Module 9 2024

03/09/2024

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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Clinical Trials

• Previously governed by the Clinical Trials Directive (CTD) 2001/20/EC, now by the Clinical Trial Regulation (CTR) EU 536/2014 • Provides guidance on the conduct of clinical trials, highlighting the importance of Good Clinical Practice (GCP) • Single system for review of clinical trials visible to all member states • Increased transparency of clinical trial materials and results • Clinical Trials Facilitation Group (CTFG) coordinate the implementation of the regulation 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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