CRED ERP 25

• Life cycle management resource and cost savings

Regulatory •

Lack of opportunity to select Rapporteur/Co-Rapporteur • “All or Nothing” procedure seen as a risk; if CHMP refuses to approve an MAA, the drug is barred from sale in every EU country • Data package may be more acceptable in some regions than others, i.e., cannot avoid countries with specific issues or concerns Commercial • Prevalence of the disease/medical condition may differ across individual countries; therefore may not be needed in all EU markets; utilise a different procedure (MRP/DCP/National) • Single MA holder, tradename and pack – possible lack of flexibility in marketing? • Pricing and reimbursement – consider interaction between regulators, Health Technology Assessment (HTA) bodies and trade associations. • Review available guidance: ICH and EU guidance, EU legislation and national treatment guidelines • Review EPARs for authorised medicinal products with similar indication(s) or issues • Check comparator(s)/Standard of Care (SOC) used, efficacy endpoints, safety profile, specification limits, stability data, toxicity profile etc. • List all known/potential issues with data package (missing data, unfavourable results etc) and rank in terms of risk to approval (critical, major, minor) • Is the proposed indication appropriately reflective of the population studied and the outcomes? • If you have orphan drug designation have you adequately assessed similarity and significant benefit? Resolved with a post-approval commitment • Justified based on the overall risk-benefit profile and unmet medical need • Determine which issues should be discussed with the (Co-)Rapporteur and/or EMA based upon the criteria assigned based on data package. Determine whether it would be appropriate for the product to be approved under ‘exceptional circumstances’ or ‘conditional approval’ • Eligibility may be subject of CHMP Scientific Advice • Eligibility should be discussed with the EMA at the pre-submission meeting and justified in the MAA. Risk Mitigation Strategy Determine which issues can/should be: • Resolved pre-submission • Data Package Gap Analysis With a cross-functional team (clinical, preclinical, CMC), conduct a gap analysis on the data package to:

eCTD eCTD-format electronic-only submissions are mandatory for centralised submissions

• The eCTD is an electronic version of the Common Technical Document (CTD) • It contains additional technical components which enable the lifecycle of individual files and of the product itself, to be managed • eCTD has: