Header

UZH-Logo

Maintenance Infos

Assessment of long-term neurodevelopmental outcome following trials of medicinal products in newborn infants


Marlow, Neil; Doyle, Lex W; Anderson, Peter; Johnson, Samantha; Bhatt-Mehta, Varsha; Natalucci, Giancarlo; Darlow, Brian A; Davis, Jonathan M; Turner, Mark A; International Neonatal Consortium (INC) (2019). Assessment of long-term neurodevelopmental outcome following trials of medicinal products in newborn infants. Pediatric Research, 86(5):567-572.

Abstract

There is significant uncertainty over the role of assessment of long-term neurodevelopmental outcome (LTO) in neonatal clinical trials. A multidisciplinary working group was established to identify key issues in this area and to make recommendations about optimal approaches to evaluate LTO in therapeutic trials in newborns, which can be developed by sponsors and investigators with other key stakeholders. A key consideration for neonatal trials is the potential for the investigational product to cause widespread effects and drives the need to assess outcome in multiple organs. Thus investigators must assess whether the product has an impact on the brain and the potential for it to cause potential effects on LTO. Critically, is assessment of LTO an important direct therapeutic target or a safety outcome? Such decisions and outcomes need to be specific to the product being studied and use published data, only considering expert opinion when prior evidence does not exist. In designing the trial, the balance of benefits, costs, and burdens of assessments to the researcher and families need to be considered. Families and parent advocates should be involved in design and execution of the study. A framework is presented for use by all key stakeholders to determine the need, nature, and duration of LTO assessments in regulatory trials involving newborn infants.

Abstract

There is significant uncertainty over the role of assessment of long-term neurodevelopmental outcome (LTO) in neonatal clinical trials. A multidisciplinary working group was established to identify key issues in this area and to make recommendations about optimal approaches to evaluate LTO in therapeutic trials in newborns, which can be developed by sponsors and investigators with other key stakeholders. A key consideration for neonatal trials is the potential for the investigational product to cause widespread effects and drives the need to assess outcome in multiple organs. Thus investigators must assess whether the product has an impact on the brain and the potential for it to cause potential effects on LTO. Critically, is assessment of LTO an important direct therapeutic target or a safety outcome? Such decisions and outcomes need to be specific to the product being studied and use published data, only considering expert opinion when prior evidence does not exist. In designing the trial, the balance of benefits, costs, and burdens of assessments to the researcher and families need to be considered. Families and parent advocates should be involved in design and execution of the study. A framework is presented for use by all key stakeholders to determine the need, nature, and duration of LTO assessments in regulatory trials involving newborn infants.

Statistics

Citations

Dimensions.ai Metrics

Altmetrics

Downloads

16 downloads since deposited on 12 Dec 2019
16 downloads since 12 months
Detailed statistics

Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Hospital Zurich > Clinic for Neonatology
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:November 2019
Deposited On:12 Dec 2019 11:43
Last Modified:01 Mar 2020 14:22
Publisher:Nature Publishing Group
ISSN:0031-3998
OA Status:Green
Free access at:PubMed ID. An embargo period may apply.
Publisher DOI:https://doi.org/10.1038/s41390-019-0526-1
PubMed ID:31398720

Download

Green Open Access

Download PDF  'Assessment of long-term neurodevelopmental outcome following trials of medicinal products in newborn infants'.
Preview
Content: Published Version
Filetype: PDF
Size: 518kB
View at publisher
Licence: Creative Commons: Attribution 4.0 International (CC BY 4.0)