Hi Tooba,
It sounds like your clinical development plan and program is progressing. You indicate that you intend to conduct trials in pediatrics and you plan to seek a label for pediatrics post adult approval; so you must have a plan. However, it looks like you are planning to start these studies and not request a deferral to start after adult approval? You also indicate you have started conducting your adult clinical trial(s) but have not indicated what phase you are in. If you are in Phase 2 but have not yet had your EOP2 meeting with FDA then you have time to generate and submit your initial PSP and have FDA comment on your plan, which complies with PREA. In the EOP2 package you must submit your PSP and get input. The FDA has guidance on the template and required information.
As for your questions,
The reference to the "required assessments" are the requirements under PREA and the following text from page 5 of the How to Comply with PREA guidance might be useful;
3 For purposes of this guidance, the term "pediatric assessment" describes the required submissions under PREA that
contain data, primarily from required pediatric clinical studies, that are adequate to assess safety and effectiveness
and support dosing and administration for claimed indications in all relevant pediatric populations (section
505B(a)(1) and (2) of the Act). Generally, the terms "pediatric assessment" and "pediatric studies" are used
interchangeably.
The question of when to have a PSP and Does the PSP have to be reviewed and approved, page 6 of the "How to Comply" guidance:
A Pediatric Plan is a statement of intent that outlines the pediatric studies (e.g.,
pharmacokinetics/pharmacodynamics, safety, efficacy) that the applicant plans to conduct. The
plan should also address the development of an age-appropriate formulation. Furthermore, it
should address whether and, if so, under what grounds, the applicant plans to request a waiver or
deferral under PREA. Applicants are encouraged to submit their pediatric plans to the Agency as
early as possible in the drug development process and to discuss these plans with the Agency at
critical points in the development process for a particular drug or biologic.
I'd say you knew the answers, it is even more critical to get input from FDA on pediatric studies as these are of concern to FDA. If you are going globally, you need to start this process early and it is complicated and longer in the EU.
All the best,
Dar
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Darlene Rosario RAC
Principal Consultant RA, Quality and Compliance
Velocity Consulting
Ventura CA
United States
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Original Message:
Sent: 04-30-2015 12:39
From: Tooba Murshedkar
Subject: Pediatric study plan (PSP)
Dear All,
Need some guidance / advice / better understanding of this
We plan on submitting a BLA for a biologic product in approx. 2-3yrs for the adult indication. We subsequently plan to update that to include a pediatric indication as well. Adult clinical trials are currently in progress. However, we are planning to conduct pediatric trials prior to the first BLA submission (these may or may not be completed by submission time). Guidance talks about submitting a PSP (A sponsor must submit the initial PSP before the date on which the sponsor submits the required assessments and not later than 60 calendar days after the date of the end-of-phase 2 meeting.) [required assessments - can someone clarify what this means]
Questions:
- Do we need to have a PSP in place if we are already conducting pediatric trials before the adult BLA submission (I'm assuming yes, but not sure)
- If needed, does the PSP have to be reviewed/approved (per the guidance) and in place prior to the submission of ANY pediatric study protocol to the IND? (again, I'm assuming yes, but not sure)
Any input would be greatly appreciated, thanks!
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Tooba M
United States
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