Module 9 2024

03/09/2024

Clinical Development – Key Issues Design of clinical trial with low

Biologics

ATMPs

patient number (statistics) Dose definition / selection / justification Duration of effect AND safety: Long-term follow-up Pivotal Phase III studies ● Prospective, randomized Clinical Trial expected, but difficult to achieve ● Control arms difficult to establish Confounding factors ● Administration procedures (e.g. surgery, bridging chemotherapy) ● Hospital procedures for manufacturing

Large, blind, randomized pivotal trial

Unblind (non-randomized) pivotal trial

Standard design

Adaptative design

FIM → Ph I → Ph II → Ph III → MA

FIM → (Ph I/II → (Ph III)) → MA

Conditional , Exception MA

Full MA

PASES , Registry establishment

● Concomitant, non standardized therapies (immunesuppression)

The Organisation for Professionals in Regulatory Affairs

41

Risk Based Approach

The Organisation for Professionals in Regulatory Affairs

42

21