CRED ERP 25

02/07/2025

Scientific advice

• Integral part of drug development

• Agencies participating in ICH generally offer opportunities to obtain advice

• By protecting/advancing public health, this includes support for innovation

• Parallel: e.g. EMA + FDA, EMA + HTA

Specific areas e.g.

•

• FDA: Rare disease endpoint advancement (RDEA) pilot program, CMC Development and readiness pilot (CDRP) Program

• PMDA: English Submission pilot

• EMA: Portfolio and technology meetings

The Organisation for Professionals in Regulatory Affairs

15

Scientific advice from a Sponsor’s perspective

New medicines are usually intended for a global market

• US is the largest single market…for now

Gateway to first clinical milestone & commercialisation

Key stakeholder and risk management

Diverse but usually inconsistent scientific advice

National vs regional (EMA)

•

• Regulators + Health Technology Assessment

Consider medical device/IVD co-development aspects and HTA input

The Organisation for Professionals in Regulatory Affairs

16

8