27
Views
2
CrossRef citations to date
0
Altmetric
Review

Pharmacoeconomic review of recombinant human DNase in the management of cystic fibrosis

, , &
Pages 49-59 | Published online: 09 Jan 2014

References

  • Heijerman HG, van Rossem RN, Bakker W Effect of rhDNase on lung function and quality of life in adult cystic fibrosis patients. Neth.j Merl 46(6), 293–297 (1995).
  • Harms HK, Matouk E, Tournier G et al Multicenter, open-label study of recombinant human DNase in cystic fibrosis patients with moderate lung disease. DNase International Study Group. Fenian: Pulmonol. 26(3), 155–161 (1998).
  • Sheppard MN, Nicholson AG. The pathology of cystic fibrosis. CL117: Diag. Pathol. 8(1), 50–59 (2002).
  • Ten-Kate PL. Cystic fibrosis in The Netherlands. Int. J: Epidemiol. 6(1), 23 (1977).
  • Davis PB, Drumm M, Konstan MW. Cystic fibrosis. Am J: Respir. Grit. Cate Ailed.. 154(5), 1229–1256 (1996).
  • Menzin J, Oster G, Davies L et al. A multinational economic evaluation of rhDNase in the treatment of cystic fibrosis. Int. J. Technol Assess. Healthcare 12 (1), 52–61 (1996).
  • Johnson JA, Connolly MA, Jacobs P, Montgomery M, Brown NE, Zuberbuhler P Cost of care for individuals with cystic fibrosis: a regression approach to determining the impact of recombinant human DNase. Pharmacotherapy19(10), 1159–1166 (1999).
  • Goa KL, Lamb H. Domase a. A review of pharmacoeconomic and quality-of-life aspects of its use in cystic fibrosis. PharmacoEconomics 12 (3), 409–422 (1997).
  • Nasr SZ, Welsch CC. Disability and quality of life in cystic fibrosis from early age to adulthood. J. Adolesc. Health 19 (6), 381–383 (1996).
  • Morgan WJ, Butler SM, Johnson CA et al Epidemiologic study of cystic fibrosis: design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosis in the US and Canada. Pecliatr Pulmonol 28(4), 231–241 (1999).
  • Kerem E, Reisman J, Corey M, Canny GJ, Levison H. Prediction of mortality in patients with cystic fibrosis. N Engl. J. Med. 326(18), 1187–1191 (1992).
  • Sun i R, Grieve R, Normand C et al Effects of hypertonic saline, alternate day and daily rhDNase on healthcare use, costs and outcomes in children with cystic fibrosis. Thorax57 (10), 841–846 (2002).
  • •Important pharmacoeconomic study.
  • van Koolwijk LM, Uiterwaal CS, van der Laag J, Hoekstra JH, Gulmans VA, van der Ent CK. Treatment of children with cystic fibrosis: central, local or both? Acta Paecliatr 91(9), 927–972 (2002).
  • McIntyre AM. Dornase a and survival of patients with cystic fibrosis. Hasp. Med. 60(10), 736–739 (1999).
  • ••Formal cost-effectiveness study using datafrom Oster and colleagues and Fuchs and colleagues.
  • Konstan MW, Butler SM, Schidlow DV, Morgan WJ, Julius JR, Johnson CA. Patterns of medical practice in cystic fibrosis: part I. Evaluation and monitoring of health status of patients. Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Pecliatr Pulmonol 28(4), 242–247 (1999).
  • Oster G, Huse DM, Lacey MJ, Regan MM, Fuchs HJ. Effects of recombinant human DNase therapy on healthcare use and costs in patients with cystic fibrosis. Ann Pharmacother 29(5), 459–464 (1995).
  • ••Important pharmacoeconomic study.
  • Johnson JA, Connolly M, Zuberbuhler P, Brown NE. Health-related quality of life for adults with cystic fibrosis: a regression approach to assessing the impact of recombinant human DNase. Pharmacotherapy 20 (10), 1167–1174 (2000).
  • Ratjen F, Tummler B. Comparison of the in vitro and in vivo response to inhaled DNase in patients with cystic fibrosis. Thorax54(1), 91 (1999).
  • King M, Dasgupta B, Tomkiewicz RP, Brown NE. Rheology of cystic fibrosis sputum after in vitro treatment with hypertonic saline alone and in combination with recombinant human deoxyribonuclease I. Am J. Respir. Cat. Carr Med. 156(1), 173–177 (1997).
  • Laube BL, Auci RM, Shields DE et al Effect of rhDNase on airflow obstruction and mucociliary clearance in cystic fibrosis. Arn. I Respir. Crit. Care Med 153(2), 752–760 (1996).
  • Zahm JM, Girod de Bentzmann S, Deneuville E et al Dose-dependent in vitro effect of recombinant human DNase on rheological and transport properties of cystic fibrosis respiratory mucus. Eur Respir. 8(3), 381–386 (1995).
  • Koch C, McKenzie SG, Koplowitz H et al International practice patterns by age and severity of lung disease in cystic fibrosis: data from the Epidemiologic Registry of Cystic Fibrosis (ERCF). Pecliatr Pulmonol 24(2), 147–154 (1997).
  • Geller DE. Aerosolized dornase alfa in cystic fibrosis: is there a role in the management of patients with early obstructive lung disease? Pecliatr Pulmonol 24(2),155–1558 (1997).
  • Davis PB. Evolution of therapy for cystic fibrosis. N. Eng. Med 331(10), 672–673 (1994).
  • Spencer H, Jaffe A. Newer therapies for cystic fibrosis. Cur Paed 13(4), 259–263 (2003).
  • Ramsey BW. Management of pulmonary disease in patients with cystic fibrosis. N Eng. Med 335(3), 179–188 (1996).
  • •Extensive review of strategies to manage cystic fibrosis.
  • Shak S, Capon DJ, Hellmiss R, Marsters SA, Baker CL. Recombinant human DNase I reduces the viscosity of cystic fibrosis sputum. Proc. Natl Acad. Sci. USA 87(23), 9188–9192 (1990).
  • Zahm JM, Galabert C, Chaffin A, Chazalette JP, Grosskopf C, Puchelle E. Improvement of cystic fibrosis airway mucus transportability by recombinant human DNase is related to changes in phospholipid profile. Am. J. Respir. Cat. Carr Med 157(6 Pt 1), 1779–1784 (1998).
  • de Boer AH, Le Brun PP, van der Woude HG, Hagedoorn P, Heijerman HG, Frijlink HW. Dry powder inhalation of antibiotics in cystic fibrosis therapy. Part 1. Development of a powder formulation with colistin sulfate for a special test inhaler with an air classifier as deagglomeration principle. Eur I Pharm. Biopharm. 54(1), 17–24 (2002).
  • Aitken ML, Burke W, McDonald G, Shak S, Montgomery AB, Smith A. Recombinant human DNase inhalation in normal subjects and patients with cystic fibrosis. A Phase I study. JAIVIA 267(14), 1947–1951 (1992).
  • Ranasinha C, Assoufi B, Shak S et al Efficacy and safety of short-term administration of aerosolised recombinant human DNase I in adults with stable stage cystic fibrosis. Lancet 342(8865), 199–202 (1993).
  • Shah PI, Bush A, Canny GJ et al Recombinant human DNase I in cystic fibrosis patients with severe pulmonary disease: a short-term, double-blind study followed by 6 months open-label treatment. Eur Respir. 8(6), 954–958 (1995).
  • Shah PL, Scott SF, Fuchs HJ, Geddes DM, Hodson ME. Medium term treatment of stable stage cystic fibrosis with recombinant human DNase I. Thorax 50(4), 333–338 (1995).
  • Shah PL, Scott SF, Geddes DM, Hodson ME. Two years experience with recombinant human DNase I in the treatment of pulmonary disease in cystic fibrosis. Respir. Med. 89(7), 499–502 (1995).
  • Milla CE. Long-term effects of aerosolised rhDNase on pulmonary disease progression in patients with cystic fibrosis. Thorax 53(12), 1014–1017 (1998).
  • Le Brun PP, de Boer AH, Mannes GP et al Dry powder inhalation of antibiotics in cystic fibrosis therapy. Part 2. Inhalation of a novel colistin dry powder formulation: a feasibility study in healthy volunteers and patients. Eur .j Phatm. Biopharm. 54(1), 25–32 (2002).
  • •Comparison of nebulization and dry powder inhalation (DPD in cystic fibrosis.
  • Duddu SP, Sisk SA, Walter YH et al Improved lung delivery from a passive dry powder inhaler using an Engineered PulmoSphere powder. Pharm. Res. 19(5), 689–695 (2002).
  • Dunbar C, Scheuch G, Sommerer K, DeLong M, Verma A, Batycky R. hi vitro and in vivo dose delivery characteristics of large porous particles for inhalation. int. j Pharm. 245(1-2), 179–189 (2002).
  • de Boer AH, Hagedoorn P, Gjaltema D, Goede J, Frijlink HW. Air classifier technology (ACT) in dry powder inhalation. Part 1. Introduction of a novel force distribution concept (FDC) explaining the performance of a basic air classifier on adhesive mixtures. int. j Pharm. 260(2), 187–200 (2003).
  • de Boer AH, Hagedoorn P, Gjaltema D, Goede J, Kussendrager KD, Frijlink HW. Air classifier technology (ACT) in dry powder inhalation. Part 2. The effect of lactose carrier surface properties on the drug-to-carrier interaction in adhesive mixtures for inhalation. Int. j Pharm. 260(2), 201–216 (2003).
  • Maa YFN, Hsu, C. Spray-coating of rhDNase on lactose: effect of system design, operational parameters and protein formulation. Int. j Pharm. (144), 47–59 (1996).
  • Chan HK, Clark A, Gonda I, Mumenthaler M, Hsu C. Spray dried powders and powder blends of recombinant human deoxyribonuclease (rhDNase) for aerosol delivery. Pharm. Res. 14 (4), 431–437 (1997).
  • Chan HK, Gonda I. Solid state characterization of spray-dried powders of recombinant human deoxyribonuclease (rhDNase). Pharm. Sd. 87(5), 647–654 (1998).
  • Hinrichs WL, Prinsen MG, Frijlink HW. Inulin glasses for the stabilization of therapeutic proteins. int. j Pharm. 215(1–2), 163–174 (2001).
  • •Basic science paper on facilitating DPI technology.
  • Robson M, Abbott J, Webb K, Dodd M, Walsworth-Bell J. A cost description of an adult cystic fibrosis unit and cost analyses of different categories of patients. Thorax 47(9), 684–689 (1992).
  • Wildhagen MF, Hilderink HB, Verzijl JG et al Costs, effects and savings of screening for cystic fibrosis gene carriers. Epidemiol Comm. Health 52 (7), 459–467 (1998).
  • •Comprehensive cost-effectiveness analysis in the field, however, not specifically directed at recombinant human DNase.
  • Wildhagen ME, Verheij JB, Verzijl JG et al. Cost of care of patients with cystic fibrosis in The Netherlands in 1990–1991. Thorax 51(3), 298–301 (1996).
  • CA, Butler SM, Konstan MW Breen TJ, Morgan WJ. Estimating effectiveness in an observational study: a case study of dornase a in cystic fibrosis. The Investigators and Co-ordinators of the Epidemiologic Study of Cystic Fibrosis. J: Pediatz: 134(6), 734–739 (1999).
  • Robertson CE How do we choose a therapeutic regimen in cystic fibrosis? Thorax57 (10), 839–840 (2002).
  • Conway SR Recombinant human DNase (rhDNase) in cystic fibrosis: is it cost effective? Atch Dis. Child.77 (1), 1–3 (1997).
  • Grieve R, Thompson S, Normand C, Suri R, Bush A, Wallis C. A cost-effectiveness analysis of rhDNase in children with cystic fibrosis. Int. J. Technol Assess. Health. 19(1), 71–79 (2003).
  • College voor Zorgverzekeringen (CvZ.) Beoordeling van de aanspraak op dornase alfa (Pulmozyme). (1999).
  • endorf DA, McGarry LJ, Watrous ML, Oster G. Use of rhDNase therapy and costs of respiratory-related care in patients with cystic fibrosis. Ann Phatmacother 34(3), 304–308 (2000).
  • Fuchs HJ, Borowitz DS, Christiansen DH et al Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl. J. Med 331(10), 637–642 (1994).
  • •Often used as the basis for pharmacoeconomic assessments.
  • Lieu TA, Ray GT, Farmer G, Shay GF. The cost of medical care for patients with cystic fibrosis in a health maintenance organization. Pediatrics 103(6), 72 (1999).
  • Wagener JS, Rock MJ, McCubbin MM, Hamilton SD, Johnson CA, Ahrens RC. Aerosol delivery and safety of recombinant human deoxyribonuclease in young children with cystic fibrosis: a bronchoscopic study. Pulmozyme Pediatric Broncoscopy Study Group. J. Pecliatr 133(4), 486–491 (1998).
  • Marshall BC, Samuelson WM. Basic therapies in cystic fibrosis. Does standard therapy work? Clin. Chest Med. 19(3), 487–504 (1998).
  • Tonelli MR, Aitken ML. New and emerging therapies for pulmonary complications of cystic fibrosis. Drugs 61(10), 1379–1385 (2001).
  • Britt° MT, Kotagal UR, Hornung RW, Atherton HD, Tsevat J, Wilmott RVV. Impact of recent pulmonary exacerbations on quality of life in patients with cystic fibrosis. Chest 121(1), 64–72 (2002).
  • Geddes DM, Shah PL. Where we are now with rhDNase? Lancet 353(9166), 1727 (1999).
  • Cobos N, Danes I, Gartner S, Gonzalez M, Linan S, Arnau JM. DNase use in the daily care of cystic fibrosis: who benefits from it and to what extent? Results of a cohort study of 199 patients in 13 centres. DNase National Study Group. Eur j Pecliatr 159(3), 176–181 (2000).
  • Robinson PJ. Domase alfa in early cystic fibrosis lung disease. Pecliatr Pulmonol 34(3), 237–241 (2002).
  • Robinson M, Hemming AL, Moriarty C, Eberl S, Bye PT Effect of a short course of rhDNase on cough and mucociliary clearance in patients with cystic fibrosis. Pecliatr Pulmonol 30(1), 16–24 (2000).
  • Burrows JA, Bunting JP, Masel PJ, Bell SC. Nebulised domase alpha: adherence in adults with cystic fibrosis. J. Cystic Fibmsis 1(4), 255–259 (2002).
  • Hjelmgren J, Berggren F, Andersson Health economic guidelines—similarities, differences and some implications. Value Health 4(3), 225–250 (2001).
  • Suri R, Metcalfe C, Lees B et al. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet 358(9290), 1316–1321 (2001).
  • Diot P, Palmer LB, Smaldone A, DeCelie- Germana J, Grimson R, Smaldone GC. RhDNase I aerosol deposition and related factors in cystic fibrosis. Am. J. Respir. Crit. Care Med. 156(5), 1662–1668 (1997).
  • Fiel SB, Fuchs HJ, Johnson C, Gonda I, Clark AR. Comparison of three jet nebulizer aerosol delivery systems used to administer recombinant human DNase Ito patients with cystic fibrosis. The Pulmozyme rhDNase Study Group. Chest 108(1), 153–156 (1995).
  • •Overview of pharrnacoeconomics of drug delivery in asthma, in particular DPI versus nebulization.
  • de Boer AH, Molema G, Frijlink HW. Pulmonary drug delivery: delivery to and through the lung. In: Drug Targetting. Molema G, Meijer DKF (Eds). Wiley-VCH, Germany, 53–87 (2001).
  • Massie RJ, Mellis CM. The economic aspects of drug delivery in asthma. PharmacoEconomits 11(5), 398–407 (1997).
  • Gale EA. Two cheers for inhaled insulin. Lancet 357(9253), 324–325 (2001).
  • Cappelleri JC, Cefalu WT, Rosenstock J, Kourides IA, Gerber RA. Treatment satisfaction in Type 2 diabetes: a comparison between an inhaled insulin regimen and a subcutaneous insulin regimen. Clin. Ther 24(4), 552–564 (2002).
  • Patton JS, Bukar J, Nagarajan S. Inhaled insulin. Adv. Drug- Del. Rev 35(2–3), 235–247. (1999).
  • Skyler JS, Cefalu WT, Kourides IA et aL Efficacy of inhaled human insulin in Type 1 diabetes mellitus: a randomised proof-of-concept study. Lancet357(9253), 331–335. (2001).

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.