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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1 vote

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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2 votes

Advancing Pediatric Drug Development

Diversity and Inclusion

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Diversity in clinical trials is imperative to better understand the safety and efficacy profile of novel pediatric therapies and to reduce disparities and advance equal access and opportunities. Yet, the extent to which individuals from minority racial and ethnic groups, across different educational backgrounds, and those with intellectual disabilities and other cognitive impairments are represented in pediatric clinical... more »

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6 votes

Advancing Pediatric Drug Development

Pediatric biomarkers

There is extreme interest in identifying pediatric specific biomarkers for multiple disease states including NAFLD, heart failure, chronic kidney disease (CKD).

• Biomarkers may serve as alternatives to invasive diagnostic techniques (e.g. Liver biopsy in NAFLD), early indicators of disease progression, and predictors of treatment efficacy. Does the pooled database support this?

• Specifically, what is the utility of... more »

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4 votes

Advancing Pediatric Drug Development

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

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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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2 votes

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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2 votes

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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2 votes

Advancing Pediatric Drug Development

Steroid-resistant acute graft-versus-host disease in pediatric populations

In steroid-resistant graft versus host disease, what are the expected distributions of main characteristics (age, underlying disease, aGvHD grade and organs affected, source of the graft, graft compatibility, type of conditioning regimen, distribution of steroid-resistance ... ) ? Which ones differ importantly from the adult population ?

Some characteristics are known to differ between age groups in aGvHD, such as the... more »

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3 votes

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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2 votes

Advancing Pediatric Drug Development

Longitudinal natural history of pediatric disease

Can the pooled databases be used to better understand the longitudinal natural history of pediatric disease states such as Nonalcoholic fatty liver disease (NAFLD), including differences between pediatric and adult onset, pathophysiology, mechanism and disease progression/long term sequelae?

• Considering the recently published paper on the long term complications observed in the TODAY study, long term cardiovascular... more »

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3 votes

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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3 votes