Phase 1 including first-in-human clinical trials to test biomarker-guided medicines or multi-modal treatment interventions for patients with rare or v...

HORIZON-MISS is a Horizon Europe Research and Innovation Action under the EU Cancer Mission funding Phase 1, including first-in-human, multicentre clinical trials of biomarker-guided medicines or multi-modal interventions for rare or very rare cancers, with an indicative topic budget of €24 million and expected EU contribution per project of €7–€9 million (indicative 3 projects). Applications must be submitted via the EU Funding & Tenders Portal by 21 September 2027 (single-stage) and are open to consortia of legal entities established in EU Member States and Horizon Europe Associated Countries. Proposals must include final preclinical validation if still needed, documented regulatory advice, an Information on Clinical Studies annex, robust patient and stakeholder involvement, data deposited in the European Health Data Space catalogue and adherence to open science and UNCAN.eu sharing, and successful projects will join the Diagnosis and Treatment cluster. Funding is provided as a lump-sum RIA with payments linked to work package completion and evaluated on Excellence, Impact and Implementation with thresholds of 4/5 per criterion (cumulative 12/15).

Phase 1 first‑in‑human trials for biomarker‑guided medicines in rare cancers

Call at a glance

HORIZON-MISS-2027-02-CANCER-03

Horizon Europe Research and Innovation Action (RIA) funding to finalise preclinical validation and run phase 1, including first‑in‑human, multi‑centre clinical trials of biomarker‑guided medicines or multi‑modal interventions for patients with rare or very rare cancers or subtypes. Projects must follow open science, deposit metadata in the EU Health Data Space catalogue, use European research infrastructures, and plan to join the EU Cancer Mission Diagnosis and treatment cluster.

Deadline:21 September 2027, 17:00 Brussels time ¹

1. 1What it funds: preclinical work where still needed and phase 1 clinical trials (including first‑in‑human) to test safety, early efficacy and patient‑reported outcomes of biomarker‑guided medicines or multi‑modal treatments for rare/very rare cancers; data must be disaggregated and made FAIR.
2. 2Who can apply: consortia of organisations established in EU Member States or Horizon Europe Associated Countries (research centres, hospitals/clinical trial sites, SMEs, biotech companies, charities/foundations, patient organisations and other relevant stakeholders). National/regional health authorities and regulatory advice must be included or evidenced.
3. 3Key requirements: proof of regulatory advice on trial design; dedicated clinical study annex (use provided template); commitment to join the Diagnosis and treatment cluster and budget for networking; open science and UNCAN.eu availability of outputs.

This is a single‑stage RIA under the Cancer Mission with lump‑sum funding rules; applicants must complete the detailed budget table and clinical study template in the submission system. Projects are expected to facilitate access to subsequent trials or compassionate use pathways and to support later validation or commercialisation of promising interventions ¹.

Footnotes

1. 1Official topic page and application details are on the EU Funding & Tenders Portal Topic page.

Funding Opportunity Overview

Title: Phase 1 including first-in-human clinical trials to test biomarker-guided medicines or multi-modal treatment interventions for patients with rare or very rare cancers or cancer subtypes. Call: Supporting the implementation of the Cancer Mission. Programme: Horizon Europe, Missions. Topic ID: HORIZON-MISS. Type of action: HORIZON-RIA (Research and Innovation Actions), implemented as a Horizon Lump Sum Grant. Status: Forthcoming. Planned opening date: 10 February 2027. Deadline: 21 September 2027 at 17:00 Brussels time (single-stage). Official topic page: HORIZON-MISS-2027-02-CANCER-03 topic page.

Scope and Expected Outcomes

Purpose: To finalise preclinical validation where still required and to conduct phase 1, including first-in-human, multicentre clinical trials for biomarker-guided medicines or multi-modal treatment interventions targeting rare or very rare cancers or subtypes, within a precision oncology paradigm across EU Member States and Associated Countries.

• Patient access and benefit: Enable patients with rare or very rare cancers/subtypes to access promising, tailored biomarker-guided medicines or multi-modal interventions via subsequent clinical trials or national/regional compassionate use programmes.
• Innovation pipeline: Provide researchers, clinicians, innovators, startups, spin-offs/spin-outs, SMEs, charities/foundations and cross-sector professionals with access to innovative technologies, medical devices, or promising biomarker-guided medicines for further validation or commercialisation.
• Health system readiness: Generate early safety and efficacy evidence to support further testing and adoption of affordable biomarker-guided medicines or multi-modal interventions in European healthcare systems.

Mandatory Technical/Scientific Elements

• Preclinical (if still relevant): Finalise preclinical validation of promising biomarker-guided drugs for rare or very rare cancer indications using in vivo and/or ex vivo and/or in silico models; consider drug repurposing; disaggregate preclinical and clinical data by tumour biology, sex, gender, age, and other relevant variables such as socio-economic status or ethnicity.
• Clinical trials: Validate early safety and efficacy in phase 1 (including first-in-human) multicentre trials; consider innovative designs such as decentralised, basket, umbrella, roll-over/extension, and adaptive trials.
• Endpoints: Define primary and secondary endpoints to support safety, efficacy and patient-reported outcomes; co-define endpoints with patients and caregivers via participatory, socially innovative research approaches.
• Data and open science: Describe all datasets with metadata in the EU dataset catalogue of the European Health Data Space; leverage European research infrastructures; adhere to open science principles; make tools and models available via the future UNCAN.eu platform.
• Regulatory: Include proof of advice from regulators on the clinical trial design; provide full clinical study details in the dedicated annex template required by the submission system (Information on clinical studies).
• Stakeholder mix: Ensure an appropriate mix across disciplines, include regional and national health authorities, and robust patient and caregiver involvement.
• Mission clustering: Budget for networking, meetings, and joint activities; successful projects will join the EU Cancer Mission Diagnosis and treatment cluster, with Commission-facilitated coordination.
• Knowledge Centre on Cancer: Build on resources from the Knowledge Centre on Cancer (European Commission JRC), including the European Guidelines and Quality Assurance Schemes for cancer screening/diagnosis and ECIS/ECIR resources.

Definitions and context referenced by the topic:Rare cancers account for approximately 24% of all cancers; definitions and incidence are referenced to ESMO and RARECARE resources. The topic cites evidence on outcomes of patients in contemporary phase I trials and encourages applicants to consider ongoing EU-funded repurposing initiatives (e.g., REMEDI4ALL, REPO4EU). Innovative trial designs are explicitly listed (decentralised, basket, umbrella, roll-over/extension, adaptive). See official work programme extract within the Missions 2026-2027 document for the full topical narrative: Horizon Europe Missions 2026-2027 (Part 12).

Budget, Contribution and Timeline

Legal-financial set-up: Lump sum contributions per work package fixed in the grant agreement; payments upon completion of work packages; no reporting of actual costs; the granting authority may object to transfer/exclusive licensing of results up to 4 years after the action ends (Annex 5 specific provision). Detailed guidance on lump sums and the authorising decision are available: Lump sums - what do I need to know?; Decision authorising the use of lump sums ¹.

Who Should Apply

Eligible Applicant Types

• Universities and higher education institutions with oncology, pharmacology, biomarker and trial methodology expertise.
• Research and technology organisations and public research institutes.
• Hospitals, comprehensive cancer centres, clinical research networks, and academic clinical trial units capable of first-in-human and multicentre phase 1 trials.
• Startups, spin-offs/spin-outs and SMEs in biotech/medtech, diagnostics, digital health, drug discovery, and trial technologies.
• Large enterprises, including pharma and medtech companies, where relevant to biomarkers, therapeutics, or devices.
• Nonprofits, charities and foundations active in cancer research or patient support.
• Public authorities and health authorities at regional and national levels (as consortium partners/associated partners) to facilitate system adoption and alignment.
• Patient organisations and civil society actors for participatory endpoint definition and patient-reported outcomes integration.

Eligibility follows Horizon Europe General Annex B: standard collaborative consortia rules apply. International organisations or entities from non-associated third countries may participate; funding is typically for EU Member States and Associated Countries, with specific provisions for some non-EU/non-Associated Countries as per the Programme Guide.

Consortium Requirement

Consortium. Horizon Europe RIA collaborative projects generally require at least three independent legal entities, each established in a different EU Member State or Horizon Europe Associated Country. Given the clinical scope, inclusion of clinical sites across multiple countries and regulators’ advice is expected; applicants must annex clinical study details using the prescribed template.

Geographic Eligibility and Countries Mentioned

Beneficiary scope: EU Member States and Associated Countries to Horizon Europe. The topic repeatedly references activity across EU Member States and Associated Countries and requires multicentre clinical trials suitable for this geography. No specific countries are individually named; the scope is pan-European within MS/AC.

Target Sectors and Technologies

• Cancer, precision oncology and rare/very rare cancer subtypes.
• Biotech/medtech, including biomarker-guided therapeutics and multi-modal interventions.
• Clinical research and trial innovation: adaptive, basket, umbrella, decentralised and roll-over/extension designs; first-in-human operations.
• Digital/data and research infrastructures: EU Health Data Space dataset catalogues, European research infrastructures, and UNCAN.eu platform for tool/model access.
• Drug repurposing and pharmacology; pharmacokinetics and pharmacodynamics.
• Medical devices and potentially diagnostics where part of multi-modal strategies.
• Open science and data stewardship aligned with European infrastructures.

Project Stage and TRL Expectations

Expected maturity: late preclinical finalisation if still needed followed by early clinical validation. Projects are expected to progress candidates into phase 1, including first-in-human multicentre clinical trials, focusing on safety, early efficacy signals and patient-reported outcomes, with robust trial design and regulatory advice documented.

Application Modality and Process

• Application type: Open call, single-stage submission via the EU Funding & Tenders Portal: EU Funding & Tenders Portal.
• Evaluation criteria and thresholds: Excellence, Impact, Implementation; threshold 4.0 per criterion; cumulative 12.0.
• Templates and annexes: Use the standard Part A/B forms and upload the required annexes, including the clinical studies template for any clinical study and the detailed lump sum budget table.
• Model Grant Agreement: HORIZON Lump Sum MGA; payments linked to completion of work packages rather than actual costs.

Nature of Support, Co-funding and Success Rates

Nature of support: Monetary grant under Horizon Europe RIA with lump sum funding. RIA standard funding rate is 100% of eligible costs approximated in the lump sum. Co-funding: Not required unless the consortium proposes total estimated eligible costs exceeding the requested EU contribution. Success rates: Not specified in the topic; applicants should consult historical Mission Cancer call statistics or the Horizon Dashboard for indicative competitiveness.

Obligations, Compliance, and Data/Regulatory Requirements

• Regulatory advice: Include proof of advice from regulators on the trial design; sponsors should plan CTIS registration and ethics/regulatory approvals per the clinical studies template guidance.
• Clinical study annex: Applicants must complete and upload the Information on Clinical Studies template detailing rationale, objectives, endpoints, population, sample size/power, design, interventions, procedures, governance, operational feasibility, sites/recruitment, data management, safety and reporting obligations. Template: Information on clinical studies (HE) template.
• Mandatory clinical deliverables per study: Study initiation package before first enrolment; midterm recruitment report at 50% accrual; report on status of posting results in registries.
• Open science and FAIR data: Datasets must be described with metadata in the EU dataset catalogue of the European Health Data Space; tools and models to be made available via the future UNCAN.eu platform; leverage European research infrastructures.
• Patient and caregiver engagement: Co-creation of endpoints, participatory research models, inclusion of patient-reported outcomes; social innovation approaches are required.
• Data disaggregation: By tumour biology, sex, gender, age and other relevant variables such as socio-economic measures or ethnicity.
• Mission coordination: Successful proposals will participate in the EU Cancer Mission Diagnosis and treatment cluster and should budget for coordination meetings, networking, joint activities.
• IP and results: The granting authority may object to transfers or exclusive licensing of results up to 4 years post-action end, as per MGA Annex 5.
• Ethics: Address all ethics issues in the application; ensure compliance with EU and national laws for clinical research and data protection.

Budgeting and Lump Sum Mechanics

Applicants propose a lump sum based on estimated eligible costs per work package, beneficiary and cost category using the mandatory detailed budget table (Excel) provided in the submission system. Experts assess reasonableness versus activities. Payments are released for completed work packages during reporting; no actual-cost reporting; simplified financial reporting; focus on technical completion of agreed tasks/work packages. Detailed guidance: Lump sums - what do I need to know?.

Evaluation and Selection

• Single-stage evaluation by independent experts against Excellence, Impact, Implementation.
• Thresholds: 4 per criterion; cumulative threshold 12.
• Budget checks: Experts verify lump sum estimations are reasonable/non-excessive and aligned with activities; recommendations may adjust the lump sum during grant preparation without affecting the score unless serious issues are found.
• Indicative timeline: As per General Annex F; refer to the portal for updates.

Categorisation Answers (Detailed)

Eligible Applicant Types:Universities, research institutes, hospitals and comprehensive cancer centres, clinical trial units, startups, spin-offs and spin-outs, SMEs, large enterprises in pharma/medtech, nonprofits and charities/foundations, public authorities and health authorities, patient organisations and NGOs, European research infrastructures participants, and multidisciplinary consortia including data/ICT expertise.

Funding Type:Grant under Horizon Europe, Research and Innovation Action (RIA), implemented as a Lump Sum Grant according to the Horizon Lump Sum MGA.

Consortium Requirement:Consortium. Collaborative RIA requiring at least three independent legal entities each established in a different EU Member State or Associated Country. Given the clinical nature, multicentre clinical sites across countries are expected; regulatory and health authority stakeholders should be included.

Beneficiary Scope (Geographic Eligibility):EU Member States and Horizon Europe Associated Countries. Some non-EU/non-Associated Countries may participate with specific national funding provisions as per the Horizon Europe Programme Guide; automatic EU funding is generally limited to MS/AC.

Target Sector:Cancer and healthcare; precision oncology; biotech/medtech; clinical research and trial design innovation; biomarkers and targeted therapeutics; medical devices and multi-modal interventions; data/open science and research infrastructures.

Mentioned Countries:Regionally specified as EU Member States and Associated Countries; no individual countries explicitly named in the topic text. Geographic reach is pan-European within MS/AC.

Project Stage:Transition from late preclinical validation (if still required) to early clinical validation in phase 1, including first-in-human multicentre trials; focus on safety, early efficacy, and patient-reported outcomes with innovative designs.

Funding Amount:Expected EU contribution per project: €7,000,000 to €9,000,000. Topic budget: €24,000,000 in 2027.

Application Type:Open call; single-stage submission via the EU Funding & Tenders Portal with mandatory templates and annexes.

Nature of Support:Money. Non-repayable grant funding under a lump sum scheme; standard RIA funding rate 100% of eligible estimated costs encompassed in the lump sum.

Application Stages:1 stage. Single-stage submission and evaluation.

Success Rates:Not disclosed for this topic. Horizon Europe Missions calls are competitive; applicants should consult Horizon dashboards and past Mission Cancer calls for indicative competitiveness.

Co-funding Requirement:No co-funding required under RIA 100% funding rate for eligible estimated costs in the lump sum. If total project costs exceed the EU contribution, additional resources must be covered by the consortium.

Templates and Application Structure

Applicants must use the standard Horizon Europe forms in the Submission System. The following call-specific templates and documents are required or strongly expected given the clinical nature:

• Information on clinical studies (HE) annex: A mandatory annex for proposals involving clinical studies. It must cover per clinical study: Title/acronym/identifier; study rationale and current knowledge; outcomes from completed/ongoing studies; MoA evidence; primary/secondary objectives; study population and inclusion/exclusion criteria with justification; sample size and power calculation; design (controlled/uncontrolled; randomised; blinded/open; parallel/crossover; innovative designs such as small-population personalised or adaptive platform trials); intervention type; schedule of procedures and participation duration; scientific advice and HTA interactions; adherence to clinical, safety and methodological guidelines; patient/carer involvement; regulatory intelligence and ethics strategy across jurisdictions; sponsor identity and governance boards; IMP/IMD availability, manufacturing, labelling, storage, logistics and import/export; recruitment strategy, monitoring and mitigation; clinical sites across countries and selection criteria; competitive recruitment management and evidence of site performance; additional supplies for study procedures; data management plan (standards, capture, verification, central collection, cleaning, analysis, reporting, security); safety, ethics and quality reporting obligations; sponsor-responsibility items supported by third parties and oversight; milestone plan for submissions, approvals, site initiations, recruitment completion, last-patient-last-visit, analysis and reporting; mandatory deliverables (study initiation package, midterm recruitment report, posting results report). Template: Information on clinical studies (HE) template.
• Detailed lump sum budget table (Excel): Mandatory annex for lump sum topics. Cost estimations per beneficiary and work package by cost category; automatic generation of lump sum shares; alignment with the Horizon personnel cost dashboard; ensure reasonableness and provide justification in the Any comments tab if above dashboard values.
• Part B content expectations: Excellence (state-of-the-art; objectives; methodology; sex/gender dimension; patient engagement; innovation potential; trial design rigor and feasibility), Impact (pathways for further trials, compassionate use and uptake; contribution to Mission objectives; exploitation/commercialisation; access/affordability; policy alignment; open science, data and UNCAN.eu contributions), Implementation (work plan with WPs aligned to clinical development, regulatory, manufacturing/supply, sites/recruitment, data management/biostatistics, ethics/safety, dissemination/exploitation, management and coordination including Mission cluster participation; consortium roles and capacities; risk management and contingency planning for recruitment/supply/regulatory hurdles).
• Model Grant Agreement: HORIZON-AG-LS (Lump Sum MGA). Guidance: Lump sums - what do I need to know?.
• Call-specific instructions: Detailed budget table (HE LS) and Information on clinical studies (HE) must be uploaded as annexes. Use the application form available in the Submission System; consult the Standard evaluation form (HE RIA, IA) in the Reference Documents.

Partnering and Support

• Partner search: Publish announcements on the Portal for this forthcoming topic after login if you are a LEAR, Account Administrator, self-registrant, or have a public profile.
• National Contact Points (NCPs): Obtain advisory support for Horizon Europe participation, including in third countries; see the Programme Guide and NCP directories.
• Enterprise Europe Network: SME-focused support including EU research funding guidance.
• Research Enquiry Service, IPR Helpdesk, and standards bodies (CEN-CENELEC, ETSI) are available for regulatory/IP/standardisation advice.

Summary Explanation

This Horizon Europe Mission Cancer RIA funds rigorous, multicentre, phase 1 including first-in-human trials to test biomarker-guided medicines or multi-modal interventions for rare and very rare cancers. Many patients with these cancers face late diagnoses, few options, and limited trial access despite promising disease control rates in early trials. The topic requires applicants to finalise any remaining preclinical validation, then design and run innovative early clinical trials using contemporary methodologies suited to small, molecularly defined populations. Endpoints must encompass safety, early efficacy and patient-reported outcomes co-created with patients and caregivers. Projects must secure documented regulatory advice and comprehensively plan operational feasibility spanning IMP/device manufacturing and logistics, site selection and recruitment across MS/AC, data standards and management, and safety/ethics reporting. Open science is foundational: datasets must be catalogued for the European Health Data Space; tools/models must be shared via UNCAN.eu; and European research infrastructures should be leveraged. Proposals must plan participation in the EU Cancer Mission Diagnosis and treatment cluster and draw on Knowledge Centre on Cancer resources to align and scale impact. Funding is delivered as a lump sum RIA at 100% of eligible estimated costs, with payments linked to the completion of work packages rather than actual-cost reporting, simplifying administration and focusing on scientific and clinical delivery. The call targets strong, multidisciplinary consortia including hospitals and cancer centres, academic and industrial innovators, SMEs, patient organisations, and health authorities, capable of rapidly generating robust early evidence to unlock subsequent clinical testing, compassionate use where appropriate, and eventual health system adoption across Europe.

Footnotes

1. 1For the legal basis and guidance on lump sum funding under Horizon Europe, see the Commission decision authorising lump sums and the participant guide: Decision authorising the use of lump sum contributions under Horizon Europe: ec.europa.eu; Lump sums - what do I need to know?: ec.europa.eu.

HORIZON-MISS-2027-02-CANCER-03: Phase 1 Clinical Trials for Rare Cancers

Opportunity Overview

This call under the EU Cancer Mission supports Phase 1 clinical trials, including first-in-human studies, to test biomarker-guided medicines or multi-modal treatment interventions for patients with rare or very rare cancers or cancer subtypes. It addresses the limited treatment options and late diagnosis challenges for these patients, who represent about 24% of all cancers in Europe.

Key Dates:Planned opening: 10 February 2027. Deadline: 21 September 2027 at 17:00 Brussels time. Single-stage submission.

Funding Details:Total indicative budget: €24 million. Expected EU contribution per project: €7-€9 million. Indicative number of projects: 3. Type of action: HORIZON-RIA (Research and Innovation Actions). Funding as HORIZON Lump Sum Grant.

Expected Outcomes

Proposals must contribute to all of the following: access for patients to promising treatments via trials or compassionate use; access for researchers, physicians, innovators, SMEs, charities to technologies for validation or commercialisation; early safety and efficacy evidence for healthcare providers and policymakers to support affordable interventions in healthcare systems.

Scope and Requirements

Proposals must address all specified activities: finalise preclinical validation of promising biomarker-guided drugs for rare/very rare cancers using in vivo/ex vivo/in silico models, considering drug repurposing (e.g., REMEDI4ALL, REPO4EU); validate safety/efficacy in Phase 1 multi-centre trials including first-in-human, with innovative designs (e.g., basket/umbrella/adaptive trials); define endpoints with patients/caregivers for safety, efficacy, patient-reported outcomes.

• Disaggregate data by tumour biology, sex, gender, age, socio-economic status, ethnicity.
• Register datasets in EU Health Data Space catalogue; follow open science principles; share via UNCAN.eu.
• Include proof of regulator advice on trial design; involve multi-disciplinary stakeholders and health authorities; use dedicated clinical study annex template.
• Join Diagnosis and Treatment cluster; budget for networking/meetings; build on Knowledge Centre on Cancer (KCC) resources.

Eligibility and Who Can Apply

Open to legal entities from EU Member States and Associated Countries. Consortia must demonstrate financial/operational capacity. Detailed conditions in General Annexes A-C. Multi-disciplinary mix required, including regulators, health authorities, patients/caregivers. SMEs, startups, spin-offs, charities encouraged.

Evaluation Criteria

Evaluation per General Annexes D-F. Focus on alignment with Cancer Mission, feasibility of Phase 1 trials, open science, patient engagement.

Application Process

1. 1Use submission system; include clinical study details in dedicated annex.
2. 2Provide detailed budget table for lump sum; adhere to page limits/layout in Annexes A/E.
3. 3Upload Information on Clinical Studies template as separate annex.
4. 4Model Grant Agreement: HORIZON-AG-LS (Lump Sum).

Lump Sum Funding:Payments based on work package completion, not actual costs. Detailed budget estimation required, including personnel, subcontracting, purchases; 25% flat indirect costs. No financial reporting/audits on costs.

Key Considerations for Applicants

• Rare cancers: ~24% of cases per ESMO/RARECARE; lower survival rates.
• Patient involvement essential for endpoints via participative models.
• Synergies with EU projects (e.g., REMEDI4ALL, REPO4EU); join Cancer Mission clusters.
• Official portal: EU Funding & Tenders Portal. Work Programme: HE WP 2026-2027 Missions.

Successful projects must collaborate in Diagnosis and Treatment cluster, with Commission-facilitated coordination. Build on KCC (JRC-hosted) for EU alignment.

Footnotes

1. 1Rare cancers defined per ESMO/RARECARE; ~650,000 new EU cases/year. See primary portal for templates/guides.