The efficiency-frontier approach for health economic evaluation versus cost-effectiveness thresholds and internal reference pricing: combining the best of both worlds?

References

• Weisbrod BA. The health care quadrilemma: an essay on technological change, insurance, quality of care, and cost containment. J Econ Lit. 1991;29(2):523–552.
   Web of Science ®Google Scholar
• Paris V, Belloni A, Value in pharmaceutical pricing. OECD Health Working Papers, No. 63. 2013, Paris: OECD Publishing.
   Google Scholar
• Deutscher Bundestag. Gesetzentwurf der Bundesregierung: entwurf eines Gesetzes zur Strukturreform im Gesundheitswesen (Gesundheits-Reformgesetz - GRG); Drucksache 11/2493. 1988; Available from: dipbt.bundestag.de/doc/btd/11/024/1102493.pdf.
   Google Scholar
• Busse R, Schreyogg J, Henke KD. Regulation of pharmaceutical markets in Germany: improving efficiency and controlling expenditures? Int J Health Plann Manage. 2005;20(4):329–349.
   PubMed Web of Science ®Google Scholar
• Vd Schulenburg JM. The German health care system at the crossroads. Health Econ. 1994;3(5):301–303.
   PubMed Web of Science ®Google Scholar
• Deutscher Bundestag. Antwort der Bundesregierung auf die Kleine Anfrage der Abgeordneten Birgitt Bender, Elisabeth Scharfenberg, Dr. Harald Terpe, Kerstin Andreae und der Fraktion BÜNDNIS 90/DIE GRÜNEN; Drucksache 16/4927; Ausgestaltung der Kosten-Nutzen-Bewertung von Arzneimitteln; Drucksache 16/5027. 2007 [cited 2014 Apr 02]; Available from: http://dipbt.bundestag.de/dip21/btd/16/050/1605027.pdf.
   Google Scholar
• Deutscher Bundestag. Gesetzentwurf der Fraktionen der CDU/CSU und SPD: entwurf eines Gesetzes zur Stärkung des Wettbewerbs in der gesetzlichen Krankenversicherung (GKV-Wettbewerbsstärkungsgesetz – GKV-WSG); Drucksache 16/3100. 2006 2014 Apr 02]; Available from: http://dip21.bundestag.de/dip21/btd/16/031/1603100.pdf.
   Google Scholar
• National Institute for Health and Care Excellence. Guide to the methods of technology appraisal 2013. London, UK: NICE; 2013.
   Google Scholar
• Schwarzer R, Rochau U, Saverno K, et al. Systematic overview of cost-effectiveness thresholds in ten countries across four continents. J Comp Eff Res. 2015;4(5):485–504.
   PubMed Web of Science ®Google Scholar
• Deutscher Ethikrat. Nutzen und Kosten im Gesundheitswesen: zur normativen Funktion ihrer Bewertung; Stellungnahme. Berlin: Deutscher Ethikrat; 2011.
   Google Scholar
• Dietz U. Kosten-Nutzen-Bewertung: ein Kommentar von Ulrich Dietz, Leiter des Referats Arzneimittelversorgung, Bundesministerium für Gesundheit. Gesundheitswesen. 2009;71(S 01):S52–S53.
   Google Scholar
• Deter G. Die Kosten-Nutzen-Bewertung von Arzneimitteln als Rechtsproblem. Medizinrecht. 2010;28(4):249–255.
   Google Scholar
• Caro JJ, Nord E, Siebert U, et al. The efficiency frontier approach to economic evaluation of health-care interventions. Health Econ. 2010;19(10):1117–1127.
   PubMed Web of Science ®Google Scholar
• Institute for Quality and Efficiency in Health Care. General methods for the assessment of the relation of benefits to costs. 2009 19.Nov. [cited 2014 Mar 14]; Available from: https://www.iqwig.de/download/General_Methods_for_the_Assessment_of_the_Relation_of_Benefits_to_Costs.pdf.
   Google Scholar
• Institute for Quality and Efficiency in Health Care. General methods: version 4.2. Cologne: IQWiG; 2015.
   Google Scholar
• Deutscher Bundestag. Gesetzentwurf der Fraktionen der CDU/CSU und FDP: entwurf eines Gesetzes zur Neuordnung des Arzneimittelmarktes in der gesetzlichen Krankenversicherung Arzneimittelmarktneuordnungsgesetz – AMNOG); Drucksache 17/2413. 2010 [cited 2014 Apr 02]; Available from: http://dip21.bundestag.de/dip21/btd/17/024/1702413.pdf.
   Google Scholar
• Drummond M, Jönsson B, Rutten F, et al. Reimbursement of pharmaceuticals: reference pricing versus health technology assessment. Eur J Health Econ. 2011;12(3):263–271.
   PubMed Web of Science ®Google Scholar
• Carone G, Schwierz C, Xavier A, Cost-containment policies in public pharmaceutical spending in the EU. Economic Papers 461. 2012, Brussels: European Union.
   Google Scholar
• Acosta A, Ciapponi A, Aaserud M, et al. Pharmaceutical policies: effects of reference pricing, other pricing, and purchasing policies. Cochrane Database Syst Rev. 2014;(10):CD005979.
   PubMed Web of Science ®Google Scholar
• Bos JM, Postma MJ. Using pharmacoeconomics for policy making: is rational decision making enhanced by applying thresholds for cost-effectiveness? Expert Rev Pharmacoecon Outcomes Res. 2004;4(3):247–250.
   PubMedGoogle Scholar
• Neumann PJ, Cohen JT, Weinstein MC. Updating cost-effectiveness–the curious resilience of the $50,000-per-QALY threshold. N Engl J Med. 2014;371(9):796–797.
   PubMed Web of Science ®Google Scholar
• Institute for Quality and Efficiency in Health Care. Health economic evaluation of venlafaxine, duloxetine, bupropion, and mirtazapine compared to further prescribable pharmaceutical treatments: documentation and evaluation of comments on the preliminary report; commission no. G09-01 [German]. 2013; Available from: https://www.iqwig.de/download/G09-01_DWAV_Kosten-Nutzen-Bewertung-von-Venlafaxin-Duloxetin-Bupropion-u….pdf.
   Google Scholar
• Brouwer WBF, Rutten FFH. The efficiency frontier approach to economic evaluation: will it help german policy making? Health Econ. 2010;19(10):1128–1131.
   PubMed Web of Science ®Google Scholar
• Sculpher M, Claxton K. Sins of omission and obfuscation: iQWiG’s guidelines on economic evaluation methods. Health Econ. 2010;19(10):1132–1136.
   PubMed Web of Science ®Google Scholar
• Wasem J, Kosten-Nutzen-Bewertung von Arzneimitteln: Eine unvermeidbare Abwägung, in Deutsches Ärzteblatt.
   Google Scholar
• Webb DJ, Walker A. Value-based pricing of drugs in the UK. Lancet. 2007;369(9571):1415–1416.
   PubMed Web of Science ®Google Scholar
• Claxton K, Briggs A, Buxton MJ, et al. Value based pricing for NHS drugs: an opportunity not to be missed? BMJ. 2008;336(7638):251–254.
   PubMed Web of Science ®Google Scholar
• Gandjour A. Presenting Germany’s drug pricing rule as a cost-per-QALY rule. Health Care Manag Sci. 2012;15(2):103–107.
   PubMed Web of Science ®Google Scholar
• Institute for Quality and Efficiency in Health Care. Health economic evaluation of venlafaxine, duloxetine, bupropion, and mirtazapine compared to further prescribable pharmaceutical treatments: final report; commission no. G09-01. 2013; Available from: https://www.iqwig.de/download/G09-01_Executive-summary-of-final-report_Health-economic-evaluation-of-venlafaxine-duloxetine-bupropion-mirtazapine.pdf.
   Google Scholar
• Weinstein M, Zeckhauser R. Critical ratios and efficient allocation. J Public Econ. 1973;2(2):147–157.
   Google Scholar
• Weinstein MC. A QALY is a QALY–or is it? J Health Econ. 1988;7(3):289–290.
   PubMed Web of Science ®Google Scholar
• Detsky AS, Laupacis A. Relevance of cost-effectiveness analysis to clinicians and policy makers. JAMA. 2007;298(2):221–224.
   PubMed Web of Science ®Google Scholar
• Grocott R. Applying programme budgeting marginal analysis in the health sector: 12 years of experience. Expert Rev Pharmacoecon Outcomes Res. 2009;9(2):181–187.
   PubMedGoogle Scholar
• Bertram MY, Lauer JA, De Joncheere K, et al. Cost-effectiveness thresholds: pros and cons. Bull World Health Organ. 2016;94(12):925–930.
   PubMed Web of Science ®Google Scholar
• Dolan P, Shaw R, Tsuchiya A, et al. QALY maximisation and people’s preferences: a methodological review of the literature. Health Econ. 2005;14(2):197–208.
   PubMed Web of Science ®Google Scholar
• Gafni A, Birch S. Incremental cost-effectiveness ratios (ICERs): the silence of the lambda. Soc Sci Med. 2006;62(9):2091–2100.
   PubMed Web of Science ®Google Scholar
• Claxton K, Sculpher M, Palmer S, et al. Causes for concern: is NICE failing to uphold its responsibilities to all NHS patients? Health Econ. 2015;24(1):1–7.
   PubMed Web of Science ®Google Scholar
• Klingler C, Shah SMB, Barron AJG, et al. Regulatory space and the contextual mediation of common functional pressures: analyzing the factors that led to the German Efficiency Frontier approach. Health Policy. 2013;109(3):270–280.
   PubMed Web of Science ®Google Scholar
• Institute for Quality and Efficiency in Health Care. General methods: version 5.0. Cologne: IQWiG; 2017.
   Google Scholar
• Neyt M, Van Brabandt H. The importance of the comparator in economic evaluations working on the efficiency frontier. Pharmacoeconomics. 2011;29(11):913–916.
   PubMed Web of Science ®Google Scholar
• Drummond MF, Sculpher MJ, Torrance GW, et al. Methods for the economic evaluation of health care programme.. 3rd ed. Oxford: Oxford University Press; 2005.
   Google Scholar
• Rebscher H, Schellhammer S, Kopp T, et al. [Evaluation of cancer therapy from the perspective of a statutory health insurance]. Urologe A. 2011;50(12):1584–1590.
   PubMedGoogle Scholar
• Anderson JP, Bush JW, Chen M, et al. Policy space areas and properties of benefit-cost/utility analysis. JAMA. 1986;255(6):794–795.
   PubMed Web of Science ®Google Scholar
• Black WC. The CE plane: a graphic representation of cost-effectiveness. Med Decis Making. 1990;10(3):212–214.
   PubMed Web of Science ®Google Scholar
• Weinstein MC, Torrance G, McGuire A. QALYs: the basics. Value Health. 2009;12(Suppl 1):S5–S9.
   PubMed Web of Science ®Google Scholar
• Danner M, Hummel JM, Volz F, et al. Integrating patients’ views into health technology assessment: analytic hierarchy process (AHP) as a method to elicit patient preferences. Int J Technol Assess Health Care. 2011;27(4):369–375.
   PubMed Web of Science ®Google Scholar
• Marsh K, Lanitis T, Neasham D, et al. Assessing the value of healthcare interventions using multi-criteria decision analysis: a review of the literature. Pharmacoeconomics. 2014;32(4):345–365.
   PubMed Web of Science ®Google Scholar
• Caro JJ. Methods of economic evaluation for the German statutory healthcare system. Pharmacoeconomics. 2009;27(3):263–264.
   PubMedGoogle Scholar
• Hoyle M. Accounting for the drug life cycle and future drug prices in cost-effectiveness analysis. Pharmacoeconomics. 2011;29(1):1–15.
   PubMed Web of Science ®Google Scholar
• Greiner W, Kuhlmann A, Schwarzbach C. Ökonomische Beurteilung des Effizienzgrenzenkonzeptes. Gesundh ökon Qual manag. 2010;15(05):241–250.
   Google Scholar
• Birch S, Gafni A. Information created to evade reality (ICER): things we should not look to for answers. Pharmacoeconomics. 2006;24(11):1121–1131.
   PubMed Web of Science ®Google Scholar
• Gandjour A, Gafni A. The German method for setting ceiling prices for drugs: in some cases less data are required. Expert Rev Pharmacoecon Outcomes Res. 2011;11(4):403–409.
   PubMed Web of Science ®Google Scholar
• Gandjour A, Gafni A, Schlander M. Determining the price for pharmaceuticals in Germany: comparing a shortcut for IQWiG’s efficiency frontier method with the price set by the manufacturer for ticagrelor. Expert Rev Pharmacoecon Outcomes Res. 2014;14(1):123–129.
   PubMed Web of Science ®Google Scholar
• Davis S, Assessing technologies that are not cost-effective at a zero price. NICE Decision Support Unit Methods Development. 2014, London: National Institute for Health and Care Excellence (NICE).
   Google Scholar
• Cleemput I, Neyt M, Van de Sande S, et al. Belgian guidelines for economic evaluations and budget impact analyses: second edition. KCE Reports 2012 [cited 2014 Apr 02]; 183C:[ Available from: http://kce.fgov.be/sites/default/files/page_documents/KCE_183C_economic_evaluations_second_edition_0.pdf.
   Google Scholar
• Haute Autorité de Santé. A methodological guide: choices in methods for economic evaluation. 2012 [cited 2014 Apr 02]; Available from: http://www.has-sante.fr/portail/upload/docs/application/pdf/2012-10/choices_in_methods_for_economic_evaluation.pdf.
   Google Scholar
• Drummond M. Twenty years of using economic evaluations for drug reimbursement decisions: what has been achieved? J Health Polit Policy Law. 2013;38(6):1081–1102.
   PubMed Web of Science ®Google Scholar
• Carrera P, Mj IJ. Are current ICER thresholds outdated? Valuing medicines in the era of personalized healthcare. Expert Rev Pharmacoecon Outcomes Res. 2016;16(4):435–437.
   PubMed Web of Science ®Google Scholar
• Farrell MJ. The measurement of productive efficiency. J R Stat Soc Ser A Stat Soc. 1957;120:253–290.
   Web of Science ®Google Scholar
• Karlsson G, Johannesson M. The decision rules of cost-effectiveness analysis. Pharmacoeconomics. 1996;9(2):113–120.
   PubMed Web of Science ®Google Scholar
• Goeree R, O’Brien BJ, Blackhouse G, et al. Cost-effectiveness and cost-utility of long-term management strategies for heartburn. Value in Health. 2002;5(4):312–328.
   PubMed Web of Science ®Google Scholar
• Murray CJ, Lauer JA, Hutubessy RCW, et al. Effectiveness and costs of interventions to lower systolic blood pressure and cholesterol: a global and regional analysis on reduction of cardiovascular-disease risk. Lancet. 2003;361(9359):717–725.
   PubMed Web of Science ®Google Scholar
• Barton GR, Briggs AH, Fenwick EAL. Optimal cost-effectiveness decisions: the role of the cost-effectiveness acceptability curve (CEAC), the cost-effectiveness acceptability frontier (CEAF), and the expected value of perfection information (EVPI). Value in Health. 2008;11(5):886–897.
   PubMed Web of Science ®Google Scholar
• Kamae MS, Kamae I, Cohen JT, et al. Regression analysis on the variation in efficiency frontiers for prevention stage of HIV/AIDS. J Med Econ. 2011;14(2):187–193.
   PubMedGoogle Scholar
• Zeckhauser R, Shepard D. Where now for saving lives? Law Contemp Probl. 1976;40(4):5–45.
   Google Scholar
• Weinstein MC, Stason WB. Foundations of cost-effectiveness analysis for health and medical practices. N Engl J Med. 1977;296(13):716–721.
   PubMed Web of Science ®Google Scholar
• Stapf-Finé H. Das IQWiG-Institut im Blickpunkt: umstrittene Kosten-Nutzen-Bewertung von Arzneien. Soziale Sicherheit. 2008;6-7:227–232.
   Google Scholar
• Neumann PJ, Greenberg D. Is the United States ready for QALYs? Health Aff (Millwood). 2009;28(5):1366–1371.
   PubMed Web of Science ®Google Scholar
• Gerber A, Dintsios CM. A distorted picture of IQWiG methodology. Health Aff (Millwood). 2010;29(1):220–221.
   PubMed Web of Science ®Google Scholar
• Dintsios CM, Gerber A. Some essential clarifications: iQWiG comments on two critiques of the efficiency frontier approach. Health Econ. 2010;19(10):1139–1141.
   PubMed Web of Science ®Google Scholar
• Sandmann F, Gerber-Grote A, Lhachimi S. Factors that led to the implementation of the efficiency frontier approach in health economic evaluation in Germany: do not avoid the elephant in the room. Comment on Klingler et al. (Health Policy 109 (2013) 270-280). Health Policy. 2013;112(3):297–298.
   PubMed Web of Science ®Google Scholar
• Klingler C, Shah SMB, Barron AJG, et al. Factors that led to the implementation of the efficiency frontier approach to health economic evaluation in Germany: let’s talk more about the elephant. Health Policy. 2013;112(3):299–300.
   PubMed Web of Science ®Google Scholar
• Gandjour A. Drug pricing and control of health expenditures: a comparison between a proportional decision rule and a cost-per-QALY rule. Int J Health Plann Manage. 2015;30(4):395–402.
   PubMed Web of Science ®Google Scholar
• Muhlbacher AC, Sadler A. The probabilistic efficiency frontier: a framework for cost-effectiveness analysis in Germany put into practice for hepatitis C treatment options. Value Health. 2017;20(2):266–272.
   PubMed Web of Science ®Google Scholar
• World Health Organization. Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research. Copenhagen: WHO Regional Office for Europe; 2015.
   Google Scholar
• Stollenwerk, B., Lhachimi SK, Briggs A, et al. Communicating the parameter uncertainty in the IQWiG efficiency frontier to decision-makers. Health Econ. 2015 Apr;24(4):481–490. doi: 10.1002/hec.3041.
   Google Scholar
• Corro Ramos, I., Lhachimi SK, Gerber-Grote A, et al. Cost recommendation under uncertainty in IQWiG’s efficiency frontier framework. Med Decis Making. 2017 Feb;37(2):162–172. doi: 10.1177/0272989X16636856.
   Google Scholar
• Briggs AH. Handling uncertainty in cost-effectiveness models. Pharmacoeconomics. 2000;17(5):479–500.
   PubMed Web of Science ®Google Scholar
• Briggs AH, Weinstein MC, Fenwick EAL, et al. Model parameter estimation and uncertainty analysis: a report of the ISPOR-SMDM modeling good research practices task force working group-6. Med Decis Making. 2012;32(5):722–732.
   PubMed Web of Science ®Google Scholar
• McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ. 2005;331(7523):1016–1019.
   PubMedGoogle Scholar
• Franken M, Koopmanschap M, Steenhoek A. Health economic evaluations in reimbursement decision making in the Netherlands: time to take it seriously? Z Evid Fortbild Qual Gesundhwes. 2014;108(7):383–389.
   PubMedGoogle Scholar
• Ognyanova D, Zentner A, Busse R. Pharmaceutical reform 2010 in Germany. Eurohealth. 2011;17(1):11–13.
   Google Scholar
• Gerber A, Stock S, Dintsios CM. Reflections on the changing face of German pharmaceutical policy: how far is Germany from value-based pricing? Pharmacoeconomics. 2011;29(7):549–553.
   PubMed Web of Science ®Google Scholar
• Horn H, Nink K, McGauran N, et al. Early benefit assessment of new drugs in Germany - Results from 2011 to 2012. Health Policy. 2014;116(2–3):147–153.
   PubMed Web of Science ®Google Scholar
• Gerber-Grote A, Sandmann FG, Zhou M, et al. Decision making in Germany: is health economic evaluation as a supporting tool a sleeping beauty? Zeitschrift für Evidenz, Fortbildung und Qualität im Gesundheitswesen. 2014;108(7):390–396.
   PubMedGoogle Scholar
• Lauenroth VD, Stargardt T. Pharmaceutical pricing in Germany: how is value determined within the scope of AMNOG? Value Health. 2017;20(7):927–935.
   PubMed Web of Science ®Google Scholar
• Franken M, Nilsson F, Sandmann F, et al. Unravelling drug reimbursement outcomes: a comparative study of the role of pharmacoeconomic evidence in Dutch and Swedish reimbursement decision making. Pharmacoeconomics. 2013;31(9):781–797.
   PubMed Web of Science ®Google Scholar
• Garrison LP Jr., Towse A, Briggs A, et al. Performance-based risk-sharing arrangements-good practices for design, implementation, and evaluation: report of the ISPOR good practices for performance-based risk-sharing arrangements task force. Value Health. 2013;16(5):703–719.
   PubMed Web of Science ®Google Scholar
• Folland S, Goodman AC, Stano M. The economics of health and health care. Vol. 6. New Jersey: Pearson Prentice Hall; 2010.
   Google Scholar