The ethical framework for performing research with rare inherited neurometabolic disease patients.
The need for performing clinical trials to develop well-studied and appropriate medicines for inherited neurometabolic disease patients faces ethical concerns mainly raising from four aspects: the diseases are rare; include young and very young patients; the neurological impairment may compromise th...
| Publicado en: | European Journal of Pediatrics Vol. 176; no. 3; pp. 395 - 406 |
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| Autores principales: | , , , , , , , , , , , , , , , , , , , |
| Formato: | research systematic review Journal Article |
| Publicado: |
Springer Nature
Mar2017
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| Acceso en línea: | Ver este registro en EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=121387438&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 121387438 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 03406199 CR1 jtl: European Journal of Pediatrics issn: 03406199 maglogo: N pubinfo: dt: Mar2017 vid: 176 iid: 3 pid: 237 pub: Springer Nature place: New York, New York artinfo: ui: 121387438 121387438 143939803 NLM28093642 121387438 10.1007/s00431-017-2852-9 NLM28093642 121387438 ppf: 395 ppct: 11 formats: fmt: @attributes: type: P tig: atl: The ethical framework for performing research with rare inherited neurometabolic disease patients. aug: au: Giannuzzi, Viviana Devlieger, Hugo Margari, Lucia Odlind, Viveca Ragab, Lamis Bellettato, Cinzia D'Avanzo, Francesca Lampe, Christina Cassis, Linda Cortès-Saladelafont, Elisenda Cazorla, Ángels Barić, Ivo Cvitanović-Šojat, Ljerka Fumić, Ksenija Dali, Christine Bartoloni, Franco Bonifazi, Fedele Scarpa, Maurizio Ceci, Adriana Odlind, Viveca Lena affil: Fondazione Per la Ricerca Farmacologica Gianni Benzi Onlus , Via Abate Eustasio 30 70010 Valenzano Italy sug: subj: Research, Medical Ethical Issues Consent Legislation and Jurisprudence Nervous System Diseases Disease Attributes Metabolic Diseases Clinical Trials Ethical Issues Human European Union Child Europe Systematic Review Child: 6-12 years ab: The need for performing clinical trials to develop well-studied and appropriate medicines for inherited neurometabolic disease patients faces ethical concerns mainly raising from four aspects: the diseases are rare; include young and very young patients; the neurological impairment may compromise the capability to provide 'consent'; and the genetic nature of the disease leads to further ethical implications. This work is intended to identify the ethical provisions applicable to clinical research involving these patients and to evaluate if these cover the ethical issues. Three searches have been performed on the European regulatory/legal framework, the literature and European Union-funded projects. The European legal framework offers a number of ethical provisions ruling the clinical research on paediatric, rare, inherited diseases with neurological symptoms. In the literature, relevant publications deal with informed consent, newborn genetic screenings, gene therapy and rights/interests of research participants. Additional information raised from European projects on sharing patients' data from different countries, the need to fill the gap of the regulatory framework and to improve information to stakeholders and patients/families.Conclusion: Several recommendations and guidelines on ethical aspects are applicable to the inherited neurometabolic disease research in Europe, even though they suffer from the lack of a common ethical approach. What is Known: • When planning and conducting clinical trials, sponsors and researchers know that clinical trials are to be performed according to well-established ethical rules, and patients should be aware about their rights. • In the cases of paediatric patients, vulnerable patients unable to provide consent, genetic diseases' further rules apply. What is New: • This work discusses which ethical rules apply to ensure protection of patient's rights if all the above-mentioned features coexist. • This work shows available data and information on how these rules have been applied. pubtype: Academic Journal doctype: research systematic review Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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