Module 1 2021

30/06/2021

Challenges & Opportunities

The Organisation for Professionals in Regulatory Affairs

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Strategic challenges & opportunities

• Patient-relevant outcomes: how to generate patient-relevant data to support major contribution to patient  patients at the center of R&D, steering development needs; • Innovative study designs: facilitate a single development plan acceptable for different stakeholders across the globe; • Balance regulatory and access with evidence generation: dynamic post-approval setting. How to manage expectations of all stakeholders? • Regulatory decision-making with ‘real world evidence’ data: e.g. registry data/natural history of the disease – what is their place in regulation? How to optimize richness of data sources and technology. • Patient access: early access programs and accelerated post-marketing access • Significant benefit : increasing challenge to argue for significant benefit in orphan conditions: difficult to generate improved innovation in rare diseases • Cross-stakeholder collaboration: understanding of the unmet medical need in rare diseases and efforts to collaborate (e.g. rare disease cluster, expedited pathways like PRIME, HTA EMA SA).

The Organisation for Professionals in Regulatory Affairs

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