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Orphan drug considerations in Health Technology Assessment ineight European countries

Tordrup, David, Tzouma, Victoria and Kanavos, Panos ORCID: 0000-0001-9518-3089 (2014) Orphan drug considerations in Health Technology Assessment ineight European countries. Rare Diseases and Orphan Drugs: An International Journal of Public Health, 1 (3). pp. 86-97.

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Health Technology Assessment (HTA) is used to assess the value of new technologies and by producing coverage recommendations it indirectly controls the uptake of new technologies in many European countries. While HTA generally relies on a robust assessment of the clinical cost-effectiveness of a new technology, the clinical and economic evidence required for this purpose is often not available for Orphan Drugs (ODs), partly because of challenges related to the recruitment of patients to participate in clinical trials. A number of European HTA agencies have started to implement specific policies to address the challenges related to evidence requirements for the case of ODs. In this study, we map out the policies currently in place in eight European countries regarding HTA and its application to the case of ODs and explore the implications these policies have for coverage decisions.

Item Type: Article
Official URL:
Additional Information: © 2014 The Authors
Divisions: European Institute
Social Policy
LSE Health
Subjects: H Social Sciences > HV Social pathology. Social and public welfare. Criminology
R Medicine > RA Public aspects of medicine > RA0421 Public health. Hygiene. Preventive Medicine
T Technology > T Technology (General)
Date Deposited: 05 Sep 2014 13:56
Last Modified: 15 Sep 2023 15:02
Funders: European Union Seventh Framework Programme (FP7/2007-2013) under grant agreement n° 305690 (Rare Best Practices)

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