European Plans Crystallize for Globalizing Clinical Trials

The approach of EU regulators to clinical trials is about to undergo another shift, with the emphasis this time on promoting international trials.

An ad-lib comment at the end of August from the European Commission's director general for health was one clear indication. Appearing before the European Parliament to defend the Commission's record in tackling COVID-19, Sandra Gallina threw in her view of the future of trials at the end of her remarks. "We need a new approach to clinical trials—more unified," she said. "They are too national or local or guided by fashion." And, she added, "We need more comparative trials."

Her views—expressed, importantly, as the Commission is finalizing its proposal for a review of its pharmaceutical rules—reflect concerns expressed during the pandemic over the proliferation of small trials, and the absence of coordination. The Commission's own "lessons learned" summary in mid-2021 already acknowledged that "A fragmented and divided approach to clinical trials across Europe since the beginning of the pandemic has hampered efforts to track and analyze variants." It went on: "The need for larger and more coordinated clinical trials has become all the more acute in the short to medium term." And looking ahead, it said: "The permanent establishment of a large-scale, flexible, well-resourced platform for multicenter trials would help establish a structured approach to EU clinical trials for future crises."

The EU's recent strengthening of the European Medicines Agency (EMA)—in force since early 2022—explicitly included provision for the agency to take a more active role in overseeing trials—at least in emergency situations such as pandemics. The pandemic "highlighted the need to coordinate assessments and conclusions on multinational clinical trials," says the legislation. It empowers a special taskforce within the agency to provide scientific advice on clinical trials design and product development as well as the rolling review of emerging evidence "to allow a more efficient assessment of medicinal products including vaccines during public health emergencies."

"Experience with clinical trials during the COVID-19 pandemic revealed a tremendous amount of duplication of investigations on the same interventions, a high number of small trials, under-representation of important population subgroups, based on gender, age, ethnicity, or medical comorbidities, and a lack of collaboration, leading to a risk that research will be wasted," says the preamble to the legislation.

The legislation focuses specifically on responding to cross-border health threats, and is designed to provide scientific support "to facilitate clinical trials for medicinal products intended to treat, prevent, or diagnose the disease causing the public health emergency". But the thinking among regulators goes way beyond just tackling emergencies. Even the Commission's 2021 "lessons learnt" summary observed that the strengthened support and simplification of clinical trials it was envisioning could be "an important tool in the fight against cancer or other diseases."

Now, further evidence of European regulators' intentions comes in a workplan they have just published—on the same day that Gallina was addressing the parliament. This sets out the program for the initiative they have jointly created on Accelerating Clinical Trials in the EU, known as ACT EU. Of the dozen "priorities" for 2022-2026, priority No. 2 includes, as a central aspect of the plan, "further development of a scheme to promote larger, multinational trials specifically in the academic setting." Officials speak of "a support process specifically aimed at academic sponsors in order to make the EU a more attractive region to conduct clinical research."

The ACT-EU workplan is no mere think-tank report. The initiative, launched at the start of 2022, fully involves the European Commission, EMA, and the parallel Heads of Medicines Agencies in Europe. Its chosen priorities reflect the recommendations of the European Medicines Regulatory Network strategy to 2025 and the European Commission’s Pharmaceutical Strategy for Europe, and aim "to transform clinical trials in Europe" and "to better integrate clinical research in the European health system." Work on the scheme is scheduled to start at the beginning of 2023, and by 2024 it should see the launch of a "one-stop shop for information on academic support."