WP11 - PUMA development


Workpackage lead and partners:

WP leader:
Mr Paul Breen, BrePco

WP partners:

Workpackage objectives: 

BrePco will act as the sponsor of the study, as such the company's objectives with respect to this clinical trial are:

  • Ensure pharmacovigilance
  • Perform all regulatory activities related to the project including filing for a PUMA and US orphan drug approval with FDA presuming a successful outcome to the trial
  • Provide clinical supply material
  • Provide clinical trial insurance
  • Commercialize formulations suitable for use in a neonatal population


Workpackage description:

Regulatory Tasks:

  • Conduct pre-submission meeting with proposed RMS/EMEA
  • Conduct pre-submission meeting with US FDA & file NDA
  • Prepare and submit regulatory portion of PIP, provide ongoing point of contact to support submission
  • Prepare modules (1 through 5) for EMA (and equivalent documents for US FDA)
  • Copy and compile dossier and RTQ's (and equivalent documents for US FDA)
  • Prepare fully compliant eCTD and/or NeeS submissions (and equivalent documents for US FDA)
  • Support application through chosen procedure and respond to questions during assessment
  • Finalize DCP/MRP/CP (Include dealing with national issues and translation) and US 505(b)(ii) submission


Clinical Supplies

  • Determine formulation specifications and supply requirements
  • Contract with manufacturing site
  • Ensure IMP-QP release
  • Manage delivery of supplies to various sites



  • Purchase and maintain appropriate clinical trial insurance policy on behalf of the consortium



  • Arrange systems and SOP to ensure quality standards for recording and managing adverse events



  • Conduct focus groups and appropriate market research to understand label requirements, distribution channel issues
  • Identify commercial partners for each country, develop and implement appropriate license and commercial agreements with each
  • Ensure marketing partners fulfil marketing diligence requirements.
  • Insurance certificate         
  • Supply contract (or MOI) with clinical trials manufacturer
  • PIP documentation
  • Clinical trial supplies
  • Marketing strategy
  • PUMA filing