Multi-criteria decision analysis (MCDA): Testing a proposed MCDA framework for orphan drugs

Orphanet Journal of Rare Diseases

Volumn 12, Issue 1, 2017, Pages

  Schey, C ^a,b   Krabbe, P F M ^c   Postma, M J ^a,c   Connolly, M P ^a,b  

^a UNIVERSITY OF GRONINGEN   (Netherlands)

^b Global Market Access Solutions (GMAS)   (Switzerland)

^c UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

Author keywords

Lennox Gastaut syndrome; MCDA; Mucopolysaccharidosis II; Multi criteria decision analysis; Myelodysplastic syndromes; Orphan drugs; Paroxysmal nocturnal haemoglobinuria; Pulmonary arterial hypertension; Reimbursement

Indexed keywords

ORPHAN DRUG;

ADULT; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DRUG COST; DRUG LEGISLATION; FOLLOW UP; HUMAN; MULTI CRITERIA DECISION ANALYSIS; REIMBURSEMENT; SCORING SYSTEM; DECISION SUPPORT SYSTEM; DRUG DEVELOPMENT; DRUG MANUFACTURE; HUNTER SYNDROME; LENNOX GASTAUT SYNDROME; METABOLISM; MYELODYSPLASTIC SYNDROME; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PULMONARY HYPERTENSION;

DECISION SUPPORT TECHNIQUES; DRUG DISCOVERY; HEMOGLOBINURIA, PAROXYSMAL; HYPERTENSION, PULMONARY; LENNOX GASTAUT SYNDROME; MUCOPOLYSACCHARIDOSIS II; MYELODYSPLASTIC SYNDROMES; ORPHAN DRUG PRODUCTION;

EID: 85011258323     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-016-0555-3     Document Type: Article

References (41)

1. Drummond MF. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007;23(1):36-42.
2. Scottish Medicines Consortium. Alglucosidase alfa (Myozyme®): https://www.scottishmedicines.org.uk/SMC-Advice/Advice/alglucosidase-alfa-50mg-powder-Myozyme--352-07-/alglucosidase-alfa-Myozyme--352-07-. Accessed Apr 2016
3. Picavet E, Cassiman D, Simoens S. What is known about the cost-effectiveness of orphan drugs? Evidence form cost-utility analyses. J Clin Pharm Ther. 2015;40:304-7.
4. Sorenson C, Drummond M, Kanavos P. Ensuring value for money in healthcare. The role of health technology assessment in the European Union. European Observatory on Health Systems and Policies. 2008: http://www.euro.who.int/-data/assets/pdf-file/0011/98291/E91271.pdf. Accessed Mar 2016
5. Cleemput I, Neyt M, Thiry N, De Laet C, Leys M. Using threshold values for cost per quality-adjusted life-year gained in healthcare decisions. Int J Technol Assess Health Care. 2011;27(1):71-6. http://www.eurohex.eu/bibliography/pdf/Cleemput-2011-2235638016/Cleemput-2011.pdf. Accessed Mar 2016.
6. Appleby J, Devlin N, Parkin D. NICE's cost effectiveness threshold. BMJ. 2007;335:358-9.
7. Cohen JP, Felix A. Are payers treating orphan drugs differently? J Market Access Health Policy. 2014;2:23513. http://www.jmahp.net/index.php/jmahp/article/download/23513/31873.
8. Hughes-Wilson W, 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. 2012;7:74. http://www.ojrd.com/content/7/1/74. Accessed Oct 2013.
9. Belton V, Stewart TJ. Multiple criteria decision analysis: an integrated approach. Dordrecht: Kluwer; 2002.
10. Carver SJ. Integrating multi-criteria evaluation with geographical information systems. Int J Geogr Inf Syst. 1991;5(3):321-39.
11. Hallerbach W, Spronk J. The relevance of MCDM for financial decisions. J Multi-Criteria Decis Anal. 2003;11(4/5):187-95.
12. Linkov I, Moberg E. Multi-criteria decision analysis: environmental applications and case studies. 2013. CRC Press. Taylor & Francis Group. ISBN 9781439853184 - CAT# K12453.
13. Youngkong S, Teerawattananon Y, Tantivess S, Baltussen R. Multi-criteria decision analysis for setting priorities on HIV/AIDS interventions in Thailand. Health Res Policy Syst. 2012;10:6. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3310796/.
14. Baltussen R, Mikkelsen E, Tromp N, Hurtig A, Byskov J, Olsen O, Baeroe K, Hontelez JA, Singh J, Norheim O. Balancing efficiency, equity and feasbility of HIV treatment in South Africa - development of programmatic guidance. Cost Eff Resour Alloc. 2013;11:26. http://www.resource-allocation.com/content/11/1/26.
15. Angelis A, Kanavos P. Applying Multiple Criteria Decision Analysis (MCDA) in the context of HTA: an experimental case study on metastatic colorectal cancer. J Pharm Policy Pract. 2015;8 Suppl 1:O8. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4602173/. Accessed Mar 2016.
16. National Institute for Health and Care Excellence. NICE to assess high cost drugs for rare conditions: https://www.nice.org.uk/News/Article/nice-to-assess-high-cost-drugs-for-rare-conditions. Accessed Mar 2016
17. Barham L. Orphan Medicines. Special Treatment required? 2020 Health.org: http://socialwelfare.bl.uk/subject-areas/services-activity/health-services/2020health/147277orphan-medicines-2012.pdf. Accessed Mar 2016
18. Thokala P, Devlin N, Marsh K, Baltussen R, Boysen M, Kalo Z, Longrenn T, Mussen F, Peacock S, Watkins J, Ijzerman M. 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;19:1-13. https://www.ispor.org/Multi-Criteria-Decision-Analysis-guideline.pdf. Accessed Mar 2016.
19. Simoens S. Market access of orphan drugs and the role of multi-criteria decision making. Orphanet J Rare Dis. 2012;7 Suppl 2:A26. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3504597/. Accessed Mar 2016.
20. Johri M. Can Cost-Effectiveness Analysis Integrate Concerns For Equity? Systematic Review. Int J Technol Assess Health Care. 2012;28(2):125-32.
21. Baltussen R, Niessen L. Priority setting of health interventions: the need for multi-criteria decision analysis. Cost Eff Resour Alloc. 2006;4:14.
22. Wahlster P, Goedghebeur M, Kriza C, Niederlander C, Kolominsky-Rabas P, and on behalf of the National Leading-Edge Cluster Medical Technologies "Medical Valley EMN". BMC Health Services Research 2015 15:262: http://bmchealthservres.biomedcentral.com/articles/10.1186/s12913-015-0930-0. Accessed Mar 2016
23. Orr S, Wolff J, Morris S. What Values Should Count in HTA for New Medicines under Value Based Pricing in the UK? UCL Centre for Philosophy, Justice and Health. 2011: https://www.ucl.ac.uk/health-humanities/docs/mcda.pdf. Accessed Mar 2016.
24. 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. http://eprints.lse.ac.uk/65148/1/Angelis-Value-based%20assessment.pdf. Accessed Mar 2016.
25. Adlunlin G, Diaby V, Montero AJ, Xiao H. Multicriteria decision analysis in oncology. Health Expect. 2015;18(6):1812-26.
26. Drummond M, Tarricone R, Torbica A. Assessing the added value of health technologies: reconciling different perspectives. Value Health. 2013;16:S7-13. http://www.valueinhealthjournal.com/article/S1098-3015(12)04158-7/pdf. Accessed Jan 2016.
27. Sculpher M. Evaluating the cost-effectiveness of interventions designed to increase the utilization of evidence-based guidelines. Fam Pract. Oxford University Press. 2000;17 Suppl 1:S26-31.
28. Tan-Torres Edejer T, Baltussen R, Adam T, Hutubessy R, Acharya DB, Evans DB, Murray CJL. Making Choices in Health: WHO Guide to cost-effectiveness analysis: http://www.who.int/choice/publications/p-2003-generalised-cea.pdf. Accessed Sept 2016
29. Evidence and Value: Impact on Decision Making (EVIDEM). EVIDEM 2.3. Decision Criteria: https://www.evidem.org/docs/2014/EVIDEM-v2-3-Decision-criteria-2013-12-24.pdf. Accessed Jan 2016
30. Radaelli G, Lettieri E, Masella C. Implementation of EUNETHA core model® in Lombardi: The VTS Framework. Int J Technol Assess Health Care. 2014;30(1):105-12.
31. Tony M, Wagner M, Khoury H, Rindress D, Papastavros T, Oh P, Goetghebeur MM. Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada. BMC Health Serv Res. 2011;11:329. http://www.biomedcentral.com/1472-6963/11/329. Accessed Nov 2015.
32. Wasfelt M, Fadeel B, Henter J. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med. 2006;260(1):1-10. http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2796.2006.01666.x/pdf. Accessed July 2014.
33. NICE highly specialised technologies guidance: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-highly-specialised-technologies-guidance. Accessed Mar 2016
34. Boon W, Martins L, Koopmanschap M. Governance of conditional reimbursement practices in the Netherlands. Health Policy. 2010;119(2):180-5.
35. Mousseau V, Dias L. Valued outranking relations in ELECTRE providing manageable disaggregation procedures. Eur J Oper Res. 2004;156:476-82.
36. Thokala P, Duenas A. Multiple criteria decision analysis for health technology assessment. Value Health. 2012;15:1172-81.
37. 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.
38. Birch S, Gafni A. Information created to evade reality (ICER) - things we should not look to for answers. Pharmacoeconomics. 2006;24:1121-31.
39. Gafni A, Birch S. Incremental cost-effectiveness ratios (ICERs): the silence of the lambda. Soc Sci Med. 2006;62:2091-100.
40. Scottish Medicines Consortium. Templates /Guidance for Submission. Budget Impact Template: https://www.scottishmedicines.org.uk/Submission-Process/Submission-guidance-and-forms/Templates-Guidance-for-Submission/Templates-Guidance-for-Submission. Accessed Nov 2016
41. Expert Roundtable. Expert Roundtable on HTA of Orphan Drugs. 2012. Facilitated by Shire Pharmaceuticals: https://www.yumpu.com/en/document/view/30976389/expert-roundtable-on-hta-of-orphan-drugs-shire. Accessed Oct 2015