TY - JOUR AU - Drummond, M. F. PY - 2007 DA - 2007// TI - Assessing the economic challenges posed by orphan drugs JO - Int J Technol Assess Health Care VL - 23 UR - https://doi.org/10.1017/S0266462307051550 DO - 10.1017/S0266462307051550 ID - Drummond2007 ER - TY - STD TI - 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 UR - https://www.scottishmedicines.org.uk/SMC_Advice/Advice/alglucosidase_alfa_50mg_powder__Myozyme___352-07_/alglucosidase_alfa__Myozyme____352-07_ ID - ref2 ER - TY - JOUR AU - Picavet, E. AU - Cassiman, D. AU - Simoens, S. PY - 2015 DA - 2015// TI - What is known about the cost-effectiveness of orphan drugs? Evidence form cost-utility analyses JO - J Clin Pharm Ther VL - 40 UR - https://doi.org/10.1111/jcpt.12271 DO - 10.1111/jcpt.12271 ID - Picavet2015 ER - TY - STD TI - 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 UR - http://www.euro.who.int/__data/assets/pdf_file/0011/98291/E91271.pdf ID - ref4 ER - TY - JOUR AU - Cleemput, I. AU - Neyt, M. AU - Thiry, N. AU - Laet, C. AU - Leys, M. PY - 2011 DA - 2011// TI - Using threshold values for cost per quality-adjusted life-year gained in healthcare decisions JO - Int J Technol Assess Health Care VL - 27 UR - https://doi.org/10.1017/S0266462310001194 DO - 10.1017/S0266462310001194 ID - Cleemput2011 ER - TY - JOUR AU - Appleby, J. AU - Devlin, N. AU - Parkin, D. PY - 2007 DA - 2007// TI - NICE’s cost effectiveness threshold JO - BMJ VL - 335 UR - https://doi.org/10.1136/bmj.39308.560069.BE DO - 10.1136/bmj.39308.560069.BE ID - Appleby2007 ER - TY - JOUR AU - Cohen, J. P. AU - Felix, A. PY - 2014 DA - 2014// TI - Are payers treating orphan drugs differently? JO - J Market Access Health Policy VL - 2 UR - https://doi.org/10.3402/jmahp.v2.23513 DO - 10.3402/jmahp.v2.23513 ID - Cohen2014 ER - TY - JOUR AU - Hughes-Wilson, W. PY - 2012 DA - 2012// TI - 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? JO - Orphanet J Rare Dis VL - 7 UR - https://doi.org/10.1186/1750-1172-7-74 DO - 10.1186/1750-1172-7-74 ID - Hughes-Wilson2012 ER - TY - BOOK AU - Belton, V. AU - Stewart, T. J. PY - 2002 DA - 2002// TI - Multiple criteria decision analysis: an integrated approach PB - Kluwer CY - Dordrecht UR - https://doi.org/10.1007/978-1-4615-1495-4 DO - 10.1007/978-1-4615-1495-4 ID - Belton2002 ER - TY - JOUR AU - Carver, S. J. PY - 1991 DA - 1991// TI - Integrating multi-criteria evaluation with geographical information systems JO - Int J Geogr Inf Syst VL - 5 UR - https://doi.org/10.1080/02693799108927858 DO - 10.1080/02693799108927858 ID - Carver1991 ER - TY - JOUR AU - Hallerbach, W. AU - Spronk, J. PY - 2003 DA - 2003// TI - The relevance of MCDM for financial decisions JO - J Multi-Criteria Decis Anal VL - 11 ID - Hallerbach2003 ER - TY - STD TI - Linkov I, Moberg E. Multi-criteria decision analysis: environmental applications and case studies. 2013. CRC Press. Taylor & Francis Group. ISBN 9781439853184 - CAT# K12453. ID - ref12 ER - TY - JOUR AU - Youngkong, S. AU - Teerawattananon, Y. AU - Tantivess, S. AU - Baltussen, R. PY - 2012 DA - 2012// TI - Multi-criteria decision analysis for setting priorities on HIV/AIDS interventions in Thailand JO - Health Res Policy Syst VL - 10 UR - https://doi.org/10.1186/1478-4505-10-6 DO - 10.1186/1478-4505-10-6 ID - Youngkong2012 ER - TY - JOUR AU - Baltussen, R. AU - Mikkelsen, E. AU - Tromp, N. AU - Hurtig, A. AU - Byskov, J. AU - Olsen, O. AU - Baeroe, K. AU - Hontelez, J. A. AU - Singh, J. AU - Norheim, O. PY - 2013 DA - 2013// TI - Balancing efficiency, equity and feasbility of HIV treatment in South Africa – development of programmatic guidance JO - Cost Eff Resour Alloc VL - 11 UR - https://doi.org/10.1186/1478-7547-11-26 DO - 10.1186/1478-7547-11-26 ID - Baltussen2013 ER - TY - JOUR AU - Angelis, A. AU - Kanavos, P. PY - 2015 DA - 2015// TI - Applying Multiple Criteria Decision Analysis (MCDA) in the context of HTA: an experimental case study on metastatic colorectal cancer JO - J Pharm Policy Pract VL - 8 UR - https://doi.org/10.1186/2052-3211-8-S1-O8 DO - 10.1186/2052-3211-8-S1-O8 ID - Angelis2015 ER - TY - STD TI - 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 UR - https://www.nice.org.uk/News/Article/nice-to-assess-high-cost-drugs-for-rare-conditions ID - ref16 ER - TY - STD TI - 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 UR - http://socialwelfare.bl.uk/subject-areas/services-activity/health-services/2020health/147277orphan_medicines_2012.pdf ID - ref17 ER - TY - JOUR AU - Thokala, P. AU - Devlin, N. AU - Marsh, K. AU - Baltussen, R. AU - Boysen, M. AU - Kalo, Z. AU - Longrenn, T. AU - Mussen, F. AU - Peacock, S. AU - Watkins, J. AU - Ijzerman, M. PY - 2016 DA - 2016// TI - Multiple criteria decision analysis for health care decision making—An introduction: report 1 of the ISPOR MCDA emerging good practices task force JO - Value Health VL - 19 UR - https://doi.org/10.1016/j.jval.2015.12.003 DO - 10.1016/j.jval.2015.12.003 ID - Thokala2016 ER - TY - JOUR AU - Simoens, S. PY - 2012 DA - 2012// TI - Market access of orphan drugs and the role of multi-criteria decision making JO - Orphanet J Rare Dis VL - 7 ID - Simoens2012 ER - TY - JOUR AU - Johri, M. PY - 2012 DA - 2012// TI - Can Cost-Effectiveness Analysis Integrate Concerns For Equity? Systematic Review JO - Int J Technol Assess Health Care VL - 28 UR - https://doi.org/10.1017/S0266462312000050 DO - 10.1017/S0266462312000050 ID - Johri2012 ER - TY - JOUR AU - Baltussen, R. AU - Niessen, L. PY - 2006 DA - 2006// TI - Priority setting of health interventions: the need for multi-criteria decision analysis JO - Cost Eff Resour Alloc. VL - 4 UR - https://doi.org/10.1186/1478-7547-4-14 DO - 10.1186/1478-7547-4-14 ID - Baltussen2006 ER - TY - STD TI - 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 Research201515:262: http://bmchealthservres.biomedcentral.com/articles/10.1186/s12913-015-0930-0. Accessed Mar 2016 UR - http://bmchealthservres.biomedcentral.com/articles/10.1186/s12913-015-0930-0 ID - ref22 ER - TY - STD TI - 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. UR - https://www.ucl.ac.uk/health-humanities/docs/mcda.pdf ID - ref23 ER - TY - JOUR AU - Angelis, A. AU - Kanavos, P. PY - 2016 DA - 2016// TI - 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 JO - PharmacoEconomics VL - 34 UR - https://doi.org/10.1007/s40273-015-0370-z DO - 10.1007/s40273-015-0370-z ID - Angelis2016 ER - TY - JOUR AU - Adlunlin, G. AU - Diaby, V. AU - Montero, A. J. AU - Xiao, H. PY - 2015 DA - 2015// TI - Multicriteria decision analysis in oncology JO - Health Expect VL - 18 UR - https://doi.org/10.1111/hex.12178 DO - 10.1111/hex.12178 ID - Adlunlin2015 ER - TY - JOUR AU - Drummond, M. AU - Tarricone, R. AU - Torbica, A. PY - 2013 DA - 2013// TI - Assessing the added value of health technologies: reconciling different perspectives JO - Value Health VL - 16 UR - https://doi.org/10.1016/j.jval.2012.10.007 DO - 10.1016/j.jval.2012.10.007 ID - Drummond2013 ER - TY - STD TI - 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.  ID - ref27 ER - TY - STD TI - 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 UR - http://www.who.int/choice/publications/p_2003_generalised_cea.pdf ID - ref28 ER - TY - STD TI - 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 UR - https://www.evidem.org/docs/2014/EVIDEM-v2-3-Decision-criteria-2013-12-24.pdf ID - ref29 ER - TY - JOUR AU - Radaelli, G. AU - Lettieri, E. AU - Masella, C. PY - 2014 DA - 2014// TI - Implementation of EUNETHA core model® in Lombardi: The VTS Framework JO - Int J Technol Assess Health Care VL - 30 UR - https://doi.org/10.1017/S0266462313000639 DO - 10.1017/S0266462313000639 ID - Radaelli2014 ER - TY - JOUR AU - Tony, M. AU - Wagner, M. AU - Khoury, H. AU - Rindress, D. AU - Papastavros, T. AU - Oh, P. AU - Goetghebeur, M. M. PY - 2011 DA - 2011// TI - 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 JO - BMC Health Serv Res VL - 11 UR - https://doi.org/10.1186/1472-6963-11-329 DO - 10.1186/1472-6963-11-329 ID - Tony2011 ER - TY - JOUR AU - Wasfelt, M. AU - Fadeel, B. AU - Henter, J. PY - 2006 DA - 2006// TI - A journey of hope: lessons learned from studies on rare diseases and orphan drugs JO - J Intern Med VL - 260 UR - https://doi.org/10.1111/j.1365-2796.2006.01666.x DO - 10.1111/j.1365-2796.2006.01666.x ID - Wasfelt2006 ER - TY - STD TI - 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 UR - https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-highly-specialised-technologies-guidance ID - ref33 ER - TY - JOUR AU - Boon, W. AU - Martins, L. AU - Koopmanschap, M. PY - 2010 DA - 2010// TI - Governance of conditional reimbursement practices in the Netherlands JO - Health Policy VL - 119 UR - https://doi.org/10.1016/j.healthpol.2014.10.013 DO - 10.1016/j.healthpol.2014.10.013 ID - Boon2010 ER - TY - JOUR AU - Mousseau, V. AU - Dias, L. PY - 2004 DA - 2004// TI - Valued outranking relations in ELECTRE providing manageable disaggregation procedures JO - Eur J Oper Res. VL - 156 UR - https://doi.org/10.1016/S0377-2217(03)00120-6 DO - 10.1016/S0377-2217(03)00120-6 ID - Mousseau2004 ER - TY - JOUR AU - Thokala, P. AU - Duenas, A. PY - 2012 DA - 2012// TI - Multiple criteria decision analysis for health technology assessment JO - Value Health VL - 15 UR - https://doi.org/10.1016/j.jval.2012.06.015 DO - 10.1016/j.jval.2012.06.015 ID - Thokala2012 ER - TY - JOUR AU - Sussex, J. AU - Rollet, P. AU - Garau, M. AU - Schmitt, C. AU - Kent, A. AU - Hutchings, A. PY - 2013 DA - 2013// TI - A pilot study of multicriteria decision analysis for valuing orphan medicines JO - Value Health VL - 16 UR - https://doi.org/10.1016/j.jval.2013.10.002 DO - 10.1016/j.jval.2013.10.002 ID - Sussex2013 ER - TY - JOUR AU - Birch, S. AU - Gafni, A. PY - 2006 DA - 2006// TI - Information created to evade reality (ICER) – things we should not look to for answers JO - Pharmacoeconomics VL - 24 UR - https://doi.org/10.2165/00019053-200624110-00008 DO - 10.2165/00019053-200624110-00008 ID - Birch2006 ER - TY - JOUR AU - Gafni, A. AU - Birch, S. PY - 2006 DA - 2006// TI - Incremental cost-effectiveness ratios (ICERs): the silence of the lambda JO - Soc Sci Med VL - 62 UR - https://doi.org/10.1016/j.socscimed.2005.10.023 DO - 10.1016/j.socscimed.2005.10.023 ID - Gafni2006 ER - TY - STD TI - 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 UR - https://www.scottishmedicines.org.uk/Submission_Process/Submission_guidance_and_forms/Templates-Guidance-for-Submission/Templates-Guidance-for-Submission ID - ref40 ER - TY - STD TI - 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 UR - https://www.yumpu.com/en/document/view/30976389/expert-roundtable-on-hta-of-orphan-drugs-shire ID - ref41 ER -