CRED Understanding Clinical Development 2024

14/10/2024

Development Phases and Objectives

Therapeutic Exploratory (most typical Phase II study)

Establish Therapeutic Use

● Explore use for the targeted indication

● Refine understanding of benefit/risk relationship in general, of special populations and/or environments

● Estimate dosage for subsequent studies

● Identify less common adverse reactions

● Provide basis for confirmatory study design, endpoints, methodologies

● Refine dosing recommendation

● Post Authorisation Efficacy Studies (PAES) and Post Authorisation Safety Studies (PASS) studies are formal post approval requirements to investigate these aspects

Therapeutic Confirmatory (most typical Phase III study)

● Demonstrate/confirm efficacy

● Establish safety profile and dose-response relationship

● Provide adequate basis for assessing benefit/risk relationship to support licensing

The Organisation for Professionals in Regulatory Affairs

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Objectives of Confirmatory Trials

• Inform use (Labeling / SmPC)

Regulatory approval

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Reimbursement

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• Communication to medical community/ patients/ society

The Organisation for Professionals in Regulatory Affairs

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