Advocating for drug development in newborn infants.

Autor: Allegaert K; Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium; Department of Development and Regeneration, KU Leuven, Leuven, Belgium; Department of Hospital Pharmacy, Erasmus Medical Center, 3015GD Rotterdam, the Netherlands. Electronic address: karel.allegaert@kuleuven.be., Mitra S; Division of Neonatology, Department of Pediatrics, University of British Columbia, Vancouver, Canada., Smits A; Department of Development and Regeneration, KU Leuven, Leuven, Belgium; Neonatal Intensive Care Unit, University Hospitals Leuven, Leuven, Belgium., Turner MA; Institute of Lifecourse and Medical Sciences, University of Liverpool, Liverpool Health Partners, Liverpool, United Kingdom; conect4children Stichting, Utrecht, the Netherlands.
Jazyk: angličtina
Zdroj: Early human development [Early Hum Dev] 2024 Dec; Vol. 199, pp. 106136. Date of Electronic Publication: 2024 Oct 29.
DOI: 10.1016/j.earlhumdev.2024.106136
Abstrakt: Neonatal care needs more robust guidance on pharmacotherapy, (formulation, dosage regimen, safety and efficacy information). This requires structured advocacy. We therefore discuss advocacy related to improving information about medicines including current practices, clinical trials, the current setting, and trial preparedness. This steps can improve neonatal drug development by generating evidence, particularly if a programmatic approach (identify dosing, eligibility criteria, and outcomes) to evidence generation is followed. Trial design should be guided by the intended use of the medicine and the benefits/risks that the study participant is exposed to. Regulatory trials (explanatory, controlled environment, internal validity, endpoints reflect clinically important outcomes, strong causal evidence) are sometimes necessary. However, some research questions are best addressed with informative trials. In either case, trial design can be supported by real world data and evidence, extrapolation from other subpopulations, or physiologically-based pharmacokinetic modeling. Data management, safety reporting, and management of drugs should be specified and proportionate. Trial design and conduct also necessitate awareness of Good Clinical Practice specific to neonates. Relevant aspects include protocol and trial design, research skills and interactions with Ethics Committees or Institutional Research Boards, capacities and competences needed within the research team, and aspects related to consent and recruitment.
Competing Interests: Declaration of competing interest The research activities of A. Smits are supported by a Senior Clinical Investigatorship of the Research Foundation Flanders (FWO) (18E2H24N).
(Copyright © 2024 Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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