Show simple item record McCormack P, Woods S, Aartsma-Rus A, Hagger L, Herczegfalvi, Ágnes 2014-11-14T09:25:48Z 2014-11-14T09:25:48Z 2013
dc.identifier 84874122651
dc.identifier.citation; journalVolume=2013; journalTitle=PLOS CURRENTS;
dc.identifier.uri doi:10.1371/
dc.description.abstract Drug trials in children engage with many ethical issues, from drug-related safety concerns to communication with patients and parents, and recruitment and informed consent procedures. This paper addresses the field of neuromuscular disorders where the possibility of genetic, mutation-specific treatments, has added new complexity. Not only must trial design address issues of equity of access, but researchers must also think through the implications of adopting a personalised medicine approach, which requires a precise molecular diagnosis, in addition to other implications of developing orphan drugs. It is against this background of change and complexity that the Project Ethics Council (PEC) was established within the TREAT-NMD EU Network of Excellence. The PEC is a high level advisory group that draws upon the expertise of its interdisciplinary membership which includes clinicians, lawyers, scientists, parents, representatives of patient organisations, social scientists and ethicists. In this paper we describe the establishment and terms of reference of the PEC, give an indication of the range and depth of its work and provide some analysis of the kinds of complex questions encountered. The paper describes how the PEC has responded to substantive ethical issues raised within the TREAT-NMD consortium and how it has provided a wider resource for any concerned parent, patient, or clinician to ask a question of ethical concern. Issues raised range from science related ethical issues, issues related to hereditary neuromuscular diseases and the new therapeutic approaches and questions concerning patients rights in the context of patient registries and bio-banks. We conclude by recommending the PEC as a model for similar research contexts in rare diseases.
dc.relation.ispartof urn:issn:2157-3999
dc.title Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases
dc.type Journal Article 2014-11-14T09:24:55Z
dc.language.rfc3066 en
dc.identifier.mtmt 2372763
dc.identifier.pubmed 23330068
dc.contributor.department Semmelweis Egyetem
dc.contributor.institution Semmelweis Egyetem

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