Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework

Social Science and Medicine

Volumn 188, Issue , 2017, Pages 137-156

  Angelis, Aris ^a   Kanavos, Panos ^a  

^a LONDON SCHOOL OF ECONOMICS   (United Kingdom)

Author keywords

Decision theory; European health policy; Health Technology Assessment; Multiple Criteria Decision Analysis; New medicines; Pharmaceuticals; Value Based Assessment; Value framework

Indexed keywords

ASSESSMENT METHOD; DRUG; HEALTH POLICY; MULTICRITERIA ANALYSIS;

ARTICLE; BIOMEDICAL TECHNOLOGY ASSESSMENT; DECISION MAKING; DECISION THEORY; DISEASE SEVERITY; DRUG COST; DRUG SAFETY; DRUG SCREENING; DRUG TOLERABILITY; HEALTH INSURANCE; MEDICAL EXPERT; METHODOLOGY; PATIENT COMPLIANCE; PROSPECTIVE PAYMENT; REIMBURSEMENT; SCIENTIFIC LITERATURE; SOCIOECONOMICS; TREATMENT OUTCOME; DECISION SUPPORT SYSTEM; DRUG APPROVAL; EVALUATION STUDY; HUMAN; PROCEDURES;

EUROPE;

DECISION MAKING; DECISION SUPPORT TECHNIQUES; DRUG APPROVAL; EVALUATION STUDIES AS TOPIC; HUMANS;

EID: 85026473174     PISSN: 02779536     EISSN: 18735347     Source Type: Journal    
DOI: 10.1016/j.socscimed.2017.06.024     Document Type: Article

References (156)

1. Advance-HTA, Advance-HTA Webpage. 2013 (www.advance-hta.eu).
2. Al, M.J., Feenstra, T.L., Hout, B.A.V., Optimal allocation of resources over health care programmes: dealing with decreasing marginal utility and uncertainty. Health Econ. 14 (2005), 655–667.
3. Amaya-Amaya, M., Gerard, K., Ryan, M., Discrete choice experiments in a nutshell. Ryan, M., Gerard, K., Amaya-Amaya, M., (eds.) Using Discrete Choice Experiments to Value Health and Health Care, 2008, Springer, Dordrecht, The Netherlands.
4. Anderson, J.L., Heidenreich, P.A., Barnett, P.G., Creager, M.A., Fonarow, G.C., Gibbons, R.J., et al. ACC/AHA statement on cost/value methodology in clinical practice guidelines and performance measures: a report of the American College of Cardiology/American Heart Association task force on performance measures and task force on practice guidelines. J. Am. Coll. Cardiol. 63 (2014), 2304–2322.
5. Angelis, A., Kanavos, P., Value Assessment Methods and Application of Multiple Criteria Decision Analysis for HTA. 2014, 1st Advance-HTA Capacity Building Workshop in Eastern Europe, Warsaw, Poland.
6. Angelis, A., Kanavos, P., Using Multiple Criteria Decision Analysis: Conceptual Framework and Model. 2015, 2nd Advance-HTA Capacity Building Workshop in Eastern Europe, Warsaw, Poland.
7. Angelis, A., Kanavos, P., Critique of the American Society of Clinical Oncology Value Assessment Framework for cancer treatments: putting methodologic robustness first. J. Clin. Oncol. 34:24 (August 2016), 2935–2936, 10.1200/JCO.2015.64.9673.
8. Angelis, A., Kanavos, P., Value-Based Assessment of new medical technologies: towards a robust methodological framework for the application of multiple criteria decision analysis in the context of Health Technology Assessment. Pharmacoeconomics 34 (2016), 435–446.
9. Angelis, A., Lange, A., Kanavos, P., Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries. Eur. J. Health Econ., 2017 Mar 16, 10.1007/s10198-017-0871-0.
10. Angelis, A., Kanavos, P., Montibeller, G., Resource allocation and priority setting in health care: a multi-criteria decision analysis problem of value?. Global Pol., 8(Suppl S2), March 2017, 10.1111/1758-5899.12387.
11. Antoñanzas, F., Terkola, R., Postma, M., The value of medicines: a crucial but vague concept. Pharmacoeconomics 34:12 (2016 Dec), 1227–1239.
12. Aronson, J.K., Ferner, R.E., Clarification of terminology in drug safety. Drug Saf. 28 (2005), 851–870.
13. Arrow, K., The economic implications of learning by doing. Rev. Econ. Stud., 29, 1962.
14. Bach, P., DrugAbacus App. 2015, Memorial Sloan Kettering Cancer Center (http://app.drugabacus.org/abacus-mskcc).
15. Baltussen, R., Niessen, L., Priority setting of health interventions: the need for multi-criteria decision analysis. Cost Eff. Resour. Alloc. C/E, 4, 2006 14–14.
16. Bana e Costa, C.A., De Corte, J.-M., Vansnick, J.-C., MACBETH. Int. J. Inf. Technol. Decis. Mak. 11 (2012), 359–387.
17. Bana e Costa, C.A., Oliveira, M.D., A multicriteria decision analysis model for faculty evaluation. Omega 40 (2012), 424–436.
18. Bana e Costa, C.A., Vansnick, J.-C., MACBETH — an interactive path towards the construction of cardinal value functions. Int. Trans. Oper. Res., 1, 1994, 489.
19. Bana e Costa, C.A., Vansnick, J.-C., Applications of the MACBETH approach in the framework of an additive aggregation model. J. Multi-Criteria Decis. Anal. 6 (1997), 107–114.
20. Bazerman, M., Judgment in Managerial Decision Making. 1998, John Wiley, New York.
21. Beauchamp, T.L., Methods and principles in biomedical ethics. J. Med. Ethics 29 (2003), 269–274.
22. Becker, M.H., Maiman, L.A., Strategies for enhancing patient compliance. J. Community Health 6 (1980), 113–135.
23. Belton, V., Stewart, T., Multiple Criteria Decision Analysis: an Integrated Approach. 2002, Kluwer Academic Publishers, Dordrecht.
24. Berkeley, D., Humphreys, P., Structuring decision problems and the ‘bias heuristic’. Acta Psychol. 50 (1982), 201–252.
25. Biomarkers Definitions Study Group, Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin. Pharmacol. Ther. 69 (2001), 89–95.
26. Bridges, et al. Conjoint analysis applications in health–a checklist: a report of the ISPOR good research practices for conjoint analysis task force. Value Health 14:4 (2011 Jun), 403–413, 10.1016/j.jval.2010.11.013.
27. Brouwer, W., Van Exel, J., Baker, R., Donaldson, C., The new myth. PharmacoEconomics 26 (2008), 1–4.
28. Buede, D.M., Structuring value attributes. Interfaces 16 (1986), 52–62.
29. Buyse, M., Molenberghs, G., Burzykowski, T., Renard, D., Geys, H., The validation of surrogate endpoints in meta-analyses of randomized experiments. Biostatistics 1 (2000), 49–67.
30. Centre for Reviews and Dissemination, Systematic Reviews: CRD's Guidance for Undertaking Reviews in Health Care. 2009, University of York.
31. Chen, T.-H., Li, L., Kochen, M.M., A systematic review: how to choose appropriate health-related quality of life (HRQOL) measures in routine general practice?. J. Zhejiang Univ. Sci. B 6 (2005), 936–940.
32. Cherepanov, D., Palta, M., Fryback, D.G., Underlying dimensions of the five health-related quality-of-life measures used in utility assessment: evidence from the National Health Measurement Study. Med. Care 48 (2010), 718–725.
33. Cherny, N.I., Sullivan, R., Dafni, U., Kerst, J.M., Sobrero, A., Zielinski, C., et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann. Oncol. Off. J. Eur. Soc. Med. Oncol./ESMO 26 (2015), 1547–1573.
34. Claxton, K., Should multi-criteria decision analysis (MCDA) replace cost effectiveness analysis (CEA) for evaluation of health care coverage decisions?. ISPOR 16th Annual European Congress, 2013 Dublin.
35. Claxton, K., Posnett, J., An economic approach to clinical trial design and research priority- setting. Health Econ. 5 (1996), 513–524.
36. Clemen, R.T., Making Hard Decisions; an Introduction to Decision Analysis. 1996, Belmont, Duxbury.
37. Cramer, J., Mattson, R., Cramer, J., Spilker, B., (eds.) Monitoring Compliance with Antiepileptic Drug Therapy, 1991, Raven Press, New York, 123–137.
38. de Bekker-Grob, E.W., Ryan, M., Gerard, K., Discrete choice experiments in health economics: a review of the literature. Health Econ. 21 (2012), 145–172.
39. Department for Communities and Local Government, Multi-criteria Analysis: a Manual. 2009.
40. DH, Department of Health, (eds.) A New Value-based Approach to the Pricing of Branded Medicines: a Consultation, 2010 London.
41. DH, Department of Health, (eds.) A New Value-based Approach to the Pricing of Branded Medicines: Government Response to Consultation, 2011 London.
42. Diaby, V., Goeree, R., How to use multi-criteria decision analysis methods for reimbursement decision-making in healthcare: a step-by-step guide. Expert Rev. Pharmacoeconomics Outcomes Res. 14 (2014), 81–99.
43. Dolan, J.G., Multi-criteria clinical decision support: a primer on the use of multiple criteria decision making methods to promote evidence-based, patient-centered healthcare. The Patient 3 (2010), 229–248.
44. Drummond, M., McGuire, A., Economic Evaluation in Health Care: Merging Theory with Practise. 2001, Oxford University Press, USA.
45. Drummond, M., Sculpher, M., Claxton, K., Stoddart, G., Torrance, G., Methods for the Economic Evaluation of Health Care Programmes. 2015, Oxford University Press.
46. European Commission, Commission Regulation (EC) No. 507/2006 on the conditional marketing authorization for medicinal products for human use. Eur. Comm. Off. J. Eur. Union, 30.3.2006 L 92/6–L92/9, Available via: http://ec.europa.eu/health//sites/health/files/files/eudralex/vol-1/reg_2006_507/reg_2006_507_en.pdf.
47. Eddy, D.M., Clinical decision making: from theory to practice. The individual vs society. Resolving the conflict. JAMA, 265, 1991, 2399.
48. Eldridge, S., Ashby, D., Bennett, C., Wakelin, M., Feder, G., Internal and external validity of cluster randomised trials: systematic review of recent trials. BMJ 336 (2008), 876–880.
49. EMA, Report of the CHMP Working Group on Benefit–risk Assessment Models and Methods., 2007, European Medicines Agency.
50. EMA, Reflection Paper on Benefit–risk Assessment Methods in the Context of the Evaluation of Marketing Authorisation Applications of Medicinal Products for Human Use. 2008, European Medicines Agency.
51. EMA, Benefit-risk Methodology Project. Work Package 3 Report: Field Tests., 2011, European Medicines Agency.
52. EMA, Benefit-risk Methodology Project. Work Package 4 Report: Benefit-risk Tools and Processes., 2012, European Medicines Agency.
53. Emanuel, E.J., Justice and managed care. Four principles for the just allocation of health care resources. Hastings Cent. Rep. 30 (2000), 8–16.
54. ®. Version 2.1 2015. European Network for Health Technology Assessment (https://meka.thl.fi/htacore/).
55. European Parliament, Regulation (EC) No 141/2000 of the European Parliament and of the Council of December 16, 1999 on orphan medicinal products. Eur. Parliam. Off. J. Eur. Communities 18 (22.1.2000), 1–9 Avilable via: http://ec.europa.eu/health//sites/health/files/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf.
56. Felli, et al. A multiattribute model for evaluating the benefit-risk profiles of treatment alternatives. Med. Decis. Making 29:1 (2009), 104–115.
57. FDA, Guidance for Industry: Premarketing Risk Assessment. 2005, US Food and Drug Administration, Center for Drug Evaluation and Research.
58. Fleck, L., Healthcare justice and rational democratic deliberation. Am. J. Bioeth. AJOB 1 (2001), 20–21.
59. Fleming, T., Design Issues in Colorectal Cancer Trials: Surrogate Endpoints and Non-inferiority Trials. 2003, Endpoints Workshop: Colorectal Cancer: Food and Drug Administration.
60. Franco, L., Montibeller, G., Problem structuring for multicriteria decision analysis interventions analysis interventions. Wiley Encycl. Oper. Res. Manag. Sci., 15 JUN 2010, 10.1002/9780470400531.eorms0683.
61. Gabrielli, W., von Winterfeldt, D., Are Importance Weights Sensitive to the Range of Alternatives in Multiattribute Utility Measurement?. Research report 78–6, 1978, University of Southern California, Social Science Research Institute.
62. George, J., Phun, Y.-T., Bailey, M.J., Kong, D.C.M., Stewart, K., Development and validation of the medication regimen complexity index. Ann. Pharmacother. 38 (2004), 1369–1376.
63. Glasgow, R., Vogt, T., Boles, S., Evaluating the public health impact of health promotion interventions: the RE-AIM framework. Am. J. Public Health Aff. 89 (1999), 1322–1327.
64. Godwin, M., Ruhland, L., Casson, I., MacDonald, S., Delva, D., Birtwhistle, R., et al. Pragmatic controlled clinical trials in primary care: the struggle between external and internal validity. BMC Med. Res. Methodol., 3, 2003 28–28.
65. Goetghebeur, M.M., Wagner, M., Khoury, H., Levitt, R.J., Erickson, L.J., Rindress, D., Evidence and value: impact on DEcisionMaking–the EVIDEM framework and potential applications. BMC Health Serv. Res., 8, 2008, 270.
66. Goetghebeur, et al. Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal. Med. Decis. Making 32:2 (2012 Mar-Apr), 376–388.
67. Goodwin, P., Wright, G., Decision Analysis for Management Judgement. 1997, John Wiley & Sons, Chichester.
68. Gordon, M., Kass, M., Validity of standard compliance measures in glaucoma compared with an electronic eyedrop monitor. Cramer, J., Spilker, B., (eds.) Patient Compliance in Medical Practice and Clinical Trials, 1991, Raven Press, New York, 163–173.
69. Guindo, L.A., Wagner, M., Baltussen, R., Rindress, D., van Til, J., Kind, P., et al. From efficacy to equity: literature review of decision criteria for resource allocation and healthcare decisionmaking. Cost Eff. Resour. Alloc. C/E, 10, 2012 9–9.
70. Guitouni, A., Martel, J.-M., Tentative guidelines to help choosing an appropriate MCDA method. Eur. J. Oper. Res. 109 (1998), 501–521.
71. Guyatt, G., Evidence-based medicine: a new approach to teaching the practice of medicine. JAMA, 268, 1992.
72. Guyatt, G.H., Feeny, D.H., Patrick, D.L., Measuring health-related quality of life. Ann. Intern. Med. 118 (1993), 622–629.
73. Guyatt, G.H., Oxman, A.D., Vist, G.E., Kunz, R., Falck-Ytter, Y., Alonso-Coello, P., et al. GRADE: an emerging consensus on rating quality of evidence and strength of recommendations. BMJ (Clin. Res. Ed.) 336 (2008), 924–926.
74. Hammond, J., Keeney, R., Raiffa, H., Smart Choices: a Practical Guide to Making Better Decisions. 1999, Harvard University Press, Cambridge.
75. Hansson, L.F., Norheim, O.F., Ruyter, K.W., Equality, explicitness, severity, and rigidity: the Oregon plan evaluated from a Scandinavian perspective. J. Med. Philos. 19 (1994), 343–366.
76. Higgins, J.P.T., Altman, D.G., Gøtzsche, P.C., Jüni, P., Moher, D., Oxman, A.D., et al. The Cochrane Collaboration's tool for assessing risk of bias in randomised trials. BMJ (Clin. Res. Ed.), 343, 2011 d5928–d5928.
77. HM, Treasury, The Green Book: Appraisal and Evaluation in Central Government: Treasury Guidance. 2003, TSO, London.
78. Hodgson, T.A., Meiners, M.R., Cost-of-illness methodology: a guide to current practices and procedures. Milbank Meml. Fund Q. Health Soc. 60 (1982), 429–462.
79. Howard, R.A., Decision Analysis: Applied Decision Theory. 1966, Wiley-Interscience.
80. Hughes-Wilson, et al. Paying for the orphan drug system: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?. Orphanet J. Rare Dis., 7, 2012 Sep 26, 74, 10.1186/1750-1172-7-74.
81. Johnson, F.R., Lancsar, E., Marshall, D., Kilambi, V., Mühlbacher, A., Regier, D.A., et al. Constructing experimental designs for discrete-choice experiments: report of the ISPOR conjoint analysis experimental design good research practices task force. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 16 (2013), 3–13.
82. Kanavos, P., Angelis, A., Value Assessment Methods and Application of Multiple Criteria Decision Analysis for HTA. 2014, 1st Advance-HTA Capacity Building Workshop in Latin America, Mexico City, Mexico.
83. Kanavos, P., Angelis, A., Using Multiple Criteria Decision Analysis: Conceptual Framework and Model. 2015, 2nd Advance-HTA Capacity Building Workshop in Latin America, Santiago, Chile.
84. Kass, M.A., Meltzer, D.W., Gordon, M., Cooper, D., Goldberg, J., Compliance with topical pilocarpine treatment. Am. J. Ophthalmol. 101 (1986), 515–523.
85. Keeney, R., Value Focused Thinking: a Path to Creative Decision Making. 1992, Harvard University Press, Cambridge.
86. Keeney, R., Raiffa, H., Decisions with Multiple Objectives: Preferences and Value Trade-offs. 1993, Cambridge University Press, Cambridge.
87. Keeney, R.L., Decision analysis: an overview. Oper. Res. 30 (1982), 803–838.
88. Keeney, R.L., Common mistakes in making value trade-offs. Oper. Res., 50, 2002, 935.
89. Keeney, R.L., Gregory, R.S., Selecting attributes to measure the achievement of objectives. Oper. Res. 53 (2005), 1–11.
90. Keeney, R.L., Raiffa, H., Decisions with Multiple Objectives: Performances and Value Trade-offs. 1976, Wiley, New York.
91. Kesselheim, A.S., Wang, B., Avorn, J., Defining “innovativeness” in drug development: a systematic review. Clin. Pharmacol. Ther. 94 (2013), 336–348.
92. Kruse, W., Weber, E., Dynamics of drug regimen compliance–its assessment by microprocessor-based monitoring. Eur. J. Clin. Pharmacol. 38 (1990), 561–565.
93. Linley, W.G., Hughes, D.A., Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain. Health Econ. 22 (2013), 948–964.
94. Lipsey, R., Carlow, K., Technology policy: basic concepts. Edquist, C., McKelvey, M., (eds.) Systems of Innovation: International Competitiveness, 2000, Edward Elgar, Cheltenham.
95. Malone, et al. International society for pharmacoeconomics and outcomes research comments on the American society of clinical oncology value framework. J. Clin. Oncol. 34:24 (August 2016), 2936–2937.
96. Marsh, K., Ijzerman, M., Thokala, P., Baltussen, R., Boysen, M., Kaló, Z., et al. Multiple criteria decision analysis for health care decision making-emerging good practices: report 2 of the ISPOR MCDA emerging good practices task force. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 19 (2016), 125–137.
97. Marsh, K., Lanitis, T., Neasham, D., Orfanos, P., Caro, J., Assessing the value of healthcare interventions using multi-criteria decision analysis: a review of the literature. PharmacoEconomics 32 (2014), 345–365.
98. Mauskopf, J.A., Sullivan, S.D., Annemans, L., Caro, J., Mullins, C.D., Nuijten, M., et al. Principles of good practice for budget impact analysis: report of the ISPOR Task Force on good research practices–budget impact analysis. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 10 (2007), 336–347.
99. McCabe, C., Claxton, K., Tsuchiya, A., Orphan drugs and the NHS: should we value rarity?. BMJ (Clin. Res. Ed.) 331 (2005), 1016–1019.
100. McDaniels, T.L., Gregory, R.S., Fields, D., Democratizing risk management: successful public involvement in local water management decisions. Risk Anal. Int. J. 19 (1999), 497–510.
101. Mestre-Ferrandiz, J., Mordoh, A., Sussex, J., The Many Faces of Innovation: a Report for the ABPI by the Office of Health Economics., 2012, Office of Health Economics.
102. Miners, A., Cairns, J., Waillo, A., Department of Health Proposals for Including Burden of Illness into Value Based Pricing: a Description and Critique. 2013, NICE Decision Support Unit.
103. Miners, A., Cairns, J., Wailoo, A., Department of Health Proposals for Including Wider Societal Benefits into Value Based Pricing: a Description and Critique. 2013, NICE Decision Support Unit.
104. Mirelman, A., Mentzakis, E., Kinter, E., Paolucci, F., Fordham, R., Ozawa, S., et al. Decision-making criteria among national policymakers in five countries: a discrete choice experiment eliciting relative preferences for equity and efficiency. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 15 (2012), 534–539.
105. Montibeller, G., Winterfeldt, D., Cognitive and motivational biases in decision and risk analysis. Risk Anal. Int. J. 35 (2015), 1230–1251.
106. Morgan, S., Lopert, R., Greyson, D., Toward a definition of pharmaceutical innovation. Open Med. A Peer-Reviewed, Indep. Open-Access J. 2 (2008), e4–e7.
107. Murphy, J., Coster, G., Issues in patient compliance. Drugs 54 (1997), 797–800.
108. Murray, C., Lopez, A., The Global Burden of Disease: Summary. 1996, Harvard School of Public Health on behalf of the World Health Organisation and the World Bank.
109. 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 (2007), S2–S15, 10.1002/pds.1435.
110. Mussen, F., Salek, S., Walker, S., Benefit–risk Appraisal of New Medicines: a Systematic Approach to Decision Making. 2009, John Willey, Surrey, UK.
111. Mussen, F., Salek, S., Walker, S., Phillips, L., A quantitative approach to benefit-risk assessment of medicines - part 2: the practical application of a new model. Pharmacoepidemiol. Drug Saf. 16:Suppl. 1 (2007), S16–S41.
112. National Cancer Institute, Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0. 2009, National Institutes of Health.
113. Nagy, V.T., Wolfe, G.R., Cognitive predictors of compliance in chronic disease patients. Med. Care 22 (1984), 912–921.
114. Naranjo, C.A., Busto, U., Sellers, E.M., Sandor, P., Ruiz, I., Roberts, E.A., et al. A method for estimating the probability of adverse drug reactions. Clin. Pharmacol. Ther. 30 (1981), 239–245.
115. ®) with NCCN Evidence Blocks™. 2015, National Comprehensive Cancer Network (https://www.nccn.org/evidenceblocks/).
116. Neumann, P.J., Cohen, J.T., Measuring the value of prescription drugs. N. Engl. J. Med. 373 (2015), 2595–2597.
117. NICE, Value Based Pricing: Response to the Department of Health Consultation. 2011, National Institute for Health and Clinical Excellence.
118. NICE, Consultation Paper: Value Based Assessment of Health Technologies. 2014, National Institute for Health and Care Excellence, London.
119. NICE, Responses to TA Methods Addendum Public Consultation. 2014, National Institute for Health and Care Excellence.
120. O'Brien, M.K., Petrie, K., Raeburn, J., Adherence to medication regimens: updating a complex medical issue. Med. Care Rev. 49 (1992), 435–454.
121. OHE, The Many Faces of Innovation. 2005, Office of Health Economics.
122. OHE, Response to the Department of Health's Consultation. 2011, Office of Health Economics.
123. Pazdur, Endpoints for assessing drug activity in clinical trials. Oncologist 13:Suppl 2 (2008), 19–21, 10.1634/theoncologist.13-S2-19.
124. Peacock, S.J., Richardson, J.R.J., Carter, R., Edwards, D., Priority setting in health care using multi- attribute utility theory and programme budgeting and marginal analysis (PBMA). Soc. Sci. Med. 64 (2007), 897–910.
125. Pearson, S., Framework for Payer Assessment of the Value of New Technologies: a US Approach. Seminar Briefing 16, 2015, Office of Health Economics.
126. Phillips, L., Fasolo, B., Zafiropoulos, N., Beyer, A., Is quantitative benefit-risk modelling of drugs desirable or possible?. Drug Discov. Today. Technol. 8 (2011), e1–e42.
127. Phillips, L.D., Bana e Costa, C.A., Transparent prioritisation, budgeting and resource allocation with multi-criteria decision analysis and decision conferencing. Ann. Oper. Res. 154 (2007), 51–68.
128. Porter, M.E., What is value in health care?. N. Engl. J. Med. 363 (2010), 2477–2481.
129. Postmus, D., Tervonen, T., van Valkenhoef, G., Hillege, H.L., Buskens, E., A multi-criteria decision analysis perspective on the health economic evaluation of medical interventions. Eur. J. Health Econ. 15 (2014), 709–716.
130. Raiffa, H., Decision Analysis: Introductory Lectures on Choices under Uncertainty. 1968, Addision-Wesley, Reading, MA.
131. Rawls, J., A Theory of Justice. 1999, Belknap Press of Harvard University.
132. Rosenberg, N., Inside the Black Box: Technology and Economics. 1982, Cambridge University Press, Cambridge.
133. Rosenberg, N., Keynote address: challenges for the social sciences in the new millennium. OECD Social Sciences and Innovation, 2001, OECD, Paris.
134. Rothwell, P.M., Factors that can affect the external validity of randomised controlled trials. PLoS Clin. Trials, 1, 2006, e9.
135. Rudd, P., Clinicians and patients with hypertension: unsettled issues about compliance. Am. Heart J. 130 (1995), 572–579.
136. Sackett, D.L., Rosenberg, W.M., Gray, J.A., Haynes, R.B., Richardson, W.S., Evidence based medicine: what it is and what it isn't. BMJ (Clin. Res. Ed.) 312 (1996), 71–72.
137. Savage, L., The Foundations of Statistics. 1954, Wiley, New York.
138. Schnipper, L.E., Davidson, N.E., Wollins, D.S., Tyne, C., Blayney, D.W., Blum, D., et al. American Society of Clinical Oncology statement: a conceptual framework to assess the value of cancer treatment options. J. Clin. Oncol. Off. J. Am. Soc. Clin. Oncol. 33 (2015), 2563–2577.
139. Shikiar, R., Rentz, A.M., Satisfaction with medication: an overview of conceptual, methodologic, and regulatory issues. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 7 (2004), 204–215.
140. Smith, N.A., Ley, P., Seale, J.P., Shaw, J., Health beliefs, satisfaction and compliance. Patient Educ. Couns. 10 (1987), 279–286.
141. Spahn, J., Clinical trial efficacy: what does it really tell you?. J. Allergy Clin. Immunol. 112 (2003), S102–S106.
142. Stewart, T.J., Simplified approaches for multicriteria decision making under uncertainty. J. Multi-Criteria Decis. Anal. 4 (1995), 246–258.
143. Sullivan, E. Clincal Trial Endpoints: Food and Drug Adminstration presentation. Food and Drug Adminstration. Accessed September 2013 (https://www.fda.gov/downloads/Training/ClinicalInvestigatorTrainingCourse/UCM283378.pdf).
144. Tanios, N., Wagner, M., Tony, M., Baltussen, R., van Til, J., Rindress, D., et al. Which criteria are considered in healthcare decisions? Insights from an international survey of policy and clinical decision makers. Int. J. Technol. Assess. Health Care 29 (2013), 456–465.
145. Tervonen, T., Naci, H., van Valkenhoef, G., Ades, A.E., Angelis, A., Hillege, H.L., et al. Applying multiple criteria decision analysis to comparative benefit-risk assessment: choosing among statins in primary prevention. Med. Decis. Mak. Int. J. Soc. Med. Decis. Mak. 35 (2015), 859–871.
146. Tervonen, T., van Valkenhoef, G., Buskens, E., Hillege, H.L., Postmus, D., A stochastic multicriteria model for evidence-based decision making in drug benefit-risk analysis. Stat. Med. 30 (2011), 1419–1428.
147. Thokala, P., Duenas, A., Multiple criteria decision analysis for health technology assessment. Value Health J. Int. Soc. Pharmacoeconomics Outcomes Res. 15 (2012), 1172–1181.
148. Tromp, N., Baltussen, R., Mapping of multiple criteria for priority setting of health interventions: an aid for decision makers. BMC Health Serv. Res., 12, 2012 454–454.
149. von Winterfeldt, D., Edwards, W., Decision Analysis and Behavioral Research. 1986, Cambridge University Press, Cambridge.
150. von Winterfeldt, D., Fasolo, B., Structuring decision problems: a case study and reflections for practitioners.Eur. J. Oper. Res.., 199, 2009, 857–866.
151. 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 (2009), 241–246.
152. Weinstein, M., Zeckhauser, R., Critical ratios and efficient allocation. J. Public Econ. 2 (1973), 147–157.
153. Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products, Process on Corporate Social Responsibility in the Field of Pharmaceuticals Platform on Access to Medicines in Europe. Final Report, 2013, European Commission, Brussels.
154. World Health Organisation, WHO Constitution. Geneva, 1948.
155. Wouters, O.J., Naci, H., Samani, N.J., QALYs in cost-effectiveness analysis: an overview for cardiologists. Heart (Br.h Card. Soc.) 101 (2015), 1868–1873.
156. Zhang, W., Bansback, N., Anis, A., Measuring and valuing productivity loss due to poor health: a critical review. Soc. Sci. Med. 72 (2011), 185–192.