Search results for: Orphan Drugs
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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…
Orphan Drugs Policies: A Suitable Case for Treatment
20 May 2014
…that incentives produce the intended consequences may require redefining ‘orphan disease’ or removing orphan status when a product achieves other, non-orphan, indications. ‘Probably the biggest concern about current policies for…
Access to Orphan Drugs in the EU
21 October 2010
…research that examines the impact of these measures as well as whether and how pricing and reimbursement policies affect access in practice. Martina Garau Orphan drugs, or orphan medicinal products…
Important Variations in Access to Orphan Drugs in France, Italy, Germany, Spain and the UK
14 March 2017
…Garau, M. and Mestre-Ferrandiz, J. (2009). Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries. OHE Briefing. London: Office of Health Economics. Mestre-Ferrandiz, J., Garau, M., O’Neill,…
Establishing a Reasonable Price for an Orphan Drug
11 September 2020
…for orphan drugs are likely to be smaller than for non-orphans. The study estimates differences in the research and development costs between orphan and non-orphans using the number of patients…
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….
Rare Disease Day 2024: Why Do We Care About Rare?
29 February 2024
…drugs. They found that the “standard” £20K/QALY threshold would need to increase to £78K/QALY for orphan drugs (for rare diseases of “average” rarity) and £937K for ultra-orphan drugs. While an…
Appraising Ultra-Orphan Drugs: Is Cost-per-QALY Appropriate?
22 March 2018
A new OHE report discusses NICE’s introduction of a cost-effectiveness threshold to appraise ultra-orphan drugs. A new OHE Consulting Report has been published that discusses the use of a cost-per-QALY…
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…