“For the clinical trial environment to evolve with advances in regulation, methodologies, technology and science, there is a need for multi-stakeholder discussions to drive and support change”, reflected the concept paper released by the European Commission, European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) and adopted this year. Multistakeholder engagement and nine other priority actions were set forth as part of the ACT-EU (Accelerating Clinical Trials in the EU) workplan 2022 – 2026 with the key objective of making European Union as the attractive location for innovative clinical research.

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Recent pandemic has clearly called for the need to change or rather innovate the way clinical trials are designed, regulated and executed to bring novel treatments on expedited basis to the patients. With this as the thought process, and in line with the recommendations of the European Medicines Regulatory Network (EMRN) strategy to 2025 and the European Commission’s Pharmaceutical Strategy for Europe, the ACT-EU workplan complements and supports the Clinical Trial Regulation (CTR) and the Clinical Trials Information System (CTIS) that became applicable on 31st January 2022.

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On 22nd and 23rd June 2023, two-day event was held in Amsterdam to discuss different stakeholder perspectives and the priority areas on how to develop the EU environment for better and faster clinical trials. The discussion focussed on the priorities for the year 2023: CTR, non-commercial clinical trials and the multi-stakeholder platform. The multistakeholder platform will serve as a common platform between all the key stakeholders in clinical trials including patients, healthcare professionals (HCPs), sponsors, regulators, ethics committee members, health technology assessment (HTA) bodies, payers and policy makers to discuss all the aspects of clinical trials, understand different perspectives  in plenary and ad hoc topic group meetings, enhance trust amongst different CT stakeholders and enable innovation through appropriate change in regulation(s), transparency of data and patient engagement. Additionally, other priority areas that were debated in the workshop included harmonisation of CT requirements across member states, innovative CT designs & methodologies and the need for consolidated scientific advice.

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In case of in vitro diagnostics, current lack of defined processes for the conduct of performance studies, their submission and assessment was highly argued in the meeting indicating an urgent need for the implementation of IVDR & CTR interface.

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To create an invigorated clinical trials environment, patient organisations strongly insisted on adequate representation of the patient groups in the platform including an ask for inclusiveness of special populations such as HIV patients. Better collaboration during clinical trials between patients, researchers and HCPs would also make the clinical trials less bureaucratic and more patient-centric and efficient. Especially, where the patient reported outcome measures are critical, the patients should be fully empowered with necessary tools required to capture the clinical trial data comprehensively. Also important is the publication of clinical trials data in CTIS with an access to the information of interests to the patients yet achieving right balance between required transparency and confidentiality requirements.

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Ethics in clinical research is of paramount importance; and the rights, safety, dignity and well-being of the subjects have to be protected and must prevail at all times while carrying out clinical studies. Though safeguarding this is primarily governed by the national medical research ethic committees(MRECs), there has to be good alignment between the MRECs of the different member states in the EU. Establishing a common EU ethics review platform with the representatives from each member state and harmonisation across Europe will certainly help overcoming current ethical challenges associated with novel technologies, and public health emergency situations; and enable the trials to run much faster and yet deliver quality data. Whether there is a need for randomised clinical trial always, whether the study can be done in smaller sample sizes and whether preclinical programme is good enough especially in cases of unmet medical needs were raised as some of the questions that need to be addressed while implementing ethical considerations for the newer study designs and expediting the drug development process for faster reach to the patients.

 

With regards to the non-commercial clinical trials, academic sponsors expect ACT-EU to facilitate appropriate funding schemes, multinational infrastructure, harmonisation of national legislations and site agreements across EU, and clear methodological guidance. EMA has planned methodology workshop in Nov 2023 to discuss the existing guidance, gaps and the need for additional guidance.

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In order to facilitate consistency between the scientific advice received from different member states,  EU Innovation Network (EU IN) has launched phase 2 of the Simultaneous National Scientific Advice (SNSA) pilot. This is intended to be used in situations where an applicant wishes to obtain national scientific advice from more than one national competent authority (NCA) at the same time. Benefits include a simplified procedure with a single application point, a common meeting request and a briefing book, predictable procedural timelines agreed upon at the start and clearly documented position of each member state. Europe intends to encourage the medicinal product manufacturers to seek this consolidated advice bridging the pure national scientific advice and centralised European scientific advice procedures from EMA. Although, this advice is not legally binding, having preliminary opinion from the competent authorities is certainly beneficial. In order to prepare for the public health emergencies, the Emergency Task Force (ETF) has been established that provides expedited scientific advice support to the potential applicants, thereby ensuring faster access of medicines to the patients.