Module232025

5/7/2025

Confirmatory phase : ‘pivotal’ or ‘registration’ efficacy and safety study • Any study design (controlled/uncontrolled, withdrawal, enrichment designs , etc) can be acceptable if justified.  What is ethical, feasible, methodologically sound  Drug’s MOA is relevant for placement in therapeutic algorithms (add-on, second line).  Based on aim of therapy: curative, symptom control, long-term disease/symptom control • One study will be sufficient if results statistically compelling • Interim analysis : potentially reduce time to registration but usually discouraged, concerns on maturity of data  If duration shorter than in adult studies already conducted, it should be justified

Phase 3

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Study population

• Defined by inclusion/exclusion criteria, relevant to clinical practice • Disease natural history + drug’s target maturation/expression • Stage of disease including previous or concomitant therapy • Use of biomarkers for patient selection : availability of global screening (genetic diseases) • Age  Sufficient numbers in different age groups – not only peak age groups based on the incidence of target disease  Staggered enrolment based on age may be needed (but not always)  Adolescents should be included in adult studies unless otherwise justified

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