The European challenges of funding orphan medicinal products

Márta Szegedi, Tamás Zelei, Francis Arickx, Anna Bucsics, Emanuelle Cohn-Zanchetta, Jurij Fürst, Maria Kamusheva, Pawel Kawalec, Guenka Petrova, Juraj Slaby, Ewa Stawowczyk, Milan Vocelka, Ingrid Zechmeister-Koss, Z. Kaló, Mária Judit Molnár

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

Background: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). Objectives: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. Methods: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. Results: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures. Conclusions: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.

Original languageEnglish
Article number184
JournalOrphanet Journal of Rare Diseases
Volume13
Issue number1
DOIs
Publication statusPublished - Nov 6 2018

Fingerprint

Orphaned Children
Health Expenditures
European Union
Budgets
Rare Diseases
Delivery of Health Care
Pharmaceutical Preparations
Eastern Europe
Bulgaria
Hungary
Austria
Pharmacies
Czech Republic
Belgium
Poland
France

Keywords

  • Equity
  • European Union
  • Funding
  • Orphan medicinal products
  • Patient access
  • Reimbursement

ASJC Scopus subject areas

  • Genetics(clinical)
  • Pharmacology (medical)

Cite this

Szegedi, M., Zelei, T., Arickx, F., Bucsics, A., Cohn-Zanchetta, E., Fürst, J., ... Molnár, M. J. (2018). The European challenges of funding orphan medicinal products. Orphanet Journal of Rare Diseases, 13(1), [184]. https://doi.org/10.1186/s13023-018-0927-y

The European challenges of funding orphan medicinal products. / Szegedi, Márta; Zelei, Tamás; Arickx, Francis; Bucsics, Anna; Cohn-Zanchetta, Emanuelle; Fürst, Jurij; Kamusheva, Maria; Kawalec, Pawel; Petrova, Guenka; Slaby, Juraj; Stawowczyk, Ewa; Vocelka, Milan; Zechmeister-Koss, Ingrid; Kaló, Z.; Molnár, Mária Judit.

In: Orphanet Journal of Rare Diseases, Vol. 13, No. 1, 184, 06.11.2018.

Research output: Contribution to journalArticle

Szegedi, M, Zelei, T, Arickx, F, Bucsics, A, Cohn-Zanchetta, E, Fürst, J, Kamusheva, M, Kawalec, P, Petrova, G, Slaby, J, Stawowczyk, E, Vocelka, M, Zechmeister-Koss, I, Kaló, Z & Molnár, MJ 2018, 'The European challenges of funding orphan medicinal products', Orphanet Journal of Rare Diseases, vol. 13, no. 1, 184. https://doi.org/10.1186/s13023-018-0927-y
Szegedi M, Zelei T, Arickx F, Bucsics A, Cohn-Zanchetta E, Fürst J et al. The European challenges of funding orphan medicinal products. Orphanet Journal of Rare Diseases. 2018 Nov 6;13(1). 184. https://doi.org/10.1186/s13023-018-0927-y
Szegedi, Márta ; Zelei, Tamás ; Arickx, Francis ; Bucsics, Anna ; Cohn-Zanchetta, Emanuelle ; Fürst, Jurij ; Kamusheva, Maria ; Kawalec, Pawel ; Petrova, Guenka ; Slaby, Juraj ; Stawowczyk, Ewa ; Vocelka, Milan ; Zechmeister-Koss, Ingrid ; Kaló, Z. ; Molnár, Mária Judit. / The European challenges of funding orphan medicinal products. In: Orphanet Journal of Rare Diseases. 2018 ; Vol. 13, No. 1.
@article{32e53fff1adc4ca1aa0c6bb40c382d0c,
title = "The European challenges of funding orphan medicinal products",
abstract = "Background: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). Objectives: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. Methods: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. Results: In 2015 29.4-92.8{\%} of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41{\%} of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066{\%} of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51{\%} of the public pharmaceutical budget, and 0.44-0.96{\%} of public healthcare expenditures. Conclusions: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.",
keywords = "Equity, European Union, Funding, Orphan medicinal products, Patient access, Reimbursement",
author = "M{\'a}rta Szegedi and Tam{\'a}s Zelei and Francis Arickx and Anna Bucsics and Emanuelle Cohn-Zanchetta and Jurij F{\"u}rst and Maria Kamusheva and Pawel Kawalec and Guenka Petrova and Juraj Slaby and Ewa Stawowczyk and Milan Vocelka and Ingrid Zechmeister-Koss and Z. Kal{\'o} and Moln{\'a}r, {M{\'a}ria Judit}",
year = "2018",
month = "11",
day = "6",
doi = "10.1186/s13023-018-0927-y",
language = "English",
volume = "13",
journal = "Orphanet Journal of Rare Diseases",
issn = "1750-1172",
publisher = "BioMed Central",
number = "1",

}

TY - JOUR

T1 - The European challenges of funding orphan medicinal products

AU - Szegedi, Márta

AU - Zelei, Tamás

AU - Arickx, Francis

AU - Bucsics, Anna

AU - Cohn-Zanchetta, Emanuelle

AU - Fürst, Jurij

AU - Kamusheva, Maria

AU - Kawalec, Pawel

AU - Petrova, Guenka

AU - Slaby, Juraj

AU - Stawowczyk, Ewa

AU - Vocelka, Milan

AU - Zechmeister-Koss, Ingrid

AU - Kaló, Z.

AU - Molnár, Mária Judit

PY - 2018/11/6

Y1 - 2018/11/6

N2 - Background: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). Objectives: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. Methods: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. Results: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures. Conclusions: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.

AB - Background: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). Objectives: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. Methods: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. Results: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures. Conclusions: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.

KW - Equity

KW - European Union

KW - Funding

KW - Orphan medicinal products

KW - Patient access

KW - Reimbursement

UR - http://www.scopus.com/inward/record.url?scp=85056134921&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85056134921&partnerID=8YFLogxK

U2 - 10.1186/s13023-018-0927-y

DO - 10.1186/s13023-018-0927-y

M3 - Article

C2 - 30396361

AN - SCOPUS:85056134921

VL - 13

JO - Orphanet Journal of Rare Diseases

JF - Orphanet Journal of Rare Diseases

SN - 1750-1172

IS - 1

M1 - 184

ER -