Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations.
Journal
Drug discovery today
ISSN: 1878-5832
Titre abrégé: Drug Discov Today
Pays: England
ID NLM: 9604391
Informations de publication
Date de publication:
07 2020
07 2020
Historique:
received:
03
02
2020
revised:
08
04
2020
accepted:
15
04
2020
pubmed:
29
4
2020
medline:
31
8
2021
entrez:
29
4
2020
Statut:
ppublish
Résumé
To maintain orphan drug status at the time of market authorization, orphan medicinal products (OMPs) need to be assessed for all criteria, including significant benefit, by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). Subsequently, health technology assessment (HTA) organizations evaluate the same OMPs in their relative effectiveness assessments (REAs). This review investigates the similarities and differences between the two frameworks for six HTA organizations, including the European Network for HTA. We discuss differences between both assessment frameworks within five domains (clinical evidence used, patient population, intervention, comparators, and outcome measures) for all drugs. Five illustrative cases studies were selected for a qualitative review.
Identifiants
pubmed: 32344040
pii: S1359-6446(20)30160-4
doi: 10.1016/j.drudis.2020.04.012
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1223-1231Informations de copyright
Copyright © 2020 The Authors. Published by Elsevier Ltd.. All rights reserved.