Assessment of Long Term Neurodevelopmental Outcome Following Trials of Medicinal Products in Newborn Infants

Importance: There is significant uncertainty over the assessment of long-term neurodevelopmental outcome (LTO) in neonatal clinical trials. Objective: To identify key issues in the assessment of LTO. Design: To examine optimal approaches to evaluate LTO for therapeutic trials in newborns. Sponsors and investigators will develop the study (including LTO) with other key stakeholders. Method: Multidisciplinary working group report. Conclusions and Relevance: 1. The potential of the investigational product to cause widespread effects drives the need to assess outcome in multiple organs. 2. The nature of the intervention a. Does the investigational product have an impact on the brain? b. Are there potential effects of the investigational product on LTO? c. Should safety be assessed? 3. All decisions need to be specific to the product being studied using published data and only considering expert opinion when prior evidence does not exist. 4. The balance of benefits, costs, and burdens of assessments to the researcher and families need to be considered. 5. Families and parent advocates should be involved in design and execution of the study. All key stakeholders may use the framework to determine the need, nature, and duration of LTO assessments in regulatory trials involving newborn infants