Showing 4 ideas for label trending idea

Advancing Pediatric Drug Development

Translation from adults data trending idea

Community Member kudos icon 3 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 »

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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 »

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22 votes

Advancing Pediatric Drug Development

Establishment of Target/MOA safety and efficacy datasets trending idea

Community Member kudos icon + Community member
For Oncology programs which intend to submit an original marketing application and are required by FDARA (i.e., those intended for treatment of an adult cancer and with molecular targets that the FDA has determined to be substantially relevant to the growth or progression of a pediatric cancer) to submit initial Pediatric Study Plans, it would be especially useful to have access to unpublished clinical safety and efficacy... more »

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0 votes