Health Technology Assessment Guidelines and Recommendations
Across European Union Countries and the United Kingdom in Rare
Disease and Paediatric Populations

J 2026

Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations

BÁRTOVÁ, Adéla; Jiří SAMEK; Vera PINHEIRO; Barbora ŘÍHOVÁ; Joao Vasco SANTOS et al.

Základní údaje

Originální název

Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations

Autoři

BÁRTOVÁ, Adéla; Jiří SAMEK; Vera PINHEIRO; Barbora ŘÍHOVÁ a Joao Vasco SANTOS

Vydání

APPLIED HEALTH ECONOMICS AND HEALTH POLICY, CHAM, SPRINGER INTERNATIONAL PUBLISHING AG, 2026, 1175-5652

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Stát vydavatele

Švýcarsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 3.300 v roce 2024

Označené pro přenos do RIV

Organizační jednotka

Lékařská fakulta

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 10. 3. 2026 10:54, Mgr. Tereza Miškechová

Anotace

V originále

ObjectivesDue to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.MethodsHTA organisations across the 27 EU Member States and the UK were identified via INAHTA, EUnetHTA, and ISPOR reference listings. Publicly available documents were screened, and 29 relevant national guidelines were selected. A structured document analysis was performed using a predefined coding framework. Key terms were systematically searched, and content was categorised into thematic domains.ResultsAmong the 29 guidelines, 16 included references to rare disease populations, and 12 to paediatric populations. For paediatric populations, most references focused on quality-of-life measurement and proxy assessments. Adaptations for rare diseases recognised flexible cost-effectiveness thresholds (e.g. adjusted incremental cost-effectiveness ratios [ICERs] or gross domestic product [GDP]-based modifiers), tailored economic modelling, and acceptance of alternative data sources. However, significant variability was observed across countries, with no consistent pattern.ConclusionWhile several HTA bodies have introduced adjustments for paediatric and rare disease populations, guidance often remains limited and heterogeneous. The findings indicate that HTA guidelines and recommendations require further collaboration to properly define and account for the specific needs of these patients. Methodological shortcomings are mainly due to the nature of these diseases, where the limited data available are primarily from clinical practice and often lack comparative effectiveness evidence.

Návaznosti

LM2023049, projekt VaV

Název: Český národní uzel Evropské sítě infrastruktur klinického výzkumu

Investor: Ministerstvo školství, mládeže a tělovýchovy ČR, CZECRIN - Czech National Node to the European Clinical Research Infrastructure Network

MUNI/A/1722/2024, interní kód MU

Název: Specifický farmakologický výzkum v oblasti farmakokinetiky, behaviorální neuropsychofarmakologie a personalizované farmakoterapie v onkologii

Investor: Masarykova univerzita, Specifický farmakologický výzkum v oblasti farmakokinetiky, behaviorální neuropsychofarmakologie a personalizované farmakoterapie v onkologii

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

BÁRTOVÁ, Adéla; Jiří SAMEK; Vera PINHEIRO; Barbora ŘÍHOVÁ a Joao Vasco SANTOS. Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations. APPLIED HEALTH ECONOMICS AND HEALTH POLICY. CHAM: SPRINGER INTERNATIONAL PUBLISHING AG, 2026, 22 s. ISSN 1175-5652. Dostupné z: https://doi.org/10.1007/s40258-025-01008-0.

@article{2530828,
   author = {Bártová, Adéla and Samek, Jiří and Pinheiro, Vera and Říhová, Barbora and Santos, Joao Vasco},
   article_location = {CHAM},
   doi = {https://doi.org/10.1007/s40258-025-01008-0},
   language = {eng},
   issn = {1175-5652},
   journal = {APPLIED HEALTH ECONOMICS AND HEALTH POLICY},
   title = {Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations},
   url = {https://link.springer.com/article/10.1007/s40258-025-01008-0?utm_source=getftr&utm_medium=getftr&utm_campaign=getftr_pilot&getft_integrator=clarivate},
   year = {2026}
}

TY  - JOUR
ID  - 2530828
AU  - Bártová, Adéla - Samek, Jiří - Pinheiro, Vera - Říhová, Barbora - Santos, Joao Vasco
PY  - 2026
TI  - Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations
JF  - APPLIED HEALTH ECONOMICS AND HEALTH POLICY
PB  - SPRINGER INTERNATIONAL PUBLISHING AG
SN  - 11755652
UR  - https://link.springer.com/article/10.1007/s40258-025-01008-0?utm_source=getftr&utm_medium=getftr&utm_campaign=getftr_pilot&getft_integrator=clarivate
N2  - ObjectivesDue to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.MethodsHTA organisations across the 27 EU Member States and the UK were identified via INAHTA, EUnetHTA, and ISPOR reference listings. Publicly available documents were screened, and 29 relevant national guidelines were selected. A structured document analysis was performed using a predefined coding framework. Key terms were systematically searched, and content was categorised into thematic domains.ResultsAmong the 29 guidelines, 16 included references to rare disease populations, and 12 to paediatric populations. For paediatric populations, most references focused on quality-of-life measurement and proxy assessments. Adaptations for rare diseases recognised flexible cost-effectiveness thresholds (e.g. adjusted incremental cost-effectiveness ratios [ICERs] or gross domestic product [GDP]-based modifiers), tailored economic modelling, and acceptance of alternative data sources. However, significant variability was observed across countries, with no consistent pattern.ConclusionWhile several HTA bodies have introduced adjustments for paediatric and rare disease populations, guidance often remains limited and heterogeneous. The findings indicate that HTA guidelines and recommendations require further collaboration to properly define and account for the specific needs of these patients. Methodological shortcomings are mainly due to the nature of these diseases, where the limited data available are primarily from clinical practice and often lack comparative effectiveness evidence.
ER  -

BÁRTOVÁ, Adéla; Jiří SAMEK; Vera PINHEIRO; Barbora ŘÍHOVÁ a Joao Vasco SANTOS. Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations. \textit{APPLIED HEALTH ECONOMICS AND HEALTH POLICY}. CHAM: SPRINGER INTERNATIONAL PUBLISHING AG, 2026, 22 s. ISSN~1175-5652. Dostupné z: https://doi.org/10.1007/s40258-025-01008-0.

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