Advancing Pediatric Drug Development

Understanding demographics of pediatric disease states

The pooled database may be instrumental in understanding genetic predisposition to selected pediatric disease states as well as characterizing any difference in prevalence with regards to geographic region/race/ethnicity/age tier.

• This understanding may be useful in interpreted discrepancy within clinical trials, if any.

• How does global (international) data compare to the FDA pooled database? A collaborative effort... more »

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Advancing Pediatric Drug Development

How to accelerate development of drugs for pediatric patients with type 2 diabetes?

Community Member kudos icon 2 Community member
Current pharmacological treatment options for youth-onset type 2 diabetes (T2D) are inadequate and limited to insulin and metformin. Unmet need is augmented by the fact that metformin monotherapy fails in many pediatric patients during the second year of treatment. The development of pharmacological treatments for pediatric use has been challenged by several factors, including the limited numbers of available pediatric... more »

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Advancing Pediatric Drug Development

Intranasal Antipyretic development in Pediatric Poulation Edited

Pediatric patients with Respiratory illnesses generally arrive to the hospital with high fevers and quite irritable. During this time their respiratory rate and work of breathing often looks much worse until the fever is controlled and mild analgesia is established.  If normothermia can be maintained at home quickly via the use of an intranasal option, these patients may not progress in illness past the tipping point... more »

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Advancing Pediatric Drug Development

Critical questions in pediatric drug safety

1. What are the most critical and significant events in children? Which events lead to drug failures, withdrawals, regulatory warnings, or clinical guideline changes? What level of evidence is required to instigate these changes?

2. How do critical pediatric ADEs differ for low-income, disadvantaged, and/or minority populations? How does representation bias affect performance of AI/ML pediatric drug safety algorithms?... more »

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Advancing Pediatric Drug Development

Safety of intrathecal hydrocortisone trending idea

Hydrocortisone has been administered intrathecally (alone or in combination with other agents) to pediatric patients with cancer for decades. The preferred agent is typically the hydrocortisone 100 mg PLAIN (NDC 0009-0825-01).  

However, the following warning is included in the FDA-approved prescribing information: "SOLU-CORTEF Sterile Powder is contraindicated for intrathecal administration. Reports of severe medical... more »

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Advancing Pediatric Drug Development

Influence of pharmacogenomics on antiemetic response and efficacy

Does CYP2D6 genotype influence ondansetron efficacy and/or toxicity?  For example, do patients with a CYP2D6 ultra-rapid metabolizer phenotype experience more treatment failure?  Do patients with intermediate or poor metabolizer phenotype experience greater toxicity (e.g., QT prolongation, headache, constipation).

https://cpicpgx.org/guidelines/guideline-for-ondansetron-and-tropisetron-and-cyp2d6-genotype/

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Advancing Pediatric Drug Development

What variables/parameters/phenomena depend on age, and what is the link? Can it be explained by body or organ weights only? Edited

Data from J. Valentin, 2003, 'Basic anatomical and physiological data for use in radiological protection: reference values', (Annals of the ICRP, vol. 32, no. 3-4, pp. 1-277 (doi:10.1016/s0146-6453(03)00002-2)) can be used in PBPK modeling to adapt the weight of each organ to the age of the virtual patient. This allows to have differences between the adult and pediatric populations. However we did not have the relevant... more »

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Advancing Pediatric Drug Development

Intranasal/ Oral Antiseptic for Decreasing Viral Load for Common Pediatric Respiratory Viruses

Every year we encounter RSV, Rhino Entero, Para flu, etc. viruses that take quite the tole on our pediatric population.  Nurseries and daycares become super spreaders every season. The idea would be looking at the efficacy of mouth wash or intranasal antiseptic use at these facilities at the start of the day at the beginning and duration of respiratory season. On top of that can it be used for hospital patients/outpatient... more »

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Advancing Pediatric Drug Development

Comparative Evaluation of Adverse events reporting during pediatric clinical trials to that of post-market monitoring reporting

Can the database of pediatric trial data at FDA be used for a systemic review of reported adverse events from clinical trials versus the reported adverse events from the post marketing-monitoring data?

Are there any correlations between reporting of adverse events and demographic make-up of the pediatric populations who participated in clinical trials? If so, how well that it is translated into the adverse event occurrence... more »

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Advancing Pediatric Drug Development

Risk of osteonecrosis and bone fractures with long-term use of proton-pump inhibitors trending idea

Is the long-term use of proton-pump inhibitors associated with an increased risk of bone fractures, osteonecrosis (or even growth delay) in children?  If so, what are the primary determinants of risk?  For example: CYP2D6 metabolizer status, concomitant use of high-dose corticosteroids, age of onset, duration of use, other disease states/comorbidities?  

 

See also: 

https://pubmed.ncbi.nlm.nih.gov/31175146/ 

https://pubmed.ncbi.nlm.nih.gov/32176276/... more »

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