CRED Understanding Clinical Development 2024

08/10/2024

Objectives of Traditional Phase 2

Identify Key Objectives / Refine Target Product Profile (TPP)

from Hypothesis generating

Generating data to

support Ph 3

to Determine potential use

to Designing confirmatory trials (Phase III)

Continually

developing the

labelling key claims

In certain circumstances (life-threatening / seriously debilitating conditions) – Phase 2 trials may be used for an expedited filing strategy

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Summary - Confidence Building

Phase 2 studies are typically exploratory in nature (compared to the confirmatory Phase 3 studies)

Initial studies in a small number of patients (~ several hundred) (intended patient population)

Phase 2 studies are more flexible in design, in contrast with the pivotal, confirmatory registration studies

Not highly powered (80%)

●

● Testing of trial design and evaluation of statistical assumptions

● May not always be a sharp distinction between Phase II and Phase III (adaptive trial design) – Intermediate phase 2b

De-risking prior to Phase III

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