Premature births pose significant risks to infants, particularly those born at less than 28 weeks’ gestation. To address the challenges associated with premature births, researchers have developed artificial womb technology (AWT) as a potential solution. AWT aims to simulate the womb’s physiologic environment, providing a bridge from extreme preterm birth to later gestation and reducing the morbidity associated with standard neonatal intensive care unit (NICU) practices. However, ahead of a Pediatric Advisory Committee meeting, FDA staff have raised concerns that must be addressed before potential human tests of AWT can proceed.
The FDA has identified four major points for discussion regarding the potential human tests of AWT:
1. Assessing Animal Models and Gathering Additional Data
Before moving on to human trials, it is crucial to evaluate the strengths and weaknesses of the existing large animal models used to test AWT. The FDA staff is seeking clarity on what additional data is necessary to support the proof of concept for AWT.
2. Addressing Knowledge Gaps through Nonclinical Studies
To strengthen the evidence base, FDA reviewers are requesting consideration of knowledge gaps that could be addressed through nonclinical studies. Acquiring this knowledge will be essential to determine the effectiveness and safety of AWT so that it can be translated into human trials.
3. Ensuring Safety and Guideline Compliance
A critical aspect of introducing AWT to human trials is ensuring patient safety. FDA staff has highlighted the need for a discussion on whether the existing data is sufficient to support first-in-human studies and the guidelines that should be followed to ensure patient safety throughout the trial.
4. Consent and Ethical Considerations
Due to the involvement of neonatal patients, there are additional ethical considerations associated with AWT trials. The panel is expected to address the challenges of obtaining informed consent in an AWT trial and discuss any foreseeable obstacles.
Several research institutions, including the University of Michigan, Tohoku University, the University of Western Australia, and the Children’s Hospital of Philadelphia, have utilized large animal models to assess AWT. The FDA recognizes the importance of these studies; however, it emphasizes the necessity of addressing the ethical considerations associated with involving neonatal patients in research.
Any first-in-human AWT trial must demonstrate a clinically significant benefit that justifies the risks involved. The FDA reviewers highlight the importance of comparing AWT with accepted alternative treatments in terms of benefit-risk profile. Adequate data showing at least comparable benefits to current NICU standard of care are necessary to proceed with human trials.
The FDA staff also outlines the essential components that a first-in-human study of AWT should incorporate. These components include robust safety monitoring and adverse event reporting, pre-established individual and study stopping rules, oversight by an independent Data and Safety Monitoring Board (DSMB), endpoints for evaluating treatment efficacy, and an effective informed consent process. Each of these elements is vital to ensure the safety and integrity of the trial.
As AWT technology progresses, it is crucial to address the concerns raised by FDA staff to ensure the safety and efficacy of potential human trials. Evaluating existing animal models, filling knowledge gaps through nonclinical studies, establishing safety guidelines, and navigating ethical concerns surrounding consent are key areas that need to be thoroughly explored. While AWT holds promise for improving the outcomes of extremely premature infants, it is essential to prioritize patient safety and gather compelling evidence before proceeding with the next stages of clinical development. By carefully addressing these concerns, researchers and healthcare professionals can pave the way for a future in which AWT becomes a vital tool in providing optimal care for premature infants.
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