Development of a framework for enhancing the transparency, reproducibility and communication of the benefit-risk balance of medicines

Clinical Pharmacology and Therapeutics

Volumn 89, Issue 2, 2011, Pages 312-315

  Coplan, P M ^a,b   Noel, R A ^c   Levitan, B S ^d   Ferguson, J ^e   Mussen, F ^f  

^a PURDUE PHARMA L P   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c ELI LILLY AND COMPANY   (United States)

^d JOHNSON AND JOHNSON PHARMACEUTICAL RESEARCH AND DEVELOPMENT   (United States)

^e NOVARTIS PHARMA AG   (Switzerland)

^f JANSSEN RESEARCH AND DEVELOPMENT   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; CONCEPTUAL FRAMEWORK; DECISION MAKING; HUMAN; MEDICINE; OUTCOME ASSESSMENT; PRIORITY JOURNAL; REPRODUCIBILITY; RISK ASSESSMENT; RISK BENEFIT ANALYSIS;

COMMUNICATION; DECISION MAKING; DRUG INDUSTRY; PHARMACEUTICAL PREPARATIONS; PRODUCT SURVEILLANCE, POSTMARKETING; REPRODUCIBILITY OF RESULTS; RISK ASSESSMENT; STATISTICS AS TOPIC;

EID: 78951480208     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2010.291     Document Type: Article

References (30)

1. CIOMS Working Group IV. Benefit-Risk Balance for Marketed Drugs: Evaluating Safety Signals (Council for International Organizations of Medical Sciences, Geneva, Switzerland, 1998).
2. Eichler, H.G., Abadie, E., Raine, J.M. & Salmonson, T. Safe drugs and the cost of good intentions. N. Engl. J. Med. 360, 1378-1380 (2009).
3. Committee on the assessment of the US Drug Safety System; Baciu, A., Stratton, K. & Burke, S.P., Eds. The Future of Drug Safety: Promoting and Protecting the Health of the Public (National academies Press, Washington, D.C., 2006).
4. Drug safety: improvement needed in fDa's postmarket decision-making and oversight process. Report no. GaO-06-402. Washington, DC: Government accountability Office, March 2006.
5. Guo, J.J., Pandey, S., Doyle, J., Bian, B., Lis, Y. & Raisch, D.W. a review of quantitative risk-benefit methodologies for assessing drug safety and efficacy-report of the ISPOR risk-benefit management working group. Value Health 13, 657-666 (2010).
6. Breckenridge, A. Regulatory challenges, Reimbursement, And risk-benefit assessment. Clin. Pharmacol. Ther. 88, 153-154 (2010).
7. Walker, S., Mcauslane, N., Liberti, L. & Salek, S. Measuring benefit and balancing risk: strategies for the benefit-risk assessment of new medicines in a risk-averse environment. Clin. Pharmacol. Ther. 85, 241-246 (2009).
8. European Medicines agency. Report of the CHMP Working Group on Benefit- Risk assessment Models and Methods. Doc.ref. EMEa/CHMP/15404/2007. London, 19 January 2007.
9. European Medicines agency Reflection Paper on Benefit-Risk assessment Methods in the Context of the Evaluation of Marketing authorisation applications of Medicinal Products for Human Use. Doc.ref. EMEa/CHMP/15404/2007. London, 19 March 2008.
10. Eichler, H.G., Pignatti, F., Flamion, B., Leufkens, H. & Breckenridge, A. Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma. Nat. Rev. Drug Discov. 7, 818-826 (2008).
11. Psaty, B.M. & Burke, S.P. Protecting the health of the public-Institute of Medicine recommendations on drug safety. N. Engl. J. Med. 355, 1753-1755 (2006).
12. Garrison, L.P. Jr, Towse, A. & Bresnahan, B.W. assessing a structured, Quantitative health outcomes approach to drug risk-benefit analysis. Health Aff. (Millwood). 26, 684-695 (2007).
13. Levitan, B. et al. application of the BRaT framework to case studies: observations and insights. Clin. Pharmacol. Ther., This issue.
14. Lynd, L.D. & O'brien, B.J. advances in risk-benefit evaluation using probabilistic simulation methods: an application to the prophylaxis of deep vein thrombosis. J. Clin. Epidemiol. 57, 795-803 (2004).
15. Gelber, R.D., Cole, B.F., Gelber, S. & Goldhirsch, A. Comparing treatments using quality-adjusted survival: the Q-TWiST method. Am. Stat. 49, 161-9 (1995).
16. Holden, W.L., Juhaeri, J. & Dai, W. Benefit-risk analysis: a proposal using quantitative methods. Pharmacoepidemiol. Drug Saf. 12, 611-616 (2003).
17. Lynd, L.D. et al. Using the incremental net benefit framework for quantitative benefit-risk analysis in regulatory decision-making-a case study of alosetron in irritable bowel syndrome. Value Health. 13, 411-417 (2010).
18. Keeney, R.L. & Raiffa, H. Decisions with Multiple Objectives: Preferences and Value Tradeoffs (Cambridge University Press, Ny, 1993).
19. Felli, J.C., Noel, R.A. & Cavazzoni, P.A. a multiattribute model for evaluating the benefit-risk profiles of treatment alternatives. Med. Decis. Making 29, 104-115 (2009).
20. Mussen, F., Salek, S. & Walker, S. a quantitative approach to benefit-risk assessment of medicines - part 1: the development of a new model using multi-criteria decision analysis. Pharmacoepidemiol. Drug Saf. 16 (suppl. 1), S2-S15 (2007).
21. Hughes, J. Palliative care and the QaLy problem. Health Care Anal. 13, 289-301 (2005).
22. Phillips, L.D. Requisite decision modelling. J. Oper. Res. Soc. 33, 303-312 (1982).
23. Von Winterfeldt, D., Edwards, W. Decision Analysis and Behavioral Research (Cambridge University Press, Cambridge, Ny, 1986).
24. Clemen, R.T. Making Hard Decisions: An Introduction to Decision Analysis (Duxbury Press, Belmont, Ca, 1996).
25. Belton, V. & Stewart, T. J. Multiple Criteria Decision Analysis: An Integrated Approach (Kluwer, Boston, Ma, 2001)
26. Dodgson, J.S., Spackman, M., Pearman, A., & Phillips, L.D. Multi-Criteria Analysis: A Manual (Department for Communities and Local Government, London, 2009).
27. Kirkwood, C.W. Strategic Decision Making: Multiobjective Decision Analysis with Spreadsheets (Duxbury Press, Belmont, Ca, 1996).
28. European Medicines agency. The Rules Governing Medicinal Products in the European Union, Vol. 9. EMEa/115735/2004. http://ec.europa.eu/health/files/ eudralex/vol-9/pdf/vol9a-09-2008-en.pdf (2004).
29. Johnson, F.R. et al. Multiple sclerosis patients' benefit-risk preferences: serious adverse event risks versus treatment efficacy. J. Neurol. 256, 554-562 (2009).
30. Leviatan, B. a concise display of multiple end points for benefit-risk assessment. Clin. Pharmacol. Ther. (2010); e-pub ahead of print 24 November 2010.