Multi-criteria decision analysis for assessment and appraisal of orphan drugs

Frontiers in Public Health

Volumn 4, Issue SEP, 2016, Pages

  Iskrov, Georgi ^a,b   Miteva Katrandzhieva, Tsonka ^a,b   Stefanov, Rumen ^a,b  

^a MEDICAL UNIVERSITY OF PLOVDIV   (Bulgaria)

^b Institute of Rare Diseases   (Bulgaria)

Author keywords

Decision making; Health technology assessment; Multi criteria decision analysis; Orphan drugs; Rare diseases; Reimbursement

Indexed keywords

EID: 85015326662     PISSN: None     EISSN: 22962565     Source Type: Journal    
DOI: 10.3389/FPUBH.2016.00214     Document Type: Article

References (35)

1. Schnell-Inderst P, Mayer J, Lauterberg J, Hunger T, Arvandi M, Conrads-Frank A, et al. Health technology assessment of medical devices: what is different? An overview of three European projects. Z Evid Fortbild Qual Gesundhwes (2015) 109:309-18. doi:10.1016/j.zefq.2015.06.011
2. EUnetHTA. What is Health Technology Assessment (HTA). (2016). Available from: http://www.eunethta.eu/about-us/faq#t287n73
3. Wahlster P, Goetghebeur M, Kriza C, Niederländer C, Kolominsky-Rabas P; National Leading-Edge Cluster Medical Technologies 'Medical Valley EMN'. Balancing costs and benefits at different stages of medical innovation: a systematic review of multi-criteria decision analysis (MCDA). BMC Health Serv Res (2015) 15:262. doi:10.1186/s12913-015-0930-0
4. Iskrov G, Stefanov R. Criteria for drug reimbursement decision-making: an emerging public health challenge in Bulgaria. Balkan Med J (2016) 33:27-35. doi:10.5152/balkanmedj.2015.15185
5. Thokala P, Duenas A. Multiple criteria decision analysis for health technology assessment. Value Health (2012) 15:1172-81. doi:10.1016/j.jval.2012.06.015
6. Panzitta M, Bruno G, Giovagnoli S, Mendicino FR, Ricci M. Drug delivery system innovation and health technology assessment: upgrading from clinical to technological assessment. Int J Pharm (2015) 495:1005-18. doi:10.1016/j.ijpharm.2015.09.026
7. Wahlster P, Goetghebeur M, Schaller S, Kriza C, Kolominsky-Rabas P; National Leading-Edge Cluster Medical Technologies 'Medical Valley EMN'. Exploring the perspectives and preferences for HTA across German healthcare stakeholders using a multi-criteria assessment of a pulmonary heart sensor as a case study. Health Res Policy Syst (2015) 13:24. doi:10.1186/s12961-015-0011-1
8. Iskrov G, Kuncheva R, Stefanov R. Incorporation of multi-criteria decision analysis into health technology assessment: experiences and challenges from Bulgaria. In: Jakovljevic M, editor. Health Economics and Policy Challenges in Global Emerging Markets. New York, NY: Nova Science Publishers (2016). p. 41-58.
9. Rocchi A, Menon D, Verma S, Miller E. The role of economic evidence in Canadian oncology reimbursement decision-making: to lambda and beyond. Value Health (2008) 11:771-83. doi:10.1111/j.1524-4733.2007.00298.x
10. 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) 16:1163-9. doi:10.1016/j.jval.2013.10.002
11. Kolasa K, Zwolinski KM, Kalo Z, Hermanowski T. Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs. Orphanet J Rare Dis (2016) 11:23. doi:10.1186/s13023-016-0388-0
12. 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 (2016) 34:435-46. doi:10.1007/s40273-015-0370-z
13. Iskrov G, Dermendzhiev S, Miteva-Katrandzhieva T, Stefanov R. Health economic data in reimbursement of new medical technologies: importance of the socio-economic burden as a decision-making criterion. Front Pharmacol (2016) 7:252. doi:10.3389/fphar.2016.00252
14. European Union. Council recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02). OffJ Eur Union (2009) C171:7-10.
15. Iskrov G, Stefanov R. Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations. Orphan Drugs Res Rev (2014) 4:1-9. doi:10.2147/ODRR.S43409
16. Zelei T, Molnár MJ, Szegedi M, Kaló Z. Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries. Orphanet J Rare Dis (2016) 11:72. doi:10.1186/s13023-016-0455-6
17. Iskrov GG, Raycheva RD, Stefanov RS. Insight into reimbursement decision-making criteria in Bulgaria: implications for orphan drugs. Folia Med (Plovdiv) (2013) 55:80-6. doi:10.2478/folmed-2013-0032
18. Linertová R, Serrano-Aguilar P, Posada-de-la-Paz M, Hens-Pérez M, Kanavos P, Taruscio D, et al. Delphi approach to select rare diseases for a European representative survey. The BURQOL-RD study. Health Policy (2012) 108:19-26. doi:10.1016/j.healthpol.2012.08.001
19. López-Bastida J, Oliva-Moreno J, Linertová R, Serrano-Aguilar P. Social/economic costs and health-related quality of life in patients with rare diseases in Europe. Eur J Health Econ (2016) 17(Suppl 1):1-5. doi:10.1007/s10198-016-0780-7
20. Iskrov G, Jessop E, Miteva-Katrandzhieva T, Stefanov R. Budget impact of rare diseases: proposal for a theoretical framework based on evidence from Bulgaria. Georgian Med News (2015) 242:46-53.
21. Franken M, Stolk E, Scharringhausen T, de Boer A, Koopmanschap M. A comparative study of the role of disease severity in drug reimbursement decision making in four European countries. Health Policy (2015) 119:195-202. doi:10.1016/j.healthpol.2014.10.007
22. Radevic S, Kocic S, Jakovljevic M. Self-assessed health and socioeconomic inequalities in Serbia: data from 2013 National Health Survey. Front Pharmacol (2016) 7:140. doi:10.3389/fphar.2016.00140
23. van de Wetering EJ, Stolk EA, van Exel NJ, Brouwer WB. Balancing equity and efficiency in the Dutch basic benefits package using the principle of proportional shortfall. Eur J Health Econ (2013) 14:107-15. doi:10.1007/s10198-011-0346-7
24. Stolk EA, van Donselaar G, Brouwer WB, Busschbach JJ. Reconciliation of economic concerns and health policy: illustration of an equity adjustment procedure using proportional shortfall. Pharmacoeconomics (2004) 22:1097-107. doi:10.2165/00019053-200422170-00001
25. Eichler HG, Kong SX, Gerth WC, Mavros P, Jönsson B. Use of cost-effectiveness analysis in health-care resource allocation decision-making: how are cost-effectiveness thresholds expected to emerge? Value Health (2004) 7:518-28. doi:10.1111/j.1524-4733.2004.75003.x
26. Schnipper LE, Davidson NE, Wollins DS, Tyne C, Blayney DW, Blum D, et al. American Society of Clinical Oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol (2015) 33:2563-77. doi:10.1200/JCO.2015.61.6706
27. Niezen MG, de Bont A, Busschbach JJ, Cohen JP, Stolk EA. Finding legitimacy for the role of budget impact in drug reimbursement decisions. Int J Technol Assess Health Care (2009) 25:49-55. doi:10.1017/S0266462309090072
28. Mauskopf J, Chirila C, Masaquel C, Boye KS, Bowman L, Birt J, et al. Relationship between financial impact and coverage of drugs in Australia. Int J Technol Assess Health Care (2013) 29:92-100. doi:10.1017/S0266462312000724
29. Lorenc T, Tyner EF, Petticrew M, Duffy S, Martineau FP, Phillips G, et al. Cultures of evidence across policy sectors: systematic review of qualitative evidence. Eur J Public Health (2014) 24:1041-7. doi:10.1093/eurpub/cku038
30. Morel T, Arickx F, Befrits G, Siviero P, van der Meijden C, Xoxi E, et al. Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis (2013) 8:198. doi:10.1186/1750-1172-8-198
31. Iskrov G, Stefanov R. Prospects of risk-sharing agreements for innovative therapies in a context of deficit spending in Bulgaria. Front Public Health (2015) 3:64. doi:10.3389/fpubh.2015.00064
32. Garrison LP Jr, Towse A, Briggs A, de Pouvourville G, Grueger J, Mohr PE, 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):703-19. doi:10.1016/j.jval.2013.04.011
33. European Union. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. OffJ Eur Communities (2000) L18:1-5.
34. Jakovljevic M, Lazarevic M, Milovanovic O, Kanjevac T. The new and old Europe: east-west split in pharmaceutical spending. Front Pharmacol (2016) 7:18. doi:10.3389/fphar.2016.00018
35. Kaló Z, Gheorghe A, Huic M, Csanádi M, Kristensen FB. HTA implementation roadmap in central and eastern European countries. Health Econ (2016) 25(Suppl 1):179-92. doi:10.1002/hec.3298