A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare budgets. This analysis aims to compare pricin...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens
Formato:	article
Lenguaje:	EN
Publicado:	Frontiers Media S.A. 2021
Materias:	cystic fibrosis gene modulators pricing reimburesement comparative analaysis Therapeutics. Pharmacology RM1-950
Acceso en línea:	https://doaj.org/article/54c83bf9580b4210b5f605462054c3aa
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:54c83bf9580b4210b5f605462054c3aa
record_format	dspace
spelling	oai:doaj.org-article:54c83bf9580b4210b5f605462054c3aa2021-11-08T17:47:59ZA Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe1663-981210.3389/fphar.2021.746710https://doaj.org/article/54c83bf9580b4210b5f605462054c3aa2021-11-01T00:00:00Zhttps://www.frontiersin.org/articles/10.3389/fphar.2021.746710/fullhttps://doaj.org/toc/1663-9812Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare budgets. This analysis aims to compare pricing and reimbursement of aforementioned cystic fibrosis medicines, across European countries.Methods: Clinical trial registries, national databases, health technology assessment reports and grey literature of Austria, Belgium, Denmark, France, Germany, Ireland, Poland, Spain, Sweden, Switzerland, Netherlands, the United Kingdom were consulted. Publicly available prices, reimbursement statuses, economic evaluations, budget impact analyses and managed entry agreements of CFTR modulators were examined. Results: In Belgium, lowest list prices were observed for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €417 per defined daily dose (DDD) and €372 per average daily dose (ADD), respectively. Whereas, Switzerland had the lowest price for Orkambi® (lumacaftor/ivacaftor) listed at €309 per DDD. Spain had the highest prices for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €850 per DDD and €761 per ADD, whereas Orkambi® (lumacaftor/ivacaftor) was most expensive in Poland at €983 per DDD. However, list prices were subject to confidential discounts and likely varied from actual costs. In all countries, these treatments were deemed not to be cost-effective. The annual budget impact of the CFTR modulators varied between countries and depended on factors such as local product prices, size of target population, scope of costs and discounting. However, all modulators were fully reimbursed in ten of the evaluated countries except for Sweden and Poland that, respectively, granted reimbursement to one and none of the therapies. Managed entry agreements were confidential but commonly adopted to address financial uncertainties.Conclusion: Discrepancies concerning prices, reimbursement and access were detected for Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor) across European countries.Khadidja AbdallahKris De BoeckMarc DoomsSteven SimoensFrontiers Media S.A.articlecystic fibrosisgene modulatorspricingreimburesementcomparative analaysisTherapeutics. PharmacologyRM1-950ENFrontiers in Pharmacology, Vol 12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	cystic fibrosis gene modulators pricing reimburesement comparative analaysis Therapeutics. Pharmacology RM1-950
spellingShingle	cystic fibrosis gene modulators pricing reimburesement comparative analaysis Therapeutics. Pharmacology RM1-950 Khadidja Abdallah Kris De Boeck Marc Dooms Steven Simoens A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
description	Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare budgets. This analysis aims to compare pricing and reimbursement of aforementioned cystic fibrosis medicines, across European countries.Methods: Clinical trial registries, national databases, health technology assessment reports and grey literature of Austria, Belgium, Denmark, France, Germany, Ireland, Poland, Spain, Sweden, Switzerland, Netherlands, the United Kingdom were consulted. Publicly available prices, reimbursement statuses, economic evaluations, budget impact analyses and managed entry agreements of CFTR modulators were examined. Results: In Belgium, lowest list prices were observed for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €417 per defined daily dose (DDD) and €372 per average daily dose (ADD), respectively. Whereas, Switzerland had the lowest price for Orkambi® (lumacaftor/ivacaftor) listed at €309 per DDD. Spain had the highest prices for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €850 per DDD and €761 per ADD, whereas Orkambi® (lumacaftor/ivacaftor) was most expensive in Poland at €983 per DDD. However, list prices were subject to confidential discounts and likely varied from actual costs. In all countries, these treatments were deemed not to be cost-effective. The annual budget impact of the CFTR modulators varied between countries and depended on factors such as local product prices, size of target population, scope of costs and discounting. However, all modulators were fully reimbursed in ten of the evaluated countries except for Sweden and Poland that, respectively, granted reimbursement to one and none of the therapies. Managed entry agreements were confidential but commonly adopted to address financial uncertainties.Conclusion: Discrepancies concerning prices, reimbursement and access were detected for Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor) across European countries.
format	article
author	Khadidja Abdallah Kris De Boeck Marc Dooms Steven Simoens
author_facet	Khadidja Abdallah Kris De Boeck Marc Dooms Steven Simoens
author_sort	Khadidja Abdallah
title	A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
title_short	A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
title_full	A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
title_fullStr	A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
title_full_unstemmed	A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe
title_sort	comparative analysis of pricing and reimbursement of cystic fibrosis transmembrane conductance regulator modulators in europe
publisher	Frontiers Media S.A.
publishDate	2021
url	https://doaj.org/article/54c83bf9580b4210b5f605462054c3aa
work_keys_str_mv	AT khadidjaabdallah acomparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT krisdeboeck acomparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT marcdooms acomparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT stevensimoens acomparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT khadidjaabdallah comparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT krisdeboeck comparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT marcdooms comparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope AT stevensimoens comparativeanalysisofpricingandreimbursementofcysticfibrosistransmembraneconductanceregulatormodulatorsineurope
_version_	1718441472373555200

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Ejemplares similares