Pragmatic clinical trials to optimise immunotherapeutic interventions for patients with refractory cancers

Expected Outcome

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

• Patients with refractory cancers and their caregivers/families will have access to optimised, tailored and affordable immunotherapeutic interventions that increase their quality of life, across European regions, EU Member States and Associated Countries;
• Healthcare professionals and academia will have access to clinical evidence, on effectiveness of immunotherapeutic interventions, to deploy evidence-based treatment interventions with improved patient selection that improve outcomes in real life, i.e. in routine healthcare, for patients with refractory cancers who often present with co-morbidities;
• National healthcare providers, policymakers and authorities in European regions, EU Member States and Associated Countries will have the evidence to implement and reimburse optimised and affordable immunotherapeutic interventions in their healthcare systems, including in everyday medical practice.

Scope

While cancer research and innovation have generated novel treatment options, patients with refractory cancers across Europe need access to more effective, affordable and tailored cancer immunotherapeutic interventions which keep up with increasing demands in a complex and fragmented oncology healthcare landscape with spiralling healthcare costs.

Pragmatic clinical trials focus on choosing between care options. Pragmatic trials evaluate effectiveness of interventions in settings that more closely resemble routine, real-world settings, aiming to produce evidence directly applicable to clinical practice[1].

Proposals should address all the following:

• Conduct randomised or cluster-randomised academic investigator-initiated[2] pragmatic clinical trials that benefit patients with refractory cancers - at any stage of the disease, for any cancer subtype, in any age group or part of society - to deliver effective, affordable and tailored immunotherapeutic interventions for implementation by healthcare systems at the level of local communities, European regions, EU Member States and Associated Countries.
• All data should be disaggregated by sex, gender, age and other relevant variables, such as by measures of socio-economic status or ethnicity. Translational research is limited to supporting biomarker-informed patient stratification and the conduct and analyses of the proposed clinical trial(s).
• The primary and secondary endpoints of the pragmatic clinical trials should target overall survival, patient-reported outcomes and quality of life issues considered important by and for cancer patients and their caregivers/families. Such endpoints should be defined together with patients and their caregivers/families through research that stimulates social innovation and supports end-user engagement using participative research models.
• The chosen treatment 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 EU Member States and Associated Countries. Furthermore, affordability and accessibility should be taken into account.
• Applicants should include an appropriate mix of stakeholders from various disciplines, sectors[3] and regional as well as national health authorities, and provide details of the clinical study(ies) in the dedicated annex using the template provided in the submission system.

The successful proposals are expected to build on resources made available by the Knowledge Centre on Cancer (KCC)[4] to foster EU alignment and coordination.

Successful proposals will be asked to join the 'Diagnosis and Treatment' cluster for the EU Cancer Mission[5] and should include a budget for networking, attendance at meetings, and joint activities[6]. The Commission will facilitate coordination of these activities.

[1] Some examples: treatment versus active surveillance in patient management, combination of treatment interventions, determination of optimal dose and dose schedule, de-escalation of treatment intervention, comparative effectiveness of different treatment interventions.

[2] 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.

[3] such as physicians, academia, patients and their caregivers, patient representatives, engineers, behavioural scientists, SMEs, business networks, insurance companies, charities and foundations, research organisations, civil society.

[4] 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

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

[6] Examples of those activities are research or research capacity, organising joint workshops, establishing best practices, joint communication or citizen engagement activities with projects funded under other clusters and pillars of Horizon Europe, or other EU programmes, as appropriate. Proposals are not required to include details of these activities, as they will be defined during the grant agreement preparation and during the life of the project.