Challenges and opportunities for improving access to approved neonatal drugs and devices
TLDR
In this paper , the authors argue that neonatal drug and device development has lagged behind other patient populations and that providers are using drugs and devices without adequate study of safety and efficacy, which will require novel approaches and unique collaborations between multiple key stakeholders.Abstract:
Neonatal drug and device development has lagged behind other patient populations. Oftentimes, providers are using drugs and devices without adequate study of safety and efficacy. Neonates deserve dedicated drug and device development programs, which will require novel approaches and unique collaborations between multiple key stakeholders. Legislative efforts, infrastructure, clinical trial methodology, and international collaborations have all contributed to improvements in neonatal drug and device development, but more work is still needed. Leadership from neonatologists, clinical care providers, and parents is essential to implement needed changes. read more
Citations
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Advancing Precision Medicine in Paediatrics; Past, Present and Future
Abdelbaset A. Elzagallaai,Charlotte I. S. Barker,Tamorah Lewis,Ronald Cohn,Michael J. Rieder +4 more
TL;DR: In this paper , the authors consider the role of ontogeny and genetics in determining drug efficacy and safety for infants, children, and youth in the care of infants and children.
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Barriers in translating stem cell therapies for neonatal diseases.
TL;DR: In this paper , the authors explore the existing clinical evidence for stem cell therapies in neonates, highlight the barriers faced by researchers, and suggest potential solutions to move the field forward.
References
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Real-World Evidence — What Is It and What Can It Tell Us?
Rachel E. Sherman,Steven A. Anderson,Gerald J. Dal Pan,Gerry W. Gray,Thomas G. Gross,Nina L. Hunter,Lisa M. LaVange,Danica Marinac-Dabic,Peter W. Marks,Melissa A. Robb,Jeffrey Shuren,Robert Temple,Janet Woodcock,Lilly Q. Yue,Robert M. Califf +14 more
TL;DR: The FDA is developing guidance on the use of “real-world evidence” — health care information from atypical sources, including electronic health records, billing databases, and product and disease registries — to assess the safety and effectiveness of drugs and devices.
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Multidimensional Evidence Generation and FDA Regulatory Decision Making: Defining and Using “Real-World” Data
TL;DR: The widespread use of electronic health records, administrative claims, and social media and the ubiquity of smart devices have created “big data” that heretofore have not been widely utilized, but there are other potential applications.
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Drug Labeling and Exposure in Neonates
Matthew M. Laughon,Debbie Avant,Nidhi Tripathi,Christoph P. Hornik,Michael Cohen-Wolkowiez,Reese H. Clark,P. Brian Smith,William J. Rodriguez +7 more
TL;DR: To quantify progress made in neonatal studies and neonatal information in product labeling as a result of recent legislation, a cohort of drug studies between 1997 and 2010 that included neonates was identified using information available on the FDA website.
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Core outcomes in neonatology: Development of a core outcome set for neonatal research
James Webbe,James Duffy,Elsa Afonso,Iyad Al-Muzaffar,Ginny Brunton,Anne Greenough,Nigel J. Hall,Marian Knight,Jos M. Latour,Caroline Lee-Davey,Neil Marlow,Laura Noakes,Julie Nycyk,Angela Richard-Löndt,Ben Wills-Eve,Neena Modi,Chris Gale +16 more
TL;DR: A core outcome set (COS) for research involving infants receiving neonatal care in a high-income setting has been identified to help standardise outcome selection in clinical trials and ensure these are relevant to those most affected by Neonatal care.
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Safety, dosing, and pharmaceutical quality for studies that evaluate medicinal products (including biological products) in neonates
Robert M. Ward,Daniel K. Benjamin,Jeffrey S. Barrett,Karel Allegaert,Karel Allegaert,Ronald J. Portman,Jonathan M. Davis,Mark A. Turner +7 more
TL;DR: An international consortium from Canada, Europe, Japan, and the United States has developed a comprehensive, referenced White Paper to guide neonatal clinical trials of medicines – particularly early phase studies, highlighting the need to base product development on neonatal physiology and pharmacology.