Validation of fluid-derived biomarkers for the prediction and prevention of brain disorders
Treatments for some high-burden brain disorders are potentially on the horizon[[ For example, the Nature news feature (March, 2022): Could drugs prevent Alzheimer’s? These trials aim to find out. doi: https://doi.org/10.1038/d41586-022-00651-0]]. Consequently, many patients and citizens will want to know if they are eligible for these treatments. For some disorders, a definitive diagnosis is difficult, expensive and time-consuming. Simple blood or other fluid-derived (e.g. saliva, urine, sweat) tests for markers that may indicate early signs of the disorder, and which can be deployed for widespread clinical use are needed.
The brain disorders within the scope of this topic fall under two categories, namely those listed under chapters six and eight of the International Classification of Diseases[[ International Classification of Diseases 11th Revision (ICD-11), developed by the World Health Organization (WHO); Chapter 6: ‘Mental, behavioural or neurodevelopmental disorders’; Chapter 8: ‘Diseases of the nervous system’.]]. Proposals in the area of mental disorders are encouraged.
Proposals should address all of the following aspects:
- Proposals should aim to validate biomarkers that can reliably confirm early stages of the human brain disorder and guide treatment/ intervention selection[[ The biomarker should link to a clinical meaningful endpoint.]].
- Proposals should aim to provide evidence supporting the regulatory acceptance of the biomarkers[[ The European Medicines Agency (EMA) offers scientific advice to support the qualification of innovative development methods for a specific intended use in the context of research and development into pharmaceuticals.]].
- Exploitation of existing data, biobanks, registries and cohorts is expected, together with the generation of new key data.
- Inclusion of patients or patient organisations in the research is strongly encouraged, as to ensure that their views are considered.
- Sex and gender aspects, age, socio-economic, lifestyle and behavioural factors should be taken into consideration in the study.
- To enable sharing of samples, quality data and advanced analytical and digital tools, consideration should be made for using infrastructures already developed at the European[[ EU-supported infrastructures include, for example, the BBMRI-ERIC infrastructure for biobanking, the EBRAINS research infrastructure, and various platforms developed by the Innovative Medicines Initiative (IMI) and its successor the Innovative Health Initiative (IHI).]] or national level.
- To enable the management of brain disorders, consideration should be made in demonstrating the gained cost efficiency.
- SME participation is encouraged.
Applicants invited to the second stage and 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.