Addressing Legislative and Financial Hurdles in Rare Disease Medicines Accessibility: Insight From Czechia With a Focus on the Pediatric Population

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F24%3A00138332" target="_blank" >RIV/00216224:14110/24:00138332 - isvavai.cz</a>

Výsledek na webu

<a href="https://www.valueinhealthjournal.com/article/S1098-3015(24)04292-X/fulltext" target="_blank" >https://www.valueinhealthjournal.com/article/S1098-3015(24)04292-X/fulltext</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1016/j.jval.2024.10.1429" target="_blank" >10.1016/j.jval.2024.10.1429</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Addressing Legislative and Financial Hurdles in Rare Disease Medicines Accessibility: Insight From Czechia With a Focus on the Pediatric Population

Popis výsledku v původním jazyce

Advanced therapy medicinal products (ATMPs) and orphan drugs are vital for treating rare diseases, especially in pediatric patients. However, the reimbursement and financing system for these medicines in Czechia lacks sustainability and transparency. This study analyzes the legislative framework governing rare disease medicine reimbursement, with a focus on §16 of Act No 48/1997 Coll. regarding exceptional reimbursement cases. Data from University Hospital Brno highlight the growing reliance on §16, with approval rates for pediatric patients increasing dramatically between 2017 and 2021. Despite high approval rates, the overuse of §16 undermines the need for legislative reform. Significant gaps, such as impractical cost-effectiveness analysis (CEA) requirements, hinder transparency and equitable access. The findings emphasize the urgent need for legislative reforms to ensure a predictable, sustainable, and fair reimbursement system for ATMPs and orphan drugs, particularly benefiting pediatric patients.

Název v anglickém jazyce

Addressing Legislative and Financial Hurdles in Rare Disease Medicines Accessibility: Insight From Czechia With a Focus on the Pediatric Population

Popis výsledku anglicky

Advanced therapy medicinal products (ATMPs) and orphan drugs are vital for treating rare diseases, especially in pediatric patients. However, the reimbursement and financing system for these medicines in Czechia lacks sustainability and transparency. This study analyzes the legislative framework governing rare disease medicine reimbursement, with a focus on §16 of Act No 48/1997 Coll. regarding exceptional reimbursement cases. Data from University Hospital Brno highlight the growing reliance on §16, with approval rates for pediatric patients increasing dramatically between 2017 and 2021. Despite high approval rates, the overuse of §16 undermines the need for legislative reform. Significant gaps, such as impractical cost-effectiveness analysis (CEA) requirements, hinder transparency and equitable access. The findings emphasize the urgent need for legislative reforms to ensure a predictable, sustainable, and fair reimbursement system for ATMPs and orphan drugs, particularly benefiting pediatric patients.

Druh

O - Ostatní výsledky

CEP obor

—

OECD FORD obor

30200 - Clinical medicine

Projekt

<a href="/cs/project/LM2023049" target="_blank" >LM2023049: Český národní uzel Evropské sítě infrastruktur klinického výzkumu</a><br>

Návaznosti

P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)<br>S - Specificky vyzkum na vysokych skolach<br>I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2024

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů