Beyond Reimbursement Status: Availability of Advanced Therapy
Medicinal Products Across the European Union

J 2025

Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union

ČECHOVÁ, Zora; Jana KUBÁTOVÁ; Adéla BÁRTOVÁ; Jakub JAMÁRIK; Jiří SAMEK et al.

Základní údaje

Originální název

Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union

Autoři

ČECHOVÁ, Zora; Jana KUBÁTOVÁ ; Adéla BÁRTOVÁ; Jakub JAMÁRIK a Jiří SAMEK

Vydání

THERAPEUTIC INNOVATION & REGULATORY SCIENCE, HEIDELBERG, SPRINGER HEIDELBERG, 2025, 2168-4790

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30230 Other clinical medicine subjects

Stát vydavatele

Německo

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 1.900 v roce 2024

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/25:00141069

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

Cell therapy; Gene therapy; Patient access; Inequity; Pharmaceutical market; National competent authority

Štítky

14110516, 14119710, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 16. 2. 2026 09:15, Mgr. Tereza Miškechová

Anotace

V originále

BackgroundAdvanced Therapy Medicinal Products (ATMPs) represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Although authorised centrally in the European Union, their market launch differs across Member States (MS). This study aimed to describe the ATMP market availability in MS and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies.MethodsATMP availability was defined as the product launch in each MS. Data was collected through open governmental sources, databases, and communication with national competent authorities. Spearman's correlation coefficients were calculated to examine the relationship between ATMP availability and their characteristics (time since granting marketing authorisation, target patient population size, and cost).ResultsWe collected the availability data on 18 ATMPs from 23 EU MS. Market uptake varied significantly, with Germany (89%), France and Italy (61%) leading. Estonia and Latvia confirmed that no ATMP has been launched on their markets yet. Six ATMPs were available in more than one-third of the analysed MS. No significant correlation was observed between ATMP availability and analysed product characteristics except for time dependency for CAR T-cell therapies.ConclusionBeyond pricing and reimbursement processes, the ATMP commercialisation in particular MS is influenced by the marketing authorisation holder's decision and capacity. ATMPs face product-specific challenges in achieving EU-wide availability, including complex manufacturing, distribution, and administration processes. To increase the accessibility of innovative ATMP-based treatments, implementing the cross-border access framework or individual ATMP production under the hospital exemption is essential, especially in underserved MS.

Návaznosti

101059788, interní kód MU

Název: Central European Advanced Therapy and Immunotherapy Centre

Investor: Evropská unie, Central European Advanced Therapy and Immunotherapy Centre, Rozšiřování účasti a posílení ERA

90249, velká výzkumná infrastruktura

Název: CZECRIN IV

Citovat

ČECHOVÁ, Zora; Jana KUBÁTOVÁ; Adéla BÁRTOVÁ; Jakub JAMÁRIK a Jiří SAMEK. Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union. THERAPEUTIC INNOVATION & REGULATORY SCIENCE. HEIDELBERG: SPRINGER HEIDELBERG, 2025, roč. 59, č. 4, s. 728-736. ISSN 2168-4790. Dostupné z: https://doi.org/10.1007/s43441-025-00769-z.

@article{2492779,
   author = {Čechová, Zora and Kubátová, Jana and Bártová, Adéla and Jamárik, Jakub and Samek, Jiří},
   article_location = {HEIDELBERG},
   article_number = {4},
   doi = {https://doi.org/10.1007/s43441-025-00769-z},
   keywords = {Cell therapy; Gene therapy; Patient access; Inequity; Pharmaceutical market; National competent authority},
   language = {eng},
   issn = {2168-4790},
   journal = {THERAPEUTIC INNOVATION & REGULATORY SCIENCE},
   title = {Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union},
   url = {https://link.springer.com/article/10.1007/s43441-025-00769-z},
   volume = {59},
   year = {2025}
}

TY  - JOUR
ID  - 2492779
AU  - Čechová, Zora - Kubátová, Jana - Bártová, Adéla - Jamárik, Jakub - Samek, Jiří
PY  - 2025
TI  - Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union
JF  - THERAPEUTIC INNOVATION & REGULATORY SCIENCE
VL  - 59
IS  - 4
SP  - 728-736
EP  - 728-736
PB  - SPRINGER HEIDELBERG
SN  - 21684790
KW  - Cell therapy
KW  - Gene therapy
KW  - Patient access
KW  - Inequity
KW  - Pharmaceutical market
KW  - National competent authority
UR  - https://link.springer.com/article/10.1007/s43441-025-00769-z
N2  - BackgroundAdvanced Therapy Medicinal Products (ATMPs) represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Although authorised centrally in the European Union, their market launch differs across Member States (MS). This study aimed to describe the ATMP market availability in MS and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies.MethodsATMP availability was defined as the product launch in each MS. Data was collected through open governmental sources, databases, and communication with national competent authorities. Spearman's correlation coefficients were calculated to examine the relationship between ATMP availability and their characteristics (time since granting marketing authorisation, target patient population size, and cost).ResultsWe collected the availability data on 18 ATMPs from 23 EU MS. Market uptake varied significantly, with Germany (89%), France and Italy (61%) leading. Estonia and Latvia confirmed that no ATMP has been launched on their markets yet. Six ATMPs were available in more than one-third of the analysed MS. No significant correlation was observed between ATMP availability and analysed product characteristics except for time dependency for CAR T-cell therapies.ConclusionBeyond pricing and reimbursement processes, the ATMP commercialisation in particular MS is influenced by the marketing authorisation holder's decision and capacity. ATMPs face product-specific challenges in achieving EU-wide availability, including complex manufacturing, distribution, and administration processes. To increase the accessibility of innovative ATMP-based treatments, implementing the cross-border access framework or individual ATMP production under the hospital exemption is essential, especially in underserved MS.
ER  -

ČECHOVÁ, Zora; Jana KUBÁTOVÁ; Adéla BÁRTOVÁ; Jakub JAMÁRIK a Jiří SAMEK. Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union. \textit{THERAPEUTIC INNOVATION \&{} REGULATORY SCIENCE}. HEIDELBERG: SPRINGER HEIDELBERG, 2025, roč.~59, č.~4, s.~728-736. ISSN~2168-4790. Dostupné z: https://doi.org/10.1007/s43441-025-00769-z.

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