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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Advancing Pediatric Drug Development

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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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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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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What have we learnt from successful/failed pediatric oncology trials?

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Based on the pediatric oncology indications FDA has approved or rejected, what have we learnt from the successful/failed trials that might be useful for future trials (e.g. trial designs, endpoints, statistical methods)? In addition, with experience and knowledge accumulated in products approved for tissue agnostic indications, are there diseases (and drug classes) for which extrapolation (adults to adolescents, older... more »

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Off-target toxicity of intravitreal anti-vegf therapy for retinopathy of prematurity

Vascular development is a key process in ongoing postnatal organ development. Intravitreal anti-vegf in preterm infants with ROP is known to be associated with systemic suppression of anti-vegf for prolonged periods of time. Current dosing is off-label  and increasing commonly used. Off-target toxicities are not well understood.

Is this therapy associated with systemic toxicities including worsened bronchopulmonary dysplasia,... more »

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Comparison of Pediatric and Adult Oncology Safety and Efficacy Within Similar Indications

Pediatric oncology clinical trials often begin after the development or approval of a drug for adults. By pooling available pediatric clinical trials and related adult clinical trials, can we assess the safety and efficacy profiles of the drug in the two populations to identify similarity and differences? An analysis could explore the dosing regimens—both the level and frequency of administration—between adults and pediatric... more »

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External Control Arms from Historical Clinical Trial Data for Pediatric Oncology trending idea

Many pediatric oncology clinical trials fail due to lack of patient enrollment. With a low disease prevalence and ethical considerations to treat all patients, efforts should be made to minimize patients assigned to a control arm. Pooled historical control arm data from randomized clinical trials (RCTs) could be utilized to create a regulatory-grade external control arm. Using the pooled pediatric clinical trials available,... more »

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Industry Research Questions - Advancing Pediatric Drug Development

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1. Recently, the FDA has published articles citing the acceptability of extrapolation of efficacy from adults to adolescents in schizophrenia (Kalaria et al. (2019); Kalaria et al. (2020)). This approach may help to greatly accelerate pediatric studies. Can the Agency define extrapolation criteria, i.e., age range, disease characteristics, drug class, that may be leveraged across different CNS indications to position... more »

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