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Report of the PMDA-ATC & U.S. FDA Pediatric Review Webinar 2020

PMDA-ATC & U.S.FDA Pediatric Review Webinar 2020

  From September 28 to October 1, PMDA convened an online seminar entitled “PMDA-ATC & U.S. FDA Pediatric Review Webinar 2020". This webinar was designed for officials from overseas regulatory authorities involved in the review of pediatric clinical trial applications and/or new/generic drug applications for pediatric populations. A total of 27 participants from Brazil, India, Indonesia, Malaysia, Myanmar, Philippines, Saudi Arabia, Singapore, Tanzania, Thailand and Uganda joined this event (see agenda for details).

Recorded lectures by staff members from PMDA and the U.S. FDA were provided in advance of the webinar as preliminary training materials, which then were followed during the live webinar by Question & Answer sessions. The lectures covered the overview of new drug applications in PMDA and the U.S. FDA, U.S. pediatric regulations, physiology and clinical pharmacology in pediatric populations, extrapolation of efficacy from other population data (e.g. adults, foreign children), and ethical considerations for clinical trials in children.

The webinar live session opened with remarks by Mr. UZU Shinobu, Director of PMDA-ATC followed by Dr. SAKIYAMA Michiyo, PMDA Webinar coordinator and Dr. ALEXANDER John of U.S. FDA.    On the second and third day, the attendees worked in groups exploring case studies provided by PMDA and the U.S. FDA on such topics as the review of a pediatric application, extrapolation, and ethical issues in pediatric clinical trial design. The participants actively engaged in all discussions throughout the webinar.

At the end of the seminar, Dr. FUJIWARA Yasuhiro, Chief Executive of PMDA handed the course completion certificate to each participant virtually by recorded video.

Photos and participant comments

< Recorded Lectures and Q&A sessions >

 Comments from the participants:

Material provided are understandable and enriched me with new approaches of dealing with pediatric formulations. Enlightens me on how USFDA operates on regulations and control of pediatric formulations.  Able to know the PMDA activities, programs, legislation to establish it and the systems and procedures.”
This session brought out clearly why pediatric-focused drug development and pediatric clinical trials are necessary in the development of safe and efficacious products for children.”
The explanation was great and can improve my knowledge about pediatric PK/PD modeling and simulation. I hope that I could use the knowledge that I gain to review of pediatric drug in my country”.
This session helped me understand when extrapolation of adult data to pediatric patients can be done.”
The explanation was great and can improve my knowledge about what expect should we look if we have pediatric foreign data.”
This was a very educative and interesting session. I was able to appreciate the unique ethical considerations of conducting pediatric clinical trials.”

< Case study by PMDA >

Comments from the participants:
“Gained experience on how PMDA conduct clinical trials particularly for pediatric populations.  Commented me on the use of ICH guidelines in many decisions.

The Case Study example was very useful in contextualizing the information you have provided to me so far. I understand and can explain the regulatory decision that PMDA reached.
This presented the opportunity to review data from a Clinical study report and use this in making a decision on whether to approve or not approve a drug for MA. It was interesting to learn more about reviewing foreign data in adults and children and how that would then be used to provide evidence for use in Children in Japan. Great session
The day 2 case study involved many learning aspects which were well explained.”
The group discussion was really a good opportunity to learn with other regulators and how the decision make process happens. I have only one point: the time was not enough to answer all the questions, so, maybe a writing round between the members of each group may contribute to time be enough in the live session.”

< Case Study by FDA >

Comments from the participants:
“I enjoyed the interaction on the ethical issues presented by Case 9. From this one case, aspects of minor increase over minimal risk were assessed for example the renal biopsy under sedation, permissible use of a placebo.  Assessment of preclinical and clinical data to determine that there is equipoise prior to the conduct of a clinical trial. Great learning experience. Thank you”
“Provided me on the knowledge how USFDA handles clinical trials based on the protocol and ICH guidelines.  Teach me the importance of ethical considerations during formulation development and conduct of clinical trials”
I think the case study brought out the ethical considerations and rationale in the designing of a paediatric research protocol”
The study case was excellent and the facilitator was great. We could learn more and understand about pediatric review.”
“This case study help to clarify more in understanding in Ethical analysis.”

<Group photo>

Overall comments from the participants

“The course was very instructive for me. It gave me an opportunity to learn about the specific concerns about pediatric population which should be considered during a NDA/ sNDA review.  Besides, I think that the group I was allocated was a case of success of working group. We could discuss almost all the questions and reach consensus in the short time we had on days 2 and 3. I will recommend this course to my colleagues and I am eager to participate in more courses offered in this format (even online).”
“I would like to thank PMDA for organizing this pediatric webinar and both the teams from PMDA and FDA for the resourceful information and guidance they gave us in spite of the unique challenges posed by the online nature of the training. I would like to say that my pediatric studies evaluation skills have been improved by the webinar and am looking forward to passing on this to my colleagues at NDA. My prayer is that if the situation allows in future, please consider us for the in person training. Many Thanks.” 
“The questions were answered pretty clearly, and it was nice to see all of the people involved in the organization of this webinar. Thank you all for the opportunity!”
“Generally great session. The questions answered about IDeg gave a great insight on the decision process at PMDA, which is insightful for other regulators.”

Information about future PMDA-ATC Seminars:

PMDA-ATC Seminars/Webinars are held periodically, with a different theme for each seminar/webinar.
The Seminar/Webinar announcements are posted on this website.

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