Comprehensive value assessment of drugs using a multi-criteria decision analysis: An example of targeted therapies for metastatic colorectal cancer treatment

English version

Autoři: Jason C. Hsu ^aff001; Jia-Yu Lin ^aff002; Peng-Chan Lin ^aff003; Yang-Cheng Lee ^aff004
Působiště autorů: School of Pharmacy and Institute of Clinical Pharmacy and Pharmaceutical Sciences, College of Medicine, National Cheng Kung University, Tainan, Taiwan ^aff001; Department of Pharmacy, National Cheng Kung University Hospital, Tainan, Taiwan ^aff002; Department of Internal Medicine, National Cheng Kung University Hospital, Tainan, Taiwan ^aff003; Department of Internal Medicine, Tainan Municipal Hospital, Tainan, Taiwan ^aff004
Vyšlo v časopise: PLoS ONE 14(12)
Kategorie: Research Article
prolekare.web.journal.doi_sk: https://doi.org/10.1371/journal.pone.0225938

Souhrn

Objective

This study is aimed toward establishing a decision-making model with multiple criteria for appraisal and reimbursement to compare the attitudes of different stakeholders toward various dimensions and criteria and to evaluate the five targeted therapies (bevacizumab, cetuximab, panitumumab, aflibercept, and regorafenib) for metastatic colorectal cancer.

Method

This study is a multi-criteria decision analysis (MCDA) using a model that includes three dimensions and nine criteria. Both the overall and individual scores of the respective targeted therapies in different dimensions and criteria were calculated. A sensitivity analysis was carried out in order to evaluate the robustness of the research results. An interview-based questionnaire survey was applied to obtain the performance information for the targeted therapies and the weights of the dimensions and criteria.

Results

Overall, the clinical dimension had the highest weight, followed by the economic dimension, and finally, the social dimension. In the clinical dimension, the “comparative efficacy” criterion had the highest weight; in the economic dimension, the “cost-effectiveness” criterion” was given the greatest importance; in the social dimension, the “social concern and patient needs” criterion was given more emphasis. The overall values ranked from high to low as follows: cetuximab (overall score 3.3666), bevacizumab (3.3043), panitumumab (3.2030), aflibercept (2.8923) and regorafenib (2.8366).

Conclusions

A comprehensive value assessment system combining “multi-dimensional criteria,” “multi-perspectives,” and an “integrative assessment” is necessary to evaluate the value of medicines. The results showed not only the order of weights of different dimensions or criteria, but also the rankings of the value of the targeted therapies.

Klíčová slova:

Economics – Drug therapy – Cancer treatment – Drug research and development – Cost-effectiveness analysis – Health insurance – Colorectal cancer – Drug policy

Zdroje

1. Sorenson C, Lavezzari G, Daniel G, Burkholder R, Boutin M, Pezalla E, et al. Advancing Value Assessment in the United States: A Multistakeholder Perspective. Value Health. 2017;20(2):299–307. Epub 2017/02/27. doi: 10.1016/j.jval.2016.11.030 28237214.

2. Finkelstein EA, Krishnan A, Doble B. Beyond cost-effectiveness: A five-step framework for appraising the value of health technologies in Asia-Pacific. Int J Health Plann Manage. 2019. doi: 10.1002/hpm.2851 31290187.

3. World Health Organization. Operational principles for good pharmaceutical procurement: Strategic objectives for good pharmaceutical procurement 1999 [cited 2018 November]. http://www.who.int/3by5/en/who-edm-par-99-5.pdf.

4. Johannesen KM, Claxton K, Sculpher MJ, Wailoo AJ. How to design the cost-effectiveness appraisal process of new healthcare technologies to maximise population health: A conceptual framework. Health Econ. 2018;27(2):e41–e54. doi: 10.1002/hec.3561 28833844.

5. van Baal P, Meltzer D, Brouwer W. Future Costs, Fixed Healthcare Budgets, and the Decision Rules of Cost-Effectiveness Analysis. Health Econ. 2016;25(2):237–48. doi: 10.1002/hec.3138 25533778.

6. International Society for Pharmacoeconomics and Outcomes Research. US Value Assessment Frameworks Initiative Documents Draft White Paper, Section 2 Value and Perspective: An Overview 2017 [updated July 7, 2017; cited 2018 November]. https://www.ispor.org/USValueAssessmentFrameworks/STF-White-Paper-Section2-DRAFT-for-Member-Review.pdf.

7. Antonijevic ZE. Optimization of Pharmaceutical R&D Programs and Portfolios Chapter 2 Clinical Aspects of Pharmaceutical Portfolio Management2015.

8. Mt-Isa S, Hallgreen CE, Wang N, Callreus T, Genov G, Hirsch I, et al. Balancing benefit and risk of medicines: a systematic review and classification of available methodologies. Pharmacoepidemiol Drug Saf. 2014;23(7):667–78. Epub 2014/05/14. doi: 10.1002/pds.3636 24821575.

9. 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. 2014;32(4):345–65. Epub 2014/02/08. doi: 10.1007/s40273-014-0135-0 24504851.

10. Kaur G, Prinja S, Lakshmi PVM, Downey L, Sharma D, Teerawattananon Y. Criteria Used for Priority-Setting for Public Health Resource Allocation in Low- and Middle-Income Countries: A Systematic Review. Int J Technol Assess Health Care. 2019:1–10. doi: 10.1017/S0266462319000473 31307561.

11. Le Gales C, Moatti JP. Searching for consensus through multi-criteria decision analysis. Assessment of screening strategies for hemoglobinopathies in southeastern France. Int J Technol Assess Health Care. 1990;6(3):430–49. Epub 1990/01/01. doi: 10.1017/s0266462300001033 2228458.

12. Dolan JG, Bordley DR. Isoniazid prophylaxis: the importance of individual values. Med Decis Making. 1994;14(1):1–8. Epub 1994/01/01. doi: 10.1177/0272989X9401400101 8152352.

13. Perez Encinas M, Fernandez MA, Martin ML, Calvo MV, Gomez-Alonso A, Dominguez-Gil A, et al. Multicriteria decision analysis for determining drug therapy for intermittent claudication. Methods Find Exp Clin Pharmacol. 1998;20(5):425–31. Epub 1998/08/14. doi: 10.1358/mf.1998.20.5.485704 9701781.

14. Wenstop F, Magnus P. Value focused rationality in AIDS policy. Health Policy. 2001;57(1):57–72. Epub 2001/05/12. doi: 10.1016/s0168-8510(01)00120-8 11348694.

15. Diaby V, Lachaine J. An application of a proposed framework for formulary listing in low-income countries: the case of Cote d'Ivoire. Appl Health Econ Health Policy. 2011;9(6):389–402. Epub 2011/10/25. doi: 10.2165/11595220-000000000-00000 22017499.

16. Danner M, Hummel JM, Volz F, van Manen JG, Wiegard B, Dintsios CM, 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):369–75. Epub 2011/10/19. doi: 10.1017/S0266462311000523 22004779.

17. Goetghebeur MM, Wagner M, Khoury H, Levitt RJ, Erickson LJ, Rindress D. 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. 2012;32(2):376–88. doi: 10.1177/0272989X11416870 21987539.

18. Youngkong S, Baltussen R, Tantivess S, Mohara A, Teerawattananon Y. Multicriteria decision analysis for including health interventions in the universal health coverage benefit package in Thailand. Value Health. 2012;15(6):961–70. Epub 2012/09/25. doi: 10.1016/j.jval.2012.06.006 22999148.

19. Assessment HT. What is health technology assessment: Center for Drug Evaluation, Taiwan; 2015 [cited 2018 November]. http://nihta.cde.org.tw/About/.

20. Erbitux(R) [package insert]. Merck KGaA, Germany, September 2015. [cited 2018 November].

21. Avastin(R) [package insert]. F. Hoffmann-La Roche Ltd, Switzerland, June 2015. [cited 2018 November].

22. Vectibix(R) [package insert]. Amgen Manufacturing Limited, Puerto Rico, March 2015. [cited 2018 November].

23. Zaltrap(R) [package insert]. Sanofi-aventis, Germany, April 2015. [cited 2018 November].

24. Stivarga(R) [package insert]. Bayer Pharma AG, Germany, April 2015. [cited 2018 November].

25. Marsh K, M IJ, Thokala P, Baltussen R, Boysen M, Kalo 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. 2016;19(2):125–37. doi: 10.1016/j.jval.2015.12.016 27021745.

26. Saaty TL. The Analytic Hierarchy Process. McGraw-Hill, New York 1980.

27. Lynn MR. Determination and quantification of content validity. Nurs Res. 1986;35(6):382–5. Epub 1986/11/01. 3640358.

28. Waltz CWaRBB. Nursing research: design, statistics and computer analysis. Philadelphia: F.A. Davis; 1981.

29. Thokala P, Devlin N, Marsh K, Baltussen R, Boysen M, Kalo Z, et al. Multiple Criteria Decision Analysis for Health Care Decision Making—An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health. 2016;19(1):1–13. doi: 10.1016/j.jval.2015.12.003 26797229.

30. Sussex J, Rollet P, Garau M, Schmitt C, Kent A, Hutchings A. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013;16(8):1163–9. doi: 10.1016/j.jval.2013.10.002 24326170.

31. Gilabert-Perramon A, Torrent-Farnell J, Catalan A, Prat A, Fontanet M, Puig-Peiro R, et al. Drug Evaluation and Decision Making in Catalonia: Development and Validation of a Methodological Framework Based on Multi-Criteria Decision Analysis (Mcda) for Orphan Drugs. Int J Technol Assess Health Care. 2017;33(1):111–20. doi: 10.1017/S0266462317000149 28434413.

32. Joint decision-making meeting and method for universal health insurance drug payment projects and payment standards. website: https://wwwnhigovtw/Content_Listaspx?n=748D4A0C0839220C&topn=3FC7D09599D25979 (access Aug 31, 2019).

33. Center for Drug Evaluation, Health Technology Assessment Methodology Guide 2014 [cited 2018 November]. https://www.ispor.org/PEguidelines/source/HTA_guidelines_Taiwan.pdf.

34. Saltz LB, Clarke S, Diaz-Rubio E, Scheithauer W, Figer A, Wong R, et al. Bevacizumab in combination with oxaliplatin-based chemotherapy as first-line therapy in metastatic colorectal cancer: a randomized phase III study. J Clin Oncol. 2008;26(12):2013–9. doi: 10.1200/JCO.2007.14.9930 18421054.

35. Heinemann V, von Weikersthal LF, Decker T, Kiani A, Vehling-Kaiser U, Al-Batran SE, et al. FOLFIRI plus cetuximab versus FOLFIRI plus bevacizumab as first-line treatment for patients with metastatic colorectal cancer (FIRE-3): a randomised, open-label, phase 3 trial. Lancet Oncol. 2014;15(10):1065–75. doi: 10.1016/S1470-2045(14)70330-4 25088940.

36. Van Cutsem E, Kohne CH, Hitre E, Zaluski J, Chang Chien CR, Makhson A, et al. Cetuximab and chemotherapy as initial treatment for metastatic colorectal cancer. N Engl J Med. 2009;360(14):1408–17. doi: 10.1056/NEJMoa0805019 19339720.

37. Bokemeyer C, Bondarenko I, Hartmann JT, de Braud F, Schuch G, Zubel A, et al. Efficacy according to biomarker status of cetuximab plus FOLFOX-4 as first-line treatment for metastatic colorectal cancer: the OPUS study. Ann Oncol. 2011;22(7):1535–46. doi: 10.1093/annonc/mdq632 21228335.

38. Cunningham D, Humblet Y, Siena S, Khayat D, Bleiberg H, Santoro A, et al. Cetuximab monotherapy and cetuximab plus irinotecan in irinotecan-refractory metastatic colorectal cancer. N Engl J Med. 2004;351(4):337–45. doi: 10.1056/NEJMoa033025 15269313.

39. Van Cutsem E, Peeters M, Siena S, Humblet Y, Hendlisz A, Neyns B, et al. Open-label phase III trial of panitumumab plus best supportive care compared with best supportive care alone in patients with chemotherapy-refractory metastatic colorectal cancer. J Clin Oncol. 2007;25(13):1658–64. doi: 10.1200/JCO.2006.08.1620 17470858.

40. Peeters M, Price TJ, Cervantes A, Sobrero AF, Ducreux M, Hotko Y, et al. Randomized phase III study of panitumumab with fluorouracil, leucovorin, and irinotecan (FOLFIRI) compared with FOLFIRI alone as second-line treatment in patients with metastatic colorectal cancer. J Clin Oncol. 2010;28(31):4706–13. doi: 10.1200/JCO.2009.27.6055 20921462.

41. Van Cutsem E, Tabernero J, Lakomy R, Prenen H, Prausova J, Macarulla T, et al. Addition of aflibercept to fluorouracil, leucovorin, and irinotecan improves survival in a phase III randomized trial in patients with metastatic colorectal cancer previously treated with an oxaliplatin-based regimen. J Clin Oncol. 2012;30(28):3499–506. doi: 10.1200/JCO.2012.42.8201 22949147.

42. Grothey A, Van Cutsem E, Sobrero A, Siena S, Falcone A, Ychou M, et al. Regorafenib monotherapy for previously treated metastatic colorectal cancer (CORRECT): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2013;381(9863):303–12. doi: 10.1016/S0140-6736(12)61900-X 23177514.

43. Li J, Qin S, Xu R, Yau TC, Ma B, Pan H, et al. Regorafenib plus best supportive care versus placebo plus best supportive care in Asian patients with previously treated metastatic colorectal cancer (CONCUR): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2015;16(6):619–29. doi: 10.1016/S1470-2045(15)70156-7 25981818.

44. Cunningham D, Lang I, Marcuello E, Lorusso V, Ocvirk J, Shin DB, et al. Bevacizumab plus capecitabine versus capecitabine alone in elderly patients with previously untreated metastatic colorectal cancer (AVEX): an open-label, randomised phase 3 trial. Lancet Oncol. 2013;14(11):1077–85. doi: 10.1016/S1470-2045(13)70154-2 24028813.

45. Loupakis F, Cremolini C, Masi G, Lonardi S, Zagonel V, Salvatore L, et al. Initial therapy with FOLFOXIRI and bevacizumab for metastatic colorectal cancer. N Engl J Med. 2014;371(17):1609–18. doi: 10.1056/NEJMoa1403108 25337750.

46. Cremolini C, Loupakis F, Antoniotti C, Lupi C, Sensi E, Lonardi S, et al. FOLFOXIRI plus bevacizumab versus FOLFIRI plus bevacizumab as first-line treatment of patients with metastatic colorectal cancer: updated overall survival and molecular subgroup analyses of the open-label, phase 3 TRIBE study. Lancet Oncol. 2015;16(13):1306–15. doi: 10.1016/S1470-2045(15)00122-9 26338525.

47. Bokemeyer C, Bondarenko I, Makhson A, Hartmann JT, Aparicio J, de Braud F, et al. Fluorouracil, leucovorin, and oxaliplatin with and without cetuximab in the first-line treatment of metastatic colorectal cancer. J Clin Oncol. 2009;27(5):663–71. doi: 10.1200/JCO.2008.20.8397 19114683.

48. Van Cutsem E, Lenz HJ, Kohne CH, Heinemann V, Tejpar S, Melezinek I, et al. Fluorouracil, leucovorin, and irinotecan plus cetuximab treatment and RAS mutations in colorectal cancer. J Clin Oncol. 2015;33(7):692–700. doi: 10.1200/JCO.2014.59.4812 25605843.

49. Taiwan National health insurance drug search system. website: https://www1nhigovtw/QueryN/Query1aspx (access: Aug 31, 2019).

50. Oliveira MD, Mataloto I, Kanavos P. Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art. Eur J Health Econ. 2019;20(6):891–918. doi: 10.1007/s10198-019-01052-3 31006056.

51. Marsh K, Thokala P, Youngkong S, Chalkidou K. Incorporating MCDA into HTA: challenges and potential solutions, with a focus on lower income settings. Cost Eff Resour Alloc. 2018;16(Suppl 1):43. doi: 10.1186/s12962-018-0125-8 30455602.

52. Terkola R, Antonanzas F, Postma M. Stakeholder opinions on value in healthcare. Eur J Hosp Pharm. 2019;26(2):79–84. doi: 10.1136/ejhpharm-2017-001295 31157104.

53. Soprovich A, El Kurdi S, Eurich DT. An analysis of redactions in Canada’s Common Drug Review Clinical Review Reports and how they relate to the patients’ voice. BMJ Open. 2017;7(9):e015497. doi: 10.1136/bmjopen-2016-015497 28893743.

54. V.; B, T. S. Multiple criteria decision analysis: an integrated approach: Springer Science & Business Media. 2002.

55. Angelis A, Kanavos P. Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework. Soc Sci Med. 2017;188:137–56. doi: 10.1016/j.socscimed.2017.06.024 28772164.

56. Angelis A, Montibeller G, Hochhauser D, Kanavos P. Multiple criteria decision analysis in the context of health technology assessment: a simulation exercise on metastatic colorectal cancer with multiple stakeholders in the English setting. BMC Med Inform Decis Mak. 2017;17(1):149. doi: 10.1186/s12911-017-0524-3 29073892.