Showing 10 ideas for label trending idea

Advancing Pediatric Drug Development

Translation from adults data trending idea

Community Member kudos icon + Community member
The common approach to dose selection in pediatric population when only adults data is available is population PKPD (PBPK) modelling.

If age and weight are not identified as covariates for the model, if the flat exposure-response curve is observed in adult population, if in vitro tests and biology theory claim that there are no effects of growth and maturation on the observed levels of PD-markers (or components of the... more »

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9 votes

Advancing Pediatric Drug Development

Predictability of Adult Biomarkers Across Pediatric Disease and Therapeutic Areas trending idea

While there is general recognition of the need for pediatric-specific biomarkers, sponsors and researchers are often left with adult biomarkers out of convenience or necessity. It would be helpful to have a broader understanding of the suitability of this occurrence for dose selection and/or clinical endpoint utility across pediatric disease states, therapeutic area, drug class and developmental status to drive the effort... more »

Voting

12 votes

Advancing Pediatric Drug Development

MRD as a surrogate for Overall Survival trending idea

MRD as a general measure of tumor burden has multiple potential regulatory and clinical uses as a biomarker; however, the evidence to support the clinical validity of MRD as a biomarker varies across hematologic cancer types and patient populations. In pediatric patients with relapsed/refractory AML, MRD-negative CR has not yet been established as an intermediate endpoint likely to predict overall survival. To investigate... more »

Voting

22 votes

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 »

Voting

1 vote

Advancing Pediatric Drug Development

Reducing or eliminating placebo burden on pediatric patients in areas of unmet need trending idea

Community Member kudos icon 34 Community member
i. With the goal of reducing or eliminating placebo burden on pediatric patients while maintaining FDA's high standards for the development of safe and effective medicines, it is recommended that FDA conduct analyses of placebo and natural history data from both successful and unsuccessful clinical trials in pediatric rare diseases. The aims of the research should be to: 1) consider published, peer-reviewed literature... more »

Voting

27 votes

Advancing Pediatric Drug Development

Data requirement to facilitate Pediatric Drug Development trending idea

How could we maximize use of all available data (e.g., existing clinical data in adults, in pediatric population in other diseases, MOA of drug, etiology of the disease, maturation of drug target, safety data in adults and pediatric patients on similar compounds, preclinical data, in vitro data etc.), to minimize data requirement from clinical trial in neonate and older pediatric population.

Voting

7 votes

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 »

Voting

2 votes