Advancing Pediatric Drug Development

The quality and quantity of reports of suspected ADRs from young people

Spontaneous adverse drug reaction reports to the UK regulatory agency (MHRA) have shown a difference in both the type of drugs and reactions reported, when comparing reports about young people, and those that originated from a young person themselves (Bhoombhla 2020). The reactions that were identified by young people included serious mental health issues. This difference may also be true in clinical trials - but it would... more »

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Advancing Pediatric Drug Development

Identification of Key Gaps in Neonatal and Infant Ontogenic Data for Drug Metabolism and Response

The FDA likely has the largest repository of pediatric dose-exposure and exposure-response data of any entity, including these data for the same drug in multiple age groups. For key drugs / drug classes and metabolic enzymes, the developmental profile of metabolism and drug target could be investigated, with the goal of identifying key enzymes /  transporters where there is insufficient data.

The answering of this question... more »

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Advancing Pediatric Drug Development

Biomarkers and Surrogate Endpoints In Clinical Trials

For DMD clinical trials, most of the associated clinically meaningful endpoints take long time (5-7 years) to show significant improvement. That makes the idea of using biomarkers and surrogate endpoints in this type of clinical trials very practical. It would be a good practice to use surrogate endpoints in clinical trial conditional on follow-up study to prove clinically meaningful endpoints in a long duration.

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Advancing Pediatric Drug Development

Effect of Vitamin D Supplementation on Bone Health During Cancer Treatment

Oncology therapies (steroids, methotrexate) and supportive care medications (proton pump inhibitors?) are part of a multi-modal approach to curing pediatric acute lymphoblastic leukemia and also contribute to osteopenia/osteoporosis.  Pediatric patients are different from adult patients in the way that their bones are still maturing during oncology treatment.  

What are the rates of osteopenia/osteoporosis and/or AVN... more »

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Advancing Pediatric Drug Development

Making the medicine taste OK

 Flavouring and sweetening are often required to enhance the palatability/acceptability of paediatric products that are intended for delivery as oral liquid dosage forms. What flavours and sweeteners have been used in applications for products that were accepted by the agency and what evidence was provided on the acceptability of those products to the paediatric patients?

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Childhood Obesity: Preventing chronic disease via pharmaceutical intervention

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It is well know that obesity leads to many chronic diseases. We also know that genetic, behavioral and environmental factors contribute to this continuously growing disease. However, there is insufficient investigation into if pharmaceutical intervention in children could stop and reverse obesity in children, and in turn prevent long term chronic diseases.

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Decentralized Clinical Trials Edited

Pooled analyses can only be conducted if the included studies use the same study design and statistical models, and if their respective populations were homogeneous.

Decentralized clinical trials can improve trial homogeneity as well as participant diversity, eliminate geographic barriers, allow for participation in clinical trials through local practices, allow for local subsequent testing, and eliminate participant... more »

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Advancing Pediatric Drug Development

COVID-19 Best COVID-19 Therapeutic Treatment

For children under 12 who are COVID-19 positive (+), what medical therapeutics treatments have shown most efficacy against COVID-19 which prevented or reduced hospitalization?  

Hopefully this can be done by MIDD (meta-analysis) from hospitals, urgent care, and doctor's office who submitted protocol treatments used by the various facilities.

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Advancing Pediatric Drug Development

Biliary Atresia potential treatment other than liver transplantation

Current there is no cure for biliary atresia other than liver transplantation.  From the current clinical studies world wide, we believe  that targeting liver fibrosis and cirrhosis will ultimately help those children with this rare disease. And such treatment could be expanded to the general population for any cause related liver cirrhosis. 

 

Our preliminary research has found out that targeting TGF-beta and IL-2... more »

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