Module 1 2021

25/06/2021

Case Study 1 – Acceptance of novel endpoints for regulatory filling Question Is there precedence for the regulatory acceptance of a novel composite clinical endpoint in registrational trials for an immuno- inflammatory disease Notes Information requested as part of a new development program and following intelligence of competitor using this new end-point.

Outcome/Application Only competitor is using the specific end-point. Regulatory uptake of composite primary end-points not common. Information used to define regulatory strategy

Tools Literature review (PubMed), competitor web-site, FDA guidance, EPARs, TrialTrove

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Case Study 2 – EU/US Orphan designation in sub- set population in a non-orphan disease Question Would a product under

Notes No current treatments are available to serve this patient population. Request for examples Outcome Possible, but under very defined circumstances in both EU and US. Examples were identified and shared with the product team

development qualify for orphan designation if used to treat rare sub-set of patients in a non- orphan disease? Tools Regulations/Directives, EC notice, EU guidance, FDA CFR, FDA webinars

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