Title: Access to Orphan and Ultra-Orphan Drugs in China

Mechanism of coordinated access to orphan drugs

Context Although the EU Council stated[1] that “All health systems in the EU aim to make provision, which is patient-centered and responsive to individual need”, numerous sources show important and unacceptable differences in access to orphan drugs in the Member States of the European Union (EU COM [2], EURORDIS [3,4], BE EU Presidency [5,8], EU Council [6]). With this regard, in the context of...

Access To Orphan Drugs In Greece During Economic Crisis.

Background & Objectives Orphan Drugs (ODs) are medicinal products intended for diagnosis, prevention or treatment of rare diseases. Access to ODs is crucial for patients' health and quality of life. However, patients claim that access to orphan drugs is sometimes restricted, a phenomenon that has grown during the economic crisis. The aim of this study was to identify current problems and future...

Developing a Patient-Directed Policy Framework for Managing Orphan and Ultra-Orphan Drugs Throughout Their Lifecycle

INTRODUCTION Policy decisions related to orphan and ultra-orphan drugs challenge traditional decision-making processes and often frustrate those affected by them. In general, these drugs are associated with significant uncertainties around clinical benefit, 'value for money', affordability, and 'adoption/diffusion', all of which arise from a lack of available high-quality evidence. Increasingly...

Orphan drugs revisited.

Commentary Orphan drugs revisited

C. MCCABE, A. TSUCHIYA, K. CLAXTON and J. RAFTERY From the Section of Health Economics and Decision Science, School of Health and Related Research, University of Sheffield, Department of Economics, University of Sheffield, Sheffield, Department of Economics and Related Studies, University of York, York, and Wessex Institute for Health Research and Development, University of Southampton, Southam...