Hostname: page-component-586b7cd67f-rdxmf Total loading time: 0 Render date: 2024-11-28T04:19:25.089Z Has data issue: false hasContentIssue false
  • English
  • Français

Contribution of economic evaluation to decision making in early phases of product development: A methodological and empirical review

Published online by Cambridge University Press:  01 October 2008

Susanne Hartz  and
Jürgen John
Susanne Hartz
Affiliation:
Friedrich-Schiller-University of Jena
Jürgen John
Affiliation:
Helmholtz Zentrum München, Institute of Health Economics and Healthcare Management
Get access Rights & Permissions [Opens in a new window]

Abstract

Background: Economic evaluation as an integral part of health technology assessment is today mostly applied to established technologies. Evaluating healthcare innovations in their early states of development has recently attracted attention. Although it offers several benefits, it also holds methodological challenges.

Objectives: The aim of our study was to investigate the possible contributions of economic evaluation to industry's decision making early in product development and to confront the results with the actual use of early data in economic assessments.

Methods: We conducted a literature research to detect methodological contributions as well as economic evaluations that used data from early phases of product development.

Results: Economic analysis can be beneficially used in early phases of product development for various purposes including early market assessment, R&D portfolio management, and first estimations of pricing and reimbursement scenarios. Analytical tools available for these purposes have been identified. Numerous empirical works were detected, but most do not disclose any concrete decision context and could not be directly matched with the suggested applications.

Conclusions: Industry can benefit from starting economic evaluation early in product development in several ways. Empirical evidence suggests that there is still potential left unused.

Keywords

Economic evaluationEarly dataResearch and developmentInnovation
Type
GENERAL ESSAYS
Information
International Journal of Technology Assessment in Health Care , Volume 24 , Issue 4 , October 2008 , pp. 465 - 472
Copyright
Copyright © Cambridge University Press 2008

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1. Álvarez, JS. Estudios de farmacoeconomía: ¿por qué, cómo, cuándo y para qué? MEDIFAM. 2001;11:147–155.Google Scholar
2. Annemans, L, Genesté, B, Jolain, B. Early modelling for assessing health and economic outcomes of drug therapy. Value Health. 2000;3:427–434.CrossRefGoogle ScholarPubMed
3. Bombardier, C, Maetzel, A. Pharmaeconomic evaluation of new treatments: Efficacy versus effectiveness studies? Ann Rheum Dis. 1999;58 (suppl I):I82-I85.CrossRefGoogle Scholar
4. Bonate, PL. Clinical trial simulation in drug development. Pharmaceutical Res. 2000;17:252–256.CrossRefGoogle ScholarPubMed
5. Briggs, A, Sculpher, MJ, Claxton, KP. Decision modelling for health economic evaluation. 1st ed. Oxford: Oxford University Press; 2006.CrossRefGoogle Scholar
6. Chilcott, J, Brennan, A, Booth, A, Karnon, J, Tappenden, P. The role of modelling in prioritising and planning clinical trials. Health Technol Assess. 2003;7:iii, 1–125.CrossRefGoogle ScholarPubMed
7. Claxton, KP, Posnett, J. An economic approach to clinical trial design and research priority-setting. Health Econ. 1996;5:513–524.3.0.CO;2-9>CrossRefGoogle ScholarPubMed
8. Claxton, KP, Sculpher, MJ. Using value of information analysis to prioritize health research. some lessons from recent UK experience. Pharmacoeconomics. 2006;24:1055–1068.CrossRefGoogle ScholarPubMed
9. Clemens, K, Garrison, LP Jr, Jones, A, Macdonald, F. Strategic use of pharmacoeconomic research in early drug development and global pricing. Pharmacoeconomics. 1993;4:315–322.CrossRefGoogle ScholarPubMed
10. DiMasi, JA. Risks in new drug development: Approval success rates for investigational drugs. Clin Pharmacol Ther. 2001;69:297–307.CrossRefGoogle ScholarPubMed
11. DiMasi, JA. The value of improving the productivity of the drug development process. Pharmacoeconomics. 2002;20 (suppl 3):1–10.CrossRefGoogle ScholarPubMed
12. DiMasi, JA, Caglarcan, E, Wood-Armany, M. Emerging role of pharmacoeconomics in the research and development decision-making process. Pharmacoeconomics. 2001;19:753–766.CrossRefGoogle ScholarPubMed
13. DiMasi, JA, Hansen, RW, Grabowski, HG. The price of innovation: New estimates of drug development costs. J Health Econ. 2003;22:151–185.CrossRefGoogle Scholar
14. Dranitsaris, G, Leung, P. Using decision modeling to determine pricing of new pharmaceuticals: The case of neurokinin-1 receptor antagonist antiemetics for cancer chemotherapy. Int J Technol Assess Health Care. 2004;20:289–295.CrossRefGoogle ScholarPubMed
15. Drummond, MF, Jefferson, TO. Guidelines for authors and peer reviewers of economic submissions to the BMJ. The BMJ Economic Evaluation Working Party. BMJ. 1996;313:275–283.CrossRefGoogle Scholar
16. Evers, S, Goossens, M, de Vet, H, van Tulder, M, Ament, A. Criteria list for assessment of methodological quality of economic evaluations: Consensus on Health Economic Criteria. Int J Technol Assess Health Care. 2005;21:240–245.CrossRefGoogle ScholarPubMed
17. Girard, P. Clinical trial simulation: A tool for understanding study failures and preventing them. Basic Clin Pharmacol Toxicol. 2005;96:228–234.CrossRefGoogle Scholar
18. Grabowski, H. The effect of pharmacoeconomics on company research and development decisions. Pharmacoeconomics. 1997;11:389–397.CrossRefGoogle ScholarPubMed
19. Gregson, N, Sparrowhawk, K, Mauskopf, J, Paul, J. Pricing medicines: Theory and practice, challenges and opportunities. Nat Rev Drug Discov. 2005;4:121–130.CrossRefGoogle ScholarPubMed
20. Hessel, FP. Economic evaluation of the artificial liver support system MARS in patients with acute-on-chronic liver failure. Cost Eff Resour Alloc. 2006;4:16.CrossRefGoogle ScholarPubMed
21. Hill, S, Freemantle, N. A role for two-stage pharmacoeconomic appraisal? Is there a role for interim approval of a drug for reimbursement based on modelling studies with subsequent full approval using phase III Data? Pharmacoeconomics. 2003;21:761–767.CrossRefGoogle Scholar
22. Holford, NH, Kimko, HC, Monteleone, JP, Peck, CC. Simulation of clinical trials. Annu Rev Pharmacol Toxicol. 2000;40:209–234.CrossRefGoogle ScholarPubMed
23. Hughes, DA, Walley, T. economic evaluations during early (phase II) drug development. A role for clinical trial simulations? Pharmacoeconomics. 2001;19:1069–1077.CrossRefGoogle ScholarPubMed
24. Kerwin, R. Discontinued drugs in 2005: Schizophrenia drugs. Expert Opin Investig Drugs. 2006;15:1487–1495.CrossRefGoogle ScholarPubMed
25. Krall, RL, Engleman, KH, Ko HC, Peck CC. Clinical trial modeling and simulation—Work in progress. Drug Inf J. 1998;32:971–976.CrossRefGoogle Scholar
26. Langley, P. In all probability. Script Magazine. 2003(June):22–24.Google Scholar
27. Langley, P. Focusing pharmacoeconomic activities: Reimbursement or the drug life cycle? Curr Med Res Opin. 2004;20:181–188.CrossRefGoogle ScholarPubMed
28. Lee, KK. Discontinued drugs in 2005: Pulmonary-allergy, dermatological, gastrointestinal and arthritis drugs. Expert Opin Investig Drugs. 2006;15:1497–1505.CrossRefGoogle ScholarPubMed
29. Leidl, R. Innovation—A health economic perspective. Eur J Clin Pharmacol. 2005;61:693. Abstract.Google Scholar
30. Mark, DB, Simons, TA. Economic end points in Phase II trials. Am Heart J. 2000;139:S155-S157.CrossRefGoogle ScholarPubMed
31. Mauskopf, J, Schulman, K, Bell, L, Glick, H. A strategy for collecting pharmacoeconomic data during phase II/III clinical trials. Pharmacoeconomics. 1996;9:264–277.CrossRefGoogle ScholarPubMed
32. Miller, P. Role of pharmacoeconomic analysis in R&D decision making. Pharmacoeconomics. 2005;23:1–12.CrossRefGoogle Scholar
33. Philips, Z, Ginnelly, L, Sculpher, M et al. Review of guidelines for good practice in decision-analytic modelling in health technology assessment. Health Technol Assess. 2004;8: iii–iv, ix–xi, 1–158. Review.CrossRefGoogle ScholarPubMed
34. Poland, B, Wada, R. Combining drug-disease and economic modelling to inform drug development decisions. Drug Discov Today. 2001;6:1165–1170.CrossRefGoogle ScholarPubMed
35. Schachter, AD, Ramoni, MF, Baio, G, Roberts, TG Jr, Finkelstein, SN. Economic evaluation of a Bayesian model to predict late-phase success of new chemical entities. Value Health. 2007;10:377–385.CrossRefGoogle ScholarPubMed
36. Sculpher, M, Drummond, M, Buxton, M. The iterative use of economic evaluation as part of the process of health technology assessment. J Health Serv Res Policy. 1997;2:26–30.CrossRefGoogle ScholarPubMed
37. Seidman, A, Portenoy, R, Yao, TJ et al. , Quality of life in phase II trials: A study of methodology and predictive value in patients with advanced breast cancer treated with paclitaxel plus granulocyte colony-stimulating factor. J Natl Cancer Inst. 1995;87:1316–1322.CrossRefGoogle ScholarPubMed
38. Stallard, N. Decision-theoretic designs for phase III clinical trials allowing for competing studies. Biometrics. 2003;59:402–409.CrossRefGoogle Scholar
39. Sternbergh, CW III, Money, SR. Hospital cost of endovascular versus open repair of abdominal aortic aneurysms: A multicenter study. J Vasc Surg. 2000;31:237–244.CrossRefGoogle ScholarPubMed
40. Tanneberger, S, Lelli, G, Martoni A, Piana E, Pannuti F. The antiemetic efficacy and the cost-benefit ratio of ondansetron calculated with a new approach to health technology assessment (real cost-benefit index). J Chemother. 1992;4:326–331.CrossRefGoogle ScholarPubMed
41. Taylor, RS, Drummond, MF, Salkeld, G, Sullivan, SD. Inclusion of cost effectiveness in licensing requirements of new drugs: The fourth hurdle. BMJ. 2000;329:972–975.CrossRefGoogle Scholar
42. WHO. International statistical classification of diseases and related health problems, 10th revision, Version for 2007. http://www.who.int/classifications/apps/icd/icd10online/. Accessed 13 July 2007.Google Scholar
43. Wirtz, V, Cribb, A, Barber, N. Reimbursement decisions in health policy—extending our understanding of the elements of decision-making. Health Policy. 2005;73:330–338.CrossRefGoogle ScholarPubMed
44. Yan, EC, Chen, L. A cost-related approach for evaluating drug development programs. Stat Med. 2004;23:2863–2873.CrossRefGoogle ScholarPubMed