1) Mapping the hurdles for the clinical applications of Advanced Therapy Medicinal Products (ATMPs)

Deadline model single-stage

Planned opening date 12 January 2023

Deadline date 13 April 2023 17:00:00 Brussels time

ExpectedOutcome:

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 6 “Maintaining an innovative, sustainable and globally competitive health industry”. To that end, proposals under this topic should aim to deliver results that are directed towards and contributing to all of the following expected outcomes:

• Challenging aspects of regulation, policy, safety, efficacy, manufacturing, organisation, infrastructure, decision-making, and commercialisation are identified for speeding up the equitable clinical applications of ATMPs.
• European regulatory frameworks are adapted to novel scientific progress, especially those related to platform approaches, genome editing, interface with medical devices, artificial intelligence.
• Competent authorities in the Member States can propose adapted pricing and reimbursement schemes that allow European citizens to benefit from novel ATMPs.
• Academic and SME developers and manufacturers of ATMPs have an increased knowledge of the regulatory aspects.
• The decentralised manufacturing of ATMPs is consistent across health care centres.

Scope:

New pioneering treatments called Advanced Therapy Medicinal Products (ATMPs), including cell and gene therapies, have the potential to bring new cures to patients affected by diseases with limited or no available treatments. However, several hurdles impede or slow down the access of ATMPs to patients in the EU and Associated Countries. These include e.g. regulatory challenges, underlying scientific uncertainties, differences in assessing the values of ATMPs by the various Health Technology Agencies (HTA), difficulties to perform randomised-controlled clinical trials or to obtain long-term safety and effectiveness data, the lack of harmonised approaches to the reimbursement of the high upfront costs by health systems, manufacturing processes, etc.

The proposals should address all of the following activities:

• Map the regulatory, safety and efficacy assessment, manufacturing, organisational and infrastructural needs to improve the translation of ATMPs from preclinical development to clinical use.
• Address the gaps and uncertainties in regulatory and policy aspects pertinent to complex innovative ATMPs.
• Address predictivity of preclinical data for safety and efficacy testing of ATMPs. Improved novel models could be proposed.
• Tackle decision-making processes relating to ATMPs, such as for instance the assessment of their values, the demonstration of the long-term safety and effectiveness, or new pricing and reimbursement frameworks.
• Propose opportunities for an improved knowledge of the regulatory processes among academic ATMP developers.
• Involve regulatory authorities, Health Technology Agencies (HTA), clinicians, ethics committees, and patients, with the aim to ensure higher clinical use of ATMPs. The findings of the project will be available to competent authorities, ATMP developers and manufacturers as well as to national/regional funding agencies.

2) Clinical trials of combined Advanced Therapy Medicinal Products (ATMPs)

Deadline model single-stage

Planned opening date 12 January 2023

Deadline date 13 April 2023 17:00:00 Brussels time

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.