Spring Intro 2023

16/02/2023

Applying combined clinical and regulatory knowledge

• Are CDP endpoints compatible with intended claims? • Is the proposed population compatible? • Are there enough patients in the trial / programme?

SmPC claims

• Are you using the right statistical approach? • Is the design robust (will regulators criticise it)?

Statistics

• What is mandatory for regulators? • What is preferred by regulators? • What is scientifically necessary? • What is ethically possible?

Control groups

Consider ICH, EMA, national CA and FDA guidance

Published guidance

Are the overall plans in line with expectations?

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Specific regulatory input to clinical trials

• Does the duration of toxicology studies support the duration of the clinical trial? • Is the toxicity profile compatible with the trial (benefit-risk profile)?

Toxicology data

• Does the authorised IMP shelf life cover the trial…? • …or will you need to manufacture more drug / extend the authorised shelf life during the study?

IMP shelf-life

• Are the changes substantial or non-substantial? • How can delays to the trial be prevented?

Protocol amendments

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Document your advice and the final decision

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