Dornase alfa: A review of pharmacoeconomic and quality-of-life aspects of its use in cystic fibrosis

PharmacoEconomics

Volumn 12, Issue 3, 1997, Pages 409-422

  Goa, Karen L ^a   Lamb, Harriet ^a  

^a ADIS INTERNATIONAL LIMITED   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

DORNASE ALFA;

COST BENEFIT ANALYSIS; CYSTIC FIBROSIS; DISEASE SEVERITY; HUMAN; LUNG DISEASE; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW;

EID: 0030921204     PISSN: 11707690     EISSN: None     Source Type: Journal    
DOI: 10.2165/00019053-199712030-00011     Document Type: Review

References (62)

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
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
4. Witt DM, Anderson L. Dornase alfa: a new option in the management of cystic fibrosis. Pharmacotherapy 1996 Jan-Feb; 16: 40-8
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
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
7. Cramer GW, Bosso JA. The role of dornase alfa in the treatment of cystic fibrosis. Ann Pharmacother 1996 Jun; 30: 656-61
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
9. Perras C, Otten N. Pulmozyme: clinical and economic impacts [online]. Canadian Coordinating Office for Health Techno- logy 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
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
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
13. Shale DJ. The cost of care in cystic fibrosis. Thorax 1992 Sep; 47: 673
14. Robert G, Stevens A, Colin-Jones D. Dornase alfa for cystic fibrosis [letter]. BMJ 1995 Sep 23; 311: 813
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
16. Garber AM, Fenerty JP. Costs and benefits of prenatal screening for cystic fibrosis. Med Care 1991 May; 29: 473-89
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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.
35. Marra CA, Carleton BC, Basmadjian D, et al. Comment: cost of rhDNase in cystic fibrosis [letter]. Ann Pharmacother 1995 Oct; 29: 1050-1
36. Bates RD, Nahata MC. Cost of rhDNase in cystic fibrosis. Ann Pharmacother 1995 Oct; 29: 1051
37. Brown TER, Glennie JL, Carleton BC. A pharmacoeconomic evaluation of DNase in cystic fibrosis [abstract], Pharmacotherapy 1996 Jan-Feb; 16: 134-5
38. Tetley TD, Rogers DF. Development of new treatments for lung disease. Respir Med 1996 Jan; 90: 5-23
39. Tullis DE, Guyatt GH. Quality of life in cystic fibrosis. Pharmacoeconomics 1995 Jul; 8: 23-33
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
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
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
43. Orenstein DM, Nixon PA, Ross EA, et al. The quality of well-being in cystic fibrosis. Chest 1989; 95: 344-7
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
45. Götz I, Eichler I, Götz M. Quality of life in adults with cystic fibrosis [letter]. Thorax 1997 Apr; 52: 397
46. Congleton J, Hodson M, Duncan-Skingle F. Quality of life in adults with cystic fibrosis [reply, letter]. Thorax 1997; 52: 397
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
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
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
50. FDA approves Pulmozyme for advanced cystic fibrosis. Scrip 1996 Dec 10; 2188: 17
51. Eddy DM. Principles for making difficult decisions in difficult times. JAMA 1994 Jun 8; 271 (22): 1792-8
52. Nishimura LY, Shane R. Therapeutic dilemmas: an approach to the management of expensive pharmaceutical advances. Pharmacoeconomics 1994; 6 (6): 498-505
53. Eddy DM. Health system reform: will controlling costs require rationing services? JAMA 1994 Jul 27: 272 (4): 324-8
54. Eddy DM. Rationing resources while improving quality: how to get more for less. JAMA 1994 Sep 14; 272 (10): 817-24
55. Spencer D, Weller P. Dornase-alfa for cystic fihrosis [letter]. Lancet 1995 May 20; 345: 1307
56. Davis PB. Evolution of therapy for cystic fibrosis. N Engl J Med 1994 Sep 8; 331: 672-3
57. Kanga JF. Dornase alfa therapy in cystic fibrosis: who should get it? Chest 1996 Oct; 110: 871-2
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
60. Pharmac funding. GP Weekly 1997 Mar 12: 2
61. Johnanesson M. The Australian guidelines for subsidisation of pharmaceuticals: the road to cost-effective drug prescribing? Pharmacoeconomics 1992; 2 (5): 355-62
62. Collier J. Dornase-alfa and orphan drugs [letter]. Lancet 1995 Sep 2; 346: 633