Costs, effects, and savings of screening for cystic fibrosis gene carriers.

STUDY OBJECTIVE: Evaluating the costs, effects, and savings of several strategies for cystic fibrosis (CF) gene carrier screening. DESIGN: A general model for evaluating prenatal, preconceptional, school, and neonatal carrier screening was constructed. For prenatal and preconceptional screening, two strategies were evaluated: single entry and double entry two step couple screening. Firstly, the Dutch situation was evaluated prospectively; subsequently the results were generalised to other carrier frequencies. SETTING: Prospective simulation model. MAIN RESULTS: Of all screening strategies, neonatal carrier screening gives most carrier couples an informed choice concerning reproduction. If the parents of carrier newborns would not be tested however, prenatal screening detects most carrier couples. Prenatal and single entry preconceptional screening programmes have a favourable cost-savings balance in the Netherlands under a wide range of assumptions. For double entry preconceptional screening and neonatal screening, high enough values of uptake of screening, prenatal diagnosis, and induced abortion are necessary. School carrier screening does not have a favourable cost-savings balance. CONCLUSIONS: If a CF screening programme is judged to be useful on individual and social grounds, costs considerations are no obstacle for prenatal and single entry preconceptional screening.

[Preconception screening of parents and carrier state of cystic fibrosis in the Netherlands: expenses and savings]. / Preconceptionele screening van paren op dragerschap van cystische fibrose in Nederland: kosten en besparingen.

OBJECTIVE: To evaluate effects, costs and savings for a preconceptional couple screening programme for cystic fibrosis (CF) carriers. SETTING: State University Groningen, the Netherlands. DESIGN: Prospective theoretical evaluation. METHOD: A decision tree and an arithmetic model were constructed for two different strategies of preconceptional CF screening of couples: 'single entry two step' (SETS; start by testing one partner), and 'double entry two step' (DETS; test both partners). The difference between costs of screening and costs of CF illness prevented by screening was determined. RESULTS: DETS couple screening with the assumptions used for e.g. sensitivity and use of options can detect 81.5% of carrier couples in the Netherlands (against 70% for SETS), but results in twice as many positive/negative couples as SETS couple screening. The maximum number of carrier couples identified when screening 100,000 couples would be 88, resulting in a decrease of the number of children with CF of 25 each year. The costs of screening equal the savings if approximately 8,000 couples are screened yearly in the Netherlands. CONCLUSIONS: There are no financial objections to preconceptional couple screening in the Netherlands, even with an uptake ratio of around 10%. Whether screening for CF carriers should be offered or not should be decided on the basis of non-financial arguments.

The nonhospital costs of care of patients with CF in The Netherlands: results of a questionnaire.

Cystic fibrosis (CF) causes a relatively high medical consumption. A large part of the treatment takes place at home. Because data regarding nonhospital care are lacking, we wished to determine the costs of care of patients with CF outside the hospital. A questionnaire was sent to 73 patients with CF from two Dutch hospitals (response rate 64%, 14 children and 33 adults). Average consumption and average costs per patient per year were calculated for children and adults for six categories: nonhospital medical care; domestic help; diet; travelling because of CF; medication; and devices and special facilities at home, work or school. The average nonhospital costs of care amounted to Pounds 4,641 per child per year (range Pounds 712-13,269) and Pounds 10,242 per adult (range Pounds 1,653-26,571). Nonhospital medical care for children and adults accounted for, respectively, 8 and 5% of these costs, domestic help for 15 and 9%, diet for 10 and 7%, travelling because of CF for 4 and 8%, medication for 63 and 67%, and devices and special facilities at home, work or school for 1 and 4%. Nonhospital costs of care of cystic fibrosis are very high and amount to 50% of the total (medical and nonmedical) lifetime costs of cystic fibrosis.

Cost of care of patients with cystic fibrosis in The Netherlands in 1990-1.

BACKGROUND: Research on the cost of care of patients with cystic fibrosis is scarce. The aim of this study was to estimate the costs using age-specific medical consumption from real patient data. METHODS: The age-specific medical consumption of patients with cystic fibrosis in The Netherlands in 1991 was estimated from a survey of medical records and a patient questionnaire. A distinction was made between costs of hospital care, hospital and non-hospital medication, and home care. Costs per year were obtained by multiplying the yearly amount of care and the costs per unit. RESULTS: On average the annual cost of a patient with cystic fibrosis in 1991 was 10,908 pounds (hospital care 42%, medication 37%, home care 20%). The cost of care of cystic fibrosis in The Netherlands, with approximately 1000 patients, is estimated at 10.9 million pounds per year, which is 0.07% of the total health care budget. The cost of care of a patient up to the age of 35 is estimated at 614,587 pounds. When year-to-year survival is taken into account and future costs are discounted to the year of birth with a yearly discount rate of 5%, the cost of care of a patient with cystic fibrosis is estimated at 164,365 pounds for 1991. This estimate will be used in a prospective evaluation of screening for cystic fibrosis carriers. CONCLUSIONS: The cost of care of patients with cystic fibrosis estimated by age-specific medical consumption of real patients is higher than that estimated by non-age-specific medical consumption and/or expert opinions.

Comparison of single-entry and double-entry two-step couple screening for cystic fibrosis carriers.

Both single-entry two-step (SETS) couple screening and double-entry two-step (DETS) couple screening have been recommended as methods to screen for cystic fibrosis gene carriers. In this paper we compare the expected results from both types of screening. In general, DETS results in a higher detection rate of couples in which both partners are carriers, but also in a higher proportion of couples with only one identified carrier who have a significant remaining risk in their offspring.

[Continuous intravenous home treatment of airway infections using ceftazidime administration via portable pump in patients with cystic fibrosis; a multicenter study]. / Continue intraveneuze thuisbehandeling van luchtweginfecties met ceftazidim via een draagbare pomp bij patiënten met cystische fibrose; een multicentrisch onderzoek.

OBJECTIVE: To study the feasibility and applicability of Home intravenous antibiotic treatment (HIVAT) in cystic fibrosis (CF) patients in the Netherlands. DESIGN: Prospective, multicentre study in CF patients with an exacerbation of a Pseudomonas aeruginosa pulmonary infection using a computerised, ambulatory pump for continuous infusion. METHOD: The effects of HIVAT were studied during one year in 24 CF patients (9 male, 15 female; mean age 23.3 years, range 7-52), who received a total of 39 courses of continuous ceftazidime 100 mg/kg/24 hrs i.v. for 3 weeks delivered by a pump. The treatment was preceded by a 2-3 days in-hospital instruction period. The treatment was supervised by the clinic without home visits. Clinical data and quality of life questionnaires were collected at the start and at the end of treatment and one month later. Statistical analysis was performed using Wilcoxon's rank-sum test for paired samples. RESULTS: No major problems were encountered. The patients as well as their physicians preferred home treatment over in-hospital treatment. 35 out of 37 evaluable courses in 23 patients were efficacious. There were statistically significant improvements in clinical condition and quality of life during the treatment. This improvement lasted at least until one month after the end of treatment. The costs of HIVAT were 48-63% less than the same antibiotic treatment in the hospital. CONCLUSION: HIVAT was feasible and could be applied without major problems in the Netherlands. It proved safe, efficacious and cost-effective.