CRED Understanding Clinical Development 2024

08/10/2024

Study Design Considerations

– What are the important design considerations – Proof of Mechanism/Proof of Concept – Adaptive Trial Design – Comparator

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Proof of Concept - Study Design Considerations

⚫ Proof of Concept/Proof of Principle (POC/POP) – ‘Standard Design’

⚫ To determine whether a new molecule has any clinical benefit and thus worthy of further testing / investment of resources (progression to Phase III)

⚫ Usually placebo controlled, but there are examples of where this is not possible (e.g. oncology)

Straight forward efficacy evaluation

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⚫ Limited statistical power (typically around 80%), however a clear outcome is required

⚫ Studies may also take the form of:

⚫ Single arm trial

⚫ Non-comparative randomized study

⚫ Randomized Active Comparator study

⚫ Head to head superiority study

Both likely to be under-powered BUT, enables an early go/no-go decision

⚫ Comparative efficacy ⚫ An adaptive trial design

Seamless Phase (1/2 or) 2/3

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⚫ Two stage design based on based on biomarker presence

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