Advancing Pediatric Drug Development

Assess correlation between Objective Response, Overall Survival and Progression free survival in pediatric cancer.

The proposal is to use pooled databases and/or meta-analyses techniques to establish correlations between key phase 2 endpoints such as Objective response and late phase¬† endpoints such as Progression free Survival and Overall Survival as well as correlations between Progression Free Survival and Overall Survival. Those correlations would be established at the study level for the most frequent pediatric cancers. A better... more »

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Advancing Pediatric Drug Development

Can the FDA databases be used for qualification of biomarkers or surrogate endpoints to be used instead of clinical endpoints

In some indications the established primary clinical endpoint may be a difficult to obtain (for example because it requires a biopsy). Could one use the FDA data to qualify biomarkers or surrogate endpoints for use in pediatric trials instead? If such data don't exist, could one plan prospectively to generate such data over time?

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Advancing Pediatric Drug Development

Placebo Controlled Trials, Biomarkers and Expression of Known Targets

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Potential Research Question #1: For serious diseases, placebo controlled trials represent a significant challenge. Parents and investigators in many therapeutic areas are hesitant to enroll their children in them and this results in enrollment delays and prevents sponsors from generating the data needed to fully inform prescribers on the risks/benefits of a particular treatment (and contributes to increases in off-label... more »

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Advancing Pediatric Drug Development

Reducing Pediatric Clinical Trial Burden or Elimination

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Are there adult PK characteristics (i.e., certain elimination pathways, variability as determined by %CV on parameters or other popPK metrics) that reliably allow for accurate pediatric predictions that could routinely lead to either a reduction in pediatric trial complexity (e.g., shorter trials, fewer participants, fewer PK time points) or ability to leverage PK data instead of studying younger age groups altogether?... more »

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Advancing Pediatric Drug Development

Establishment of Synthetic Control Arms

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Both within and across companies, is there an opportunity to more effectively leverage existing data from pediatric patients. Thinking about the heme malignancy world, especially multiple myeloma, there's a heavy reliance on ALL for pediatric studies. Occasionally there are master protocols run by a group like the Children's Oncology Group (COG), but separate studies for each drug are very common. In the case of comparative... more »

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Establishment of Target/MOA safety and efficacy datasets

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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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Efficiencies in pediatric clinical trials of multiple indications with a single compound

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While the use of a single compound in multiple oncologic conditions based on mechanism of action has been an accepted approach in pediatric drug development for many years, we are now beginning to see single agents with a specific MOA being explored for multiple indications crossing therapeutic areas, e.g., immunology, nephrology, neurology, endocrinology, hematology, hepatology, dermatology including conditions such... more »

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Advancing Pediatric Drug Development

Developing the second or third drug

The first drug for a particular condition can be studied in a placebo-controlled trial, but if that drug is approved, how can later and possibly better agents be developed? Unless the condition is trivial (in which case the need for medication may be questionable anyway), placebo-controlled trials are no longer ethically-justifiable, so active comparator trials are required, but that is likely to increase the sample size... more »

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Advancing Pediatric Drug Development

The level of information provided

Are information sheets becoming so large that they are defeating the purpose for which they were created? Information sheets are continuing to grow, often because sponsors' legal teams insist upon providing progressively more information to reduce the prospect of litigation, in addition to data privacy requirements. However, a clear difference exists between the provision of data and its assimilation.

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