International differences in patient access to ultra-orphan drugs
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DOI: 10.1016/j.hlpt.2017.12.001
Abstract: Abstract Objectives Reimbursement recommendations on (orphan) drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient… read more here.
Keywords: status; orphan; orphan drugs; patient access ... See more keywords
OP21 Enhancing Capability: Patient Impact In Ultra-Orphan Conditions
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DOI: 10.1017/s026646231900093x
Abstract: Introduction Written evidence is submitted to the National Institute for Heath and Care Excellence (NICE) by patient organisations for all ultra-orphan evaluations. To enhance the capability of patient involvement at NICE and to further develop… read more here.
Keywords: orphan conditions; committee; impact; evidence ... See more keywords
Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs
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DOI: 10.1186/s13023-017-0690-5
Abstract: BackgroundThe unmet medical needs of individuals with very rare diseases are high. The clinical trial designs and evaluation methods used for ‘regular’ drugs are not applicable in the clinical development of ultra-orphan drugs ( read more here.
Keywords: development ultra; clinical development; ultra orphan; orphan drugs ... See more keywords