Clinical trials

The advance of highly innovative, increasingly personalized therapies in hematology requires:  

  • Novel clinical trial designs   
  • More flexible, adaptive regulatory frameworks   
  • Improved data generation to support decision-making, both during and after clinical studies  

EHA’s priorities are to work with regulators, sponsors, clinical research organizations, and other stakeholders on improving the:  

  • Quality, patient-centricity, and efficiency of clinical trials 
  • Facilitation of academic investigators and patient involvement

Clinical Trials Regulation (CTR)  

CTR sets out harmonized rules for the assessment and supervision of clinical trials across the European Union.   

CTR streamlines the clinical trials application process so that submitting an approval application per Member State is no longer needed. Clinical trials sponsors now just need to submit a single application via the new Clinical Trials Information System (CTIS).   

This allows for quicker and more efficient processes, facilitating the operation of larger and multinational trials. 

Modernized legislative framework 

The legislation also 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 

Accelerating Clinical Trials in the European Union (ACT EU) initiative   

ACT EU is a regulatory partnership formed by the European Commission, the European Medicines Agency (EMA), and the Heads of Medicines Agencies (HMA). It seeks to improve the design, efficiency, and effectiveness of clinical trials.  

The ACT EU initiative collaborates with sponsors, healthcare professionals, patients, and ethics committees through a structural multi-stakeholder platform (ACT EU MSP). This collaborative platform was established as a means for clinical trial stakeholders and regulators to build trust and exchange views.  

EHA and ACT EU  

Improving the EU’s regulatory framework for clinical trials is a key precondition for improving access to innovative therapies for hematology patients across Europe.   

We‘ve been part of the MSP since its launch when we presented the work of the Coalition for Reducing Bureaucracy in Clinical Trials.   

To ensure structured and comprehensive involvement in ACT EU, we’ve 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 (RBinCT)   

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’re meant to protect: clinical trial quality and patient safety.  

Coalition Recommendations 

The Coalition for RBinCT is led by EHA and works on a series of concrete and consensus-based recommendations. The objectives of these are to:  

  • Reduce the administrative burden on academic investigators   
  • Increase patient centricity   
  • Harmonize requirements across the EU   
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