Advertisement

The lag between effectiveness and cost-effectiveness evidence of new drugs

Implications for decision-making in health care
  • Boyka StoykovaEmail author
  • Michael Drummond
  • Marco Barbieri
  • Jos Kleijnen
Original papers

Abstract

A new drug is approved for use if its efficacy and safety have been demonstrated. However, healthcare decision makers may also require data on the cost-effectiveness of new drugs if they are to make informed decisions about their place in therapy. Cost-effectiveness evidence may lag behind the effectiveness data in terms of its availability. We explored the timeliness of delivering cost-effectiveness information about new drugs with established effectiveness and significant financial impact. Drugs were identified, based on guidance documents and reports published by the UK National Institute for Clinical Excellence (NICE), and the following data were collected: dates of publication of first effectiveness and cost-effectiveness evidence, methodology of the cost-effectiveness analysis, quality scores of the clinical studies. Eighteen guidance documents on the use of new drugs/drug groups published by NICE by October 2001 covered 30 health technologies, which were included in the analysis. The analysis of the evidence showed that their effectiveness had been demonstrated in the last 12 years, with only two exceptions. However, cost-effectiveness evidence had been published for 21 (70%) of the technologies. The cost-effectiveness was estimated in 52.4% of cases using models. The good quality effectiveness evidence lagged behind the first effectiveness evidence by 1.40 years (95% CI 0.57–2.23), while the mean lag between the first effectiveness evidence and the first cost-effectiveness publications was estimated as 3.20 years (95% CI 1.76–4.65). Cost-effectiveness evidence thus often lags behind the effectiveness evidence. As a result healthcare decision makers are sometimes in a position of having to take decisions without having adequate cost-effectiveness data at their disposal.

Keywords

Decision making Cost-effectiveness Guidance Evidence Lag 

Notes

Acknowledgements

The Health Economics Research Group at Brunel University, the NHS Centre for Reviews and Dissemination and the Centre for Health Economics receive funding from the Department of Health in England. M.B. was the recipient of a Research fellowship supported by Schering-Plough Inc. The authors are grateful to Prof. Martin Buxton for helpful comments.

References

  1. 1.
    Burls A, Clark W, Stewart A, Preston C, Bryan S, Jefferson T et al (2001) Zanamivir for the treatment of influenza in adults. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  2. 2.
    Chilcott J, Wight J, Jones M, Tappenden P (2001) The clinical effectiveness and cost-effectiveness of pioglitazone for type 2 diabetes mellitus Available at: http://www.nice.org.uk/ (accessed 31October 2001)
  3. 3.
    Clegg A, Bryant J, Nicholson T, Gerrard K, McIntyre L, de Broe S, et al (2000) Clinical and cost-effectiveness of donepezil, rivastigmine and galantamine for Alzheimer's disease: a rapid and systematic review. Health Technol Assess 5(1)Google Scholar
  4. 4.
    Department of Health (1999) Faster access to modern treatment: how NICE appraisal will work. Department of Health: LeedsGoogle Scholar
  5. 5.
    Dinnes J, Cave C, Huang S, Major K, Milne R (2001) The effectiveness and cost-effectiveness of temozolomide for the treatment of recurrent malignant glioma. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  6. 6.
    Drummond M, O'Brien B, Stoddart G, Torrance G (1997) Methods for the economic evaluation of health care programmes. 2nd edn. Oxford University Press: New YorkGoogle Scholar
  7. 7.
    Forbes C, Shirran L, Bagnall AM, Duffy S, ter Riet G (2001) A rapid and systematic review of clinical effectiveness and cost-effectiveness of topotecan for ovarian cancer. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  8. 8.
    Health Economic Evaluations Database (2001) OHE-IFPMA Database Ltd, NovemberGoogle Scholar
  9. 9.
    Jadad AR, Moore RA, Carroll D, Jenkinson C, Reynolds DJ, Gavaghan DJ, McQuay HJ (1996) Assessing the quality of reports of randomised clinical trials: is blinding necessary? Control Clin Trials 17:.1–12Google Scholar
  10. 10.
    Lister-Sharp D, McDonagh MS, Khan KS, Kleijnen J (2000) A rapid and systematic review of the effectiveness and cost-effectiveness of the taxanes used in the treatment of advanced breast and ovarian cancer. Health Technol Assess 4(17)Google Scholar
  11. 11.
    Lord J, Paisley S (2000) The clinical effectiveness and cost effectiveness of methylphenidate for hyperactivity in childhood, version 2. National Institute for Clinical Excellence: LondonGoogle Scholar
  12. 12.
    Lord J, Paisley S, Taylor R (2000) The clinical effectiveness and cost effectiveness of rosiglitazone for type 2 diabetes mellitus. National Institute for Clinical Excellence: LondonGoogle Scholar
  13. 13.
    McDonagh M, Bachmann L, Golder S, Kleijnen J, ter Riet G (2000) A rapid and systematic review of the clinical effectiveness and cost-effectiveness of glycoprotein IIb/IIIa antagonists in the medical management of unstable angina. Health Technol Assess 4(30)Google Scholar
  14. 14.
    NHS (2001) Economic evaluation database. Available at: http://agatha.york.ac.uk/nhsdhp.htm (accessed 1 December 2001)
  15. 15.
    NICE Appraisal Team (2001) The clinical effectiveness and cost-effectiveness of celecoxib, rofecoxib, meloxicam and etodolac (Cox II inhibitors) for rheumatoid arthritis and osteoarthritis. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  16. 16.
    O'Meara S, Riemsma R, Shirran L, Mather L, ter Riet G (2001) A systematic review of the clinical effectiveness and cost-effectiveness of orlistat in the management of obesity. Available at: http://www.nice.org.uk/cat.asp?c=15712 (accessed 19 October 2001)
  17. 17.
    O'Meara S, Riemsma R, Shirran L, Mather L, ter Riet G (2001) A systematic review of the clinical effectiveness and cost-effectiveness of sibutramine in the management of obesity. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  18. 18.
    Scott D, Clegg A, Sidhu M, Hewitson P, Waugh N (2001) Clinical and cost-effectiveness of paclitaxel, docetaxel, gemcitabine and vinorelbine in lung cancer. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  19. 19.
    Sculpher M, Fenwick E, Claxton K (2000) Assessing quality in decision analytic cost-effectiveness models. Pharmacoeconomics 17:461–477PubMedGoogle Scholar
  20. 20.
    Sheldon TA (1996) Problems of using modelling in the economic evaluation of health care. Health Econ, 5:1–11Google Scholar
  21. 21.
    Shepherd J, Waugh N, Hewitson P (2000) Combination therapy (interferon alfa and ribavirin) in the treatment of chronic hepatitis c: a rapid and systematic review. Health Technol Assessment 4(33)Google Scholar
  22. 22.
    Smith, R (1999) What is publication? BMJ 318:142PubMedGoogle Scholar
  23. 23.
    Stewart A, Sandercock J, Bryan S, Hyde C, Fry-Smith A, Burls A (2001) The clinical effectiveness and cost-effectiveness of riluzole for motor neurone disease. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  24. 24.
    Sullivan SD, Lyles A, Luce B, Gricar J (2001) AMCP guidance for submissions of clinical and economic evaluation data to support formulary listing in United States health plans and pharmacy benefit management organizations. J Manage Care Pharm 7:272–282Google Scholar
  25. 25.
    Wake B, Bryan S, Barton P, Fry-Smith A, Davenport C, Song F et al (2001) Fludarabine as second line therapy for B-cell chronic lymphocytic leukamia. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)
  26. 26.
    Ward S, Bansback N, Morris E, Calvert N (2001) A review of clinical and cost-effectiveness of gemcitabine for the treatment of pancreatic cancer. Available at: http://www.nice.org.uk/ (accessed 31 October 2001)

Copyright information

© Springer-Verlag 2003

Authors and Affiliations

  • Boyka Stoykova
    • 1
    • 4
    Email author
  • Michael Drummond
    • 2
  • Marco Barbieri
    • 2
  • Jos Kleijnen
    • 3
  1. 1.Health Economics Research GroupBrunel UniversityUxbridgeUK
  2. 2.Centre for Health EconomicsUniversity of YorkHeslingtonUK
  3. 3.NHS Centre for Reviews and DisseminationUniversity of YorkHeslingtonUK
  4. 4.Health Economics Research GroupBrunel UniversityUxbridgeUK

Personalised recommendations