Promises of New Support—in Cash, Not Just Comments—for European Clinical Trials

Anyone interested in exploring the use of complex clinical trials or who has skin in the game of running trials for combined advanced therapy medicinal products (ATMPs) may find something coming their way in new European opportunities for funding and support over the coming months. A leaked draft of the health plans under consideration for the next stage of the European Union's Horizon research program gives some insight into what is likely to result later in the year in a formal invitation for proposals.

The EU wants—according to the leaked text—to create a "diverse and comprehensive" landscape of multi-country adaptive platform trials that is "able to study multiple interventions in a disease or condition in a perpetual manner, thus allowing modification to the trial after its initiation without undermining its validity and integrity." That itself is no secret: it has been a part of European doctrine for close on a decade. But it has been given new impetus by attempts to tackle the coronavirus pandemic, because of the potential to assess medical countermeasures for infectious diseases. "The conduct of perpetual adaptive platform trials, with the in-built agility to pivot when an epidemic strikes, is key to be prepared for infectious disease epidemics or pandemics," says the document.

So the EU is now contemplating putting new money behind innovative designs of clinical studies suited for pandemic preparedness, in order to place additional expertise within the clinical research community when the need arises. It is also looking favorably on efforts to create trial sites across multiple countries with the capacity to deliver robust clinical evidence in a diverse European population. They don't even have to be created primarily for studies of pandemics. Adaptive clinical platform trials that are implemented routinely outside of an epidemic or pandemic context could qualify for support if they are designed to be ready also for rapid assessment of novel diagnostics, therapeutics, or vaccines in the face of an epidemic or pandemic.

Successful proposals are likely to be able to demonstrate the ability to sustain multi-country adaptive platform trials with adequate trial implementation capacity, laboratory analysis capacity, and a harmonized approach to the collection, storage, sharing, and analysis of data. They will take into account sex, gender, age, ethnicity, and socioeconomic factors, and hurdles related to ethical, administrative, regulatory, and logistical aspects "should be anticipated and addressed." A focus on the European regulatory environment is another ingredient specified in the plan, and an awareness of the EU view of the global context would not go amiss, as the current draft text unashamedly says: "Proposals should strengthen the leading role of the EU in clinical research preparedness for future epidemics and pandemics."

The topic is linked to the concept of the European clinical trial networks, which was developed in the context of the COVID-19 pandemic, and currently encompasses a network for COVID therapeutic trials and a network for COVID vaccine trials. In addition, the EU recently gave a new formal status to a European clinical research network that had been in development since before the pandemic. The leaked draft contains plans for backing and sustaining European clinical trial networks for pandemic preparedness and response "through well-established coordination mechanisms between EU adaptive platform trials." That would include fostering a trusted environment for the exchange of trial plans, challenges, and early findings.

Proposals should consider the coordination of adaptive platform trials addressing diverse trial target populations (such as primary care or hospitalized patients) as well as different possible medical countermeasures (including therapeutics and vaccines), within or across networks, which could include performance study plans for diagnostics, says the leaked draft. Connections with relevant initiatives such as the Coalition for Epidemic Preparedness Innovations or the World Health Organization should also be considered in any proposals.

Raised ATMP profile

More specific areas of clinical trials are also covered in the draft plans. They include support—in the millions of dollars per project—for developers of combined ATMPs, as part of an overall bid to unlock "the full potential of new tools, technologies, and digital solutions for a healthy society." The project funding would be available for development of ATMPs with one or more medical devices in which the cellular or tissue part contains either viable cells or tissues, or non-viable cells or tissues liable for exerting the primary action on the human body. Research should focus on Phase II clinical trials and above on products in advanced stages of clinical development that have already completed safety studies and where the technologies are ready to undergo interventional clinical trials in patients or end users for assessing the usability and clinical performance. Where the technologies have demonstrable safety and performance profiles, they could also benefit it they are ready to undergo clinical validation for inclusion into guidelines for specific clinical pathways.

The combined ATMPs addressed should be more effective than current state-of-the-art solutions on the European market owing to improved features like personalization, accuracy, reliability, and usability, and should contribute to long-term sustainability of European health systems by providing faster and more affordable treatment, the draft plan remark optimistically. What the EU is looking for with this support, it says, is to provide innovative treatment options with demonstrated health benefits for unmet medical needs, as well as greater scientific certainty on proposed therapeutic approaches. Again, the EU self-interest gets a look-in: "EU companies should also get a better market position in the field," says the draft.

Proposals are likely to be invited on innovative ATMP treatments for any medical condition that are ready to be assessed for clinical efficacy in terms of performance and benefit in a specific indication on a large number of patient cohorts. Products for rare diseases and products with already existing market solutions would not be eligible for this topic.

Europe's Horizon research support stretches widely across areas as diverse as environment and energy, but health has inevitably risen up the agenda in the wake of the coronavirus pandemic, and the move toward coordinating health research and innovation among European countries is gaining strength. The aims are not just to find new ways to prevent diseases and develop better diagnostics and more effective therapies, but to use personalized medicine approaches and promote the take up of innovative health technologies.