Showing 10 ideas for label trending idea

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 »

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27 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

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

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

Rare Disease Aggregate Control Arm Data to Alleviate Pediatric Patient Burden trending idea

Placebo- and active-controlled trials are considered the "gold standard" of well-controlled trials. However, for rare pediatric diseases, this imposes a large burden on families who are desperate for new treatments that could impact the unmet needs of the disease and do not want to enroll their children in a trial in which they may not gain access to the treatment with the potential to provide greater benefit (see for... more »

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9 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 »

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2 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 »

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1 vote

Advancing Pediatric Drug Development

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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0 votes