Skip to main content
SpringerLink
Log in

Dornase Alfa

A Review of Pharmacoeconomic and Quality-of-Life Aspects of its Use in Cystic Fibrosis

  • Adis Pharmacoeconomic Drug Evaluation
  • Published:
PharmacoEconomics Aims and scope Submit manuscript

Summary

Cystic fibrosis (CF) is a fatal hereditary disease; patients with CF have an average lifespan of 30 years. By cleaving neutrophil-derived DNA, dornase alfa (recombinant human deoxyribonuclease I) decreases the adhesiveness and viscoelasticity of sputum in the infected lungs of patients with CF. As a result, respiratory function is improved in patients with all degrees of disease severity, and the relative risk of pulmonary exacerbations is reduced in patients with mild to moderate disease.

Resource utilisation (days spent in hospital or receiving parenteral antibiotics) in patients with mild to moderate disease is also reduced by dornase alfa, as evidenced by a placebo-controlled trial in >900 patients. Cost savings generated by these reductions in resource use during 24 weeks of dornase alfa therapy offset about 17 to 37.5% of the acquisition cost of the drug, depending on local cost data for various countries. Reductions in resource utilisation with dornase alfa have not been observed in patients with severe disease.

Available cost-effectiveness and cost-utility analyses are not fully published. One analysis estimated that the incremental cost of avoiding one hospitalisation was about $Can15 000 relative to standard therapy after 1 year of treatment. Informal analysis in the UK suggests a cost per quality-adjusted life-year of £25 000 for dornase alfa.

Some quality-of-life (QOL) domains (mainly cough frequency and chest congestion) have shown modest improvement in patients treated with dornase alfa, mainly those with mild CF. Persuasive evidence of QOL benefit is lacking in those with more severe disease.

Identifying patients most likely to benefit from dornase alfa therapy is essential to maximise clinical and cost benefits. The lack of a demonstrated reduction in resource utilisation in patients with severe CF makes its use more difficult to justify economically in this group than in those with less severe disease. However, in the absence of other treatments for this group, economic considerations must be weighed against clinical benefits.

In conclusion, the acquisition cost of dornase alfa is partially offset by savings gained by reducing resource utilisation in patients with mild to moderate CF, and the drug appears to improve quality of life in some patients, mostly those with less severe disease. However, in the absence of guidance from definitive cost-effectiveness analyses, individual healthcare providers must make their own decisions about how best to provide dornase alfa to patients with CF in a rational and cost-justifiable manner.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. Bryson HM, Sorkin EM. Dornase alfa: a review of its pharmacological properties and therapeutic potential in cystic fibrosis. Drugs 1994 Dec; 48: 894–906

    Article  PubMed  CAS  Google Scholar 

  2. National Institutes of Health. Consensus development statement: genetic testing for cystic fibrosis [online]. National Institutes of Health; 1997 Apr 14–16. Available from: http://odp.nih.gov/consensus/statements/cdc/106/106stmt.html [accessed 1997 Jun 13]

  3. Ramsey BW. Management of pulmonary disease in patients with cystic fibrosis. N Engl J Med 1996 Jul 18; 335: 179–88

    Article  PubMed  CAS  Google Scholar 

  4. Witt DM, Anderson L. Dornase alfa: a new option in the management of cystic fibrosis. Pharmacotherapy 1996 Jan–Feb; 16: 40–8

    PubMed  CAS  Google Scholar 

  5. Zhao C, Wang I, Lehrer RI. Widespread expression of beta-defensin hBD-1 in human secretory glands and epithelial cells. FEBS Lett 1996; 396: 319–22

    Article  PubMed  CAS  Google Scholar 

  6. Smith JJ, Travis SM, Greenberg EP, et al. Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell 1996 Apr 19; 85: 229–36

    Article  PubMed  CAS  Google Scholar 

  7. Cramer GW, Bosso JA. The role of dornase alfa in the treatment of cystic fibrosis. Ann Pharmacother 1996 Jun; 30: 656–61

    PubMed  CAS  Google Scholar 

  8. Hodson ME. Aerosolized dornase alfa (rhDNase) for therapy of cystic fibrosis. Am J Respir Crit Care Med 1995 Mar; 151 (Pt 2): S70–4

    PubMed  CAS  Google Scholar 

  9. Perras C, Otten N. Pulmozyme: clinical and economic impacts [online]. Canadian Coordinating Office for Health Technology Assessment; 1996 Feb. Available from: URL: http://www.ccohta.ca/pubs/english/pulmozym [Accessed 1997 Jul 8]

  10. Koch C, McKenzie SG, Kaplowitz H, et al. International practice patterns by age and severity of lung disease in cystic fibrosis: data from the Epidemiological Registry of Cystic Fibrosis (ERCF). Pediatr Pulmonol 1997. In press

    Google Scholar 

  11. Levy LD, Durie PR, Pencharz PB, et al. Effects of long-term nutritional rehabilitation on body composition and clinical status in malnourished children and adolescents with cystic fibrosis. J Pediatr 1985 Aug; 107: 225–30

    Article  PubMed  CAS  Google Scholar 

  12. Geller DE. Aerosolized dornase alpha in cystic fibrosis: is there a role in the management of patients with early obstructive lung disease? Pediatr Pulmonol 1997. In press

    Google Scholar 

  13. Shale DJ. The cost of care in cystic fibrosis. Thorax 1992 Sep; 47: 673

    Article  PubMed  CAS  Google Scholar 

  14. Robert G, Stevens A, Colin-Jones D. Dornase alfa for cystic fibrosis [letter]. BMJ 1995 Sep 23; 311: 813

    Article  PubMed  CAS  Google Scholar 

  15. Ginsberg G, Blau H, Kerem E, et al. Cost-benefit analysis of a national screening programme for cystic fibrosis in an Israeli population. Health Econ 1994 Jan–Feb; 3: 5–23

    Article  PubMed  CAS  Google Scholar 

  16. Garber AM, Fenerty JP. Costs and benefits of prenatal screening for cystic fibrosis. Med Care 1991 May; 29: 473–89

    Article  PubMed  CAS  Google Scholar 

  17. Royal College of Physicians. Cystic fibrosis in adults. Recommendations for care of patients in the UK. J R Coll Physicians Lond 1991 Jan; 25(1): 12–5

    Google Scholar 

  18. Rous E, Coppel A, Haworth J, et al. A purchaser experience of managing new expensive drugs: interferon beta. BMJ 1996 Nov 9; 313: 1195–6

    Article  PubMed  CAS  Google Scholar 

  19. Wildhagen MF, Verheij JBGM, Verzijl JG, et al. The nonhospital costs of care of patients with CF in The Netherlands: results of a questionnaire. Eur Respir J 1996; 9: 2215–9

    Article  PubMed  CAS  Google Scholar 

  20. Robson M, Abbott J, Webb K, et al. A cost description of an adult cystic fibrosis unit and cost analyses of different categories of patients. Thorax 1992 Sep; 47: 684–9

    Article  PubMed  CAS  Google Scholar 

  21. Wildhagen MF, Verheij JBGM, Hilderink HBM, et al. Cost of care of patients with cystic fibrosis in The Netherlands in 1990–1. Thorax 1996 Mar; 51: 298–301

    Article  PubMed  CAS  Google Scholar 

  22. Ramsey BW, Astley SJ, Aitken ML, et al. Efficacy and safety of short-term administration of aerosolized recombinant human deoxyribonuclease in patients with cystic fibrosis. Am Rev Respir Dis 1993 Jul; 148: 145–51

    PubMed  CAS  Google Scholar 

  23. 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 1993 Jul 24; 342: 199–202

    Article  PubMed  CAS  Google Scholar 

  24. 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. N Engl J Med 1994; 331(10): 637–42

    Article  PubMed  CAS  Google Scholar 

  25. Mellis CJ, Australian rhDNase Phase IIIb Study Group. Effect of rhDNase on quality of life and spirometry in patients with cystic fibrosis [abstract]. Aust N Z J Med 1997 Apr; 27: 257

    Article  Google Scholar 

  26. Romano L, Antonelli M, Gaiero A, et al. Audit on one year treatment with recombinant human deoxyribonuclease in 97 CF patients [abstract]. Chest 1996 Oct; 110 Suppl.: 156S

    Google Scholar 

  27. Shah PL, Scott SF, Geddes DM, et al. Two years experience with recombinant human DNase I in the treatment of pulmonary disease in cystic fibrosis. Respir Med 1995 Aug; 89: 499–502

    Article  PubMed  CAS  Google Scholar 

  28. McCoy K, Hamilton S, Johnson C. Effects of 12-week administration of dornase alfa in patients with advanced cystic fibrosis lung disease. Chest 1996 Oct; 110: 889–95

    Article  PubMed  CAS  Google Scholar 

  29. 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 six months open-label treatment. Eur Respir J 1995 Jun; 8: 954–8

    PubMed  CAS  Google Scholar 

  30. Wilmott RW, Amin RS, Colin AA, et al. Aerosolized recombinant human DNase in hospitalized cystic fibrosis patients with acute pulmonary exacerbations. Am J Respir Crit Care Med 1996 Jun; 153 (Pt 1): 1914–7

    PubMed  CAS  Google Scholar 

  31. Dornase alfa prescribing information. Genentech, Inc. South San Francisco, USA 1996

  32. Oster G, Huse DM, Lacey MJ, et al. Effects of recombinant human DNase therapy on healthcare use and costs in patients with cystic fibrosis. Ann Pharmacother 1995 May; 29: 459–64

    PubMed  CAS  Google Scholar 

  33. Menzin G, Oster G, Davies L, et al. A multinational economic evaluation of rhDNase in the treatment of cystic fibrosis. Int J Technol Assess Health Care 1996; 12(1): 52–61

    Article  PubMed  CAS  Google Scholar 

  34. Graf von der Schulenberg J-M, Greiner W, von der Hardt H. Socioeconomic evaluation of the influence of rhDNase on the costs of treating respiratory-tract infections in patients with cystic fibrosis [in German]. Med Klin 1995 Apr 15; 90: 220–4.

    Google Scholar 

  35. Marra CA, Carleton BC, Basmadjian D, et al. Comment: cost of rhDNase in cystic fibrosis [letter]. Ann Pharmacother 1995 Oct; 29: 1050–1

    PubMed  CAS  Google Scholar 

  36. Bates RD, Nahata MC. Cost of rhDNase in cystic fibrosis. Ann Pharmacother 1995 Oct; 29: 1051

    PubMed  CAS  Google Scholar 

  37. Brown TER, Glennie JL, Carleton BC. A pharmacoeconomic evaluation of DNase in cystic fibrosis [abstract]. Pharmacotherapy 1996 Jan–Feb; 16: 134–5

    Google Scholar 

  38. Tetley TD, Rogers DF. Development of new treatments for lung disease. Respir Med 1996 Jan; 90: 5–23

    Article  Google Scholar 

  39. Tullis DE, Guyatt GH. Quality of life in cystic fibrosis. Pharmacoeconomics 1995 Jul; 8: 23–33

    Article  PubMed  CAS  Google Scholar 

  40. Ramsey BW, Boat TF. Outcome measures for clinical trials in cystic fibrosis. Summary of a Cystic Fibrosis Foundation Consensus Conference. J Pediatr 1994 Feb; 124: 177–92

    Article  PubMed  CAS  Google Scholar 

  41. Kaplan RM, Anderson JP, Wu AW, et al. The quality of well-being scale: applications in AIDS, cystic fibrosis and arthritis. Med Care 1989 Mar; 27 (3 Suppl.): S27–43

    Article  PubMed  CAS  Google Scholar 

  42. Orenstein DM, Pattishall ED, Nixon PA, et al. Quality of well-being before and after antibiotic treatment of pulmonary exacerbation in patients with cystic fibrosis. Chest 1990 Nov; 98(5): 1081–4

    Article  PubMed  CAS  Google Scholar 

  43. Orenstein DM, Nixon PA, Ross EA, et al. The quality of well-being in cystic fibrosis. Chest 1989; 95: 344–7

    Article  PubMed  CAS  Google Scholar 

  44. Czyzewski DI, Mariotto MJ, Bartholomew LK, et al. Measurement of quality of well being in a child and adolescent cystic fibrosis population. Med Care 1994 Sep; 32: 965–72

    Article  PubMed  CAS  Google Scholar 

  45. Götz I, Eichler I, Götz M. Quality of life in adults with cystic fibrosis [letter]. Thorax 1997 Apr; 52: 397

    PubMed  Google Scholar 

  46. Congleton J, Hodson M, Duncan-Skingle F. Quality of life in adults with cystic fibrosis [reply, letter]. Thorax 1997; 52: 397

    Google Scholar 

  47. de Jong W, Kaptein AA, van der Schans CP, et al. Quality of life in patients with cystic fibrosis. Pediatr Pulmonol 1997; 23: 95–100

    Article  PubMed  Google Scholar 

  48. Munzenberger PJ, Van Wagnen CA, Abdulhamid I, et al. Impact of dornase alfa on the quality of life in patients with mild cystic fibrosis [abstract]. Pharmacotherapy 1996 May–Jun; 16: 515

    Google Scholar 

  49. Heijerman HGM, van Rossem RN, Bakker W. Effect of rhDNase on lung function and quality of life in adult cystic fibrosis patients. Neth J Med 1995 Jun; 46: 293–7

    Article  PubMed  CAS  Google Scholar 

  50. FDA approves Pulmozyme for advanced cystic fibrosis. Scrip 1996 Dec 10; 2188: 17

    Google Scholar 

  51. Eddy DM. Principles for making difficult decisions in difficult times. JAMA 1994 Jun 8; 271(22): 1792–8

    Article  PubMed  CAS  Google Scholar 

  52. Nishimura LY, Shane R. Therapeutic dilemmas: an approach to the management of expensive pharmaceutical advances. Pharmacoeconomics 1994; 6(6): 498–505

    Article  PubMed  CAS  Google Scholar 

  53. Eddy DM. Health system reform: will controlling costs require rationing services? JAMA 1994 Jul 27; 272(4): 324–8

    Article  PubMed  CAS  Google Scholar 

  54. Eddy DM. Rationing resources while improving quality: how to get more for less. JAMA 1994 Sep 14; 272(10): 817–24

    Article  PubMed  CAS  Google Scholar 

  55. Spencer D, Weller P. Dornase-alfa for cystic fibrosis [letter]. Lancet 1995 May 20; 345: 1307

    Article  PubMed  CAS  Google Scholar 

  56. Davis PB. Evolution of therapy for cystic fibrosis. N Engl J Med 1994 Sep 8; 331: 672–3

    Article  PubMed  CAS  Google Scholar 

  57. Kanga JF. Dornase alfa therapy in cystic fibrosis: who should get it? Chest 1996 Oct; 110: 871–2

    Article  PubMed  CAS  Google Scholar 

  58. Dornase alfa price list. Red Book, 1996

  59. Gumery L, Edenborough F, Stableforth D, et al. Physiotherapy and nebulised drug use in a Birmingham adult cystic fibrosis unit: patient practice, knowledge and adherence [abstract no. P91]. Thorax 1996; 51 Suppl. 3: A48

    Google Scholar 

  60. Pharmac funding. GP Weekly 1997 Mar 12: 2

    Google Scholar 

  61. Johnanesson M. The Australian guidelines for subsidisation of Pharmaceuticals: the road to cost-effective drug prescribing? Pharmacoeconomics 1992; 2(5): 355–62

    Article  Google Scholar 

  62. Collier J. Dornase-alfa and orphan drugs [letter]. Lancet 1995 Sep 2; 346: 633

    Article  PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Adis International Limited, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, Auckland 10, New Zealand

    Karen L. Goa & Harriet Lamb

Authors
  1. Karen L. Goa
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Harriet Lamb
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Karen L. Goa.

Additional information

Various sections of the manuscript reviewed by: B. Carleton, Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, British Columbia, Canada; A. Clarke, Institute of Medical Genetics, University Hospital of Wales, Heath Park, Cardiff, Wales; D. Czyzewski, Department of Psychiatry and Behavioral Sciences, Baylor College of Medicine, Texas Children’s Hospital, Texas Medical Center, Houston, Texas, USA; S. Fiel, Allegheny University Hospital — MCP Division, Pulmonary/Critical Care Medicine, Philadelphia, Pennsylvania, USA; G. Ginsberg, Department of Data Analysis, Division of Computing and Information, Ministry of Health, Jerusalem, Israel; H.G.M. Heijerman, Adult Cystic Fibrosis Centre, Department of Pulmonology, Leyenburg Hospital, The Hague, The Netherlands; J.F. Kanga, Division of Pediatric Pulmonology, University of Kentucky, Lexington, Kentucky, USA; N.J.W. Lewis, Lewis Consulting Group, Inc., Wilmington, Delaware, USA; G. Oster, Policy Analysis, Inc., Brookline, Massachusetts, USA; D.J. Shale, Section of Respiratory Medicine, Department of Medicine, University of Wales College of Medicine, Llandough Hospital, Penarth, Cardiff, Wales.

Rights and permissions

Reprints and permissions

About this article

Cite this article

Goa, K.L., Lamb, H. Dornase Alfa. Pharmacoeconomics 12, 409–422 (1997). https://doi.org/10.2165/00019053-199712030-00011

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.2165/00019053-199712030-00011

Keywords

Search

Navigation