Clinical trials of combined Advanced Therapy Medicinal Products (ATMPs)

ExpectedOutcome:

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 5 “Unlocking the full potential of new tools, technologies and digital solutions for a healthy society”. To that end, proposals under this topic should aim for delivering results that are directed towards and contributing to several of the following Expected Outcomes:

• Healthcare providers increase their knowledge on the potential of combined ATMPs and get access to innovative treatment options with demonstrated health benefits for unmet medical needs;
• Developers and manufacturers of combined ATMPs obtain scientific evidence on the proposed therapeutic approach;
• Patients benefit from new advanced therapies delivered through the combined ATMPs;
• EU companies get a better market position in the field of combined ATMPs.

Scope:

The subjects of this topic are combined ATMPs (Advanced Therapy Medicinal Products) according to the definition of the ATMP-regulation (EU 1394/2007, Article 2d). Such combined ATMPs are composed of an ATMP and one or more medical devices or one or more active implantable medical devices, and their cellular or tissue part must either contain viable cells or tissues, or non-viable cells or tissues liable for exerting the primary action on the human body.

The combined ATMPs should be more effective than current state-of-the-art solutions on the European market owing to improved features like personalisation, accuracy, reliability and usability and contribute to long-term sustainability (faster and affordable) of European health systems.

Research should focus on advanced stages of clinical development with regulatory work on the Medical Device part completed and safety studies of the combination product in an advanced stage.

Proposals should address all of the following activities:

• Phase 2 clinical trials and above of combined ATMPs focussing on:
  • technologies ready to undergo interventional clinical trials in patients/end users assessing the usability and clinical performance, and/or
  • technologies that have demonstrable safety/performance profiles and should undergo clinical validation in view of their inclusion into guidelines for specific clinical pathways.
• Delivery of safe and clinically validated combined ATMPs that are compliant with current European regulatory requirements. The related regulatory work should be considered as an essential component and the proposed work should involve consultation/interaction with competent regulatory agencies such as the European Medicines Agency (EMA) or national regulatory agency. Applicants are encouraged to seek regulatory and/or Health Technology Assessment (HTA) advice as appropriate.

The topic invites proposals that include innovative treatments for any medical condition excluding rare diseases that are ready to be assessed for clinical efficacy (performance and clinical benefit) in a specific indication on a big number of patient cohorts; already existing market solutions are not in the scope of this topic.

Sex and gender aspects, age, socio-economic, lifestyle and behavioural factors and any other non-health related individual attributes should be taken into consideration. SME participation is strongly encouraged.

Applicants envisaging to include clinical studies should provide details of their clinical studies in the dedicated annex using the template provided in the submission system. See definition of clinical studies in the introduction to this work programme part.