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1.  Orphan drugs expenditure in the Netherlands in the period 2006–2012 
Background
The relatively low budget impact of orphan drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of orphan drugs can still be substantial. In this study, we assess the uptake and budget impact of orphan drugs in the Netherlands.
Methods
We examined the number of orphan drugs, the number of patients and budget impact of orphan drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient orphan drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending.
Results
The number of orphan drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available drugs had an individual budget impact of more than €10 million per year.
Conclusions
Individual budget impact of orphan drugs is often limited, although exceptions exist. However, in total, the budget impact of orphan drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for orphan drugs in the future.
doi:10.1186/s13023-014-0154-0
PMCID: PMC4195995  PMID: 25304026
Orphan drugs; Uptake; Budget impact; Reimbursement
2.  Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease 
Background
Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease. However, the costs of therapy are very high. In this paper, we assess the cost-effectiveness of enzyme replacement therapy in infantile Pompe disease.
Methods
A patient simulation model was used to compare costs and effects of ERT with costs of effects of supportive therapy (ST). The model was filled with data on survival, quality of life and costs. For both arms of the model, data on survival were obtained from international literature. In addition, survival as observed among 20 classic-infantile Dutch patients, who all received ERT, was used. Quality of life was measured using the EQ-5D and assumed to be the same in both treatment groups. Costs included the costs of ERT (which depend on a child’s weight), infusions, costs of other health care utilization, and informal care. A lifetime time horizon was used, with 6-month time cycles.
Results
Life expectancy was significantly longer in the ERT group than in the ST group. On average, ST receiving patients were modelled not to survive the first half year of life; whereas the life expectancy in the ERT patients was modelled to be almost 14 years. Lifetime incremental QALYs were 6.8. Incremental costs were estimated to be € 7.0 million, which primarily consisted of treatment costs (95%). The incremental costs per QALY were estimated to be € 1.0 million (range sensitivity analyses: € 0.3 million - € 1.3 million). The incremental cost per life year gained was estimated to be € 0.5 million.
Conclusions
The incremental costs per QALY ratio is far above the conventional threshold values. Results from univariate and probabilistic sensitivity analyses showed the robustness of the results.
doi:10.1186/1750-1172-9-75
PMCID: PMC4038090  PMID: 24884717
Pompe disease; Infants; Enzyme replacement therapy; Cost-effectiveness; Quality adjusted lifeyears
3.  How Work Impairments and Reduced Work Ability are Associated with Health Care Use in Workers with Musculoskeletal Disorders, Cardiovascular Disorders or Mental Disorders 
Purpose the aim of this study was to explore how work impairments and work ability are associated with health care use by workers with musculoskeletal disorders (MSD), cardiovascular disorders (CVD), or mental disorders (MD). Methods in this cross-sectional study, subjects with MSD (n = 2,074), CVD (n = 714), and MD (n = 443) were selected among health care workers in 12 Dutch organizations. Using an online questionnaire, data were collected on individual characteristics, health behaviors, work impairments, work ability, and consultation of a general practitioner (GP), physiotherapist, specialist, or psychologist in the past year. Univariate and multivariate logistic regression analyses were performed to explore the associations of work impairments and work ability with health care use. Results lower work ability was associated with a higher likelihood of consulting any health care provider among workers with common disorders (OR 1.05–1.45). Among workers with MSD work impairments increased the likelihood of consulting a GP (OR 1.55), specialist (OR 2.05), and physical therapist (OR 1.98). Among workers with CVD work impairments increased the likelihood of consulting a specialist (OR 1.94) and physical therapist (OR 2.73). Among workers with MD work impairments increased the likelihood of consulting a specialist (OR 1.79) and psychologist (OR 1.82). Conclusion work impairments and reduced work ability were associated with health care use among workers with MSD, CVD, or MD. These findings suggest that addressing work-related problems in workers with common disorders may contribute in reducing health care needs.
doi:10.1007/s10926-013-9492-3
PMCID: PMC4229647  PMID: 24390780
Work ability; Work impairments; Health care utilization; Musculoskeletal disease; Cardiovascular disease; Mental disorders
4.  Systematic review of available evidence on 11 high-priced inpatient orphan drugs 
Background
Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, cost-effectiveness and budget impact for orphan drugs.
Methods
A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient orphan drugs listed on the Dutch policy rule on orphan drugs. For included studies, we determined the type of study and various study characteristics.
Results
A total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the orphan drugs. Eight studies described the cost-effectiveness of an orphan drug; an equal number described an orphan drug’s budget impact.
Conclusions
Despite the often heard claim that RCTs are not feasible for orphan drugs, we found that an RCT was available in 60% of orphan drugs investigated. Cost-effectiveness and budget impact analyses for orphan drugs are seldom published.
doi:10.1186/1750-1172-8-124
PMCID: PMC3751719  PMID: 23947946
Rare diseases; Orphan drugs; Evidence based medicine; Clinical effectiveness; Cost-effectiveness; Budget impact
5.  Burden of illness of Pompe disease in patients only receiving supportive care 
Background
Pompe disease is an orphan disease for which enzyme replacement therapy (ERT) recently became available. This study aims to estimate all relevant aspects of burden of illness—societal costs, use of home care and informal care, productivity losses, and losses in health-related quality of life (HRQoL)—for adult Pompe patients only receiving supportive care.
Methods
We collected data on all relevant aspects of burden of illness via a questionnaire. We applied a societal perspective in calculating costs. The EQ-5D was used to estimate HRQoL.
Results
Eighty adult patients (87% of the total Dutch adult Pompe population) completed a questionnaire. Disease severity ranged from mild to severe. Total annual costs were estimated at €22,475 (range €0–169,539) per adult Pompe patient. Patients on average received 8 h of home care and 19 h of informal care per week. Eighty-five percent of the patients received informal care from one or more caregivers; 40% had stopped working due to their disease; another 20% had reduced their working hours. HRQoL for Pompe patients who only received supportive care was estimated at 0.72, 17% lower than the Dutch population at large.
Conclusions
Adult Pompe disease is associated with a considerable burden of illness at both the societal and patient levels. The disease leads to substantial costs and dependency on medical devices, home care, and informal care, and has a high impact on the patient’s social network. In addition, patients are limited in their ability to work and have significantly reduced HRQoL.
doi:10.1007/s10545-011-9320-x
PMCID: PMC3173621  PMID: 21499718
6.  Cost-consequence analysis of remifentanil-based analgo-sedation vs. conventional analgesia and sedation for patients on mechanical ventilation in the Netherlands 
Critical Care  2010;14(6):R195.
Introduction
Hospitals are increasingly forced to consider the economics of technology use. We estimated the incremental cost-consequences of remifentanil-based analgo-sedation (RS) vs. conventional analgesia and sedation (CS) in patients requiring mechanical ventilation (MV) in the intensive care unit (ICU), using a modelling approach.
Methods
A Markov model was developed to describe patient flow in the ICU. The hourly probabilities to move from one state to another were derived from UltiSAFE, a Dutch clinical study involving ICU patients with an expected MV-time of two to three days requiring analgesia and sedation. Study medication was either: CS (morphine or fentanyl combined with propofol, midazolam or lorazepam) or: RS (remifentanil, combined with propofol when required). Study drug costs were derived from the trial, whereas all other ICU costs were estimated separately in a Dutch micro-costing study. All costs were measured from the hospital perspective (price level of 2006). Patients were followed in the model for 28 days. We also studied the sub-population where weaning had started within 72 hours.
Results
The average total 28-day costs were €15,626 with RS versus €17,100 with CS, meaning a difference in costs of €1474 (95% CI -2163, 5110). The average length-of-stay (LOS) in the ICU was 7.6 days in the RS group versus 8.5 days in the CS group (difference 1.0, 95% CI -0.7, 2.6), while the average MV time was 5.0 days for RS versus 6.0 days for CS. Similar differences were found in the subgroup analysis.
Conclusions
Compared to CS, RS significantly decreases the overall costs in the ICU.
Trial Registration
Clinicaltrials.gov NCT00158873.
doi:10.1186/cc9313
PMCID: PMC3219979  PMID: 21040558

Results 1-6 (6)