[HTML][HTML] A systematic review of moral reasons on orphan drug reimbursement
BM Zimmermann, J Eichinger… - Orphanet Journal of Rare …, 2021 - Springer
Background The number of market approvals of orphan medicinal products (OMPs) has
been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for …
been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for …
[HTML][HTML] Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries
Background In case of orphan drugs applicability of the standard health technology
assessment (HTA) process is limited due to scarcity of good clinical and health economic …
assessment (HTA) process is limited due to scarcity of good clinical and health economic …
[BOOK][B] Application of operations research to funding decisions for treatments with rare disease
In this chapter, the focus is on the application of decision analytic tools to assist in
reimbursement decisions related to drugs for rare diseases. Focus is on the evaluative …
reimbursement decisions related to drugs for rare diseases. Focus is on the evaluative …
Opportunity cost of funding drugs for rare diseases: the cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria
D Coyle, MC Cheung, GA Evans - Medical decision making, 2014 - journals.sagepub.com
Background. Both ethical and economics concerns have been raised with respect to the
funding of drugs for rare diseases. This article reports both the cost-effectiveness of …
funding of drugs for rare diseases. This article reports both the cost-effectiveness of …
Priority setting for orphan drugs: an international comparison
ZRS Rosenberg-Yunger, AS Daar, H Thorsteinsdóttir… - Health Policy, 2011 - Elsevier
OBJECTIVES: To describe the process of priority setting for two orphan drugs–Cerezyme
and Fabrazyme–in Canada, Australia and Israel, in order to understand and improve the …
and Fabrazyme–in Canada, Australia and Israel, in order to understand and improve the …
[HTML][HTML] Scoping review on use of drugs targeting interleukin 1 pathway in DIRA and DITRA
F Gómez-García, JL Sanz-Cabanillas… - Dermatology and …, 2018 - Springer
Introduction Deficiencies in interleukin (IL)-1 receptor (IL-R) antagonist (DIRA) and IL-36R
antagonist (DITRA) are rare genetic autoinflammatory diseases related to alterations in …
antagonist (DITRA) are rare genetic autoinflammatory diseases related to alterations in …
[HTML][HTML] Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach
GK Fellows, A Hollis - Orphanet journal of rare diseases, 2013 - Springer
Background Manufacturers justify the high prices for orphan drugs on the basis that the
associated R&D costs must be spread over few patients. The proliferation of these drugs in …
associated R&D costs must be spread over few patients. The proliferation of these drugs in …
[HTML][HTML] Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia …
JI McCormick, LD Berescu, N Tadros - Orphanet Journal of Rare Diseases, 2018 - Springer
Background Public payer reimbursement for non-oncology drugs in Canada, including
orphan drugs, is based on recommendations by the Common Drug Review (CDR)(with the …
orphan drugs, is based on recommendations by the Common Drug Review (CDR)(with the …
Decision models for assessing the cost effectiveness of treatments for pediatric drug-resistant epilepsy: a systematic review of economic evaluations
J Elliott, S van Katwyk, B McCoy, T Clifford… - …, 2019 - Springer
Background Drug-resistant epilepsy affects about one-third of children with epilepsy and is
associated with high costs to the healthcare system, yet the cost effectiveness of most …
associated with high costs to the healthcare system, yet the cost effectiveness of most …
Economic evaluation of stiripentol for Dravet syndrome: a cost-utility analysis
Background Dravet syndrome is a catastrophic form of pediatric treatment-resistant epilepsy
with few effective treatment options. Stiripentol is approved for use in Canada for treatment …
with few effective treatment options. Stiripentol is approved for use in Canada for treatment …
