Use of rhDNase Therapy and Costs of Respiratory-Related Care in Patients with Cystic Fibrosis

Ollendorf, Daniel A.; McGarry, Lisa; Watrous, Marc; Oster, Gerry

doi:10.1345/aph.19159

Cited by 8 publications

(2 citation statements)

References 12 publications

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“…Furthermore, a recent US study suggested that prolonged use of rhDNase may reduce the costs of respiratory disease related care in patients with CF. 23 Our study did not provide any strong evidence that daily rhDNase reduced the rate of inpatient admissions or the duration of intravenous antibiotics compared with HS. However, it suggested that the length of stay for pulmonary exacerbations (the primary reason for admission) was on average 1.95 days longer following HS than daily rhDNase, although the confidence intervals around the estimate were wide and the difference was not statistically significant.…”

Section: Discussioncontrasting

confidence: 67%

Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis

Suri

Grieve²,

Normand³

et al. 2002

Thorax

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Background: Daily recombinant human deoxyribonuclease (rhDNase) is an established but expensive treatment in cystic fibrosis (CF). An alternative lower cost therapy is hypertonic saline (HS), which has been shown to improve lung function in short term studies. This study compares the costs and consequences of daily rhDNase with alternate day rhDNase and HS in children with CF. Methods:In an open, randomised, crossover trial, 48 children with CF were allocated consecutively to 12 weeks of once daily 2.5 mg rhDNase, alternate day 2.5 mg rhDNase, and twice daily 5 ml 7% HS. Outcomes assessed included forced expiratory volume in 1 second (FEV 1 ) and quality of life. All healthcare resource use was prospectively recorded for each patient. Unit costs were collected and combined with resource use data to give the total health service costs per patient for each treatment strategy. Results: Daily rhDNase resulted in a significantly greater increase in mean FEV 1 than HS (8%, 95% CI 2 to 14) but there was no significant difference in FEV 1 between daily and alternate day rhDNase (2%, 95% CI -4 to 9). Over a 12 week period the mean incremental costs of daily rhDNase compared with HS was £1409 (95% CI £440 to £2318), and the incremental cost of using daily rather than alternate day rhDNase was £513 (95% CI -£546 to £1510). Conclusions: Daily rhDNase is more effective than 5 ml 7% HS twice daily delivered by jet nebuliser, but significantly increases healthcare costs. Administering rhDNase on an alternate day rather than a daily basis is as effective, with a potential for cost savings.

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Section: Discussioncontrasting

confidence: 67%

Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis

Suri

Grieve²,

Normand³

et al. 2002

Thorax

View full text Add to dashboard Cite

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“…In addition, using alternate-day dornase-alfa was not a cost-effective alternative in comparison with daily treatment for the same period 20. In general, clinical trials in CF have shown improvements in lung function 21. However, the drug is considered an expensive treatment, costing £7 442 per patient per year, and not all patients benefit from it 2223.…”

Section: Practical Examples Of the Usefulness Of Pharmacoeconomic Anamentioning

confidence: 99%

Pharmacoeconomic Analysis in Saudi Arabia: An Overdue Agenda Item for Action

Al-Jazairi

Al-Qadheeb

Ajlan

2011

Annals of Saudi Medicine

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Pharmacoeconomics is a branch of health economics related to the most economical and efficient use of pharmaceuticals. Pharmacoeconomic research identifies, measures and compares the costs and outcomes (clinical, economic and humanistic) of pharmaceutical products and services. Pharmacoeconomic evaluation can play a significant role in the efficient allocation of resources in healthcare systems with constrained budgets. Countries are trying to control the rising costs of health care in their aging population. They are all asking the same question: Is the new drug good value for money; and if so, what is the society willing to pay for it? This article reviews the importance of, and the need for, adaptation of pharmacoeconomic analysis to the conditions in Saudi Arabia. It will shed some light on the important steps for converting the concept into practice, including the need for identifying the willing-to-pay (WTP) or the threshold cutoff, the existence of a real cost for each utility, the nonexistence of an pharmacoeconomic advisory forum, pharmaceutical budget allocation, and the impact of pharmaceutical marketing. It will also provide recommendations for easing any challenges that might jeopardize the conduct of such analysis in Saudi Arabia.

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Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

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A c c e p t e d M a n u s c r i p t 2 Highlights  We systematically reviewed the socioeconomic cost literature for 10 rare diseases. Direct and indirect costs incurred by patients, carers and society were searched. 77 studies were included with an unequal distribution of studies over disease types. Level of existing evidence is highest for diseases with available drug treatments. Indirect costs are in most cases of similar or higher magnitude than direct costs. found to range between 1-2 per 100,000 population (PWS, a sub-type of Histiocytosis, and EB) up to 42 per 100,000 population (Scleroderma). Overall, cost evidence on rare diseases appears to be very scarce (a total of 77 studies were identified across all diseases), with CF (n=29) and Haemophilia (n=22) being relatively well studied, compared to the other conditions, where very limited cost of illness information was available. In terms of data availability, total lifetime cost figures were found only across four diseases, and total annual costs (including indirect costs) across five diseases.Overall, data availability was found to correlate with the existence of a pharmaceutical treatment and indirect costs tended to account for a significant proportion of total costs.Although methodological variations prevent any detailed comparison between conditions, most of the rare diseases examined are associated with significant economic burden, both direct and indirect.

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Use of rhDNase Therapy and Costs of Respiratory-Related Care in Patients with Cystic Fibrosis

Abstract: Prolonged use of rhDNase may reduce costs of respiratory-related care in patients with cystic fibrosis; further study is required, however, to confirm these findings.

Cited by 8 publications

References 12 publications

Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis

Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis

Pharmacoeconomic Analysis in Saudi Arabia: An Overdue Agenda Item for Action

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

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