TY - JOUR AU - Meekings, K. N. AU - Williams, C. S. AU - Arrowsmith, J. E. PY - 2012 DA - 2012// TI - Orphan drug development: an economically viable strategy for biopharma R & D JO - Drug Discov Today VL - 17 UR - https://doi.org/10.1016/j.drudis.2012.02.005 DO - 10.1016/j.drudis.2012.02.005 ID - Meekings2012 ER - TY - JOUR AU - Wellman-Labadie, O. AU - Zhou, Y. PY - 2010 DA - 2010// TI - The US Orphan Drug Act: rare disease research stimulator or commercial opportunity? JO - Health Pol VL - 95 UR - https://doi.org/10.1016/j.healthpol.2009.12.001 DO - 10.1016/j.healthpol.2009.12.001 ID - Wellman-Labadie2010 ER - TY - JOUR AU - Denis, A. AU - Mergaert, L. AU - Fostier, C. AU - Cleemput, I. AU - Simoens, S. PY - 2010 DA - 2010// TI - Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013 JO - J Med Econ VL - 13 UR - https://doi.org/10.3111/13696998.2010.491427 DO - 10.3111/13696998.2010.491427 ID - Denis2010 ER - TY - JOUR AU - Schey, C. AU - Milanova, T. AU - Hutchings, A. PY - 2011 DA - 2011// TI - Estimating the budget impact of orphan medicines in Europe: 2 JO - Orphanet J Rare Dis VL - 6 UR - https://doi.org/10.1186/1750-1172-6-62 DO - 10.1186/1750-1172-6-62 ID - Schey2011 ER - TY - JOUR AU - Michel, M. AU - Toumi, M. PY - 2012 DA - 2012// TI - Access to orphan drugs in Europe: current and future issues JO - Expert Rev Pharmacoecon Outcomes Res VL - 12 UR - https://doi.org/10.1586/erp.11.95 DO - 10.1586/erp.11.95 ID - Michel2012 ER - TY - JOUR AU - Roos, J. C. AU - Hyry, H. I. AU - Cox, T. M. PY - 2010 DA - 2010// TI - Orphan drug pricing may warrant a competition law investigation JO - BMJ VL - 341 UR - https://doi.org/10.1136/bmj.c6471 DO - 10.1136/bmj.c6471 ID - Roos2010 ER - TY - JOUR AU - Simoens, S. PY - 2011 DA - 2011// TI - Pricing and reimbursement of orphan drugs: the need for more transparency JO - Orphanet J Rare Dis VL - 6 UR - https://doi.org/10.1186/1750-1172-6-42 DO - 10.1186/1750-1172-6-42 ID - Simoens2011 ER - TY - JOUR AU - Messori, A. AU - Cicchetti, A. AU - Patregani, L. PY - 2010 DA - 2010// TI - Orphan drugs JO - BMJ VL - 341 UR - https://doi.org/10.1136/bmj.c4615 DO - 10.1136/bmj.c4615 ID - Messori2010 ER - TY - JOUR AU - Rollet, P. AU - Lemoine, A. AU - Dunoyer, M. PY - 2013 DA - 2013// TI - Sustainable rare diseases business and drug access: no time for misconceptions JO - Orphanet J Rare Dis VL - 8 UR - https://doi.org/10.1186/1750-1172-8-109 DO - 10.1186/1750-1172-8-109 ID - Rollet2013 ER - TY - JOUR AU - Orofino, J. AU - Soto, J. AU - Casado, M. A. AU - Oyaguez, I. PY - 2010 DA - 2010// TI - Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007 JO - Appl Health Econ Health Pol VL - 8 UR - https://doi.org/10.2165/11531880-000000000-00000 DO - 10.2165/11531880-000000000-00000 ID - Orofino2010 ER - TY - JOUR AU - Aballea, S. AU - Toumi, M. AU - Vataire, A. L. AU - Millier, A. AU - Lamure, M. PY - 2010 DA - 2010// TI - Quantitative Analysis of the Influence of Disease and Product Characteristics on Orphan Drug Prices in Europe [abstract] JO - Value Health VL - 13 UR - https://doi.org/10.1016/S1098-3015(10)72389-5 DO - 10.1016/S1098-3015(10)72389-5 ID - Aballea2010 ER - TY - STD TI - Alcimed: Study on Orphan Drugs - Phase I Overview of the Conditions for Marketing Orphan drugs in Europe. [http://ec.europa.eu/health/files/orphanmp/doc/pricestudy/final_final_report_part_1_web_en.pdf] UR - http://ec.europa.eu/health/files/orphanmp/doc/pricestudy/final_final_report_part_1_web_en.pdf ID - ref12 ER - TY - JOUR AU - Denis, A. AU - Mergaert, L. AU - Fostier, C. AU - Cleemput, I. AU - Simoens, S. PY - 2010 DA - 2010// TI - A comparative study of European rare disease and orphan drug markets JO - Health Pol VL - 97 UR - https://doi.org/10.1016/j.healthpol.2010.05.017 DO - 10.1016/j.healthpol.2010.05.017 ID - Denis2010 ER - TY - STD TI - European Medicines Agency. [http://www.ema.europa.eu/] UR - http://www.ema.europa.eu/ ID - ref14 ER - TY - JOUR AU - Walpole, S. C. AU - Prieto-Merino, D. AU - Edwards, P. AU - Cleland, J. AU - Stevens, G. AU - Roberts, I. PY - 2012 DA - 2012// TI - The weight of nations: an estimation of adult human biomass JO - BMC Public Health VL - 12 UR - https://doi.org/10.1186/1471-2458-12-439 DO - 10.1186/1471-2458-12-439 ID - Walpole2012 ER - TY - STD TI - World Health Organisation: WHO Child Growth Standards: Length/height-for-age, weight-for-age, weight-for-length, weight-for-height and body mass index-for-age: Methods and development. [http://www.who.int/childgrowth/standards/technical_report/en/] UR - http://www.who.int/childgrowth/standards/technical_report/en/ ID - ref16 ER - TY - JOUR AU - Haycock, G. B. AU - Schwartz, G. J. AU - Wisotsky, D. H. PY - 1978 DA - 1978// TI - Geometric method for measuring body surface area: a height-weight formula validated in infants, children, and adults JO - J Pediatr VL - 93 UR - https://doi.org/10.1016/S0022-3476(78)80601-5 DO - 10.1016/S0022-3476(78)80601-5 ID - Haycock1978 ER - TY - STD TI - XE The World’s Favorite Currency Site. [http://www.xe.com/] UR - http://www.xe.com/ ID - ref18 ER - TY - STD TI - European Commission: Eurostat. [http://epp.eurostat.ec.europa.eu/portal/page/portal/eurostat/home/] UR - http://epp.eurostat.ec.europa.eu/portal/page/portal/eurostat/home/ ID - ref19 ER - TY - STD TI - Denis A, Simoens S, Fostier C, Mergaert L, Cleemput I: Beleid voor zeldzame ziekten en Weesgeneesmiddelen KCE reports 112A. [https://kce.fgov.be/sites/default/files/page_documents/d20091027330.pdf] UR - https://kce.fgov.be/sites/default/files/page_documents/d20091027330.pdf ID - ref20 ER - TY - STD TI - BCFI - Belgian Centre for Pharmacotherapeutic Information. [http://www.bcfi.be/] UR - http://www.bcfi.be/ ID - ref21 ER - TY - STD TI - College voor Zorgverzekeringen: Medicijnkosten. [http://www.medicijnkosten.nl/] UR - http://www.medicijnkosten.nl/ ID - ref22 ER - TY - STD TI - SÚKL - State Institute for Drug Control. [http://www.sukl.eu/] UR - http://www.sukl.eu/ ID - ref23 ER - TY - STD TI - Thériaque. [http://www.theriaque.org/] UR - http://www.theriaque.org/ ID - ref24 ER - TY - STD TI - Informazioni sui farmaci. [http://www.informazionisuifarmaci.it/] UR - http://www.informazionisuifarmaci.it/ ID - ref25 ER - TY - STD TI - AIFA - Agenzia Italiana del Farmaco. [http://www.agenziafarmaco.gov.it/] UR - http://www.agenziafarmaco.gov.it/ ID - ref26 ER - TY - BOOK PY - 2013 DA - 2013// TI - British National Formulary 65 PB - Elsevier Inc CY - London ID - ref27 ER - TY - STD TI - Belgian Treasury: Reimbursement of Drugs - Performance of Governmental Management. [https://www.ccrek.be/Docs/2014_02_TerugbetalingGeneesmiddelen.pdf] UR - https://www.ccrek.be/Docs/2014_02_TerugbetalingGeneesmiddelen.pdf ID - ref28 ER - TY - JOUR AU - Picavet, E. AU - Cassiman, D. AU - Simoens, S. PY - 2013 DA - 2013// TI - Do ultra-orphan medicinal products warrant ultra-high prices? A review JO - Orphan Drugs Res Rev VL - 3 UR - https://doi.org/10.2147/ODRR.S33085 DO - 10.2147/ODRR.S33085 ID - Picavet2013 ER - TY - JOUR AU - Cote, A. AU - Keating, B. PY - 2012 DA - 2012// TI - What is wrong with orphan drug policies? JO - Value Health VL - 15 UR - https://doi.org/10.1016/j.jval.2012.09.004 DO - 10.1016/j.jval.2012.09.004 ID - Cote2012 ER - TY - JOUR AU - Schlander, M. AU - Beck, M. PY - 2009 DA - 2009// TI - Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI JO - Curr Med Res Opin VL - 25 UR - https://doi.org/10.1185/03007990902892633 DO - 10.1185/03007990902892633 ID - Schlander2009 ER - TY - JOUR AU - Hemphill, T. A. PY - 2010 DA - 2010// TI - Extraordinary Pricing of Orphan Drugs: Is it a Socially Responsible Strategy for the U.S. Pharmaceutical Industry? JO - J Bus Ethics VL - 94 UR - https://doi.org/10.1007/s10551-009-0259-x DO - 10.1007/s10551-009-0259-x ID - Hemphill2010 ER - TY - CHAP AU - Mycka, J. M. AU - Dellamano, R. PY - 2010 DA - 2010// TI - ISPOR short course: Elements of Pharmaceutical/Biotech Pricing BT - Proceedings of the ISPOR 13th Annual European Congress ID - Mycka2010 ER - TY - JOUR AU - Hughes-Wilson, W. AU - Palma, A. AU - Schuurman, A. AU - Simoens, S. 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 - JOUR AU - Picavet, E. AU - Dooms, M. AU - Cassiman, D. AU - Simoens, S. PY - 2011 DA - 2011// TI - Drugs for rare diseases: influence of orphan designation status on price JO - Appl Health Econ Health Policy VL - 9 UR - https://doi.org/10.2165/11590170-000000000-00000 DO - 10.2165/11590170-000000000-00000 ID - Picavet2011 ER - TY - JOUR AU - Kanavos, P. AU - Nicod, E. PY - 2012 DA - 2012// TI - What is wrong with orphan drug policies? Suggestions for ways forward JO - Value Health VL - 15 UR - https://doi.org/10.1016/j.jval.2012.08.2202 DO - 10.1016/j.jval.2012.08.2202 ID - Kanavos2012 ER - TY - STD TI - U.S. Food and Drug Administration: FDA Application: Search Orphan Drug Designations and Approvals. [http://www.accessdata.fda.gov/scripts/opdlisting/oopd/] UR - http://www.accessdata.fda.gov/scripts/opdlisting/oopd/ ID - ref37 ER - TY - JOUR AU - Simoens, S. AU - Picavet, E. AU - Cassiman, D. AU - Dooms, M. PY - 2012 DA - 2012// TI - What Price do we pay for Repurposing Medicines for Rare Diseases? JO - Value Health VL - 15 UR - https://doi.org/10.1016/j.jval.2012.03.092 DO - 10.1016/j.jval.2012.03.092 ID - Simoens2012 ER - TY - STD TI - OneSource. [http://www.onesource.com/] UR - http://www.onesource.com/ ID - ref39 ER - TY - STD TI - European Observatory on Health Systems and Policies: Health Systems in Transition (HiT) series. [http://www.euro.who.int/en/about-us/partners/observatory/health-systems-in-transition-hit-series] UR - http://www.euro.who.int/en/about-us/partners/observatory/health-systems-in-transition-hit-series ID - ref40 ER - TY - STD TI - WHO Collaborating Centre for Pharmaceutical Pricing and Reimbursement Policies: Pharmaceutical Pricing and Reimbursement Information (PPRI) Report. [http://whocc.goeg.at/Literaturliste/Dokumente/BooksReports/PPRI_Report_final.pdf] UR - http://whocc.goeg.at/Literaturliste/Dokumente/BooksReports/PPRI_Report_final.pdf ID - ref41 ER - TY - BOOK AU - Martikainen, J. AU - Rajaniemi, S. PY - 2002 DA - 2002// TI - Drug reimbursement systems in EU Member States, Iceland and Norway PB - Belgian Health Care Knowledge Centre CY - Helsinki ID - Martikainen2002 ER - TY - JOUR AU - Picavet, E. AU - Annemans, L. AU - Cleemput, I. AU - Cassiman, D. AU - Simoens, S. PY - 2012 DA - 2012// TI - Market uptake of orphan drugs - a European analysis JO - J Clin Pharm Ther VL - 37 UR - https://doi.org/10.1111/j.1365-2710.2012.01364.x DO - 10.1111/j.1365-2710.2012.01364.x ID - Picavet2012 ER - TY - JOUR AU - Morel, T. AU - Arickx, F. AU - Befrits, G. AU - Siviero, P. AU - van der Meijden, C. AU - Xoxi, E. AU - Simoens, S. PY - 2013 DA - 2013// TI - 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 JO - Orphanet J Rare Dis VL - 8 UR - https://doi.org/10.1186/1750-1172-8-198 DO - 10.1186/1750-1172-8-198 ID - Morel2013 ER - TY - JOUR AU - Denis, A. AU - Mergaert, L. AU - Fostier, C. AU - Cleemput, I. AU - Simoens, S. PY - 2010 DA - 2010// TI - Issues surrounding orphan disease and orphan drug policies in Europe JO - Appl Health Econ Health Pol VL - 8 UR - https://doi.org/10.2165/11536990-000000000-00000 DO - 10.2165/11536990-000000000-00000 ID - Denis2010 ER - TY - JOUR AU - Genane, C. AU - Marinoni, G. AU - Reinaud, F. AU - Ando, G. PY - 2013 DA - 2013// TI - Impact of Indication Extensions on Pharmaceutical Prices [abstract] JO - Value Health VL - 16 UR - https://doi.org/10.1016/j.jval.2013.08.759 DO - 10.1016/j.jval.2013.08.759 ID - Genane2013 ER - TY - JOUR AU - Miyamoto, B. E. AU - Kakkis, E. D. PY - 2011 DA - 2011// TI - The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases JO - Orphanet J Rare Dis VL - 6 UR - https://doi.org/10.1186/1750-1172-6-49 DO - 10.1186/1750-1172-6-49 ID - Miyamoto2011 ER - TY - JOUR PY - 2000 DA - 2000// TI - Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products JO - Off J Eur Communities VL - L 18 ID - ref48 ER - TY - CHAP AU - Silverman, E. PY - 2013 DA - 2013// BT - Tiger in the Fiscal Room: Beware the Increasing Cost and Number of Orphan Drugs ID - Silverman2013 ER - TY - JOUR AU - Bach, P. B. PY - 2013 DA - 2013// TI - Reforming the payment system for medical oncology JO - JAMA VL - 310 UR - https://doi.org/10.1001/jama.2013.8127 DO - 10.1001/jama.2013.8127 ID - Bach2013 ER - TY - JOUR AU - Linley, W. G. AU - Hughes, D. A. PY - 2013 DA - 2013// TI - Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain JO - Health Econ VL - 22 UR - https://doi.org/10.1002/hec.2872 DO - 10.1002/hec.2872 ID - Linley2013 ER - TY - STD TI - Herper M: The World’s Most Expensive Drug. [http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html] UR - http://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html ID - ref52 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 - Vegter, S. AU - Rozenbaum, M. H. AU - Postema, R. AU - Tolley, K. AU - Postma, M. J. PY - 2010 DA - 2010// TI - Review of regulatory recommendations for orphan drug submissions in the Netherlands and Scotland: focus on the underlying pharmacoeconomic evaluations JO - Clin Ther VL - 32 UR - https://doi.org/10.1016/j.clinthera.2010.08.006 DO - 10.1016/j.clinthera.2010.08.006 ID - Vegter2010 ER - TY - JOUR AU - Kalo, Z. AU - Annemans, L. AU - Garrison, L. P. PY - 2013 DA - 2013// TI - Differential pricing of new pharmaceuticals in lower income European countries JO - Expert Rev Pharmacoecon Outcomes Res VL - 13 UR - https://doi.org/10.1586/14737167.2013.847367 DO - 10.1586/14737167.2013.847367 ID - Kalo2013 ER - TY - STD TI - Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCa-OMP): Transparent Value Framework. [http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/orphans_framework_en.pdf] UR - http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/orphans_framework_en.pdf ID - ref56 ER -