Orphan Drugs
MoCA Initiative and its Value Framework: An Opportunity to Improve Decision Making for Orphan Drugs in Europe?
18 September 2018
Orphan drugs inherently face a number of challenges in their development and value assessment. In this blog post, we describe the Mechanism of Coordinated Action (MoCA)…
Establishing a Reasonable Price for an Orphan Drug
1 July 2018
The high cost of drugs for rare diseases (‘orphan drugs’) has generated considerable debate. While there is debate in the economic literature over whether a premium…
Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence
1 March 2018
The report addresses the implications of NICE appraising treatments for very rare diseases using a cost-per-QALY gained decision rule of the type used by NICE in…
Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries
1 March 2017
The European Commission’s (EC) Orphan Medicinal Products Regulation intended to incentivise the research, development and marketing of new treatments for rare and chronically disabling or life-threatening…
Multi-Criteria Decision Analysis to Value Orphan Medicines
1 May 2013
The purpose of this research is to identify the attributes to include in a value framework for orphan medicinal products (OMPs), determine their relative importance using…
Assessment of the Impact of Orphan Medicinal Products on the European Economy and Society
1 November 2010
This report examines the effect of the European Union’s 1999 Regulation on Orphan Medicinal Products (OMPs) on the European economy and society. This study collected data…
Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries
1 November 2009
This study compares pricing and reimbursement (P&R) policy for 43 orphan medicinal products (OMPs) across seven EU Member States: France, Germany, Italy, The Netherlands, Spain, Sweden…