Abstract

Many companies that are developing investigational advanced therapy medicinal products (ATMPs) target diseases that require specific assays for diagnosis, patient selection or safety and efficacy monitoring. The implementation of the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 (IVDR),^[1] that was applicable on 26 May 2022, has significantly impacted the initiation of clinical trials (CTs) with ATMPs in Europe. This Regulation requires ATMP developers to consider in vitro diagnostic medical device (IVD) requirements and the potential need for companion diagnostics (CDx) from the early stages of drug development.

In this article, these new challenges are illustrated through three case studies of CTs involving adeno-associated virus (AAV) vector-based gene therapy for rare diseases, which require the use of IVDs, such as genotyping and anti-AAV neutralising antibody assays. Based on the experience gained from the case studies, a list of key IVD-related questions can be addressed early on in ATMP development to identify future CT requirements and potential impact on timelines and budget.