Clinical trials
The advance of highly innovative, increasingly personalized therapies in hematology requires novel clinical trial designs and more flexible, adaptive regulatory frameworks and improved data generation to support decision making both during and after clinical studies.
Improving the quality, patient centricity and efficiency of clinical trials and better facilitation of academic investigators and patient involvement are priority objectives for EHA in working with regulators, sponsors, clinical research organizations and other stakeholders.
Clinical Trials Regulation (CTR)
The Clinical Trials Regulation sets out harmonized rules for the assessment and supervision of clinical trials across the European Union. The legislation was published in April 2014 and will become fully applicable by January 31, 2025.
Streamlined applications
The Regulation streamlines the application process for clinical trials, so that submitting an approval application per Member State is no longer needed. Clinical trials sponsors will now submit a single application via the new Clinical Trials Information System (CTIS). This will allow for quicker and efficient processes, facilitating the operation of larger and multinational trials.
Modernized legislative framework
In addition, the legislation modernizes the legislative framework by:
- Fostering the sharing of information
- Incentivizing collective decision-making
- Increasing transparency with regard to clinical trial data
- Extending the silent agreement principle to the authorization process, which enhances legal certainty
- Ensuring high standards of safety for EU clinical trial participants
Improving the EU clinical trials environment: the ACT EU initiative
ACT EU (Accelerating Clinical Trials in the European Union) seeks to improve the design, efficiency, and effectiveness of clinical trials. The initiative was launched in January 2022, at the start of the transition period for application of the Clinical Trials Regulation (CTR).
ACT EU is a regulatory partnership formed by the European Commission, the European Medicines Agency (EMA), and the Heads of Medicines Agencies (HMA).
Stakeholder involvement in ACT EU
ACT EU established a multi-stakeholder platform (MSP) as a means for clinical trial stakeholders and regulators to build trust, exchange views and collaborate. The MSP will entail:
- The creation of an MSP Advisory Group
- The organization of relevant events
- The rolling out of tools designed to obtain feedback e.g. surveys and consultations
The ACT EU initiative collaborates with sponsors, healthcare professionals, patients and ethics committees through a structural multi-stakeholder platform (ACT EU MSP).
EHA’s role in ACT EU
For EHA, improving the EU’s regulatory framework for clinical trials is a key precondition for improving access to innovative therapies for hematology patients across Europe.
EHA has been part of the MSP since its launch in June 2023, when it presented the work of the Coalition for Reducing Bureaucracy in Clinical Trials. In addition, to ensure structured and comprehensive involvement in ACT EU, EHA successfully nominated Prof Tarec El-Galaly as a member of the MSP’s permanent Advisory Group (MSP AG), and Dr Lorenzo Brunetti as his alternate.
EHA-led Coalition for Reducing Bureaucracy in Clinical Trials
The cross-disciplinary Coalition for RBinCT was created in 2020 to ensure that regulatory guidelines, safety reporting requirements, and informed consent procedures do not harm what they are meant to protect: clinical trial quality and patient safety.
Coalition recommendations
The Coalition continues to work on a series of concrete and consensus-based recommendations. The key objectives of these recommendations are:
- Reducing the administrative burden on academic investigators
- Increasing patient centricity
- Harmonizing requirements across the EU
For information about the EHA-led Coalition, visit the Coalition for Reducing Bureaucracy in Clinical Trials website.
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