Spring Intro 2023

03/03/2023

Legal environment and its consequences

Exceptional circumstances Conditional approval

Orphan designation

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indication

• Well-defined, clear and widely accepted • Reflects evidence (study population) with an age restriction • Extrapolation to a wider population should be justified • Sub-groups of disease • Symptom driven vs treatment • Restriction related on the pharmacologic presentation included in section 4.2 To avoid • based on a subgroup after statistical analysis (data-mining) • Not doable in clinical practice

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