Enrichment Strategies in Pediatric Drug Development: An Analysis of Trials Submitted to the US Food and Drug Administration

Peer Reviewedhttps://deepblue.lib.umich.edu/bitstream/2027.42/146322/1/cpt971_am.pdfhttps://deepblue.lib.umich.edu/bitstream/2027.42/146322/2/cpt971.pd

Understanding Identity Changes in Psychosis: A Systematic Review and Narrative Synthesis.

BACKGROUND AND OBJECTIVE: Experiencing psychosis can be associated with changes in how people see themselves as individuals and in relation to others (ie, changes in their identity). However, identity changes receive little attention in treatment, possibly due to a lack of clarity or consensus around what identity change means in people with psychosis. We aimed to create a conceptual framework synthesizing how identity changes are understood in the psychosis literature. METHODS: Electronic databases were searched up to April 2020. Studies about identity changes among people with psychotic disorders were analyzed using narrative synthesis by a collaborative review team, including researchers from different disciplines, clinicians, and people who have experienced psychosis. RESULTS: Of 10 389 studies screened, 59 were eligible. Identity changes are understood in 5 ways as (1) characteristics of psychosis, (2) consequences of altered cognitive functioning, (3) consequences of internalized stigma, (4) consequences of lost roles and relationships, and (5) reflections of personal growth. These 5 understandings are not mutually exclusive. Across a heterogeneous literature, identity changes were mostly framed in terms of loss. CONCLUSIONS: Our conceptual framework, comprising 5 understandings, highlights the complexity of studying identity changes and suggests important implications for practice and research. For clinicians, this framework can inform new therapeutic approaches where the experience and impact of identity changes are acknowledged and addressed as part of treatment. For researchers, the conceptual framework offers a way of locating their understandings of identity changes when undertaking research in this area

Development of a neonatal adverse event severity scale through a Delphi consensus approach

BACKGROUND: Assessment of the seriousness, expectedness and causality are necessary for any adverse event (AE) in a clinical trial. In addition, assessing AE severity helps determine the importance of the AE in the clinical setting. Standardisation of AE severity criteria could make safety information more reliable and comparable across trials. Although standardised AE severity scales have been developed in other research fields, they are not suitable for use in neonates. The development of an AE severity scale to facilitate the conduct and interpretation of neonatal clinical trials is therefore urgently needed. METHODS: A stepwise consensus process was undertaken within the International Neonatal Consortium (INC) with input from all relevant stakeholders. The consensus process included several rounds of surveys (based on a Delphi approach), face-to-face meetings and a pilot validation. RESULTS: Neonatal AE severity was classified by five grades (mild, moderate, severe, life threatening or death). AE severity in neonates was defined by the effect of the AE on age appropriate behaviour, basal physiological functions and care changes in response to the AE. Pilot validation of the generic criteria revealed κ=0.23 and guided further refinement. This generic scale was applied to 35 typical and common neonatal AEs resulting in the INC neonatal AE severity scale (NAESS) V.1.0, which is now publicly available. DISCUSSION: The INC NAESS is an ongoing effort that will be continuously updated. Future perspectives include further validation and the development of a training module for users.status: publishe