Module 1 2021

30/06/2021

Patients, care givers and specialists

Few patients and often few experts and treatment centers Dedicated patients and care givers • Patients organizations Understand needs Pressure for early access

The Organisation for Professionals in Regulatory Affairs

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Health Authorities

Develop global regulatory strategy • Incl early interaction with regulators • Consider different pathways e.g. breakthrough designation, PRIME, conditional approval etc • Anticipate post-approval commitments Develop global clinical strategy • Randomized placebo-controlled clinical trials not always possible

• Agree on less conventional study designs Develop global clinical strategy • Define validated endpoints • Protocol assistance • Be aware of guidelines

• E.g. Clinical Trials in small populations (CHMP/EWP/83561/05)

The Organisation for Professionals in Regulatory Affairs

30

15