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Purpose: Although researchers have explored parental perspectives of childhood speech and language disorders, most studies have been conducted in English-speaking countries. Little is known about parental experiences across countries, where procedures of language screening and services for language disorders differ. The authors participated in the COST Action network IS1406, "Enhancing Children's Oral Language Skills Across Europe and Beyond," which provided an opportunity to conduct cross-country qualitative interviews with parents. The aim of this pilot study was to explore ways in which parents construed and described speech and language disorders across countries. Method: Semistructured qualitative interviews were conducted with parents from 10 families in 10 different countries. The data were analyzed using thematic analysis. Findings: The overall theme was "acknowledging parental expertise." The parents described, in detail, ways in which their children's speech and language (dis)abilities had an impact on the children's everyday life. Three subthemes were identified: impairment, disability, and changes over time. Conclusions: The findings suggest that, across a range of countries, parents demonstrated contextualized understandings of their children's speech and language (dis)abilities, along with the everyday functional implications of the disorders. Hence, despite not holding professional knowledge about language disorders, the voices, views, understandings, and personal experiences of parents in relation to their child's disorder should be listened to when planning therapy services.

Introduction: Marketing materials from pharmaceutical companies attempt to create a positive image of marketed, often new, medicines. To gain more insight in strategies pharmaceutical companies use to influence primary care practitioners' attitudes towards marketed medicines, we investigated the use of persuasion strategies in direct marketing mailings and advertisements from pharmaceutical companies sent to general practitioners. Methods: General practitioners in the Netherlands were recruited to collect all direct marketing mailings, meaning all leaflets, letters and other information sent by pharmaceutical industries to the practice during one month (June 2022). Direct marketing mailings and advertisements in collected medical journals concerning medicines or diseases (together called marketing materials) were analysed according to presence of one of the seven common persuasion strategies, i.e. reciprocity, consistency/commitment, social proof, liking, authority, scarcity and unity; as well as marketed medicine and year of introduction. Results: Twenty general practices collected 68 unique marketing materials concerning 37 different medicines. Direct factor Xa inhibitors (n = 12), glucagon-like peptide-1 analogues (n = 5) and sodium-glucose co-transporter 2 inhibitors (n = 4) were the most frequently marketed medicines. The median year of introduction of all marketed medicines was 2012. All seven persuasion strategies were identified, with liking (64.7% of all materials) and authority (29.4%) as most prominent strategies, followed by social proof (17.6%), unity (14.7%), scarcity (13.2%), reciprocity (11.8%) and consistency/commitment (2.9%). In addition to those strategies, we identified emotional pressure (30.9%) as one commonly used new strategy. Conclusion: Marketing materials sent to general practices use a wide range of persuasion strategies in an attempt to influence prescription behaviour. Primary care practitioners should be aware of these mechanisms through which pharmaceutical companies try to influence their attitudes towards new medicines. [ABSTRACT FROM AUTHOR]

Introduction: Current research to assess the impact that medicine shortages have on patients is limited to general aspects, such as the prevalence of shortages and product characteristics. The aim of this study is to assess the overall impact that medicine shortages have on economic, clinical, and humanistic outcomes. Methods: A cohort of all known products in shortage in the Netherlands between 2012 and 2015 were characterized by their route of administration, anatomical therapeutic chemical class, and whether they were originator or generic products. A representative sample of 324 shortages (18% of all shortages) was rated as having low, medium, or high impact on the five elements that determine the impact of shortages on patients: availability of an alternative product, underlying disease, susceptibility of the patient, costs (for patients and society at large), and number of patients affected. Ratings were converted into numerical scores per element and multiplied to obtain an overall impact score. Results: Two elements were most frequently rated as having a high impact: disease (29%) and costs (20%). Nearly half of the shortages (47%) rated high on at least one element, while nearly 10% rated high on multiple elements. Thirty percent of the shortages rated high on direct impact, which is represented by these elements: alternative product and disease. An additional 17% of the shortages rated high on indirect impact, which is represented by these elements: costs, susceptibility, and number of patients. High impact scores could not significantly be attributed to characteristics of the products in shortage. Conclusions: An assessment of the medicine shortages' impact using a framework based on economic, clinical, and economic outcomes showed that all three outcomes affect the overall impact that medicine shortages have on patients. [ABSTRACT FROM AUTHOR]

Background: Medicine shortages are often described in plain numbers, suggesting all shortages have a uniform impact. However, some shortages have a direct and serious effect on patients and need a prompt reaction from stakeholders. This study aims to create a broad framework to assess the impact of a shortage.Method: We identified high impact shortages and selected exemplary shortages which we considered our learning cases. From five learning cases, we identified elements that had a potentially profound impact on one or more of these cases. We tested data saturation on the elements with another five test cases. Based on these elements, we created a framework to assess impact of shortages on patients and presented practical examples how to rate these different elements. Subsequently, we visualised the impact of these five learning cases on patients in radar charts.Results: The five elements which we identified as potentially having a large impact were 1) alternative product, 2) disease, 3) susceptibility, 4) costs and 5) number of patients affected. The five learning cases rated high on different elements, leading to diverse and sometimes even opposite patterns of impact.Conclusion: We created a framework for assessing the impact of a medicine shortage on patients by means of five key elements. By rating these elements, an indication of the impact can be obtained. [ABSTRACT FROM AUTHOR]

Background Numerous drugs prolong the QTc interval on the ECG and potentially increase the risk of cardiac arrhythmia. This risk is clinically relevant in patients with additional risk factors. Objective The objective was to develop and validate a risk model to predict QTc interval prolongation of eligible ECGs. Setting Spaarne Gasthuis (Haarlem/Hoofddorp, The Netherlands). Method A dataset was created from ECGs recorded in patients using one or more QTc prolonging drugs, in the period January 2013 and October 2016. In the development set, independent risk factors for QTc interval prolongation were determined using binary logistic regression. Risk scores were assigned based on the beta coefficient. In the risk-score validation set, the area under the ROC-curve, sensitivity and specificity were calculated. Main outcome measure QTc interval prolongation, defined as a QTc interval > 500 ms. Results In the development set 12,949 ECGs were included and in the risk-score validation set 6391 ECGs. The proportion of ECGs with a prolonged QTc interval in patients with no risk factors in the risk-score validation set was 2.7%, while in patients with a high risk score the proportion was 26.1%. The area under the ROC curve was 0.71 (95% CI 0.68-0.73). The sensitivity and specificity were 0.81 and 0.48, respectively. Conclusion A risk model was developed and validated for the prediction of QTc interval prolongation. This risk model can be implemented in a clinical decision support system, supporting the management of the risks involved with QTc interval prolonging drugs. [ABSTRACT FROM AUTHOR]

Objectives: Antibiotic use is unnecessarily high for paediatric respiratory tract infections (RTIs) in primary care, and implementation of treatment guidelines is difficult in practice. This study aims to assess guideline adherence to antibiotic prescribing for RTIs in children and examine potential variations across Dutch general practices.Methods: We conducted a retrospective observational study, deriving data on diagnoses and prescriptions from the electronic health records-based NIVEL Primary Care Database. Patients <18 years of age with a diagnosis of fever, ear and respiratory infections (International Classification of Primary Care codes A03, H71, R72, R75, R76, R78 and R81) during 2010-12 were included. Antibiotics were linked to episodes of illness. Two types of disease-specific outcomes were used to assess adherence to national guidelines regarding antibiotic prescribing choices. Inter-practice variability in adherence was assessed with multilevel analysis.Results: Half of the episodes with RTIs with restrictive prescribing policy and 65% of episodes with pneumonia were treated with antibiotics. General practitioners prescribed antibiotics for 40% of episodes with bronchitis, even though guidelines discourage antibiotic prescribing. First-choice antibiotics were prescribed in 50%-85% of episodes with selected diseases, with lowest values for narrow-spectrum penicillins. Levels of adherence to guidelines varied widely between diagnoses and between practices.Conclusions: Most paediatric RTIs in the Netherlands continue to be treated with antibiotics conservatively. Potential aspects of concern are the inappropriate antibiotic prescribing for acute bronchitis and the underuse of some first-choice antibiotics. Continuing progress may be achieved by targeting practices with lower adherence rates to guidelines. [ABSTRACT FROM AUTHOR]

Objective To assess the trends in the incidence and prevalence rates of type 1 diabetes ( T1D) among children and adolescents in the Netherlands. Methods A population-based cohort study was conducted in the Dutch PHARMO record linkage system (1998-2011). All children and adolescents aged ≤19 yr with at least one insulin dispensing (as a proxy for T1D) were identified and the numbers of incident and prevalent cases (numerators) were calculated. Overall age-adjusted (0-19 yr) incidence and prevalence rates together with age- and sex-specific rates of T1D and their 95% confidence intervals ( CI) were calculated using data from the Dutch Central Bureau of Statistics as denominator. Trends over time were assessed using Joinpoint regression software (National Cancer Institute, Bethesda, MD, USA). Results In 2011, the overall age-adjusted incidence and prevalence rates of T1D were 25.2/100 000 (95% CI, 23.7-26.8) person-years ( PY) and 174.4/100 000 (95% CI, 170.2-178.5) children, respectively. The average annual percentage change ( AAPC) in the overall age-adjusted incidence and prevalence rate was 3.7% (95% CI, 1.8-5.7) and 3.8% (95% CI, 2.4-5.2), respectively. While during the study period the largest increases in the incidence and prevalence rates of T1D were observed for the oldest age groups (10-14 and 15-19 yr), a decreasing trend was detected for the 0- to 4-yr-old category (with AAPCs of −1.8 (95% CI, −9.9 to 7.1) and −6.9% (95% CI, −11.5 to −2.1) for incidence and prevalence, respectively). Conclusion Age-adjusted incidence (1999-2011) and prevalence rates (1998-2011) of T1D in Dutch children (aged 0-19 yr) continued to increase and a shift was observed to a later onset of the disease. [ABSTRACT FROM AUTHOR]

The article discusses the transformation of the Technische Universiteit Delft Library in the Netherlands into a Library Learning Centre using different archetypes. Topics include the increase of its visitors, the different users in the centre and the services offered by the library. Other topics are the development of the basic principles that guide staff members in line with the transformation.

Background: In 2003-2004 and 2007-2008, the regulatory banning of SSRI use in pediatrics and young adults due to concerns regarding suicidality risk coincided with negative media coverage. SSRI use trends were analyzed from 2000-2010 in the Netherlands (NL) and the UK, and whether trend changes might be associated with media coverage of regulatory warnings. Methods: Monthly SSRIs sales were presented as DDDs/1000 inhabitants/day. SSRI-use trends were studied using time-series segmented regression analyses. Timing of trend changes was compared with two periods of media coverage of warnings. Annual Dutch SSRI prescription data were analyzed by age group. Results: Trend changes in SSRI use largely corroborated with the periods of media coverage of warnings. British SSRI use declined from 3.9 to 0.7 DDDs/month (95%CI 3.3;4.5 & 0.5;0.9, respectively) before the first warning period (2003-2004). A small decrease of 20.6 DDDs/month (21.2; 20.05) was observed in Dutch SSRI use shortly after 2003-2004. From 2007-2008, British SSRI use stabilized, whilst Dutch SSRI use diminished to 20.04 DDDs/month (20.4;0.3). Stratified analyses showed a rapid decrease of 21.2 DDDs/month (22.1; 21.7) in UK paroxetine use before 2003-2004, but only a minimal change in Dutch paroxetine use (20.3 DDDs/month 20.8;0.2). Other SSRI use, especially (es)citalopram, increased during 2003-2004 in both countries. Significant reductions in Dutch paroxetine use were observed in pediatrics, adolescents, and young adults after 2003-2004. Conclusion: Changes in SSRI use (NL & UK) were associated with the timing of the combined effect of media coverage and regulatory warnings. Our long-term assessment illustrates that changes in SSRI use were temporal, drug-specific and more pronounced in pediatrics and young adults. The twofold increase in SSRI use over one decade indicates that regulatory warnings and media coverage may come and go, but they do not have a significant impact on the overall upward trend of SSRI use as a class in both countries. [ABSTRACT FROM AUTHOR]

Background: At present, there is insufficient evidence to guide appropriate management of women with preterm prelabor rupture of membranes (PPROM) near term. Methods and Findings: We conducted an open-label randomized controlled trial in 60 hospitals in The Netherlands, which included non-laboring women with .24 h of PPROM between 34+0 and 37+0 wk of gestation. Participants were randomly allocated in a 1:1 ratio to induction of labor (IoL) or expectant management (EM) using block randomization. The main outcome was neonatal sepsis. Secondary outcomes included mode of delivery, respiratory distress syndrome (RDS), and chorioamnionitis. Patients and caregivers were not blinded to randomization status. We updated a prior meta-analysis on the effect of both interventions on neonatal sepsis, RDS, and cesarean section rate. From 1 January 2007 to 9 September 2009, 776 patients in 60 hospitals were eligible for the study, of which 536 patients were randomized. Four patients were excluded after randomization. We allocated 266 women (268 neonates) to IoL and 266 women (270 neonates) to EM. Neonatal sepsis occurred in seven (2.6%) newborns of women in the IoL group and in 11 (4.1%) neonates in the EM group (relative risk [RR] 0.64; 95% confidence interval [CI] 0.25 to 1.6). RDS was seen in 21 (7.8%, IoL) versus 17 neonates (6.3%, EM) (RR 1.3; 95% CI 0.67 to 2.3), and a cesarean section was performed in 36 (13%, IoL) versus 37 (14%, EM) women (RR 0.98; 95% CI 0.64 to 1.50). The risk for chorioamnionitis was reduced in the IoL group. No serious adverse events were reported. Updating an existing meta-analysis with our trial results (the only eligible trial for the update) indicated RRs of 1.06 (95% CI 0.64 to 1.76) for neonatal sepsis (eight trials, 1,230 neonates) and 1.27 (95% CI 0.98 to 1.65) for cesarean section (eight trials, 1,222 women) for IoL compared with EM. Conclusions: In women whose pregnancy is complicated by late PPROM, neither our trial nor the updated meta-analysis indicates that IoL substantially improves pregnancy outcomes compared with EM. [ABSTRACT FROM AUTHOR]

The article presents a study which aims to assess the impact of reimbursement restriction on benzodiazepine use in patients diagnosed with anxiety or sleeping disorder. The study identified 13,596 patients with an incident diagnosis of anxiety or sleeping disorder taken from the database of Netherlands Information Network of General Practice (LINH). Result shows that reimbursement restriction has led to a moderate decrease in the number of incident diagnoses and initiation of benzodiazepine use.

Introduction: There is some evidence that the beneficial effects of HMG-CoA reductase inhibitors (statins) in the elderly are at least comparable to the effects in middle-aged people. However, several studies have shown prescription rates of statins to be significantly lower in the elderly than in younger populations. Objective: The aim of the present study was to monitor statin prescribing trends in the elderly in the Netherlands over time in terms of prevalence, incidence, type of statin, dose prescribed and adherence to clinical guidelines. Methods: The database of a community pharmacy in Utrecht, which includes prescription data for approximately 11 000 people, was analysed to investigate trends in statin prescriptions from January 1999 to December 2008. The 1-year prevalence and incidence of statin use stratified by age were determined for each calendar year. Rate ratios (RRs) and 95% confidence intervals were calculated with 1999 as the reference year. Furthermore, the following trends of interest were calculated for each calendar year: the percentage of statin users prescribed simvastatin or atorvastatin, the median dose of simvastatin and atorvastatin prescribed, and the percentage of simvastatin users prescribed a dosage of 40mg/day (which is recommended by the Dutch multidisciplinary guideline). Results: The 1-year prevalence of statin use in medication users aged ⩾50 years increased from 13.9% in 1999 to 22.8% in 2008 (RR 1.6; 95% CI 1.4, 1.9; p<0.001). Overall, the lowest prevalence (5.1% in 1999 and 15.2% in 2008) and incidence rates (3.2% in 2000 and 4.2% in 2008) were found in patients aged ⩾80 years. Before 2006, simvastatin was the most commonly prescribed statin, but the number of users declined as the percentage of patients with new simvastatin prescriptions decreased (from 43.4% in 2000 to 36.5% in 2005) and the percentage of patients treated with new atorvastatin prescriptions increased (from 37.7% in 2000 to 47.3% in 2005). As from 2006, when the Dutch multidisciplinary guideline for Cardiovascular Risk Management was introduced, recommending treatment with a daily simvastatin dose of 40mg, the number of simvastatin users increased again and most treatment-naive patients were sorted on simvastatin (62.3% in 2006, increasing to 66.7% in 2008). The median simvastatin dose increased from 10mg in 1999 to 20mg in 2001, remaining at the same dose until 2008, and appeared to be related to the patient's age. From 2006, patients aged ⩾80 years were the least likely group to receive the recommended dose of 40mg simvastatin daily (10.0-20.0% of simvastatin users aged 80 years compared with 32.5-36.9% of simvastatin users aged 60-69 years). Conclusion: Despite the benefits of statin treatment previously reported in older patients, the prevalence and incidence of statin use were lower in elderly patients compared with younger patients. In addition, lower dosages of statins were prescribed. These findings suggest the beneficial effects of statins in the elderly observed in clinical trials may not be achieved in everyday practice. [ABSTRACT FROM AUTHOR]

To assess dosing and determinants of the choice of statins among starters of statins. Data were obtained from the PHARMO database comprising pharmacy and linked hospital discharge records of approximately 300 000 subjects in the Netherlands. All new users of statins in 1998 were selected. Patient characteristics and drug regimens were compared between starters of different statins. Odds ratios (OR) and 95% confidence intervals (CI) were calculated using polytomous logistic regression modelling, using the start of simvastatin therapy as reference category. In 1998, 1738 patients started using simvastatin (41.1%), pravastatin (23.1%), fluvastatin (11.9%), atorvastatin (22.8%) or cerivastatin (1.0%). Compared with starters with simvastatin [mean dose 1.02 ± 0.39 defined daily doses (DDDs)], starters with pravastatin (1.27 ± 0.56 DDDs) and atorvastatin (1.43 ± 0.59 DDDs) received higher doses ( P < 0.001), whereas users of fluvastatin (0.78 ± 0.37 DDDs) and cerivastatin (0.81 ± 0.30 DDDs) received lower doses ( P < 0.001). Patients already using CYP3A4 inhibitors more frequently received fluvastatin (OR = 1.80; 95% CI 1.11, 2.94), metabolized by non-CYP3A4 pathways, and atorvastatin (OR = 1.62; 95% CI 1.06, 2.47), which is metabolized by CYP3A4, than simvastatin. Statin doses were not adjusted when prescribed to patients using CYP3A4 inhibitors. Many patients starting statin therapy did not receive a statin of first choice. The coadministration of potentially interacting drugs may have led to a change in statin choice, but not in dosage lowering. These findings suggest that the quality of statin therapy could be improved. [ABSTRACT FROM AUTHOR]

To assess recent trends in undertreatment of hypercholesterolaemia (1998–2002). Data were obtained from the third cross-sectional examination of the Monitoring Project on Risk Factors for Chronic Diseases ( n = 4878; age 31–70 years), conducted in the Netherlands. Treatment eligibility was established according to Dutch guidelines. Data from the second examination (1993–1997) were used to assess time trends. The association between demographic variables, cardiovascular disease risk factors, drug use and lipid-lowering medication was assessed using multivariable logistic regression. Overall, 45.9%[95% confidence interval (CI) 41.4, 50.4] of respondents eligible for treatment were treated, and 17.4% (95% CI 13.9, 20.9) were both treated and controlled (1998–2002). Treatment increased significantly after 1995, showed a slight decrease in subsequent years until 2000, when treatment increased again. Subgroups less frequently treated for primary prevention included among others males [odds ratio (OR) = 0.08; 95% CI 0.03, 0.21], younger patients (OR = 0.93 per year; 95% CI 0.88, 0.98), diabetics (OR = 0.19; 95% CI 0.07, 0.56), untreated hypertensives (OR = 0.21; 95% CI 0.09, 0.49) and current smokers (OR = 0.09; 95% CI 0.03, 0.25). In secondary prevention, patients with a history of stroke were less likely to receive treatment (OR = 0.41; 95% CI 0.18, 0.94) compared with patients with a history of ischaemic heart disease. Treatment of hypercholesterolaemia has steadily increased over the past 10 years in the Netherlands. However, at present still less than one out of two eligible for treatment is treated, and only about one out of six is both treated and controlled. [ABSTRACT FROM AUTHOR]

Background: Persistence with lipid-lowering drug use is important in order for patients to gain full treatment benefit. The withdrawal of cerivastatin from the market may have affected persistence due to the fear of serious adverse effects.Objective: To assess failure of patients to continue lipid-lowering drug use following the withdrawal of cerivastatin.Methods: A cohort study including 3.5 months follow-up after the withdrawal of cerivastatin in August 2001 was conducted using data from community pharmacies in The Netherlands, covering a population of approximately 600,000 subjects. Patients selected for inclusion in the index group were current users of cerivastatin on August 10, 2001 (the date that cerivastatin was withdrawn from the market). Reference patients were gender, age and pharmacy or region matched patients who were using any HMG-CoA reductase inhibitor other than cerivastatin on the same date. The main outcome measure was discontinuation of lipid-lowering drug use. To assess whether discontinuation had increased in the reference group, discontinuation rates were compared with discontinuation rates in the previous year. Data on these rates in 2000 were obtained from the population-based PHARMO record linkage system.Results: A total of 31 pharmacies of the research network (response rate 86.1%) provided medication histories of 234 current users of cerivastatin and 431 matched patients using any other HMG-CoA reductase inhibitor. In addition, 352 current users of cerivastatin and 704 matched patients using any other HMG-CoA reductase inhibitor were obtained from the PHARMO database. Overall, 13.7% of subjects in the cerivastatin group (n=586) and 9.5% in the reference group (n=1135) discontinued lipid-lowering medication (adjusted odds ratio [OR] 1.44; 95% CI 1.04-2.00). The rate of discontinuation in the reference group was comparable to this rate in the previous year. Discontinuation was more prevalent in women who had been taking cerivastatin (adjusted OR 1.74; 95% CI 1.09-2.78), those receiving low doses of cerivastatin (adjusted OR 2.45; 95% CI 1.20-4.97), those who received their last cerivastatin prescription from a specialist (adjusted OR 1.92; 95% CI 1.02-3.60) and those who had recently started using cerivastatin (adjusted OR 2.80; 95% CI 0.98-7.98), although the latter was not statistically significant.Conclusions: Failure to continue lipid-lowering drug use was higher in patients using cerivastatin than in users of other HMG-CoA reductase inhibitors, especially in women, those using low doses of HMG-CoA reductase inhibitors and recent starters of lipid-lowering medication. The prevention of unwarranted discontinuation of drugs due to market withdrawal should be a joint task of healthcare providers, industry and regulatory bodies. [ABSTRACT FROM AUTHOR]

Assesses time trends in lipid lowering drug use in the Netherlands. Data from pharmacy records of approximately 300,000 people in the Netherlands from 1991-98; Estimated incidence of lipid lowering drug use; Issue of whether lipid lowering medication was targeted to the appropriate patients.

In this article the design of three master programs (MSc in Pharmacy) and two postgraduate specialization programs for community or hospital pharmacist is described. After a preceding BSc in Pharmacy, these programs cover the full pharmacy education capacity for pharmacists in primary and secondary health care in the Netherlands. All programs use the CanMEDS framework, adapted to pharmacy education and specialization, which facilitates the horizontal integration of pharmacists' professional development with other health care professions in the country. Moreover, it is illustrated that crossing the boundary from formal (university) education to experiential (workplace) education is eased by a gradual change in time spent in these two educational environments and by the use of comparable monitoring, feedback, and authentic assessment instruments. A reflection on the curricula, based on the principles of the Integrative Pedagogy Model and the Self-determination Theory, suggests that the alignment of these educational programs facilitates the development of professional expertise and professional identity of Dutch pharmacists. [ABSTRACT FROM AUTHOR]

• Gene and cell therapies are frequently accompanied by large clinical uncertainties and upfront payments, introducing reimbursement challenges. • Managed entry agreements have the potential to mitigate these and create win-win-win situations for health-care payers, health-technology developers and patients. • An innovative approach prospectively prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. • One size does not fit all. Depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. • These insights offer applicability beyond gene and cell therapies, supporting sustainable health-care systems and creating patient access to innovative therapies. Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies. [ABSTRACT FROM AUTHOR]

Background: In 2012, around 400.000 patients in the Netherlands were treated with Vitamin K Antagonists (VKA) for thromboembolic diseases. Since 2011, non-VKA oral anticoagulants (NOACs) are available. NOACs do not require frequent INR monitoring which benefits patients, but also imposes a risk of reduced therapy adherence. The objective of this study is to describe uptake and patient adherence of NOACs in The Netherlands until October 2016. Methods: Prescription data for 247.927 patients across 560 pharmacies were used to describe patient profiles, uptake of NOACs among new naive patients and switch between VKA and NOACs, and calculate therapy adherence as the Proportion of Days Covered (PDC). Results: During the studied period the share of NOACs in oral anticoagulants has grown to 57% of prescriptions to new patients. More than 70% of new NOAC users were new naive patients and around 26% switched from VKA. The overall share of NOACs among starters is largest in the group of patients of 50-80 years. Calculated compliance rate for NOAC patients shows that 88% of all users are adherent with a PDC higher than 80%. Conclusions: NOAC have overtaken VKA as the major treatment prescribed to new oral anticoagulant patients, and the number of starters on VKA is decreasing. Patients are generally adherent to NOACs during the implementation phase, the period that the medication is used. Fear for inadherence by itself does not need to be a reason for not prescribing NOACs instead of VKA. [ABSTRACT FROM AUTHOR]

Reports on a study conducted in Netherlands, which revealed limits in adherence to drug prescribing guidelines to perform clinical chemistry measurements. Strategies for adherence; Ways to optimize benefit-harm balance.

Objectives: Onasemnogene Abeparvovec-xioi (AVXS-101) is a gene therapy intended for curative treatment of spinal muscular atrophy (SMA) with an expected price of around €2 000 000. The goal of this study is to perform a cost-effectiveness analysis of treatment of SMA I patients with AVXS-101 in The Netherlands including relapse scenarios.Methods: An individual-based state-transition model was used to model treatment effect and survival of SMA I patients treated with AVXS-101, nusinersen and best supportive care (BSC). The model included five health states: three health states according to SMA types, one for permanent ventilation and one for death. Deterministic and probabilistic sensitivity analyses were performed. Effects of relapsing to lower health states in the years following treatment was explored.Results: The base-case incremental cost-effectiveness ratio (ICER) for AVXS-101 versus BSC is €138 875/QALY, and €53 447/QALY for AVXS-101 versus nusinersen. If patients relapse within 10 years after treatment with AVXS-101, the ICER can increase up to 6-fold, with effects diminishing thereafter. Only relapses occurring later than 50 years after treatment have a negligible effect on the ICER. To comply with Dutch willingness-to-pay reference values, the price of AVXS-101 must decrease to €680 000.Conclusions: Based on this model, treatment with AVXS-101 is unlikely to be cost-effective under Dutch willingness-to-pay reference values. Uncertainty regarding the long-term curative properties of AVXS-101 can result in multiplication of the ICER. Decision-makers are advised to appropriately balance these uncertainties against the price they are willing to pay now. [ABSTRACT FROM AUTHOR]