ISoP is developing a pre-conference session focusing on the unique challenges and opportunities facing model-based approaches in guiding pediatric development programs for new medicines. We would like your feedback to help us understand what the key issues are facing the pharmacometrics community involved in pediatric drug development.
We have prepared a short survey that will help us understand your key issues.
1. How many years of post-graduate experience in pharmaceutical sciences do you have?
- None (currently a student)
- < 5 years
- 5 to 10 years
- 10 to 20 years
- 20 years or more
0 voters
2. In which sector did you spend most of your post-graduate experience in pharmaceutical sciences?
- Academia
- Industry
- Regulatory
- CRO
- Other
0 voters
3. How many pediatric development or pediatric clinical pharmacology programs have you been involved in?
- 0
- 1
- 2
- 3
- 4
- 5 or more
0 voters
4. How many pediatric programs have you prepared “Pediatric Study Plans” or “Pediatric Investigation Plans” for?
- 0
- 1
- 2
- 3
- 4
- 5 or more
0 voters
5. Among the pediatric programs you have been involved with, how often were pharmacometrics approaches employed?
- Always
- Often
- Sometimes
- Rarely
- Never
0 voters
6. What types of pharmacometric analyses were used in your pediatric programs? (choose all that apply)
- Population pharmacokinetic analysis
- Quantitative Systems Pharmacology
- Physiologically based pharmacokinetic modeling
- Population pharmacokinetic-pharmacodynamic analysis using a biomarker
- Exposure-response analysis using clinical endpoint
- Disease-progression modeling
- Health Economic/Outcome Research
- Other
0 voters
7. How often do the pediatric studies you are involved with fail to enroll completely?
- Always
- Often
- Sometimes
- Rarely
- Never
0 voters
8. How often do you experience resistance to using pharmacometric approaches in your pediatric programs?
- Always
- Often
- Sometimes
- Rarely
- Never
0 voters
Below is the pediatric study decision tree from the Dunne paper published in 2011 [Pediatrics 2011;128:e1242–e1249], which was adapted from “Guidance for Industry: Exposure-Response Relationships: Study Design, Data Analysis, and Regulatory Applications. Washington, DC: Food and Drug Administration; 2003”
9. Since the publication of Pediatric Study Decision Tree in 2003, roughly what percentage of your pediatric programs used each option described
Option A. No Extrapolation
- 0%
- 20%
- 40%
- 60%
- 80%
- 100%
0 voters
Option B. Partial Extrapolation
- 0%
- 20%
- 40%
- 60%
- 80%
- 100%
0 voters
Option C. Full Extrapolation
- 0%
- 20%
- 40%
- 60%
- 80%
- 100%
0 voters
10. For the pediatric programs that were based on “Option A: no extrapolation”, what type of safety and efficacy trials were conducted in children? (multiple Choices allowed)
- Non-inferior efficacy study
- Superior efficacy study
- Non-inferior safety study
- Superior safety study
- other
0 voters
11. Is the concept of “extrapolation” in the Pediatric Study Decision Tree according to the FDA or EMA guidelines, clearly understood by the pediatric team members within your organization?
- Yes
- No
0 voters
12. If you answered “no” to the question above, please provide further details on what area(s) would benefit from further clarity (click reply and type your input below).
13. What area(s) should be updated in Pediatric Study Decision Tree published in 2003 based on emerging science and knowledge? (click reply and type your input below)
14. Are there any key topics or challenges in pediatric drug development or pediatric clinical pharmacology research that you would like to include at this pre-conference? (click reply and type your input below)
15. What is the most frustrating issue you’ve faced in developing or studying drugs in children? (click reply and type your input below)
Thank you for your time and help!
Wonkyung Byon, PhD
Brenda Cirincione, PhD