A Universal Framework for the Benefit-Risk Assessment of Medicines: Is This the Way Forward?

Therapeutic Innovation and Regulatory Science

Volumn 49, Issue 1, 2015, Pages 17-25

  Walker, Stuart ^a,b   McAuslane, Neil ^a   Liberti, Lawrence ^a   Leong, James ^b,c   Salek, Sam ^b  

^a Centre for Innovation in Regulatory Science   (United Kingdom)

^b CARDIFF UNIVERSITY   (United Kingdom)

^c HEALTH SCIENCES AUTHORITY   (Singapore)

Author keywords

multicriteria decision making; universal methodology for benefit risk assessment

Indexed keywords

NEW DRUG;

ARTICLE; CONCEPTUAL FRAMEWORK; DECISION MAKING; DRUG INDUSTRY; FOOD AND DRUG ADMINISTRATION; PRIORITY JOURNAL; RISK ASSESSMENT; RISK BENEFIT ANALYSIS; SENSITIVITY AND SPECIFICITY;

EID: 84920983041     PISSN: 21684790     EISSN: 21684804     Source Type: Journal    
DOI: 10.1177/2168479014547421     Document Type: Article

References (23)

1. Center for Innovation in Regulatory Science. Measuring benefit and balancing risk: strategies for the benefit-risk assessment of new medicines in a risk-averse environment.http://www.cirsci.org/sites/default/files/June_2008.pdf. Published 2008. Accessed November 20, 2013.
2. Leong J,McAuslane N,Walker S,Salek S.Is there a need for a universal benefit-risk assessment framework for medicines? Regulatory and industry perspectives.Pharmacoepidemiol Drug Saf. 2013;22:1004-1012
3. European Medicines Agency. Reflection paper on benefit-risk assessment methods in the context of the evaluation of marketing authorization applications of medicinal products for human use.http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2010/01/WC500069634.pdf. Published 2008. Accessed November 20, 2013.
4. European Medicines Agency. Benefit-risk methodology project: development and testing of tools and processes for balancing multiple benefits and risks as an aid to informed regulatory decisions about medicinal products.http://www.ema.europa.eu/docs/en_GB/document_library/Report/2011/07/WC500109477.pdf. Published 2009. Accessed November 20, 2013.
5. European Medicines Agency. Benefit-risk methodology project: work package 2 report. Applicability of current tools and processes for regulatory benefit-risk assessment. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2010/10/WC500097750.pdf. Published 2010. Accessed November 20, 2013.
6. Guo J,Pandey S,Doyle J,Bian B,Lis Y,Raisch DW.A review of quantitative risk-benefit methodologies for assessing drug safety and efficacy: report of the ISPOR Risk-Benefit Management Working Group.Value Health. 2010;13:657-666
7. Boyd CM,Singh S,Varadhan R, et alMethods for Benefit and Harm Assessment in Systematic Reviews: Methods Research Report. Rockville, MD: Agency for Healthcare Research and Quality; 2012:
8. Mussen F,Salek S,Walker SRBenefit-Risk Appraisal of Medicines: A Systematic Approach to Decision Making. Surrey, England: Wiley; 2009:
9. Dodgson JS,Spackman M,Pearman A,Phillips LDMulti-criteria Analysis: A Manual. London, England: Department for Communities and Local Government; 2009:
10. Centre for Innovation in Regulatory Science. Visualising benefit-risk: the key to developing a framework that informs stakeholder perspective and clarity of decision making.http://www.cirsci.org/sites/default/files/CIRS_June_2011_Workshop.pdf. Published 2011. Accessed November 23, 2013.
11. Fischhoff B.Good decision making requires good communication.Drug Saf. 2012;35:983-993
12. US Food and Drug Administration. PDUFA re-authorisation performance goals and procedures fiscal years 2013 through 2017.http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf. Published 2012. Accessed November 23, 2013.
13. European Medicines Agency. Benefit-risk methodology project: work package 3 report. Field tests. http://www.emea.europa.eu/docs/en_GB/document_library/Report/2011/09/WC500112088.pdf. Published 2011. Accessed November 23, 2013.
14. European Medicines Agency. Benefit-risk methodology project: work package 4 report. Benefit-risk tools and processes. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2012/03/WC500123819.pdf. Published 2012. Accessed November 23, 2013.
15. Hammond JS,Keeney RL,Raiffa HSmart Choices: A Practical Guide to Making Better Decisions. Boston, MA: Harvard University Press; 1999:
16. Coplan PM,Noel R,Levitan B,Ferguson J,Mussen F.Development of a framework for enhancing the transparency, reproducibility and communication of the benefit-risk balance of medicines.Clin Pharmacol Ther. 2011;89:312-315
17. Levitan BS,Andrews EB,Gilsenan A, et al.Application of the BRAT framework to case studies: observations and insights.Clin Pharmacol Ther. 2011;89:217-224
18. Noel R,Hermann R,Levitan B,Watson DJ,Van Goor K.Application of the Benefit-Risk Action Team (BRAT) framework in pharmaceutical R&D: results from a pilot program.Drug Info J. 2012;46:736-743
19. Centre for Innovation in Regulatory Science. Refining the benefit-risk framework for the assessment of medicines: valuing and weighting benefit and risk parameters.http://cirsci.org/sites/default/files/CIRS_June2010_Workshop_Report.pdf. Published 2010. Accessed November 23, 2013.
20. Centre for Innovation in Regulatory Science. Building the benefit-risk toolbox: are there enough common elements across the different methodologies to enable a consensus on a scientifically acceptable framework for making benefit-risk decisions?http://www.cirsci.org/sites/default/files/CIRS%20June%202012%20Workshop%20Report%20low-res.pdf. Published 2012. Accessed November 23, 2013.
21. Philips LD,Fasolo B,Zafiropoulos N,Beyer A.Is quantitative benefit-risk modeling of drugs desirable or possible?.Drug Discov Today Technol. 2011;8:e3-e10
22. Walker S,Liberti L,McAuslane N.Refining the benefit-risk framework for the assessment of medicines: valuing and weighting benefit and risk parameters.Clin Pharmacol Ther. 2011;89:179-182
23. Mt-Isa S,Tzoulaki I,Callreus T,Micaleff A,Ashby D.Weighing benefit-risk of medicines: concepts and approaches.Drug Discov Today Technol. 2011;8:e29-e35