The impact of conventional cost-effectiveness analysis on pricing dynamics in the market of new medicines: a proposed countervailing approach

References

• Kantarjian HM, Fojo T, Mathisen M, et al. Cancer drugs in the United States: justum pretium—the just price. J clin oncol. 2013;31:3600.
   PubMed Web of Science ®Google Scholar
• Howard DH, Bach PB, Berndt ER, et al. Pricing in the market for anticancer drugs. J Econ Perspect. 2015;29:139–162
   Google Scholar
• Light DW, Kantarjian H. Market spiral pricing of cancer drugs. Cancer. 2013;119:3900–3902.
   PubMed Web of Science ®Google Scholar
• Wait S, Han D, Muthu V, et al. Towards sustainable cancer care: reducing inefficiencies, improving outcomes—A policy report from the All.Can initiative. J Cancer Policy. 2017;13:47–64.
   Web of Science ®Google Scholar
• Ghinea N, Lipworth W, Kerridge I. Propaganda or the cost of innovation? Challenging the high price of new drugs. BMJ. 2016;352.
   Google Scholar
• Light DW, Warburton R. Demythologizing the high costs of pharmaceutical research. Undefined. 2011;6:34–50.
   Google Scholar
• Avorn J. The $2.6 billion pill — methodologic and policy considerations. N Engl J Med. 2015;372:1877–1879.
   PubMed Web of Science ®Google Scholar
• Morgan S, Grootendorst P, Lexchin J, et al. The cost of drug development: a systematic review. Health Policy (New York). 2011;100:4–17.
   PubMed Web of Science ®Google Scholar
• Schlander M, Hernandez-Villafuerte K, Cheng CY, et al. How much does it cost to research and develop a new drug? A systematic review and assessment. Pharmacoeconomics. 2021;39:1243–1269.
   PubMed Web of Science ®Google Scholar
• Elkin EB, Bach PB. Cancer’s next frontier: addressing high and increasing costs. JAMA. 2010;303:1086–1087.
   PubMed Web of Science ®Google Scholar
• Morgan SG, Bathula HS, Moon S. Pricing of pharmaceuticals is becoming a major challenge for health systems. In: BMJ. 2020. p. 368.
   Google Scholar
• Capri S. High prices of new drugs: we are ready to do whatever it takes LIUC Papers in Economics. BMJ. 2020;368:l4627.
   PubMedGoogle Scholar
• Balancing Innovation and Drug Pricing - Drug Pricing Lab [Internet]. [cited 2023 Jan 20]. Available from: https://www.drugpricinglab.org/issue/balancing-innovation-and-drug-pricing/
   Google Scholar
• Claxton K, Briggs A, Buxton M, et al. Value based pricing for {NHS} drugs: an opportunity not to be missed? BMJ. 2008;336:252–254.
   Google Scholar
• Danzon P, Towse A, Value-Based Differential M-FJ. Pricing: efficient prices for drugs in a global context. Health Econ. 2015;24:294–301.
   PubMed Web of Science ®Google Scholar
• Kiernan F. The future of pharmacoeconomic policy - does value-based pricing really have a role? J Pharm Health Serv Res. 2016;7:5–9.
   Google Scholar
• Chandra A, Garthwaite C. The economics of indication-based drug pricing. N Engl J Med. 2017;377:103–106.
   PubMed Web of Science ®Google Scholar
• Mestre-Ferrandiz J, Zozaya N, Alcalá B, et al. Multi-indication pricing: nice in theory but can it work in practice? Pharmacoeconomics. 2018;36:1407–1420.
   PubMed Web of Science ®Google Scholar
• Antonanzas F, Juarez-Castello C, Rodriguez-Ibeas R. Should health authorities offer risk-sharing contracts to pharmaceutical firms? A theoretical approach. Health Econ Policy Law. 2011;6:391–403.
   PubMedGoogle Scholar
• Levaggi R, Pertile P. Which valued-based price when patients are heterogeneous? Health Econ Internet]. 2020 [cited 2021 Oct 26];29:923–935
   PubMedGoogle Scholar
• Haslam A, Kim MS, Prasad V. Overall survival for oncology drugs approved for genomic indications. Eur J Cancer. 2022;160:175–179.
   PubMedGoogle Scholar
• Mailankody S, Prasad V. Five years of cancer drug approvals: innovation, efficacy, and costs. JAMA Oncol. 2015;1:539–540.
   PubMed Web of Science ®Google Scholar
• Vivot A, Boutron I, Béraud-Chaulet G, et al. Evidence for treatment-by-biomarker interaction for FDA-approved oncology drugs with required pharmacogenomic biomarker testing. Sci Rep. 2017:7.
   PubMedGoogle Scholar
• Prasad V, Mailankody S. Research and development spending to bring a single cancer drug to market and revenues after approval. JAMA Intern Med. 2017;177:1569–1575.
   PubMed Web of Science ®Google Scholar
• Yu N, Helms Z, Bach PB. R&D costs for pharmaceutical companies do not explain elevated US drug prices. In: Health affairs blog. Health Affairs; 2017. DOI:10.1377/forefront.20170307.059036.
   Google Scholar
• Kaltenboeck A, Bach PB. Value-based pricing for drugs: theme and variations. JAMA. 2018;319:2165–2166.
   PubMed Web of Science ®Google Scholar
• OECD Health Division. Addressing challenges in access to oncology medicines analytical report [internet]. 2020 [cited 2022 Feb 17]. Available from: https://www.oecd.org/health/health-systems/addressing-challenges-in-access-to-oncology-medicines.htm.
   Google Scholar
• Levaggi L, Levaggi R. Value-based drug price schemes: a welfare analysis. J Pharm Health Serv Res Internet]. 2021 [cited 2022 Jan 24];12:357–362
   Google Scholar
• Haycox A. Why Cancer? Pharmacoeconomics. 2016;34:625–627.
   PubMed Web of Science ®Google Scholar
• Luzzatto L, Hyry HI, Schieppati A, et al. Outrageous prices of orphan drugs: a call for collaboration. Lancet Internet]. 2018 [cited 2022 Sep 20];392:791–794. Available from
   PubMed Web of Science ®Google Scholar
• Simoens S, Picavet E, Dooms M, et al. Cost-effectiveness assessment of orphan drugs a scientific and political conundrum. 2012 [cited 2022 Sep 20]; Available from: http://www.cvz.nl/binaries/.
   Google Scholar
• Workman P, Draetta GF, Schellens JHM, et al. How much longer will we put up with 100,000 cancer drugs? Cell Internet]. 2017 [cited 2022 Sep 26];168:579–583. Available from
   PubMed Web of Science ®Google Scholar
• Hollis A Orphan drug pricing and costs: a case study of kalydeco and orkambi tarification et coûts des médicaments orphelins: étude de cas sur le Kalydeco et l’Orkambi. 70] HEALTHCARE POLICY. 2019.
   Google Scholar
• Capri S, Antonanzas F. Efficiency ratio and rocketing drug prices: old concerns and new possibilities. Eur J Health Econ. 2020;21:1273–1277.
   PubMedGoogle Scholar
• Demartino PC, Miljković MD, Prasad V. Potential cost implications for all US food and drug administration oncology drug approvals in 2018. JAMA Intern Med. 2021;181(2):162–167. DOI:10.1001/jamainternmed.2020.5921.
   PubMed Web of Science ®Google Scholar
• Franzen N, Retèl VP, Schats W, et al. Evidence underlying policy proposals for sustainable anticancer drug prices: a systematic review. JAMA Oncol. 2020;6:909–916.
   PubMed Web of Science ®Google Scholar
• Delos Santos S, Witzke N, Arciero VS, et al. Reassessing the net health benefit (NHB) of FDA approved cancer drugs with evolution of evidence using the American Society of Clinical Oncology Value Framework (ASCO-VF). J Natl Compr Canc Netw. 2021;19(7):815–820. DOI:10.6004/jnccn.2020.7677.
   PubMedGoogle Scholar
• Raymakers AJN, Regier DA, Peacock SJ. Health-related quality of life in oncology drug reimbursement submissions in Canada: a review of submissions to the pan-Canadian Oncology Drug Review. Cancer. 2020;126(1):148–155. DOI:10.1002/cncr.32455.
   PubMed Web of Science ®Google Scholar
• Thomson S, Everest L, Witzke N, et al. Examining the relationship between the clinical benefit of oncology drug indications and the time from pan-Canadian Oncology Drug Review (pCODR) recommendation to public reimbursement. J Clin Oncol. 2020;38:e19360–e19360.
   Google Scholar
• Godman B, Hill A, Simoens S, et al. Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications. Expert Rev Pharmacoecon Outcomes Res Internet]. 2021 [cited 2022 Sep 26];21:527–540. Available from.;:.
   PubMed Web of Science ®Google Scholar
• Chi YL, Blecher M, Chalkidou K, et al. What next after GDP-based cost-effectiveness thresholds? Gates Open Res. 2020;4:176.
   PubMedGoogle Scholar
• Ferguson JSJ, Summerhayes M, Masters S, et al. New treatments for advanced cancer: an approach to prioritization. Br J Cancer Internet]. 2000 [cited 2022;83:1268.
   PubMed Web of Science ®Google Scholar
• Pontes C, Zara C, Torrent-Farnell J, et al. Time to review authorisation and funding for new cancer medicines in Europe? Inferences from the case of olaratumab. Appl Health Econ Health Policy Internet]. [cited 2022 Sep 26];18:5–16. Available from. 2019
   Google Scholar
• Pisana A, Wettermark B, Kurdi A, et al. Challenges and opportunities with routinely collected data on the utilization of cancer medicines. perspectives from health authority personnel across 18 European countries. front pharmacol [internet]. 2022 [cited 2022 Sep 26];13. Available from: https://pubmed.ncbi.nlm.nih.gov/35865969/.
   Google Scholar
• Jena AB, Philipson T. Cost-effectiveness analysis and innovation. J Health Econ. 2008;27:1224–1236.
   PubMedGoogle Scholar
• Coyle D, Buxton MJ, O’Brien BJ. Stratified cost-effectiveness analysis: a framework for establishing efficient limited use criteria. Health Econ. 2003;12:421–427.
   PubMed Web of Science ®Google Scholar
• Jena AB, Philipson TJ. Endogenous cost-effectiveness analysis and health care technology adoption. J Health Econ. 2013;32:172–180.
   PubMedGoogle Scholar
• Hawkins N, Scott DA. Reimbursement and value-based pricing: stratified cost-effectiveness analysis may not be the last word. Health Econ. 2011;20:688–698.
   PubMedGoogle Scholar
• Levaggi R, Moretto M, Pertile P. Static and dynamic efficiency of irreversible health care investments under alternative payment rules. J Health Econ. 2012;31:169–179.
   PubMedGoogle Scholar
• Mouelhi Y, Jouve E, Castelli C, et al. How is the minimal clinically important difference established in health-related quality of life instruments? Review of anchors and methods. Health Qual [Internet]. 2020;18(1):136. DOI:10.1186/s12955-020-01344-w.
   Google Scholar
• Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol Internet]. 2003 [cited 2022 Jun 1];56:395–407
   PubMed Web of Science ®Google Scholar
• Levaggi R. Pricing schemes for new drugs: a welfare analysis. Soc Sci Med Internet]. 2014;102:69–73
   PubMedGoogle Scholar
• Refoios Camejo R, Miraldo M, Rutten F. Cost-effectiveness and dynamic efficiency: does the solution lie within? Value Health Internet]. 2017 [cited 2021 Feb 15];20:240–243
   PubMedGoogle Scholar
• Mortimer D. The value of thinly spread QALYs. Pharmacoeconomics Internet]. 2006 [cited 2022 Jun 1];24:845–853. Available from.;:.
   PubMed Web of Science ®Google Scholar
• Levaggi R, Value-Based Pricing PP. Alternatives for personalised drugs: implications of asymmetric information and competition. Appl Health Econ Health Policy. 2020;18:357–362.
   PubMedGoogle Scholar
• Hlávka JP, Yu JC, Goldman DP, et al. The economics of alternative payment models for pharmaceuticals. Eur J Health Econ Internet]. 2021 [cited 2021 Aug 4];22:559–569
   PubMedGoogle Scholar
• Saluja R, Jiao T, Koshy L, et al. Comparing manufacturer submitted and pan-Canadian oncology drug review reanalysed incremental cost-effectiveness ratios for novel oncology drugs. Curr Oncol Internet]. 2021 [cited 2022 Jun 16];28:606–618
   PubMedGoogle Scholar
• Levaggi R, Pertile P Drug prices and incentives to innovation by the pharmaceutical industry. Pharmaceutical prices in the 21st century [Internet]. 2015. p. 389–401. [cited 2023 Jan 20].Available from: http://www.scopus.com/inward/record.url?eid=2-s2.0-84943396525&partnerID=MN8TOARS
   Google Scholar
• Filson DA. Markov-perfect equilibrium model of the impacts of price controls on the performance of the pharmaceutical industry. RAND J Econ. 2012;43:110–138.
   Google Scholar
• Gravelle HSE. Ex post Value Reimbursement for Pharmaceuticals. Med Decis Making Internet]. 1998;18:S27–S38.
   PubMedGoogle Scholar
• Prescription drug prices in the United States Are 2.56 times those in other countries | RAND [internet]. [cited 2022 Sep 30]. Available from: https://www.rand.org/news/press/2021/01/28.html.
   Google Scholar
• Danzon PM. Differential Pricing of Pharmaceuticals: theory, Evidence and Emerging Issues. Pharmacoeconomics. 2018: [Internet]. 123AD; 36:1395–1405
   PubMed Web of Science ®Google Scholar
• Lindgren P, Löfvendahl S, Brådvik G, et al. Value appropriation in hepatitis C. Eur J Health Econ Internet]. 2022 [cited 2022 Sep 26];23:1059–1070. Available from
   PubMedGoogle Scholar
• Lakdawalla DN, Phelps CE. Health technology assessment with diminishing returns to health: the generalized risk-adjusted cost-effectiveness (GRACE) approach. Value Health. 2021 Feb;24(2):244–249.
   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
• Grosse SD. Assessing cost-effectiveness in healthcare: history of the $50,000 per QALY threshold. Expert Rev Pharmacoeconomics Outcomes Res. 2008;8:165–178.
   PubMedGoogle Scholar
• Culyer A, McCabe C, Briggs A, et al. Searching for a threshold, not setting one: the role of the National institute for health and clinical excellence. J Health Serv Res Policy. 2007;12:56–58.
   PubMedGoogle Scholar
• Claxton K, Martin S, Soares M, et al. Methods for the estimation of the national institute for health and care excellence cost-effectiveness threshold. Health Technol Assess (Rockv). 2015;19:1–503.
   PubMed Web of Science ®Google Scholar
• Schwarzer R, Rochau U, Saverno K, et al. Systematic overview of cost–effectiveness thresholds in ten countries across four continents. 2015;4(5):485–504. DOI:10.2217/cer.15.38.
   Google Scholar
• Brouwer W, van Baal P, van Exel J, et al. When is it too expensive? Cost-effectiveness thresholds and health care decision-making. Eur J Health Econ. 2019 [2018 feb 20];20(2):175–180.
   PubMed Web of Science ®Google Scholar
• Paluri RK, Kasi A, Young C, et al. Second-line treatment for metastatic pancreatic cancer. Clinical advances in hematology & oncology. 2020;18(2):1–10.
   Google Scholar