Module 9 2021

Clinical

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) • Hospital procedures for manufacturing • Concomitant, non standardized therapies (immunosuppression) Non conventional design & statistics • Orphan diseases • Bayesian statistics and adaptive designs • High unmet medical need Registration • Limited clinical data and small patient population (~20 – ~200) • Licensing with restriction (PASS, Establishment of registries) Pharmacovigilance • Long follow up and risk management • Safety and efficacy endpoints Logistics

• Delivery and Supply chain • Storage at the infusion site

Novartis Pharma AG- Business use only- not for further distribution

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