OP21 Enhancing Capability: Patient Impact In Ultra-Orphan Conditions
Sign Up to like & getrecommendations! Published in 2019 at "International Journal of Technology Assessment in Health Care"
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
Defining orphan conditions in the context of the European orphan regulation: challenges and evolution
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DOI: 10.1038/nrd.2018.128
Abstract: The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for… read more here.
Keywords: orphan conditions; context european; defining orphan; conditions context ... See more keywords
Designation of orphan conditions in Europe: regulatory observations and considerations after implementation of regulation 141/2000
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DOI: 10.1080/21678707.2020.1784720
Abstract: ABSTRACT Introduction In the first 18 years of the implementation of the orphan medicinal products regulation, it has been noted that every year a steady number of orphan designations refer to new conditions, not designated… read more here.
Keywords: orphan conditions; drug development; designation orphan; regulation ... See more keywords