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 in Health

Volumn 13, Issue 4, 2010, Pages 411-417

  Lynd, Larry D ^a   Najafzadeh, Mehdi ^a   Colley, Lindsey ^a   Byrne, Michael F ^a   Willan, Andrew R ^b   Sculpher, Mark J ^c   Johnson, F Reed ^d   Hauber, A Brett ^d  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c UNIVERSITY OF YORK   (United Kingdom)

^d RTI HEALTH SOLUTIONS   (United States)

Author keywords

alosetron; benefit risk analysis; discrete event simulation; irritable bowel syndrome

Indexed keywords

ALOSETRON; PLACEBO; CARBOLINE DERIVATIVE; GASTROINTESTINAL AGENT;

ARTICLE; CLINICAL ASSESSMENT TOOL; HEALTH HAZARD; INCREMENTAL NET BENEFIT FRAMEWORK; IRRITABLE COLON; MEDICAL DECISION MAKING; PRIORITY JOURNAL; QUALITY OF LIFE; QUANTITATIVE ANALYSIS; SENSITIVITY ANALYSIS; SIMULATION; ADVERSE DRUG REACTION; COMPUTER SIMULATION; DRUG CONTROL; FEMALE; HUMAN; IRRITABLE BOWEL SYNDROME; MALE; MIDDLE AGED; PATIENT PREFERENCE; PROCEDURES; RISK ASSESSMENT; SEVERITY OF ILLNESS INDEX; STATISTICS AND NUMERICAL DATA;

CARBOLINES; COMPUTER SIMULATION; DRUG AND NARCOTIC CONTROL; DRUG-RELATED SIDE EFFECTS AND ADVERSE REACTIONS; FEMALE; GASTROINTESTINAL AGENTS; HUMANS; IRRITABLE BOWEL SYNDROME; MALE; MIDDLE AGED; PATIENT PREFERENCE; QUALITY OF LIFE; RISK ASSESSMENT; SEVERITY OF ILLNESS INDEX;

EID: 77954268317     PISSN: 10983015     EISSN: 15244733     Source Type: Journal    
DOI: 10.1111/j.1524-4733.2009.00595.x     Document Type: Article

References (26)

1. Baciu A, Stratton K, Burke S, eds. The Future of Drug Safety: Promoting and Protecting the Health of the Public.
2. Committee for Medical Products for Human Use. Reflection Paper on Benefit-Risk Assessment Methods in Context of the Evaluation of Marketing Authorisation Applications of Medicinal Products for Human Use.
3. Bender R, Blettner M. Calculating the "number needed to be exposed" with adjustment for confounding variables in epidemiological studies. J Clin Epidemiol 2002 55 : 525 530.
4. Schulzer M, Mancini GB. "Unqualified success" and "unmitigated failure": number-needed-to-treat-related concepts for assessing treatment efficacy in the presence of treatment-induced adverse events. Int J Epidemiol 1996 25 : 704 712.
5. Riegelman R, Schroth WS. Adjusting the number needed to treat: incorporating adjustments for the utility and timing of benefits and harms. Med Decis Making 1993 13 : 247 252.
6. Sinclair JC, Cook RJ, Guyatt GH, et al. When should an effective treatment be used? Derivation of the threshold number needed to treat and the minimum event rate for treatment. J Clin Epidemiol 2001 54 : 253 262.
7. Willan AR, O'Brien BJ, Cook DJ. Benefit-risk ratios in the assessment of the clinical evidence of a new therapy. Control Clin Trials 1997 18 : 121 130.
8. Chuang-Stein C, Mohberg NR, Sinkula MS. Three measures for simultaneously evaluating benefits and risks using categorical data from clinical trials. Stat Med 1991 10 : 1349 1359.
9. Lynd LD, O'Brien BJ. Advances in risk-benefit evaluation using probabilistic simulation methods: an application to the prophylaxis of deep vein thrombosis. J Clin Epidemiol 2004 57 : 795 803.
10. Garrison LP, Jr., Towse A, Bresnahan BW. Assessing a structured, quantitative health outcomes approach to drug risk-benefit analysis. Health Aff (Millwood) 2007 26 : 684 695.
11. Center for Drug Evaluation and Research. Lotronex tablets (alosetron hydrochloride): questions and answers. 2002. Available from: http://www.fda.gov/Drugs/DrugSafety/ PostmarketDrugSafetyInformationforPatientsandProviders/ucm110859.htm [Accessed December 4, 2007
12. Krinsky I, Robb AL. On approximating the statistical properties of elasticities. MIT Press 1986 68 : 715 719.
13. Chey WD, Chey WY, Heath AT, et al. Long-term safety and efficacy of alosetron in women with severe diarrhea-predominant irritable bowel syndrome. Am J Gastroenterol 2004 99 : 2195 2203.
14. Chang L, Chey WD, Harris L, et al. Incidence of ischemic colitis and serious complications of constipation among patients using alosetron: systematic review of clinical trials and post-marketing surveillance data. Am J Gastroenterol 2006 101 : 1069 1079.
15. Wolfe SG, Chey WY, Washington MK, et al. Tolerability and safety of alosetron during long-term administration in female and male irritable bowel syndrome patients. Am J Gastroenterol 2001 96 : 803 811.
16. Ladabaum U. Safety, efficacy and costs of pharmacotherapy for functional gastrointestinal disorders: the case of alosetron and its implications. Aliment Pharmacol Ther 2003 17 : 1021 1030.
17. Drummond M, Sculpher M, Torrance G, et al. Methods for Economic Evalution of Health Programs. New York: Oxford University Press, 2005.
18. National Institute for Clinical Excellence. Guide to Methods of Technology Appraisal. London: National Health Service, 2004.
19. Hillner BE. Anastrozole had a better risk-benefit profile than tamoxifen as adjuvant treatment for breast cancer in postmenopausal women. ACP J Club 2007 146 : 4.
20. Hoffmann JN, Wiedermann CJ, Juers M, et al. Benefit/risk profile of high-dose antithrombin in patients with severe sepsis treated with and without concomitant heparin. Thromb Haemost 2006 95 : 850 856.
21. Hochberg MC, Lebwohl MG, Plevy SE, et al. The benefit/risk profile of TNF-blocking agents: findings of a consensus panel. Semin Arthritis Rheum 2005 34 : 819 836.
22. Rodondi N, Bauer DC. Assessing the risk/benefit profile before recommending aspirin for the primary prevention of cardiovascular events. Am J Med 2004 117 : 528 530.
23. Barie PS, Williams MD, McCollam JS, et al. Benefit/risk profile of drotrecogin alfa (activated) in surgical patients with severe sepsis. Am J Surg 2004 188 : 212 220.
24. Clark LT. Treating dyslipidemia with statins: the risk-benefit profile. Am Heart J 2003 145 : 387 396.
25. Minelli C, Abrams KR, Sutton AJ, Cooper NJ. Benefits and harms associated with hormone replacement therapy: clinical decision analysis. BMJ 2004 328 : 71.
26. US Food and Drug Administration. Transcript: joint meeting with the risk management subcommittee of the advisory committee for pharmaceutical sciences. 2002. Available from: http://www.fda.gov/ohrms/dockets/ac/02/transcripts/3848T1. htm [Accessed February 29, 2008 ].