Spring Intro 2023

30/03/2023

Understand the bigger picture

A good understanding of the “bigger picture” is essential to formulate effective regulatory strategies: • The Medicine, The Patient, The Environment and What Success looks like

The Medicine • Molecule – is this a new active substance? • Mode of action • Pharmaceutical form and Dose • Current and planned data efficacy/safety The Patient • Disease • Population (including importance in paediatrics) • Treatment landscape • Intended line of therapy/ positioning

The Organisation for Professionals in Regulatory Affairs

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Understand the bigger picture

The Environment • Any regulatory precedent • Regulatory and treatment guidelines • Reimbursement • Risks & mitigations

What success looks like – critical factors • Speed: 1st to approval

• Maximising Intellectual Property (IP) protection • Improved therapy - better than current treatments • Differentiated label– efficacy/safety, usability/convenience • Link to the Target Product Profile

The Organisation for Professionals in Regulatory Affairs

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