CRED Understanding Clinical Development 2024

08/10/2024

Proof of Concept

⚫ Evaluate the drug’s effectiveness and safety within a specific disease or condition (target patient population)

⚫ Are there any regulatory guidance's for the disease?

⚫ Would a specific sub-set of patients benefit more?

Severe disease

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⚫ Biomarker high population (additional planning required for a companion diagnostic)

⚫ Clinical efficacy and safety evaluated over several doses (dose ranging – ideally 3 doses minimum)

⚫ Could the Ph 2 study be considered registrational (severely debilitating or life-threatening condition, high unmet medical need, no alternative therapies)?

⚫ Consider Agencies to approach for Scientific Advice

The Organisation for Professionals in Regulatory Affairs

PROOF OF CONCEPT STUDY (POC)

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Initial Safety

⚫ Start of the safety evaluation – multiple dosing and for longer study duration in patient population

Identify Adverse Event profile

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⚫ Understand precautions to be taken in Phase 3 - Reference Safety Information (RSI)/SUSAR’s

⚫ Obvious risk factors ⚫ QT-prolongation ⚫ Hepatic side effects ⚫ Renal changes

⚫ Negative effect on efficacy ⚫ Immunogenicity of proteins (Anti-Drug Antibodies – ADA) ⚫ Immunosuppression (inclusion/exclusion)

The Organisation for Professionals in Regulatory Affairs

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