Reimbursement Criteria Research Articles

Extracorporeal membrane oxygenation use, expenditure, and outcomes in taiwan from 2000 to 2010.

BackgroundNo study to date has systematically examined use, expenditure, and outcomes associated with extracorporeal membrane oxygenation (ECMO) use in Taiwan. The aim of this study was to examine ECMO use, expenditure, and outcomes during an 11-year period in Taiwan.MethodsClaims data were collected from the Taiwan National Health Insurance Research Database for patients who received ≥1 ECMO treatment between January 2000 and December 2010. Measurements included demographics, indications for ECMO use, length of hospital stay, outcome, and expenditure.ResultsA total of 3969 patients received ECMO during the study period (median age: 54.6 years). The number of patients receiving ECMO increased from 52 in 2000 to 1045 in 2010. The major indication for ECMO was cardiovascular disease (68.7%), followed by respiratory disease (17.9%). Median length of hospital stay was 13 days in 2000 and 17 days in 2010. Median expenditure (New Taiwan dollars) was $604 317 in 2000 and $673 888 in 2010. Some variables significantly differed by age, sex, hospital setting, calendar year, and indication for ECMO, and were associated with in-hospital and after-discharge mortality.ConclusionsECMO use has increased dramatically in Taiwan over the last decade. The high mortality rate of ECMO users suggested that ECMO may be being used in Taiwan for situations in which it provides no added benefit. This situation may be a reflection of the current reimbursement criteria for National Health Insurance in Taiwan. Refinement of the indications for use of ECMO is suggested.

Mixed-Methods Survey of Professional Perspectives of Music Therapy Practice in Mental Health

The field of music therapy in the United States is undergoing a transformation that has been fueled by changes in healthcare, insurance reimbursement criteria, and requirements of evidence-based research in clinical practice. In addition, requirements within the profession as a whole are changing the landscape of education and specialization. One response to these changes is evident in the training of music therapists (MTs), as the American Music Therapy Association (AMTA) and its members consider the relative advantages of creating a Master's-Level Entry (MLE) into the profession. Healthcare delivery in the field of mental health, in particular, has been transformed, as advances made in the pharmaceutical treatment of psychiatric disorders has shifted care from the acute inpatient setting to the recovery community model. This change in treatment approach has resulted in increasingly shorter length of inpatient stays. An average length of stay of a 1987 inpatient cohort was 22.6 days (Weiden & Oltson, 1995, table 1 ), but it had fallen to 8 days in 2005 (Stranges, Levit, Stocks, & Santora, 2011) and is estimated at 7.2 days in 2015 (Centers for Disease Control and Prevention, 2015).This new treatment model is reflected in the recent music therapy literature, where short-term acute stays have resulted in a need for practices that are focused on single sessions (Carr, Odell-Miller, & Priebe, 2013; Jones, 2005; Silverman, 2014'), and address immediate areas of concern (e.g., de l'Etoile, 2002; Silverman & Rosenow, 2013,) rather than provide process or depth-oriented therapeutic experiences with a goal of insight as the outcome (Carr et al., 2013). Carr et al/s (2013) systematic review of music therapy practices in psychiatric acute care in the United States and Europe provides insight into some of the ways that music therapists have adapted to these changes. These authors found that MTs conducted a high number of sessions per week, and that the music experiences they implemented required greater therapist direction, flexibility, and predictable musical structure. This review also indicated that music therapists often found it difficult to engage patients in music therapy, especially early in their care. Because of the constant changes in group participants and functioning levels, fostering group process was often not a realistic goal. Patients in the acute unit are often in crisis; thus, goals for inpatients were focused primarily on symptom management and fostering relationships with peers and healthcare professionals in the here and now (2013). Carr et al. (2013) concluded that the current music therapy models of Guided Imagery and Music (GIM), Analytical Music Therapy (AMT), and Nordoff Robbins Music Therapy (NR) have been developed with depth-oriented care in mind and may not address the needs of acute-care patients.This interpretation may be supported by recent music therapy research literature in the acute-care setting, wherein the focus has been on single sessions that use interventions such as music games (Silverman, 2005, 2014), music games and other recreational activities (Silverman & Rosenow, 2013), music psychoeducation (Silverman, 2009, 2014), and songwriting (Jones, 2005; Silverman, 2012b; Silverman & Leonard, 2012). In contrast, in the recovery or community treatment setting, MTs use music experiences in a different way, or use other music experiences that may be more suitable for longterm treatment. This includes choirs (Bailey & Davidson, 2003; Eyre, 2011), songwriting (Vander Kooij, 2009), songwriting and recording (Grocke et al., 2014), Sound Training for Attention and Memory protocol (Ceccato, Caneva, & Lamonaca, 2006), and adapted GIM (Moe, Roesen, & Raben, 2000). Although some studies have been carried out in the community setting (e.g., Grocke, Bloch, & Castle, 2009), there is a relative lack of research and clinical literature that focuses specifically on music therapy in the community setting. …

Budget impact analysis of apixaban versus other NOACs for the prevention of stroke in Italian atrial fibrillation patients

OBJECTIVE: This study aims to perform a budget impact analysis of the use of three available novel oral anticoagulant agents (NOACs) for preventing thromboembolic events in Italian patients with non‑valvular atrial fibrillation (NVAF).METHODS: Estimated Italian population of patients was run through a previously published lifetime decision tree/Markov model simulating their treatment with the available therapeutic options: dabigatran at two dose levels (110 mg/bid for the over 80 years old, 150 mg/bid for younger NVAF patients), rivaroxaban (20 mg/uid), and apixaban (5 mg/bid). Effectiveness and safety estimates derive from an adjusted indirect treatment comparison using warfarin as link. The main clinical events considered in the model are ischemic and hemorrhagic stroke, systemic thromboembolism, bleeds (both major and clinically relevant minor) and cardiovascular hospitalizations, besides treatment discontinuations. Epidemiological data and unit costs, actualized to 2013, are collected from Italian published sources. The budget impact analysis evaluates the financial impact of apixaban introduction by comparing expected 1,2, and 3 years costs in hypothetical scenarios: with and without apixaban. Italian NVAF patient population estimation is based on official apixaban reimbursement criteria, applying the characteristics of the trial population to national epidemiologic data. Numbers of patients for each regimen are estimated by projecting share evolution. Sensitivity analysis is performed on an alternative non‑experimental population of NVAF patients.RESULTS: Among available NOACs, apixaban was expected to be the least expensive in an estimated patient population of 364,000 Italian patients, allowing for savings of € 1,180,549, € 3,841,429 and € 5,368,918 at 1,2, and 3 years, respectively. Results of the simulation run on an alternative non‑experimental population of NVAF patients yields comparable estimates.CONCLUSIONS: The different safety and effectiveness profiles of the three available NOACs emerging from the adjusted indirect comparison indicate that apixaban could improve health care expenditure control while maintaining or increasing therapeutic appropriateness in the Italian NVAF population.

Persistence and adherence with dementia pharmacotherapy: relevance of patient, provider, and system factors.

This paper provides a comprehensive review of studies examining adherence and (or) persistence with dementia pharmacotherapy during the past decade, including a summary of the key patient-, drug-, system-, and provider-level factors associated with these measures. Estimates of adherence and 1-year persistence to these drugs have ranged from 34% to 94% and 35% to 60%, respectively. Though many studies reported nonsignificant associations, there are data suggesting that patient age, sex, ethnoracial background, socioeconomic status, and region-specific reimbursement criteria, as well as the extent and quality of interactions among patients, caregivers, and providers, may influence persistence with pharmacotherapy. As many studies relied on administrative data, limited information was available regarding the relevance of patient's cognitive and functional status or the importance of caregiver involvement or assistive devices to adherence or persistence.

Anaemia management in patients with chronic kidney disease: Taiwan practice guidelines.

The introduction of erythropoiesis-stimulating agents (ESAs) markedly improved the lives of many anaemic patients with chronic kidney disease (CKD). In Taiwan, the strategy of management of anaemia in patients with CKD was different from many other parts of the world. In 1996, the National Health Insurance Administration of Taiwan applied a more restrictive reimbursement criteria for ESA use in patients with CKD. ESA is to be initiated when non-dialysis CKD patients have a serum creatinine >6 mg/dL and a hematocrit <28% to maintain a hematocrit level not exceeding 30%. The maximal dose of epoetin-α or β was 20 000 U per month. The target haemoglobin range and dose limitation for ESAs were the same for dialysis CKD patients. Thus, long before randomized controlled trials showing an increased risk for cardiovascular events at nearly normal haemoglobin concentrations and higher ESA doses in CKD, nephrologists in Taiwan had avoided the use of disproportionately high dosages of ESAs to achieve a haemoglobin level of 10-11 g/dL. Moreover, intravenous iron supplementation was encouraged earlier in Taiwan in 1996, when we reached consensus on the diagnostic criteria for iron deficiency (serum ferritin <300 ng/mL and/or transferrin saturation <30%). The experience of CKD anaemia management in Taiwan demonstrated that a reasonable haemoglobin target can be achieved by using the lowest possible ESA dose and intravenous iron supplementation.

Valuing health at the end of life: A stated preference discrete choice experiment

A source of debate in the field of health care priority setting is whether health gains should be weighted differently for different groups of patients. The debate has recently focused on the relative value of life extensions for patients with short life expectancy. However, few studies have examined empirically whether society is prepared to fund life-extending end-of-life treatments that would not meet the reimbursement criteria used for other treatments.A web-based discrete choice experiment was conducted in 2012 using a sample of 3969 members of the general public in England and Wales. The study design was informed by the National Institute for Health and Care Excellence's supplementary policy for the appraisal of life-extending end-of-life treatments. The choice tasks involved asking respondents which of two hypothetical patients they would prefer to treat, assuming that the health service has enough funds to treat only one of them. Conditional logit regressions were used for modelling.Choices about which patient to treat were influenced more by the sizes of treatment gains than by patients' life expectancy without treatment. Some respondents appear to support a health-maximisation type objective throughout, whilst a small minority always seek to treat those who are worse off without treatment. The majority of respondents, however, seem to advocate a mixture of the two approaches. Overall, we find little evidence that members of the general public prefer to give higher priority to life-extending end-of-life treatments than to other types of treatment. When asked to make decisions about the treatment of hypothetical patients with relatively short life expectancies, most people's choices are driven by the size of the health gains offered by treatment.

CV3 - Economic Evaluation of Change in Reimbursement Criteria for Lipid-Lowering Drugs in Taiwan

As the international guidance for lipid control has evolved, the National Health Insurance Administration (NHIA) in Taiwan was requested to modify the current reimbursement criteria for initiation of lipid-lowering treatment. The study aims to evaluate the cost-effectiveness and the budget impact of the newly proposed reimbursement criteria on lipid-lowering drug from the NHIA’s viewpoint with a focus on the change of statins usage. In the new criteria, the cut-off points of low-density lipoprotein cholesterol (LDL-C) and total cholesterol for initiating lipid-lowering therapy for patients having diabetes or previous heart diseases (high risk group) have been lowered. A Markov decision model was constructed to examine the cost-effectiveness of the new criteria compared with the original ones. The efficacy of statins on coronary heart diseases (CHD) and stroke was obtained from the literature. The numbers of subjects who may be affected by these criteria changes and the LDL-C specific disease transition probabilities were obtained from a population-based survey conducted by the Taiwan Health Promotion Administration (HPA). Medical costs were derived from the NHI data. Both costs and health outcomes were discounted at 3%. The corresponding financial impact on NHI expenditure was also estimated. If the new criteria are applied, the number of subjects who fulfill the reimbursement criteria in the high risk group will be almost twice as many as before. This would lead to an great increase in the NHI expenditures for statins and monitoring costs, however, this could be offset by the treatment cost saved from the averted CHD and stroke cases. Therefore, the new criteria are a dominant strategy compared with the original criteria. Although the newly proposed reimbursement criteria would lead to an increase in drug expenditure of NHIA, it is expected to be a cost-saving strategy after taking into account the health benefit on preventing CHD and stroke.

Impact Laws And Decrees On Activities: The Ilda Study.

To assess whether the prescribing pattern of statins changed after reimbursement criteria revision and regional policies in a general practice in southern Italy. Analysis has been performed on a database of 123 medical practitioners that have managed an average of 190.000 inhabitants in the Campania Region (south of Italy). Prevalence of use and incidence of new treatments were calculated from Jan 2012 to Jun 2013. Statin users were stratified into three groups (Moderate Cardiovascular Risk, MR; High Cardiovascular Risk, HR; Very High Cardiovascular Risk, VHR) according to new criteria for reimbursement for lipid lowering agents revised by the Italian Drug Agency (AIFA) (Nota 13). After the reimbursement criteria revision (November 2012), the prevalence of statin use slightly decreased reaching 6.6% in the second quarter of 2013 (-14% compared to second quarter of 2012). Stratified by level of CV risk, the prevalence of statin use is reduced by 24.9% into MR, 13.1% into HR and 5.9% into VHR, while incidence of new users of 22.4%, 34 5% and 45.8% respectively. In the second quarter of 2013, atorvastatin (+45.3%) was prescribed in 57,5% of patients in MR group (+45.3%), rosuvastatin 5.9% with (-60.1%) and 1.6% with simvastatin + ezetimide (-57.5%); in HR group, 40.7% (+20.8) atorvastatin, 5.3% (-57.0%) rosuvastatin and 2.1% (- 42.3%) simvastatin + ezetimide; in VHR, 56.3% (+15.8) atorvastatin, 12.5% (-37.5%) rosuvastatin and 2.1% (-27.1%) simvastatin + ezetimide. The revision of reimbursement criteria and the regional policies led to significant changes in general practice in southern Italy resulting in a reduction in the statin use, especially in patients who could potentially benefit from it most. The results of the study provide useful information for the general practitioner about areas for improvement prescriptive.

Plerixafor prescription modalities in autologous haematopoietic stem cell mobilization in Belgium

Objectives:The efficacy and safety of plerixafor, an antagonist of the CXCR4 receptor, in combination with G-CSF has been demonstrated in patients suffering from Iymphoma and multiple myeloma (MM) eligible for autologous haematopoietic stem cell collection. However, different reimbursement criteria have been applied in different countries to select patients eligible for treatment with plerixafor. The objective of this observational study was to describe the plerixafor prescription modalities in daily practice in Belgium.Methods:This open-label, prospective, observational study was conducted in 11 Belgian centres in 114 patients with lymphoma (Hodgkin’s and non-Hodgkin’s lymphoma) or MM who were treated with plerixafor according to the SmPC between April 2011 and October 2012. Patients included in another clinical trial with plerixafor were excluded from the study.Results:The use of plerixafor in patients with MM or lymphoma was effective, with a success rate (defined as a total yield >2×106 CD34+ cells/kg) of 77%, and well tolerated (one SAE reported). Optimal collection (defined as a total yield >4×106 CD34+ cells/kg) was obtained for 43% of the study population (31% in lymphoma patients, compared to 61% in patients with MM). The use of plerixafor was in line with the SmPC and the Belgian reimbursement criteria for all patients.Conclusion:This study is showing that the use of plerixafor according to Belgian reimbursement criteria results in similar efficacy and safety as in other centres and countries worldwide.

OP0159 Microscopic Gut Inflammation in SPA is A Prognostic Factor for Initiation of Anti-TNF Therapy in Daily Practice

BackgroundMicroscopic gut inflammation frequently occurs in patients with spondyloarthritis (SpA), and is associated with a risk of evolution to Crohn's disease and ankylosing spondylitis (AS). Half of all SpA patients...

The Changing Landscape of Adult Home Noninvasive Ventilation Technology, Use, and Reimbursement in the United States

There has been an exponential increase in the use of home noninvasive ventilation (NIV). Despite growing use, there is a paucity of evidence-based guidelines and practice standards in the United States to assist clinicians in the initiation and ongoing management of home NIV. Consequently, home NIV practices are being influenced by complicated local reimbursement policies and coding. This article aims to provide a practice management perspective for clinicians providing home NIV, including Local Coverage Determination reimbursement criteria for respiratory assist devices, Durable Medical Equipment coding, and Current Procedural Terminology coding to optimize clinical care and minimize lost revenue. It highlights the need for further research and development of evidence-based clinical practice standards to ensure best practice policies are in place for this rapidly evolving patient population.

Relapse Of Severe Asthma Exacerbations After Cessation Of Omalizumab Treatment – Real Life Data

Many clinical and observational studies demonstrated effectiveness of omalizumab (OMA) in the treatment of severe asthma, but the optimal duration of the therapy remains unknown. Here we present our clinical experience on OMA cessation in routine practice. Due to new reimbursement criteria OMA therapy was interrupted in 11 subjects (6 women/5 men) this year. The mean patients’ age is 50.7±14.2 yrs, the mean severe asthma duration is 13.5±6.1 yrs. The mean OCS dose (prednisone equivalent) before OMA therapy was 23±12.2 mg/day. Six subjects have near-fatal asthma exacerbations recorded in their medical history. Patients received OMA according to the manufacturer recommendation. All had good response to OMA and asthma deterioration when doses were missed. The mean time of OMA therapy was 67.7±11.6 months. Ten patients had severe asthma exacerbation within first 5 months after OMA withdrawal. The mean time to first exacerbation was 7.4±2.6 weeks. Since OMA cessation to the time of reassessment the mean ACQ increased from 2.4±0.8 to 3.6±1.2 points, AQLQ decreased from 4.5±1 to 3.1±1.1 points, OCS dose increased from 4.15±1.2 mg/day to 33.3±13.1 mg/day, the number of exacerbations increased form 1.5±0.8 to 4.8±1.8/year/patient and the number of hospitalizations or ICU admissions increased from 0.09±0.3 to 1.5±1.2/year/patient. In nine patients OMA treatment has already been reintroduced. These data indicate, that withdrawal of OMA therapy, after successful long-term therapy, may cause severe asthma exacerbations in a certain group of patients therefore each decision regarding cessation of OMA treatment should be undertaken individually after careful weighing of benefits and risks.

Use of cognitive enhancers and associated medical care costs among patients with dementia: a nationwide study in Taiwan

Pharmaceutical therapy for patients with dementia including cholinesterase inhibitors (ChEI) and memantine is covered by Taiwan's National Health Insurance (NHI) but with strict reimbursement criteria. This study compared utilization of selected cognitive enhancers among elderly patients with dementia and estimated associated differences in medical care costs. This study used medical claims and pharmacy claims from the NHI Research Database of Taiwan from 2009 to 2011, which included all patients 65 years or older diagnosed with dementia in their outpatient or inpatient claims. Both individual-level and market-level analysis were performed to calculate the average medical costs per person and the share of drug expenditures. Generalized linear models with propensity score adjustment estimated differences in medical care costs by use of selected cognitive enhancers. Users of ChEI had the highest medication and outpatient costs but the lowest inpatient costs among all users of cognitive enhancers. However, annual adjusted total medical care costs per ChEI user were not significantly different from those who used cerebral vasodilators (CBV). In 2011, 52.4% of the elderly with dementia in Taiwan used cognitive enhancers, but among them 88.3% used CBV while 9.2% used ChEI. Among patients with dementia who used at least one cognitive enhancer, the aggregated expenditure as a share of their total drug expenditures was 9.7% in 2011. Given that CBV had a much higher utilization rate than ChEI or memantine among elderly people with dementia, the strict reimbursement policy for ChEI and memantine may need to be revisited to increase access to those drugs by patients with dementia in Taiwan.

The impact of regional co-payment and national reimbursement criteria on statins use in Italy: an interrupted time-series analysis.

BackgroundStatins are among the most commonly prescribed drugs worldwide in the prevention of cardiovascular diseases and their effectiveness is largely acknowledged. The consumption of statins increased four-fold during the 2000–2010 decade in Italy and national and regional control policies were developed. Restrictions to reimbursement were fixed at the national level, whereas co-payment was introduced in some, but not all, regions. The aim of the present study is to assess the impact of such policies on the consumption of statins in Italy between 2001–2007 among outpatients.MethodsThe statin use was measured in terms of defined daily doses per 1,000 inhabitants per day (DDD/1000 inh. day) from May 2001 to December 2007. The study was conducted in 17 out of 21 regions, nine of which had implemented a co-payment policy. Time trends in consumption before and after the introduction of co-payment policies and reimbursement criteria were examined using segmented regression analysis of interrupted time-series, adjusting for seasonal components.ResultsThe consumption of statins increased by 22.9 DDD/1000 inh. day in May 2001 to 54.7 DDD/1000 inh. day in December 2007. On average, there was a 1.7% increase in statin use each month before the national guideline changed while the increase was about 0.5% afterwards. The revision of the reimbursement criteria was associated with a significant decrease in level (coefficient = −2.80, 95% CI −3.70 to −1.90 p-value <0.001) and trend (coefficient = −0.33, 95% CI −0.37 to −0.29 p-value <0.001). The introduction of co-payment was associated with a significant change in trend of consumption so that the overall use of the drug increased by 0.04 (95% CI 0.02 to 0.07, p-value < 0.001) DDD/1000 inh. day per month in the post-intervention period, but there was no evidence of a change in level of consumption (p-value = 0.163).ConclusionsConsumption of statins in Italy increased almost three-fold during the study period. The restriction to reimbursement Interventions was associated with an immediate drop and a decrease in trend of statin use, while the regional copayment was associated with a small increase in trend of statin use.

Loss of asthma control after cessation of omalizumab treatment: real life data

IntroductionMany clinical and observational studies have demonstrated effectiveness of omalizumab (OMA) in the treatment of severe asthma, but the optimal duration of the therapy remains unknown.AimThe article presents the authors’ clinical experience on OMA cessation in routine practice.Material and methodsDue to new reimbursement criteria, OMA therapy has been interrupted in 11 subjects (6 women/5 men). The mean age of patients was 50.73 ±14.16 years, the mean time of severe asthma duration was 13.54 ±6.05 years. All of them had an excellent/good response to OMA. The duration of OMA therapy was 67.73 ±11.64 months.ResultsNine out of 11 patients had severe asthma exacerbation within the first 5 months after the OMA withdrawal. The mean time to the first severe exacerbation was 7.56 ±2.67 weeks. Between the time of OMA cessation and the time of reassessment, the mean score of Asthma Control Questionnaire increased from 2.58 ±0.71 to 3.63 ±1.26 points and the mean score of Asthma Quality of Life Questionnaire decreased from 4.3 ±1.91 to 3.18 ±1.17 points. The mean oral corticosteroids (OCS) dose increased from 4.61 ±3.0 mg/day to 33.33 ±13.12 mg/day. The number of exacerbations within the last 12 months increased from 1.6 ±0.67 to 5.2 ±1.4, and the number of hospitalizations or emergency room (ER) attendence increased from 0.11 ±0.31 to 1.56 ±1.26.ConclusionsThese data indicate that the withdrawal of OMA therapy after the successful long-term therapy may cause severe asthma exacerbations. Therefore, the decision regarding cessation of OMA treatment should be undertaken individually after careful weighing benefits and risks, especially in patients with a long history of severe asthma, treated with high doses of OCS before OMA introduction, near-fatal asthma events and/or aggravation of asthma during previous episodes of interruptions in OMA treatment.

PP179—Identification of Drug–Drug Interactions Through a Digital Health Service

e74 Volume 35 Number 8S accounting for 21.2% and 16.2% of total AED consumption (DDD 163.7 and 125.2, respectively). In the same year, oxcarbazepine and lamotrigine were the most used new AEDs (10.91% and 10.79% of total; DDD 84.1 and 83.2, respectively), while gabapentin and pregabalin exhibited the higher incidence of use. The main indication of use was epileptic disorders for older AEDs and neuropathic pain for newer AEDs. A high number of patients treated with older AEDs, in particular carbamazepine, phenobarbital, and valproic acid, received coprescription at clinically relevant interaction risk. Among newer AEDs, lamotrigine showed a high annual rate of possible interaction. Conclusion: Significant differences were shown in the prescribing pattern of newer and older medications: older AEDs were mainly used in the treatment of epileptic disorders, while newer compounds were also preferred for conditions other than epilepsy, in particular neuropathic pain. The fall in the use of newer AEDs during 2007 agreed with revised reimbursement criteria for gabapentin and pregabalin. The coprescription should be evaluated with caution and avoided if possible. Drugs at risk of interactions should be replaced with others having same indication of use. Disclosure of Interest: None declared.

PP178—Antiepileptic Drugs: Prescribing Patterns and Interaction Risk in General Practice

PP178—Antiepileptic Drugs: Prescribing Patterns and Interaction Risk in General Practice

Treatment persistence and changes in fracture risk, back pain, and quality of life amongst patients treated with teriparatide in routine clinical care in France: Results from the European Forsteo Observational Study

ObjectivesThe European Forsteo Observational Study assessed the clinical fracture incidence, back pain, quality of life (QoL), and treatment persistence amongst post-menopausal women, who were prescribed teriparatide in routine care in eight European countries. We present the results for France, with health-insurance reimbursement criteria channel teriparatide to women with severe disease and limit treatment to 18months. MethodsA representative sample of women initiating teriparatide in France was followed in routine care for 36months. We described patients’ characteristics at baseline and persistence to teriparatide (Kaplan–Meier analysis), fracture incidence, back pain, and QoL (EQ-5D) at baseline, 18 and 36months follow-up (last-observation-carried-forward (LOCF) and mixed-models-for-repeated-measures (MMRM). ResultsOne hundred and sixteen rheumatologists included 309 patients, of whom 290 (93.9%) had at least one follow-up visit. Women's mean age (standard deviation) was 74.5years (7.4) and 296 (95.8%) had greater or equal to two vertebral fractures prior to teriparatide initiation. Clinical fracture incidence, mainly vertebral fractures, decreased around 6months after teriparatide initiation, and was sustained at 36months (P=0.013) when most patients were treated by anti-resorptives. Back pain and EQ-5D measures improved significantly at 18 and 36months (P<0.0001) in the LOCF analyses but did not improve in the EQ-5D VAS measure after covariate adjustment in the MMRM model. Median treatment duration was 17.4months. ConclusionFrench women initiating teriparatide in routine care had severe osteoporosis and showed good treatment persistence, consistent with France's insurance reimbursement criteria. Improvements in fracture risk and back pain began soon after treatment and was maintained at 36months follow-up.

Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease

Glycogen storage disease type II is a rare multi-systemic disorder characterised by an intracellular accumulation of glycogen due a mutation in the acid alpha glucosidase (GAA) gene. The level of residual enzyme activity, the genotype and other yet unknown factors account for the broad variation of the clinical phenotype. The classical infantile form is characterised by severe muscle hypotonia and cardiomyopathy leading to early death. The late-onset form presents as a limb girdle myopathy with or without pulmonary dysfunction. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA) in infants is life saving. In contrast, therapeutic efficacy of rhGAA in the late-onset form is modest. High expenses of rhGAA, on-going infusions and poor pharmacokinetic efficacy raised a discussion of the cost effectiveness of ERT in late-onset Pompe disease in Switzerland. This discussion was triggered by a Swiss federal court ruling which confirmed the reluctance of a health care insurer not to reimburse treatment costs in a 67-year-old female suffering from Pompe disease. As a consequence of this judgement ERT was stopped by all insurance companies in late-onset Pompe patients in Switzerland regardless of their clinical condition. Subsequent negotiations lead to the release of a national guideline of the management of late-onset Pompe disease. Initiation and limitation of ERT is outlined in a national Pompe registry. Reimbursement criteria are defined and individual efficacy of ERT with rhGAA is continuously monitored.

OP0011 Inequalities across 46 european countries in clinical eligibility criteria for the start of a first (reimbursed) biologic in patients with rheumatoid arthritis

BackgroundIn the treatment of patients with RA, strategies that include biologics have resulted in a better outcome for patients with regard to disease activity, need for surgery and work participation....