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Rare Disease Day 2024: Why Do We Care About Rare?
29 February 2024
…event was launched by EURORDIS (Rare Disease Europe and its Council of National Alliances) in 2008, to raise awareness among the public and decision-makers about rare diseases and their impact….
Advancing Rare Disease Care: Challenges and Key Issues
28 February 2025
…and pricing and reimbursement—to support a resilient healthcare and innovation ecosystem for rare diseases. Promoting equitable access to medicines for rare diseases is a key component of the World Health…
How Much Should Society Pay for a New Orphan Drug? A Contribution to this Debate is now Available in a New OHE Research Paper
18 July 2018
…by the two main factors playing a role: (i) the cost of R&D for orphan drugs as compared to non-orphans and, (ii) patient population sizes targeted by orphans and non-orphans….
Establishing a Reasonable Price for an Orphan Drug
11 September 2020
…SMC and NICE define ultra-orphan drugs as medicines granted orphan status by the EMA and treating conditions with a prevalence of 1 in 50,000 people in England and/or Scotland –…
Establishing a Reasonable Price for an Orphan Drug
1 July 2018
…(i) the cost of R&D for orphan drugs as compared to non-orphans and, (ii) patient population sizes targeted by orphans and non-orphans. Research shows that, on average, the estimated research…
Ethical and Economic Issues in the Appraisal of Medicines for Ultra-rare (or Ultra-orphan) Conditions
13 January 2020
…and Mireia Jofre-Bonet discusses the ethical and economic issues in the appraisal of medicines for ultra-rare conditions (also known as ultra-orphan medicines). The authors examine the current literature and identify…
Rare Disease Day 2022
28 February 2022
World Rare Disease Day aims to raise awareness of rare diseases and the impact they can have on patients. This blog explores OHE’s contributions to research into rare diseases and…
MoCA Initiative and its Value Framework: An Opportunity to Improve Decision Making for Orphan Drugs in Europe?
18 September 2018
…it can be beneficial to all parties involved and how it can improve decision making for – and access to – orphan drugs in European countries. Orphan drugs inherently face…
Recent Statistics on Orphan Approvals in Scotland and England
23 August 2011
…same methodology, this new analysis focuses on decisions taken on orphan drugs by the Scottish Medicines Consortium (SMC) and the National Institute for Health and Clinical Excellence (NICE). Included are…