Evidence quality and uncertainties considered in appraisal documents of drugs for rare diseases in England and Germany: a data extraction protocol

Introduction Rare disease treatments (RDTs) promise considerable patient benefit but the evidence to demonstrate their value in health technology assessment (HTA) is often limited. HTA outcomes for RDTs vary across countries and there are differences in how uncertainty is dealt with by HTA agencies....

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Bibliographic Details
Main Authors: Ellen Nolte, John Cairns, Lea Wiedmann
Format: Article
Language:English
Published: BMJ Publishing Group 2025-02-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/15/2/e089418.full
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