, respectively. Conclusions There were substantial losses in capability wellbeing in the first year of the COVID-19 pandemic. Future research is required to understand the specific impact of particular COVID-19 restrictions on people's capabilities

Effectiveness and cost-effectiveness of a nurse-delivered intervention to improve adherence to treatment for HIV : a pragmatic, multicentre, open-label, randomised clinical trial

This trial was funded from public money by the Netherlands Organisation for Health Research and Development (ZonMW; grant number 171002208). Aardex provided support on the development of the study website. We thank all the HIV nurses and physicians from the seven HIV clinics involved in the AIMS study for their input and collaboration (Academic Medical Centre, Slotervaart hospital, and St. Lucas-Andreas hospital, all in Amsterdam; the Leiden University Medical Centre, Leiden; HAGA hospital, The Hague; Erasmus Medical Centre, Rotterdam; and Isala clinic, Zwolle), the study participants, and the Stichting HIV Monitoring (SHM) for their support in accessing the SHM database for identifying patient inclusion criteria and developing the Markov model. Finally, we thank and remember Herman Schaalma (deceased) for his contribution to the study design and grant application.Peer reviewedPostprin

A FRAX model for the assessment of fracture probability in Belgium

peer reviewedRESUME : Cette étude a pour but d’adapter à la population belge l’algorithme FRAX® récemment publié par l’Organisation Mondiale de la Santé (OMS) et permettant de calculer, dans les deux sexes, le risque absolu de fractures ostéoporotiques, à 10 ans. Nous nous sommes attachés à quantifier le risque fracturaire correspondant aux critères actuellement appliqués, en Belgique, pour le remboursement des médicaments de l’ostéoporose et à identifier les situations cliniques correspondant à une probabilité équivalente de fracture. Les probabilités fracturaires ont été calculées, à partir des incidences de fractures et de décès publiées, pour la population belge. Ces probabilités prennent en considération l’âge, le sexe, l’existence de facteurs cliniques de risque (FCR) et la densité minérale osseuse (DMO), mesurée au niveau de la zone propre du col fémoral. L’algorithme FRAX® permet d’identifier différents scénarios d’intervention, en Belgique, correspondant à un risque fracturaire identique ou supérieur à celui servant de base aux critères actuels de remboursement des médicaments. Il est donc possible de recommander une modification des attitudes actuelles, délaissant une stratégie basée sur une évaluation dichotomique de la DMO, au profit d’une intégration progressive des FCR qui permettra, in fine, une meilleure identification des patients à risque accru de fracture. Cette approche devra être substantiée par des analyses pharmaco-économiques appropriées

Comparison of statistical analysis methods for object case best–worst scaling

KLC received financial support for his research stay at the Department of Health Economics, Center for Public Health, Medical University of Vienna through the ‘Erasmus+ staff mobility grant’

Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium.

Three new therapies for spinal muscular atrophy (SMA) have been approved by the United States Food and Drug Administration and the European Medicines Agency since 2016. Although these new therapies improve the quality of life of patients who are symptomatic at first treatment, administration before the onset of symptoms is significantly more effective. As a consequence, newborn screening programs have been initiated in several countries. In 2018, we launched a 3-year pilot program to screen newborns for SMA in the Belgian region of Liège. This program was rapidly expanding to all of Southern Belgium, a region of approximately 55,000 births annually. During the pilot program, 136,339 neonates were tested for deletion of exon 7 of SMN1, the most common cause of SMA. Nine SMA cases with homozygous deletion were identified through this screen. Another patient was identified after presenting with symptoms and was shown to be heterozygous for the SMN1 exon 7 deletion and a point mutation on the opposite allele. These ten patients were treated. The pilot program has now successfully transitioned into the official neonatal screening program in Southern Belgium. The lessons learned during implementation of this pilot program are reported