Boosting the translation of biotech research into innovative health therapies

Expected Outcome

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination “Developing and using new tools, technologies and digital solutions for a healthy society”. To that end, proposals under this topic should aim to deliver results directed towards and contributing to all the following expected outcomes:

• Healthcare providers, researchers and patients get faster access to innovative therapies.
• The European Union benefits from more clinical trials being conducted with new biotech therapeutic approaches.
• The competitiveness of small and medium-sized enterprises (SMEs) from the EU and Associated Countries within the health biotech sector is strengthened.

Scope

The Commission Communication 'Building the future with nature: Boosting Biotechnology and Biomanufacturing in the EU' [1] has recently identified research and technology transfer to the market as a major challenge for the biotechnology sector. This topic aims to speed up the development of innovative biotechnology-based therapies by supporting the initial phases of clinical research. SMEs play a key role in the EU's potential to innovate, with most biotechnology-derived drugs in development being progressed by SMEs and small biotech companies. However, transitioning from drug discovery and development stages to approved products requires substantial investment and sufficient resources in different areas (e.g., manufacturing, clinical trial management, regulatory affairs, etc.), with the time needed for clinical development often exceeding 10 years[2]. This topic targets collaborative multidisciplinary consortia of SMEs, academics, clinicians and research organisations bringing together the necessary expertise to launch the clinical development of novel biotechnology-derived therapeutics. Collaboration with the relevant European research infrastructures is encouraged. This topic does not address the full clinical development needed to bring products to market but aims to support the critical transition phase from preclinical to clinical development by supporting the early clinical phases. A non-exhaustive list of biotechnology-derived therapies in scope include monoclonal antibodies, (therapeutic) vaccines, recombinant biomolecules, Advanced Therapy Medicinal Products (ATMPs), nano-based drugs, RNA therapies etc. Whole blood, blood components and other substances of human origin are not within the scope of this topic.

Proposals submitted under this topic should include all the following elements:

• A Clinical study either phase I, II or I/II depending on the appropriate stage of development.
• The proposal should convincingly demonstrate a significant economic potential of the final product(s) for the Single Market.
• A clearly defined exploitation plan, with a detailed proposed route to commercialisation, description of the intellectual property ownership and benefit for the SME(s). The plan should include an anti-shelving strategy, commercial forecasts for the product sales & revenue, and strategies for follow-up financing as well as market authorisation. The exploitation strategy should envisage a first deployment in the EU.
• Justification of the patient populations that will benefit directly from the development of the therapies. Clinical indications where potentially large patient populations could benefit will be favoured.

The maximum project duration should not exceed four years.

Applicants should provide details of their clinical studies[3] in the dedicated annex using the template provided in the submission system. As proposals under this topic are expected to include clinical studies, the use of the template is strongly encouraged.

[1] https://research-and-innovation.ec.europa.eu/document/download/47554adc-dffc-411b-8cd6-b52417514cb3_en

[2] https://go.bio.org/rs/490-EHZ-999/images/ClinicalDevelopmentSuccessRates2011_2020.pdf

[3] 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.