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 Journal of Rare Diseases
Thomas MorelSteven Simoens

Abstract

National payers across Europe have been increasingly looking into innovative reimbursement approaches - called managed entry agreements (MEAs) - to balance the need to provide rapid access to potentially beneficial orphan medicinal products (OMPs) with the requirements to circumscribe uncertainty, obtain best value for money or to ensure affordability. This study aimed to identify, describe and classify MEAs applied to OMPs by national payers and to analyse their practice in Europe. To identify and describe MEAs, national health technology assessments and reimbursement decisions on OMPs across seven European countries were reviewed and their main characteristics extracted. To fill data gaps and validate the accuracy of the extraction, collaboration was sought from national payers. To classify MEAs, a bespoke taxonomy was implemented. Identified MEAs were analysed and compared by focusing on five key themes, namely by describing the MEAs in relation to: drug targets and therapeutic classes, geographical spread, type of MEA applied, declared rationale for setting-up of MEAs, and evolution over time. 42 MEAs for 26 OMPs, implemented between 2006 and 2012 and representing a variety of MEA designs, were identified. Italy was the cou...Continue Reading

References

Jun 26, 2009·British Journal of Clinical Pharmacology·Roberta JoppiSilvio Garattini
Jan 21, 2010·PharmacoEconomics·Christopher J McCabeUNKNOWN Banff AED Summit
Apr 16, 2010·The New England Journal of Medicine·Ans T van der PloegSasa A Zivkovic
Sep 2, 2010·Applied Health Economics and Health Policy·Javier OrofinoItziar Oyagüez
Nov 18, 2010·BMJ : British Medical Journal·Jonathan C P RoosTimothy M Cox
Jan 26, 2011·International Journal of Technology Assessment in Health Care·Marianne KlempUNKNOWN HTAi Policy Forum
Mar 15, 2011·British Journal of Clinical Pharmacology·Alain G Dupont, Philippe B Van Wilder
Jun 7, 2011·JAMA : the Journal of the American Medical Association·Aaron S KesselheimJerry Avorn
Jun 21, 2011·Applied Health Economics and Health Policy·Eline PicavetSteven Simoens
Jun 21, 2011·Orphanet Journal of Rare Diseases·Steven Simoens
Sep 29, 2011·Orphanet Journal of Rare Diseases·Carina ScheyAdam Hutchings
Mar 1, 2012·PloS One·Aaron S KesselheimJerry Avorn
May 16, 2012·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Simon WalkerSteve Palmer
Aug 15, 2012·BMJ : British Medical Journal·Tony Sheldon
Oct 24, 2012·European Journal of Clinical Pharmacology·Roberta JoppiSilvio Garattini
Jan 17, 2013·Applied Health Economics and Health Policy·Brian Godman, Lars L Gustafsson
Jan 19, 2013·Applied Health Economics and Health Policy·Steven SimoensThomas Morel
Feb 21, 2013·Orphanet Journal of Rare Diseases·Saskia M RombachMarcel G W Dijkgraaf
Jul 10, 2013·Drug Discovery Today·M PutzeistH G M Leufkens
Aug 21, 2013·Orphanet Journal of Rare Diseases·Tim A KantersLeona Hakkaart
Aug 21, 2013·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Louis P GarrisonMiguel Sleeper
Oct 3, 2013·JAMA : the Journal of the American Medical Association·Brian P O'SullivanCarlos E Milla

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Citations

Apr 29, 2014·Orphanet Journal of Rare Diseases·Eline PicavetSteven Simoens
Sep 12, 2014·Orphanet Journal of Rare Diseases·Eline PicavetSteven Simoens
Dec 10, 2014·Expert Review of Clinical Pharmacology·Brian GodmanLars L Gustafsson
Oct 21, 2014·Expert Review of Pharmacoeconomics & Outcomes Research·Afschin Gandjour
May 4, 2015·Orphanet Journal of Rare Diseases·Laura GutierrezWarren Cowell
Sep 17, 2015·Orphanet Journal of Rare Diseases·Tim A KantersLeona Hakkaart
Sep 2, 2016·Applied Health Economics and Health Policy·Mondher ToumiÅsa Kornfeld
Oct 18, 2016·Frontiers in Public Health·Georgi IskrovRumen Stefanov
Apr 7, 2018·Health Expectations : an International Journal of Public Participation in Health Care and Health Policy·Andrea YoungTania Stafinski
Dec 24, 2017·Orphanet Journal of Rare Diseases·Andrea YoungTania Stafinski
Feb 9, 2018·Pharmaceutical Research·Szymon JarosławskiClaude Dussart
Nov 7, 2019·Applied Health Economics and Health Policy·Caridad PontesBrian Godman
Apr 16, 2020·Molecular Oncology·Yolande LievensCai Grau
Jul 14, 2020·Expert Review of Pharmacoeconomics & Outcomes Research·Hussain Abdulrahman Al-OmarIbrahim Abdulrahman Aljuffali
Sep 10, 2020·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Nasuh C BuyukkaramikliMen Thi Hoang
Sep 24, 2020·International Journal of Technology Assessment in Health Care·Weiwei Xu, Jing Wu
Mar 12, 2017·Orphanet Journal of Rare Diseases·Lieven AnnemansLugdivine Le Dez
Nov 28, 2018·Journal of Market Access & Health Policy·Szymon Jarosławski, Mondher Toumi
Sep 20, 2019·PharmacoEconomics·Fernando AntonanzasRoberto Rodríguez-Ibeas
Apr 7, 2018·PharmacoEconomics Open·Trevor Jozef PiatkiewiczTove Holm-Larsen
Oct 5, 2018·Applied Health Economics and Health Policy·Irene ErikssonBjörn Wettermark
Mar 1, 2017·Therapeutic Innovation & Regulatory Science·Jessica PaceWendy Lipworth
Nov 7, 2018·Orphanet Journal of Rare Diseases·Márta SzegediMária Judit Molnár
Apr 11, 2018·Frontiers in Pharmacology·Jacoline C BouvySarah Garner
Aug 1, 2020·PharmacoEconomics·Carolina Zampirolli DiasAugusto Afonso Guerra-Junior
May 10, 2017·PharmacoEconomics·Fernando AntoñanzasMaarten Postma
May 28, 2019·International Journal of Technology Assessment in Health Care·Linda MundyBrendon Kearney
Feb 26, 2020·Health Policy·Emelie AnderssonPeter Lindgren
Jan 30, 2021·International Journal of Technology Assessment in Health Care·Olina Efthymiadou, Panos Kanavos
Feb 4, 2021·International Journal of Environmental Research and Public Health·Patrizio ArmeniYllka Kodra
Mar 2, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Isaac Aranda-ReneoJulio López-Bastida

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