Imagine you are a patient participating in a clinical trial and half-way through it is modified or postponed due to an ongoing public health emergency (PHE) in your country. What would happen in the EU? To address this, in March 2025, the Accelerating Clinical Trials in the EU (ACT EU) Initiative published a draft guidance document outlining how clinical trials should be conducted during PHE and opened the document for comments. EFA, with the support from its members, replied to the consultation and shared specific recommendations from the patient perspective.
EFA welcomes EU-level action on crisis response, which is the first of its kind to reflect the current EU legislative framework and international guidelines. However, even though the EU responded well in collaboration during the COVID-19 pandemic, more measures are needed so we can be resilient and collaborative against PHE threats, and that patients, alongside healthcare professionals through their organisations are integral part of the process.
Read more about our response to the consultation below.
What does the guideline say?
The ACT EU initiative was launched in January 2022 by the European Commission, the EMA and Heads of Medicines Agencies (HMA). ACT EU is a multi-annual programme aiming to create a favourable environment for research and development in life sciences, through harmonisation, innovation and collaboration with stakeholders. This initiative shows the effort that the EU is taking post-COVID to prepare for PHE and crises. The guideline will influence the upcoming EU Biotech Act and Clinical Trial Regulations, and these efforts are also in parallel to the ongoing work of the EU’s Health Emergency and Response Authority (HERA) in crisis management.
The recommendations in the draft guidance are a way to streamline new clinical trials and changes to ongoing trials to generate clinical evidence. The guideline lays out the changes to ongoing clinical trials and adaptations and the responsibilities of all those involved in the process, from the regulators to the sponsors (pharmaceutical companies), and ultimately, patients.
EFA’s priorities
- Consider the people involved in a clinical trial as participants, but also as patients: Patients are not prioritised in the draft guideline, yet they are the most vulnerable to changes and adaptations to a clinical trial. Patients, as a specific group of trial participants should be mentioned.
- Focus on both participant and patient safety and health: Participant safety is mentioned throughout the text, however there is also a need to protect the health of those participating in the trial. EFA urges EMA to include measures which protect the health.
- Improve the definitions of ‘urgent medical need’ and when the national competent authorities can make changes ‘where necessary’: EMA needs to be clearer on the criteria for these definitions as ultimately any changes impact patients the most.
- Strengthen EMA’s regulatory support to sponsors: Sponsors are given the chance to make adaptations to the clinical trials without prior approval and this may not reflect the need from the public, unless it is clearer how, and making sure it is patient centered. This should be monitored closely by EMA.
EFA agrees with EMA that coordination at national and EU level is important to avoid unnecessary duplication, ensure optimal use of resources, and facilitate the generation of meaningful trial results and urges the EU to facilitate this coordination and collaboration, while consulting with patients and patient organisations to hear the real lived experiences of these adaptions. Patient health and safety should be protected first of all when adapting clinical trials, even in a PHE. As a community, EFA has experience from our members what happened unilaterally during Covid-19 for patients with allergies, asthma and COPD in trials and clinical practice, for instance on delaying critical diagnosis methods such as spirometry as others considered it as not safe.
EFA notes that the guidelines will be kept up to date with future EU legislation and guidance and will be revised once the Clinical Trial Regulation amendments and European Biotech Act are adopted. EFA looks forward to seeing these changes come into fruition and recommends more EU efforts to address public health crises, including involving patient organisations who have the link to their communities.
Next steps
Thank you to EMA for the opportunity to comment on this draft guidance!
Next, the ACT EU team, the Emergency Task Force (ETF) and the PHE Ethics Advisory Group will review the consultation responses and eventually the revised final publication of the guidance will be published.