European Commission: Development of New Effective Therapies for Rare Diseases

Deadline: 1-Feb-22

The European Commission (EC) has announced the Development of new effective therapies for rare diseases to develop therapies for rare diseases with no approved therapeutic option.

Proposals should focus on group (s) of rare diseases with commonalities, such as shared biological features, possibly within the same and / or across different medical areas within the rare diseases landscape. Thus, proposals should not address a single disease only (for example with an Orphacode representing a single disease).

The topic will support proposals covering several different stages in the continuum of the innovation pathway (i.e. translational, preclinical, clinical research, validation in the clinical and/or real-world setting etc.), as relevant. SME(s) participation is encouraged with the aim to strengthen the scientific and technological basis of SME(s) and valorise their innovations for the benefit of people living with a rare disease.

Activities

The proposals should address most of the following research activities:

Establish multidisciplinary collaborations between all relevant stakeholders by integrating disciplines, technological developments and existing knowledge. Integrate harmonised data from multiple sources (i.e. natural history studies/clinical trials, multi-omics, medical imaging, registries etc.) by utilising data analytics and/or other suitable methods, with the aim to understand the pathophysiology/heterogeneity of the rare diseases concerned and to identify therapeutically actionable mechanisms.
Develop and utilise relevant preclinical models and/or innovative tools/technologies to: verify molecular/cellular pathways/genes that can be therapeutically targeted, increase the confidence in the targets selection and/or perform toxicity studies. When using disease models the applicants should describe how well the model replicates the pathology or the human condition.
Develop and/or execute innovative clinical trials designs for small populations and novel approaches to assess and monitor the safety and efficacy of the proposed interventions. Such approaches may include but are not limited to: biomarkers defining robust surrogate and clinical endpoints; artificial intelligence tools/medical devices/biosensors/ companion/ complementary diagnostics for defining reliable patient reported outcomes; modelling and simulation and in-silico trials methodologies.
Carry out preclinical proof-of-concept (PoC) studies and / or multinational interventional clinical studies to demonstrate the safety and efficacy of the therapeutic interventions under study. Preclinical PoC studies should include late-stage preclinical studies (ie toxicological properties, adverse effects etc.). Clinical studies may cover all necessary development stages. Applicants should propose a clear exploitation pathway through the different necessary steps (research, manufacturing, regulatory approvals and licensing, IP management etc.) in order to accelerate marketing authorization and up take by the health systems.

Funding Information

The check will normally be done for the coordinator if the requested grant amount is equal to or greater than EUR 500,000, except for:

public bodies (entities established as a public body under national law, including local, regional or national authorities) or international organizations; and
cases where the individual requested grant amount is not more than EUR 60,000 (lowvalue grant).

Expected Outcomes

Proposals under this topic should aim for delivering results that are directed, tailored towards and contributing to some of the following expected outcomes:

Researchers and developers make the best use of the state-of-the-art knowledge and resources for a fast and effective development of new therapies for rare diseases.
Researchers and developers increase the development success rate of therapies for rare diseases by employing robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes and / or innovative clinical trials designs.
Developers and regulators move faster towards market approval of new therapies for rare diseases (with currently no approved treatment option) due to an increased number of interventions successfully tested in late stages of clinical development.
Healthcare professionals and people living with a rare disease get access to new therapeutic interventions and / or orphan medicinal products.

Eligibility Criteria

To be eligible for funding, applicants must be established in one of the eligible countries, ie:

the Member States of the European Union, including their outermost regions;
the Overseas Countries and Territories (OCTs) linked to the Member States;
eligible non-EU countries:
- countries associated to Horizon Europe;
- low- and middle-income countries.