Form 1 Substudy NICHD BPCA Prioritization Survey

BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
OMB # 0925-0766
Expiration Date: 4/2023
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In preparation for the 2020 BPCA Annual Meeting December 14-15, 2020, we would
like your feedback on therapeutic gaps in pediatric drug development in order to
inform the 2021 prioritization process. Please complete the following survey to
share your input. We look forward to your participation at the meeting!
Name/Contact Details
Name
Institution
Email Address

Specialty Area/Therapeutic Area Focus

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BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
1. Advancing Clinical Trial Design
Validated endpoints in a non-static state
New drug development and new therapeutic uses
Please provide your feedback on this topic below:

What are the remaining questions in pediatric trial designs? Where is the
innovation happening?

What guidelines do you currently use in selecting the correct trial design?

How can we improve the success of clinical trial design in pediatric drug
development clinical trials?

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What components do you think are integral to building an effective framework as
it relates to advancing clinical trial design?

BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
2. PK Modeling that Informs Dosing
More research/training on extrapolation from adult studies
Need a standard for PK modeling for drug development, safety, trials design
Need for comprehensive comparative data across various therapeutic areas
Please provide your feedback on this topic below:

Is extrapolation from adult data possible in your disease? If not, what research
(i.e., outcome measures, endpoints) remains to be done for extrapolation to
occur?

If extrapolation is not possible in your area of research, what tools, designs, or
research questions remain in your specialty?

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What components do you think are integral to building an effective framework as
it relates to PK modeling?

BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
3. Pharmacoepidemiology studies
Improved process for pharmacovigilance (therapeutic drug monitoring)
Developing toxicity profiles across various therapeutic areas
Please provide your feedback on this topic below:

Are there existing pharmacovigilance databases that are pediatric focused? If so,
how do we gather the data to accomplish the above?

How can pharmacoepidemiology be used to advance research in "omics?" Who are
the key players in this type of research?

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What components do you think are integral to building an effective framework as
it relates to pharmacoepidemiology studies?

BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
4. Pediatric Friendly Formulations
Please provide your feedback on this topic below:

How do we attract industries to this market?

To your knowledge, where are the existing needs in the development of pediatric
friendly formulations?

What components do you think are integral to building an effective framework as
it relates to pediatric friendly formulations?

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BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
5. Systems Pharmacology
Need for more research on the ontogeny of disease and treatment response including:
transporters, biomarkers, natural history data
Need for more research on the impact of ontogeny on “omics”
Please provide your feedback on this topic below:

To your knowledge, what research currently exists in the area of ‘quantitative’
systems pharmacology in pediatrics? Who is conducting this type of research?

Are there existing knowledge gaps in the above areas in your specialty?

What advantages or disadvantages have you encountered in targeted therapies in
your research?

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What components do you think are integral to building an effective framework as
it relates to systems pharmacology?

BPCA Prioritization – Identification of Knowledge Gaps in
Pediatric Drug Development
6. Biomarkers Research
Please provide your feedback on this topic below:

In your opinion, what do you define as the most ‘relevant’ biomarkers research
needed in pediatric drug development?

What components do you think are integral to building an effective framework as
it relates to biomarker selection?

Thank you for your feedback! Your input is vital to the successful implementation of the NIH
BPCA program.
If you have additional questions or comments, please send email to
bpca@infinityconferences.com.

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