Optimising the manufacturing of Advanced Therapy Medicinal Products (ATMPs)

Expected Outcome

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

• Academic and industrial developers advance processes that support the timely and robust development of Advanced Therapy Medicinal Products (ATMPs);
• Manufacturers integrate improved technologies/processes (including Artificial Intelligence solutions), analytic tools, methods including non-clinical methods and assays for more flexible manufacturing of ATMPs;
• Healthcare providers, researchers and patients get faster access to ATMPs with demonstrated health benefits for unmet medical needs;
• Companies in the EU and Associated countries get a better market position in the field of ATMP manufacturing and improve their knowledge on how to advance process improvements;
• The EU and Associated countries lay the foundations for academic centres of excellence[1] in ATMPs.

Scope

New pioneering treatments called Advanced Therapy Medicinal Products (ATMPs)[2], including cell and gene therapies, are at the cutting edge of medicines discovery. Owing to their precise nature, ATMPs embody personalised medicine and reflect a shift in medicine towards potentially one-time curative therapies instead of chronic therapies that mainly cure the symptoms but not the underlying cause of diseases.

ATMPs have undergone important technological advancements that are improving their efficacy, precision, scalability, and safety. Additionally, the disease focus of ATMPs is likely to shift further from rare diseases to more common conditions with larger patient populations. However, the development and manufacturing of ATMPs still faces important challenges, such as long development times, expensive manufacturing processes and a fragmented and dispersed biomanufacturing landscape.

The topic focuses on addressing the challenges of ATMP manufacturing, the need for highly specialised equipment and facilities, including in-process quality control and validation tests, scaling up and batch-to-batch reproducibility, whilst maintaining the efficacy of an ATMP product during the manufacturing process and/or the transition from centralised to decentralised manufacturing.

This topic aims to optimise the ATMP production where the general manufacturing process for a given medicinal product has already been established but has not been sufficiently optimised for its scale-up. Collaboration is crucial to refine the manufacturing of ATMPs, emphasising advancements in processes - including leveraging the potential of digital tools and advanced sensors -, fostering standardisation and enhancing quality controls for more efficient production and deployment of these innovative therapies, ideally covering the entire manufacturing lifecycle.

The proposals should address all the following activities for only one chosen category of ATMP as defined by Regulation 1394/2007 per proposal:

• Design an improved manufacturing process for ATMPs by: • Exploring the potential of platform technologies in manufacturing, quality control, non-clinical or clinical testing;
• Integrating either computational modelling, automation, robotics or digital/Artificial Intelligence solutions with meaningful and measurable impact;

• Verify the improved performance of the developed process, in comparison to established ones.
• Demonstrate a reduction in the timeframe and costs of manufacturing while maintaining product quality and standardisation.
• Demonstrate the translatability, scalability, and robustness of the process suitable for the flexible manufacturing (centralised or decentralised) and deployment of ATMPs by important stakeholders in a patient-centric manner, including the medical community and hospitals.
• Assess the process and methods developed for their regulatory validity and utility (for example standardised assays including for potency), taking into consideration the potential regulatory impact of the results and, as relevant, develop a regulatory strategy for generating appropriate evidence as well as engaging with regulators in a timely manner.
• Promote green and sustainable industrial production and minimise environmental impact.

Participation of small and medium-sized enterprises (SMEs) is strongly encouraged and proposals should include a commitment for first deployment in the EU.

Where relevant, proposals are warmly invited to liaise with the Coordination and Support Action (CSA) project JOIN4ATMP[3], in view of creating complementarities and potential synergies.

The Joint Research Centre (JRC) may participate as a member of the consortium selected for funding. Proposals should consider the involvement of the European Commission's JRC regarding its experience in this field and with respect to the value it could bring in providing an effective interface between research activities and pre-normative science as well as strategies and frameworks that address regulatory requirements. In that respect, the JRC will consider collaborating with any successful proposal and this collaboration, when relevant, should be established after the proposal’s approval.

Applicants envisaging to include clinical studies[4] should provide details of their clinical studies in the dedicated annex using the template provided in the submission system.

[1] A centre of excellence refers to a team with a clear focus on a particular area of research; such a centre may bring together faculty members from different disciplines and provide shared facilities.

[2] ATMPs as classified by the European Medicines Agency (EMA): https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview

[3] https://ec.europa.eu/info/funding-tenders/opportunities/portal/screen/how-to-participate/org-details/999999999/project/101137206/program/43108390/details, https://cordis.europa.eu/project/id/101137206

[4] Please note that the definition of clinical studies (see introduction to this work programme part) is broad and it is recommended that you review it thoroughly before submitting your application.