Module 9 2021

19/03/2021

General Principles

Clinical development of biopharmaceuticals still have the same requirements as other types of investigational medicinal products (IMPs) ● Development of a Target Product Profile (TPP) will drive the clinical development programme (CDP); – Design of the clinical studies that need to be conducted to obtain data to support the TPP ● Pre-MAA activities such as CTAs, DSURs, PIPs, ODD, Scientific advice, PRIME, accelerated assessment are all applicable, where relevant; ● Efficacy, safety and a favourable risk/benefit profile needs to be demonstrated; – Supported by preclinical studies, clinical trials (First-in-human/Phase 1, Phase 2 [Proof of Concept; POC] and Phase 3), post-approval studies, real-world evidence (RWE), risk management plan (RMP)

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The Organisation for Professionals in Regulatory Affairs

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Clinical Trials

Governed by the Clinical Trials Directive 2001/20/EC ● Provides guidance on the conduct of clinical trials, highlighting the importance of Good Clinical Practice (GCP) Assessment is conducted at a National Level until the Clinical Trial Regulation (EU 536/2014) becomes effective which will result in a harmonised approach ● Voluntary harmonisation procedure (VHP) was concluded as of January 2021 Clinical Trials Facilitation Group (CTFG) coordinate the implementation of the directive across the member states No distinction in CTA conduct and assessment with different IMP types (Biologic versus small molecule versus ATMP)

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The Organisation for Professionals in Regulatory Affairs

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