Pragmatic Clinical Trials on Minimally Invasive Diagnostics

Proposals under this topic should aim to deliver results that are directed and tailored towards and contribute to all of the following expected outcomes:

• Cancer patients and their caregivers have access to optimised and affordable, minimally-invasive diagnostic interventions that increase their quality of life, across European regions, Member States and Associated Countries;
• Healthcare professionals and academia deliver better outcomes through routine healthcare, including quality of life, for men and women with cancer who often suffer from sex-related co-morbidities and side-effects;
• National healthcare providers, policymakers and authorities in European regions, Member States and Associated Countries will have the evidence to implement optimised and affordable minimally-invasive diagnostics in their healthcare systems, including in everyday medical practice.

While cancer research and innovation have generated novel treatment options, cancer patients across Europe need access to minimally-invasive, patient-centred diagnostic interventions which keep up with increasing demand in a complex and fragmented oncology healthcare landscape with increasing healthcare costs.

Furthermore, the COVID-19 pandemic with its detrimental impact on cancer control has demonstrated the need for different clinical trial designs with fewer inclusion and exclusion criteria that would allow for the evaluation of real-world effectiveness, driving better and affordable diagnostic solutions that are widely accessible across European regions, Member States and Associated Countries.

Healthcare professionals and academia generate clinical evidence, by evaluating effectiveness in randomised or cluster-randomised academic investigator-initiated^[1] pragmatic clinical trials, on how to best perform and deploy evidence-based, minimally-invasive diagnostic interventions.

Pragmatic clinical trials focus on choosing between care options. Pragmatic trials evaluate effectiveness, the effect of diagnostics in routine (real-world) clinical practice.

Proposals should address all of the following:

• Design and conduct randomised or cluster-randomised academic investigator-initiated pragmatic clinical trials to deliver effective and evidence-based diagnostic interventions for implementation by healthcare systems at the level of local communities, European regions, Member States and Associated Countries, taking into account stratification, such as biology, molecular features, sex, gender, cancer stage, and age. Clinical trial design and conduct could be aided by computational, simulation and visualisation tools and technologies where appropriate.
• The chosen diagnostic intervention(s) should be adapted to the particular needs of the target population and to the specificities of the provision of care at local, regional, or national level, duly reflecting the diversity across Member States and Associated Countries. Furthermore, affordability and accessibility should be taken into account.
• The successful proposals should clearly justify and describe the evidence supporting the chosen diagnostic intervention.
• The primary and secondary endpoints of the pragmatic clinical trial should support overall survival, patient-reported outcomes and quality of life issues considered important by and for cancer patients and their caregivers.
• Such endpoints should be defined together with patients and their caregivers through research that uses open knowledge, (social) innovation systems and support end-user engagement, such as living labs^[2] or other participative research models.
• These pragmatic clinical trials should include stakeholders such as physicians, academia, patients and their caregivers, patient representatives, SMEs, insurance companies, charities and foundations, research organisations, civil society, regional and national research, innovation and health authorities.
• Successful pragmatic clinical trials, including their analyses, should be completed within 5 years from the start of the project. Translational research is not within the scope of this topic.
• In all instances, sex- and gender-related issues must be taken into account. All data should be disaggregated by sex, gender, age and other relevant variables, such as by measures of socio-economic status or ethnicity.

This topic requires the effective contribution of 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 successful proposals are expected to liaise with and build on resources made available by the Knowledge Centre on Cancer (KCC)^[3] in order to foster EU alignment and coordination.

The Commission will facilitate Mission-specific coordination through future actions, notably fostering exchanges with other proposals funded under this topic. Hence, successful applicants will be asked to join the ‘Diagnosis and Treatment’ cluster for the Mission on Cancer^[4]. In this regard, the Commission will take on the role of facilitator, including with relevant initiatives and stakeholders, if appropriate.

Therefore, proposals should include a budget for networking, attendance at meetings, and potential joint activities without the prerequisite to give details of these at this stage. Examples of these activities are the organisation of joint workshops, the exchange of knowledge, the establishment of best practices, or the initiation of joint communication activities with projects funded under other clusters and pillars of Horizon Europe, or other EU programmes, as appropriate.

The details of joint activities will be defined during the grant agreement preparation phase and during the life of the project.

[1] Clinical trials in which a health technology (e.g. a medicinal product, a medical device, an in-vitro diagnostic medical device, a surgical or other medical intervention) is tested in humans, independently from commercial interest and for public health benefits.

[2]https://enoll.org/

[3]Hosted by the European Commission's Joint Research Centre (JRC). Especially through the ’European Guidelines and Quality Assurance Schemes for Breast, Colorectal and Cervical Cancer Screening and Diagnosis‘, and the ’European Cancer Information System (ECIS)’ and the ’European Cancer Inequalities Registry (ECIR), see https://knowledge4policy.ec.europa.eu/cancer_en

[4]In order to address the objectives of the Mission on Cancer, participants will collaborate in project clusters to leverage EU-funding, increase networking across sectors and disciplines, and establish a portfolio of Cancer Mission R&I and policy actions.