Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways
and Patient Access

a 2025

Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access

ČECHOVÁ, Zora

Základní údaje

Originální název

Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access

Autoři

ČECHOVÁ, Zora

Vydání

PDA Advanced Therapy Medicinal Products Conference 2025, 2025

Další údaje

Jazyk

angličtina

Typ výsledku

Konferenční abstrakt

Obor

30104 Pharmacology and pharmacy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Označené pro přenos do RIV

Organizační jednotka

Lékařská fakulta

Příznaky

Mezinárodní význam

Změněno: 27. 8. 2025 14:46, PharmDr. Zora Čechová, Ph.D.

Anotace

V originále

Advanced Therapy Medicinal Products (ATMP) represent promising innovations in the treatment of a wide range of serious diseases, including those previously untreatable. For some patients, the administration of ATMPs, which are still under clinical evaluation, may be the only viable option when available therapies fail to adequately address their condition. This study aimed to assess the ATMP landscape in the Czech Republic, analyse the regulatory framework and processes governing the administration of non-authorised ATMPs, and identify barriers that prevent patient access to these therapies. Our analysis reveals that the Czech legal framework provides several pathways for the administration of non-authorised ATMPs, including clinical trials, compassionate use, hospital exemption, and notifications under Section 8 of the Medicinal Products Act. The study further describes their utilisation over the past four years. Notably, the hospital exemption has not been applied in practice in the Czech Republic yet and significant challenges persist, particularly in relation to the subsequent processes regarding pricing and financing. The findings suggest that while access to non-authorised ATMPs is formally implemented in the national legislation and guidelines, practical challenges remain, limiting the broader and more effective use of hospital exemption. Additionally, financing processes are not yet adequately prepared, as HTA methodology corresponds to that used for centrally authorised ATMPs. Future efforts should focus on clarifying that regulatory pathway to improve patient access to these innovative therapies, particularly for those with unmet medical needs.

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

Citovat

ČECHOVÁ, Zora. Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access. In PDA Advanced Therapy Medicinal Products Conference 2025. 2025.

@proceedings{2513977,
   author = {Čechová, Zora},
   booktitle = {PDA Advanced Therapy Medicinal Products Conference 2025},
   language = {eng},
   title = {Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access},
   url = {https://www.pda.org/global-event-calendar/event-detail/pda-advanced-therapy-medicinal-products-conference-2025-archived#agenda},
   year = {2025}
}

TY  - CONF
ID  - 2513977
AU  - Čechová, Zora
PY  - 2025
TI  - Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access
UR  - https://www.pda.org/global-event-calendar/event-detail/pda-advanced-therapy-medicinal-products-conference-2025-archived#agenda
N2  - Advanced Therapy Medicinal Products (ATMP) represent promising innovations in the treatment of a wide range of serious diseases, including those previously untreatable. For some patients, the administration of ATMPs, which are still under clinical evaluation, may be the only viable option when available therapies fail to adequately address their condition. This study aimed to assess the ATMP landscape in the Czech Republic, analyse the regulatory framework and processes governing the administration of non-authorised ATMPs, and identify barriers that prevent patient access to these therapies. Our analysis reveals that the Czech legal framework provides several pathways for the administration of non-authorised ATMPs, including clinical trials, compassionate use, hospital exemption, and notifications under Section 8 of the Medicinal Products Act. The study further describes their utilisation over the past four years. Notably, the hospital exemption has not been applied in practice in the Czech Republic yet and significant challenges persist, particularly in relation to the subsequent processes regarding pricing and financing. The findings suggest that while access to non-authorised ATMPs is formally implemented in the national legislation and guidelines, practical challenges remain, limiting the broader and more effective use of hospital exemption. Additionally, financing processes are not yet adequately prepared, as HTA methodology corresponds to that used for centrally authorised ATMPs. Future efforts should focus on clarifying that regulatory pathway to improve patient access to these innovative therapies, particularly for those with unmet medical needs.
ER  -

ČECHOVÁ, Zora. Non-Authorised ATMPs in the Czech Republic: Regulatory Pathways and Patient Access. In \textit{PDA Advanced Therapy Medicinal Products Conference 2025}. 2025.

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