CRED ERP 25

Case Study 1

TOPRA CRED EU Procedures

Although Pulmolax has a relatively good side-effect profile, in Phase 2 trials a new side effect was detected; cardiac rhythm abnormalities.

Decisions on the content of the pivotal clinical data package have as yet to be made.

There is a market for Pulmolax across the EU although EsraBio does not have the resources to market this product in all Member States.

Phase 3 studies are currently being considered.

Strategy development (Day 1 only)

Outline the regulatory strategy and practical considerations for this product in Europe including (but not limited to):

• • •

Product profile planning Procedure to be used Any meetings to be held

• Potential data gaps which need to be considered/addressed before filing • Considerations for requirements for an NCE • Data required for each of the Modules 1 to 5 • Safety considerations • Legal basis for the application with consequence for potential data exclusivity • Orphan Drug Designation considerations (data/strategic) • Implications of the Paediatric regulation (EU Regulation 1901/2006, as amended) • HTA requirements

Practical considerations (Day 2 only)

Outline the practical considerations for the submission of Pulmolax in Europe, including (but not limited to):

• Document preparation for procedure chosen • Planning for any meetings • Other stakeholders • Timings for preparation of data for each of the Modules 1 to 5 – identify critical path components • Dossier preparation • eCTD preparation • Consider procedure timings taking account of clock-stops, arbitration if applicable, answering questions, anticipation of potential questions/objections

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