Pushing and Dragging the New European Clinical Trials Rules into Shape

With the early-2023 deadline fast approaching for compulsory use by trial sponsors of the European Union's clinical trial regulation (CTR), the authorities are still trying to iron out the wrinkles in the new system. No surprise, given the complexity and scope of CTR and the multiplicity and variety of users of the clinical trial information system (CTIS) at the heart of the system. According to the European Medicines Agency (EMA), which is at the center of the turmoil, key objectives of 2022 are "for all sponsors and member states to learn and adapt to the CTR and to detect issues in CTIS so that they can be fixed" prior to the looming deadline.

So far, the majority of clinical trials submitted in CTIS have been approved without blocking issues, EMA says. But some users have experienced "technical difficulties that have been challenging," needing collaboration between the affected sponsors, the concerned member states, and EMA. In particular, technical challenges have been encountered with the CTIS workflow for some very large multi-member state applications. The CTIS implementation plan for Q4 2022 "will ensure that the system is further strengthened and that fixes are introduced for blocking issues before the CTR is made mandatory for new clinical trial applications in early 2023," EMA reassures everyone. "Testing of the system continues to develop definitive fixes."

A forum has been created to allow direct exchanges of information and discussion of user experience of CTIS, bringing together users and experts from member states, EU sponsors in academia and industry, contract research organizations (CROs), and patients and healthcare professionals (HCPs). The forum held its first meeting this month and will meet on a quarterly basis to review CTIS functionalities, system use, and development, as well as user support and knowledge acquisition.

Meanwhile, efforts continue among European regulators to strengthen the European environment for clinical trials, and the joint initiative they have launched with this intention, Accelerating Clinical Trials in the EU (ACT EU), has set out specific objectives for the next four years. These include innovation in clinical trials, robust methodologies, and collaboration across stakeholders. For 2023, the agenda foresees the creation of a process of dialogue between patients, HCPs, and academia to promote "the evolution of the clinical trials environment." There will also be new assistance for academic sponsors in mounting large, multinational clinical trials, and support for innovative clinical trial methods, including decentralized clinical trials (DCTs). EMA already hosted a workshop on DCTs in October with a focus on harmonized and transparent use of DCT elements in clinical trials that can ensure the protection of trial participants and at the same time maintain reliability of generated and reported data. Publication of a recommendation on the use of decentralized elements in clinical trials is expected before the end of the year.

The ambitions of ACT EU are limitless. By its own account it "seeks to transform how clinical trials are initiated, designed, and run," and to "develop the EU as a focal point for clinical research; promote the development of high-quality, safe, and effective medicines; and better integrate clinical research in the European health system." No wonder a lot of effort is going into pushing and dragging CTR and CTIS into life: the envisioned transformation of clinical trials in Europe is not going to happen if the EU cannot even get its rules into place on time.