Health Technology Assessment Guidelines and Recommendations Across European Union Countries and the United Kingdom in Rare Disease and Paediatric Populations
While 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.
Authors
- Vera Pinheiro (ORCID: https://orcid.org/0000-0002-1236-4798)
- Jiří Samek
- João Vasco Santos (ORCID: https://orcid.org/0000-0003-4696-1002)
- Barbora Říhová (ORCID: https://orcid.org/0000-0003-3835-9062)
- Adéla Bártová (ORCID: https://orcid.org/0000-0002-1619-217X)
Institutions
- Universidade do Porto (PT)
- Masaryk University (CZ)
- Hospital de Santo António (PT)
- Decision Sciences (United States) (US)
Publication Details
- Journal
- Applied Health Economics and Health Policy
- Published
- 2025-10-16
- DOI
- https://doi.org/10.1007/s40258-025-01008-0
- Primary Topic
- Health Systems, Economic Evaluations, Quality of Life
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Univerzita Komenského v Bratislave
- ZonMw
- Erasmus Universiteit Rotterdam
- Universiteit Utrecht
- Ministero della Salute
- Ministerstvo Zdravotnictví Ceské Republiky
- Office of the Civil Service Commission
- Emberi Eroforrások Minisztériuma
- Regione del Veneto
- Zorginstituut Nederland
- Institut National d'assurance Maladie-Invalidité
- Terveyden ja hyvinvoinnin laitos
- Gemeinsame Bundesausschuss
- Ministrstvo za zdravje
- Agenzia Italiana del Farmaco, Ministero della Salute