Comparative effectiveness research for healthcare interventions in areas of high public health need

Effective, affordable and accessible healthcare for diverse population groups is challenging and complex. For example, specific needs underlie the delivery of effective preventive actions and therapeutic treatments to a rapidly growing elderly population, often presenting comorbidities and associated polypharmacy. The paediatric population, including children born preterm, has also its specific needs in specially adjusted therapeutics and early interventions to address emerging health and developmental problems. Similar to the elderly population, the paediatric population is often excluded from many clinical trials that generate the evidence base for healthcare interventions. Women, including pregnant women, are also often under-represented in clinical studies and access to quality healthcare is frequently inadequate. Other population groups with limited access to quality healthcare and/or under-representation in clinical studies include low-income groups, and refugees. Intersectionality within these groups also needs consideration.

Proposals should address most of the following:

• Compare the use of currently existing (pharmacological, non-pharmacological and technological) healthcare interventions in specific population groups (or selected subgroups). While there is no restriction on diseases or conditions, preference will be given to proposals focusing on interventions with high public health relevance[[ Interventions addressing diseases or conditions that are particularly frequent, have a high negative impact on the quality of life of the individual and/or are associated with significant costs where savings can be achieved.]].
• Ensure acceptability and sustainability of the healthcare intervention through early involvement of ‘end users’ (e.g. patients, care providers) in the design of the study (integrating patient valued outcomes) and, where possible, in the research process including implementation. Additionally, proposals should take into account the diversity of health systems in different regions of Europe to allow large-scale uptake.
• Consider involving HTA bodies in order to create synergies and accelerate the practical implementation of the results. Where relevant, existing work of EU-funded projects such as EUnetHTA[[ https://www.eunethta.eu/]] should be also taken into account.
• Consider issues of particular relevance for the target populations, for example, multimorbidity, complex chronic conditions, polypharmacy, substance misuse, vaccine efficacy, compliance, age, gender specificities and diseases with high societal burden (including but not limited to e.g. musculoskeletal diseases and mental health disorders). Special consideration should be given to fulfilling all ethical requirements.
• For the chosen population, assess clinical and safety parameters, as well as health and socio-economic outcomes (e.g. quality of life, patient mortality, (co)morbidity, costs, and performance of the health system). Agreed core outcome sets (COS) should be used as endpoints in conditions where they already exist, in other cases, efforts should be made to agree on such COS. Consider using new instruments and methods for determining the burden of disease and for evaluating the effects of the interventions. Low-cost innovations should also be considered.
• Inclusion of patient organisations and associations of caregivers and other healthcare professionals is recommended.
• Clinical trials, including pragmatic clinical trials, observational studies, use of existing health data in different study designs, creation of large-scale databases and performing meta-analyses may be considered for this topic. Use of existing data should always be considered to add value, increase quality and increase implementation speed of the study. Regarding databases, sustainability after the proposed action's end also needs to be considered.
• The proposed research needs to take into account sex and gender aspects.

This topic requires the effective contribution of social sciences and humanities (SSH) disciplines and the involvement of SSH experts, institutions as well as the inclusion of relevant SSH expertise, in order to produce meaningful and significant effects enhancing the societal impact of the related research activities.

The Commission will ensure an overall coordination mechanism between the projects funded under this topic to catalyse the exchange of knowledge, as well as the development and adoption of best practices. Proposals are expected to budget for the attendance to regular meetings. Projects resulting from this call will be invited to share and discuss their case studies amongst themselves and with relevant stakeholders at the EU level, and necessary resources should be allocated to this task.

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.