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European Society for Paediatric Oncology

SIOP Europe / SIOPE

The European Society for Paediatric Oncology represents over 2,500 healthcare and research professionals working to improve care and outcomes for children with cancer across Europe.

Lobbying Activity

SIOPE urges childhood cancer focus in EU competitiveness funding

12 Nov 2025
Topic — Consultation on the post-2027 EU budget specifically regarding funding for European competitiveness.
Message — The organization asks the EU to prioritize childhood cancer in the competitiveness fund. They recommend supporting academia-led research and cross-border clinical networks to bridge innovation gaps.12
Why — This would provide dedicated resources for rare disease research and cross-border collaboration.34
Impact — Industrial infrastructure projects might see reduced funding as resources shift toward clinical networks.5
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Response to EU’s next long-term budget (MFF) – EU funding for competitiveness

7 Nov 2025

The European Society for Paediatric Oncology (SIOPE) welcomes the Horizon Europe (20282034) proposal and asks the Commission to make childhood cancer a clear, visible priority across Pillar IIIV by funding the research infrastructure and cross-border collaboration that delivers results. All paediatric cancers are rare and require dedicated approaches across the patient's journey to increase cure rates, improve quality of care, and eradicate inequalities in access to high quality research and care. Europe has strong care and research networks including the European Reference Network for PaedCan links childhood cancer centres across countries and European Clinical Trial Groups (ECTGs) design and run tumour-specific trials. The next step is the development of Comprehensive Childhood Cancer Infrastructures (CCCIs) that couple high-quality diagnosis, care and treatment with research and innovation. CCCIs can be recognised and expanded within ERN PaedCan. Sustaining the development of Comprehensive Childhood Cancer Infrastructures within ERN PaedCan in a manner distinct from adult-oriented initiatives will reflect the organisational reality of the childhood cancer in Europe and ensure integrated, multidisciplinary research and care. Such infrastructures are aligned with the Proposals aim to fund research and technology infrastructures within the European Research Area and should be considered as part of this effort. Paediatric drug development needs a different path from adult oncology due to rarity and small patient numbers Although 5-year survival rates are now up to 80%, with improvements in 10-year survival rates also reported, there are prevailing issues in the pace of development of treatments for paediatric cancers. To support life-saving innovation, Horizon should fund cross-border clinical trials, with a strong focus on early-phase studies for high-need tumours, and support regulatory and methodological science designed for rare-disease settings. This academic part must be coupled with a stronger pipeline for new companies. Calls in Pillar II and Pillar III should support collaborative research, including in academic settings, in paediatric drug development, including cell and gene therapies, advanced diagnostics and clinically validated AI, so ideas born in university hospitals can grow into products that reach children. Cross-border collaboration is vital to paediatric oncology and has been a key driver in improving delivery of high-quality outcomes for children with cancer to date. Horizon should resource ECTGs to run pan-European trials with shared protocols and outcome measures; fund twinning programmes so teams learn by doing and can exchange knowledge and good practices between universities, hospitals, and centres; and invest in interoperable data tools so studies can combine clinical, imaging and molecular data across countries while protecting childrens privacy. Projects such as UNCAN.eu and UNICA4EU are great examples of AI use in childhood cancer. Childhood cancer is also a case for European competitiveness. Saving children is a moral duty, but it is also an investment in the future of the continent. Healthy survivors shape Europes human capital for decades to come. A strong paediatric cancer ecosystem attracts talent, anchors high-value biotech and data jobs, and draws clinical trials to Europe. By funding ERN PaedCan, academic clinical trial networks, and the implementation of CCCIs the EU can unlock the potential of multi-country trials, support the paediatric oncology workforce through twinning and enable further development of innovative therapies including ATMPs Horizon Europe can help more children and young people with cancer access vital care and research, reduce inequalities between European countries, and make Europe a place where children with cancer will not only survive, but have a bright future.
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Response to European Innovation Act

2 Oct 2025

SIOPE welcomes the European Innovation Act as a potentially useful instrument to shift gears in paediatric oncology innovation, and our aim is clear: to see more start-ups and biotech companies sustaining and scaling up innovation for children with cancer, namely start-ups focused on specific paediatric oncology drug development, including ATMPs. Each year in Europe, over 35,000 children and young people are diagnosed with cancer and over 6,000 lose their lives. There are an estimated 500,000 childhood cancer survivors in Europe today, with numbers growing substantially yearly; at least two-thirds of them experience long-term adverse side-effects into adulthood. There are still persistent inequalities in access to treatment, care and research, with an estimated 20% gap in childhood cancer survival rates between Western and Eastern Europe. Since the implementation of the EU Paediatric Regulation, only 16 anti-cancer medicines (mainly adult cancer drugs) have been authorised for a paediatric cancer indication, in contrast to over 150 for adult cancers only. As capability attracts investment, we ask the European Innovation Act to recognise and further enhance the EU-wide research infrastructures for children and adolescents with cancer. This can be achieved acknowledging strategic paediatric networks delivering care and research across Europe as engines of innovation. Both the European Reference Network for Paediatric Oncology (ERN PaedCan) and SIOPEs European Clinical Trial Groups represent leading actors in this field. ERN PaedCan, as a Europe-wide driver of high-quality care linked with the adoption of standard of care treatment, can support the update of innovation. To allow this the EU should develop and expand Comprehensive Childhood Cancer Infrastructures within ERN PaedCan so every Member State can couple high-quality care with continuous clinical and translational research; and scale twinning programs to spread knowledge and competence, raise trial readiness, and reduce inequalities in access and outcomes. To support data sharing infrastructure, we ask for a workable, harmonised, and predictable European Health Data Space for rare paediatric cancers, built on federated initiatives such as UNCAN.eu and outcomes-oriented efforts like UNICA4EU, so researchers and innovators can rely on high-quality datasets and use clinically validated AI for discovery, diagnosis and decision support. At the same time, we urge them to enable hospitals to run early pilots and adopt clinically valuable tools, thus sending clear, credible demand signals that make child-focused innovation possible. Over the last 30 years, the understanding of childhood cancers biology has significantly increased due to translational research and complete genome sequencing techniques. Specific potential therapeutic targets are now identified, and incentivising the development of therapies is needed to stimulate market interest. We ask that the Biotech framework explicitly recognises the need to stimulate development of medicines for childhood cancers. Through establishing clear incentives, such as a first-in-child incentive, bespoke therapies, including CAR-T and other advanced modalities, would have paths from lab to market. Enabling support for the wider infrastructure needed in the development of paediatric oncology therapies, including practical support for academic research centres, start-ups and SMEs to access clinical networks, trial-ready sites and hospital clinical trial capabilities is essential. With a clearer and simplified regulatory framework, specific incentives, hospital-led adoption pathways, strong networks and trustworthy data, Europe can increase publicprivate partnerships, grow the number of start-ups focusing on life threatening paediatric rare diseases, and bring innovative therapies, diagnostics and digital tools to young patients faster and more fairly across all Member States.
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Meeting with Ekaterina Zaharieva (Commissioner) and

19 Sept 2025 · Childhood cancer

Response to Critical Medicines Act

4 Jul 2025

The European Society for Paediatric Oncology (SIOPE) welcomes the EUs ongoing efforts of complementing the measures present in the EU Pharmaceutical legislation with the Critical Medicines Act (CMA) to address critical medicine shortages impacting childhood cancer patients. Each year in Europe, more than 35,000 children and young people are diagnosed with cancer and more than 6,000 young patients lose their lives to this disease. There are an estimated 500,000 childhood cancer survivors in Europe today, with numbers growing substantially each year; at least two-thirds of them experience long-term adverse side-effects into adulthood. There are still persistent inequalities in access to treatment, care and research across Europe, with an estimated 20% gap in childhood cancer survival rates between Western and Eastern Europe. Since the implementation of the EU Paediatric Regulation, only 16 anti-cancer medicines have been authorised for a specific paediatric cancer indication, in contrast to over 150 for adult cancers. We strongly support the provisions within the CMA aimed at improving medicine availability and accessibility. We welcome the European Commissions commitment to strengthening incentives for swifter launches and earlier access to both novel and essential anticancer medicines. We particularly support the dedicated provisions on medicine shortages and the establishment of the Union List of Critical Medicinal Products. However, we urge the European Commission to explicitly include paediatric oncologists in the consultation on and update of such a list. We also emphasize that for the CMA to effectively address the needs of childhood cancer patients, there must be explicit clarity regarding the definition and criteria used to establish "medicines of common interest." Currently, only medicines that have undergone a Joint Clinical Assessment (JCA) under the Health Technology Assessment (HTA) Regulation qualify fully for key procurement measures, such as Commission-led and joint procurement. This narrow eligibility criterion will cut off all currently available and used essential childhood cancer medicines from EU-level procurement since they will not have been assessed through the JCA process, which only started in January 2025. A striking example is blinatumomab, authorized in August 2018, which has become a vital treatment for relapsed B-cell acute lymphoblastic leukaemia (ALL) in children. Significant inequalities already exist in access to such medicines, of which blinatumomab is an exemplar, and these disparities will persist and likely worsen under the current proposal. Restricting the inclusion of such life-saving treatments from CMA procurement mechanisms would represent a devastating setback for children with cancer, particularly in Member States with limited market attractiveness and ongoing shortages. To conclude, we urge the European Commission to take three essential actions to ensure that the Critical Medicines Act truly delivers for children and adolescents with cancer: providing a clear definition of medicines of common interest that explicitly includes paediatric oncology medicines; revising the current restrictive criterion tying procurement tools to the JCA process as it excludes the majority of essential childhood cancer medicines; and ensuring the formal involvement of paediatric oncology healthcare professionals in the definition and continuous update of the Critical Medicines List. Only with these adjustments can the EU secure equitable, timely, and sustainable access to life-saving medicines for all children and adolescents with cancer across its Member States.
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Meeting with Adam Jarubas (Member of the European Parliament, Committee chair)

30 Jun 2025 · Tackling Childhood Cancer: Cross Border Healthcare & Research

Meeting with Maya Matthews (Head of Unit Health and Food Safety)

20 Jun 2025 · Discussion on the identification of clinical experts to participate in joint scientific consultations and joint clinical assessments under the EU Regulation on health technology assessment.

European Society for Paediatric Oncology demands childhood cancer focus

6 Jun 2025
Topic — A new EU framework designed to accelerate biotechnology development from laboratories to markets.
Message — The organization requests targeted financial incentives and simplified regulatory pathways for academic developers. They advocate for harmonized EU quality standards to support local hospital-based manufacturing.12
Why — These changes would help academic institutions overcome financial barriers to develop authorized therapies.3
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Meeting with Olivér Várhelyi (Commissioner) and

28 May 2025 · Childhood cancer

Paediatric oncology society urges EU to fund cross-border trials

15 Apr 2025
Topic — The European Commission is developing a new multi-disciplinary strategy for life sciences.
Message — SIOPE requests reimbursement for cross-border clinical trials to improve patient access. They call for better regulatory support for academic-led research and drug innovation. The group also advocates for harmonised data rules and interoperable digital systems.12
Why — This would secure funding and regulatory recognition for non-profit research and development.3
Impact — Pharmaceutical companies may face stricter requirements if the EU limits regulatory waivers.4
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Meeting with Oliver Schenk (Member of the European Parliament)

26 Mar 2025 · Childhood Cancer

Paediatric oncology group urges EU to protect essential medicines

27 Feb 2025
Topic — This initiative addresses supply chain vulnerabilities to strengthen the supply of critical medicines.
Message — The organization requests the inclusion of essential paediatric oncology treatments in the Critical Medicines List. They also want paediatric cancer experts consulted during legislative updates.12
Why — This would safeguard the availability of off-patent medicines and reduce clinical risks.34
Impact — Children and adolescents currently face health risks due to persistent medicine shortages.56
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Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament) and European Cancer Organisation and EUROPEAN ORGANISATION FOR RARE DISEASES

15 Jan 2025 · Intergroup on cancer and rare diseases

Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament)

31 Oct 2024 · Introductory meeting

Response to Health technology assessment – Joint scientific consultations on medicinal products for human use

21 Oct 2024

The European Society for Paediatric Oncology (SIOP Europe or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. With more than 2,500 members across 35 European countries, SIOPE is leading the way to ensure the best possible care and outcomes for all children and adolescents with cancer in Europe. SIOPE, a member of the HTA stakeholder group, have been following developments under the HTA regulation and welcome the publication of the draft implementing act for the joint scientific consultations (JSC) of medicinal products. We remain hopeful that the HTA regulation will improve the evaluation of paediatric oncology drugs, including ATMPs. Our community (academia and clinical experts) is ready to work with the EU Commission, EMA, HTA/Payers and Industry to facilitate robust, transparent and timely scientific consultations which contribute to the development of innovative life-saving treatments. The following points are of particular importance in the implementing act: Streamlining of HTA & EMA Processes: The efforts made to streamline timelines, processes and outcomes of the HTA JSCs and EMA early scientific advice procedures, as per recital 7,8 & 12, is a positive aspect. Such efforts are important in supporting the generation of sound and supportive early guidance that drives the development of innovative life-saving treatments. Identification of experts: SIOPE encourages the early identification of experts, as per recital 9. Professional societies such as SIOPE, will play a major role in contribution of experts in the implementation of the HTA regulation. It is vital to have early information on applications, including therapeutic indications, in order identify suitable experts in a timely manner. In terms of the sources for gathering clinical experts, SIOPE is continuing to engage with the HTA Stakeholder Network and is building awareness of the HTA Regulation amongst our members. To strengthen the representation of expertise, we welcome the opportunity for general input in JSC on disease and therapeutic area from patient organisations, healthcare professional organisations or clinical and learned societies from the HTA Stakeholder Network. Timeline of JSC: More clarity on the timeline of JSCs is important to understand, particularly timelines for input of clinical experts. The timeframe in which individual experts will be expected to respond to requests for input, as per Article 10, would be valuable information and will help organisations in preparing their expert members as to the expected workloads and processes. Capacity building amongst experts: Due to the role expected for individual experts, further support for training and capacity building would enable organisation such as SIOPE to adequately prepare our community. EU funded projects such as EUCAPA and ALADDIN have been opportunities for clinical experts to learn more about HTA evaluations and their future role. Engagement of JSCs and Paediatric Investigation Plans (PIPs): In terms of the specific needs of paediatric medicines, allowing for early JSC during stepwise Paediatric Investigation Plan (PIP) development is a major opportunity which should be considered in an area of high unmet paediatric needs, i.e. paediatric rare diseases such childhood cancers. As per the EU regulation 1901/2006, PIPs ensure timely advice, agreement and support for paediatric cancer medicine development by medicines developers. The new EU revised pharmaceutical package aims to implement stepwise PIP, currently being piloted by EMA, in order to streamline the development of paediatric medicines. JSC interaction with stepwise PIPs offers another chance to drive innovation for high-need populations by defining jointly what data and evidence is required for EMA evaluation and Health Technology assessments.
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Meeting with Kathleen Funchion (Member of the European Parliament)

1 Oct 2024 · Introductory Meeting

Meeting with Tilly Metz (Member of the European Parliament)

13 Sept 2024 · Cancer

Response to Health technology assessment – Cooperation with the European Medicines Agency

24 Jul 2024

The European Society for Paediatric Oncology (SIOP Europe or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. With more than 2,500 members across 35 European countries, SIOPE is leading the way to ensure the best possible care and outcomes for all children and adolescents with cancer in Europe. The implementing act on EMA cooperation and exchange information in the context of the HTA regulation details important areas of collaboration. Involvement of experts in EMA central authorisation procedure and Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs): Foreseeing the involvement of clinical experts in EMA processes and HTA joint work for the same health technology is most welcome. Cooperation between EMA and the HTA secretariat should encourage transparency and allow cooperation with clinical experts in both processes. Managing the inclusion and transparency is of additional importance for ultra rare diseases, where the pool of clinical expertise may be smaller. Planning and forecasting of the Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs): The notification from the EMA on information on upcoming submissions of initial applications for marketing authorisation, information on variations of existing marketing authorisations for a new indication and estimated number of scientific advice procedures is an important element in the sound implementation of the joint HTA work. The HTA secretariat should use this information effectively to facilitate the cooperation of clinical excerpts in the JSCs and JCAs as early as possible. In the case of JSCs, cooperation can facilitate the early discussions of data requirements to satisfy both EMA and HTA requirements. In the case of JCAs, the involvement of clinical experts in the scoping phase and generation of the PICOs is key to ensure the JCA reports can include as much relevant evidence as feasible. Information exchange related to the identification of individual experts for Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs): The sharing of information potentially suitable experts from the EMA to the HTA secretariat is welcome and will be another important avenue for the identification of clinical experts. SIOPE as part of the HTA Stakeholder Network will be implementing a process to identify experts from the paediatric oncology community, proactively ensuring that experts from all disease areas can be engaged when needed.
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Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament)

18 Jul 2024 · Introductory Meeting

Response to Health technology assessment – Procedural rules for the assessment and management of conflicts of interest in joint wo

26 Jun 2024

The European Society for Paediatric Oncology (SIOP Europe or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. With more than 2,500 members across 35 European countries, SIOPE is leading the way to ensure the best possible care and outcomes for all children and adolescents with cancer in Europe. SIOPE have been following developments under the HTA regulation and welcome the publication of the draft implementing act for assessing and managing conflicts of interest. Ahead of the beginning of the joint work under the HTA regulation, it is important for our Community to understand how individual experts will be assessed for potential conflicts of interest ahead of their inclusion in the joint work. SIOPE is committed to support in the process of identifying clinical experts, as well as supporting our parent and patient community through our partnership with Childhood Cancer International Europe in this endeavour. To ensure good standing of the joint work to begin in 2025, a robust and effective conflict of interest management process is needed. SIOPE welcomes the balance struck by the European Commission in setting out the considerations and mitigation measures in the case that conflicts of interest arise. It is important that conflict of interest are managed in a clear and transparent manner to ensure inclusion of best available clinical expertise. To support inclusion of our Communitys expertise and prevent unintended delays in the joint work to the it is important to consider the following: As a rare disease community, it is vital that mitigation measures when potential Conflicts of Interests may arise are flexible enough to ensure clinical expertise is included in the evaluation of health technology products. This is likely to occur with ultra rare diseases. To ensure that clinical experts are supported in their duty to provide declarations of interest, extended timeframes for when additional information is sought would be welcome. Where work is being carried out in parallel with other agencies, e.g European Medicines Agency, align the required COI processes. As the inclusion of clinical experts is an important element to ensure robust outcomes of the joint work, SIOPE are preparing for our role in identifying experts. This will also include training and awareness building on HTA and the joint work our members will be taking part in. SIOPEs role in this regard should contribute greatly to ensure that sufficient paediatric oncology expertise is available for the joint work.
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SIOPE Urges Faster EU Clinical Assessments for Pediatric Cancer Medicines

28 Mar 2024
Topic — Implementing rules for joint clinical assessments of medicinal products across the EU.
Message — SIOPE requests involving clinical and patient experts early to accelerate children's access to treatments. They seek guidance on using real-world evidence and single-arm trials for rare pediatric cancers.12
Why — Standardized assessments would help pediatric oncologists secure faster reimbursement for new life-saving therapies.3
Impact — Fragmented national assessment systems currently delay vital treatment access for children and their families.4
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Meeting with Tilly Metz (Member of the European Parliament, Shadow rapporteur)

13 Feb 2024 · Pharmaceutical legislation

SIOPE Urges GDPR Harmonisation to Protect Paediatric Cancer Research

8 Feb 2024
Topic — Review of the General Data Protection Regulation six years after its entry into application.
Message — The organization recommends harmonised interpretation of GDPR rules to remove barriers to cross-border research. They request one-time broad consent for data reuse and treating pseudonymised data as anonymised. They also advocate for removing explicit consent requirements for population-based cancer registries.1234
Why — Streamlined rules would facilitate international data exchange necessary for scientific breakthroughs in rare diseases.56
Impact — Patients seeking individual control over every research use of their data may lose oversight.78
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Response to Interim evaluation of the EU4Health Programme 2021-2027

9 Jan 2024

The European Society for Paediatric Oncology (SIOP Europe, or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. The rarity and specificity of childhood cancers combined with their high overall burden in Europe require funding opportunities specifically addressing this disease area. SIOPE welcomes: Increased funding of EU4Health relative to its predecessor programmes Consistent presence of the cancer header/topic in each annual work programme (WP) Calls entirely focusing on childhood, adolescent and young adult cancers (CAYACs), particularly in WP 2024 where a substantial amount is foreseen across several initiatives specifically targeting this area Specification of childhood cancer activities in several overarching cancer calls and the disaggregation of expected results by age, e.g. Mental Health and cancer (WP 2023), Continuity in programme planning e.g. EU Network of Youth Cancer Survivors (WP 2024) building on EU-CAYAS-NET (WP 2021) Funding for sustainability of European Reference Networks, in particular ERN PaedCan Strong link with Europes Beating Cancer Plan (EBCP) and synergies with Horizon Europe, in particular the Cancer Mission To further advance the vision of the EBCP and its Childhood Cancer Spotlight as well as the Cancer Missions priority on childhood cancer, SIOPE recommends: Consistently ring-fencing/reserving funding to childhood cancer projects in all relevant cancer calls, and/or pre-specifying the inclusion of activities and indicators linked to childhood cancer A consistent sub-topic on childhood cancer under the transversal cancer header would strengthen momentum towards addressing the specificities of childhood cancer and tackling inequalities. In WP 2023, joint actions on comprehensive cancer infrastructures are allocated EUR 130,000,000 EUR. However, the childhood cancer sector does not directly fit into this network concept. SIOPE has been running an own initiative project to describe the organization of paediatric oncology (OCEAN). There is a need to specifically strengthen, support and fund the paediatric oncology infrastructure delivering care and research for children and adolescents in Europe. SIOPE is also engaging with the EU and the OECD regarding childhood cancer data sources and classification in relation to ECIS and ECIR. There is a need to support comprehensive collection of up-to-date epidemiological data on childhood cancers across Europe, incl. quality improvement, and to make Paediatric Cancer Country Profiles publicly available. Specific calls to strengthen the paediatric oncology sector across current structures and information systems would add considerable value and tie in with already existing initiatives of specialist scientific societies. Overall, we are encouraged by the trend towards increased funding opportunities and allocations to childhood cancer specifically, in particular in WP 2024, as well as by the consideration given to project continuity. The major persisting challenge and specificity of childhood cancers consistently require dedicated and appropriately sized allocations, and calls of a general cancer nature may disadvantage projects in this highly specialised field. Results can be live-saving for thousands of children in Europe and beyond (e.g. EU-US cooperation, global health) and demonstrate how the EU champions childhood cancer and pioneers cross-border cooperation in this field for a strong EU Health Union. Through the years, paediatric cancer has received instrumental support from EU programmes. We are inspired by the opportunity to further build on the achievements with EU4Health and advance towards a brighter future for children and adolescents with cancer.
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Paediatric cancer doctors seek 12-year exclusivity for child-specific medicines

8 Nov 2023
Topic — EU consultation on revising pharmaceutical legislation to ensure affordable access and innovation.
Message — They seek 12-year market exclusivity for orphan drugs addressing high unmet needs in children. They request that reducing toxicity be recognized as meeting an unmet medical need. They want professional organizations systematically included in regulatory consultation processes.123
Why — This would secure their formal role in regulation and incentivize development of safer childhood treatments.45
Impact — National health budgets may face higher costs due to extended market exclusivity periods for medicines.6
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Meeting with Deirdre Clune (Member of the European Parliament)

10 Oct 2023 · Cancer

Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur)

18 Aug 2023 · Directive on Medicinal products for human use

Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur)

9 Jun 2023 · Directive on Medicinal products for human use

Response to Ex-post evaluation of Horizon 2020

29 Jul 2022

The European Society for Paediatric Oncology (SIOP Europe or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. PROGRAMME DESIGN Paediatric oncology is a specific rare disease field with large unmet needs. We are aware of 9 paediatric oncology projects funded by H2020. In relation to the SIOPE Strategic Plan objectives, these include: Equal access (TREL), Precision medicine (PRIMAGE), Innovation and Tumour biology (ITCC-P4), Survivorship (PanCareFollowUp, PanCareSurPass). Only one project addressing innovation in a specific paediatric malignancy – hepatoblastoma – was funded (CHILTERN). Projects on other high-need childhood cancers, such as neuroblastoma, diffuse intrinsic pontine glioma and sarcomas, were not selected. The need for research on human and social sciences in the field of childhood cancers was not sufficiently addressed in H2020. Most calls were organised under broad themes, which may have limited funding opportunities in high-need specialised fields. Thus, the paediatric oncology sector would have benefitted from more calls fully dedicated to this disease area. IMPLEMENTATION Project size is overall appropriate to create multidisciplinary teams with strong synergies. Standardised processes help beneficiaries deliver, but challenges arise for smaller / inexperienced organisations. Written terms of reference for the interaction between the EU project officer and the coordinating partner could help streamline implementation. The EU online portal for beneficiaries holds important potential but may duplicate efforts, e.g. where email is also used. External factors e.g. COVID-19 created complexities such as meeting delays and lower in-person participation in favour of virtual engagement, which may be considered less effective. RESULTS, IMPACT, SUSTAINABILITY Networks and data repositories established through H2020 projects should be pursued by investing in their sustainability. This requires appropriate-sized funding and subsequent calls to build on the achieved results, incl. through professional educational opportunities. Clear programme guidance on secure data sharing after project end would be of value. DISSEMINATION & EXPLOITATION OF RESULTS SIOPE ensured efficient dissemination of selected paediatric oncology projects to the broad European clinical, translational and basic paediatric oncology research community. This was enhanced by partnership with CCI Europe to effectively reach patients, parents, and survivors. Activities adapted to COVID-19 challenges by focusing more on online branding and dissemination rather than in-person events. Moving beyond purely quantitative reporting – i.e. number of dissemination activities, to integrate qualitative aspects would help evaluate project impact. Dissemination and exploitation should go hand in hand. Follow-up funding schemes, focused on accelerating results to market, are worth exploring. It should be possible to claim open access costs when papers are published after project end to relieve the financial burden on partner institutions. COSTS & BENEFITS FOR APPLICANTS Although simplification of some H2020 processes has been welcome, partners still report complexities in proposal preparation and project delivery. Further adaptations to administrative requirements are needed. Project budgets should reflect rising costs. Some flexibility in budget allocation, incl. between project work packages, can help maximise impact and sustainability. RELEVANCE, COHERENCE, EU VALUE ADDED, EFFICIENCY, EFFECTIVENESS Research projects in childhood cancers as a rare disease area have a strong EU value added. Results can be relevant for other illnesses (e.g. C4C project). Some stakeholders report overlapping calls concerning oncology, with potential for fragmentation. This places increased importance on ongoing dialogue with the community across the programme lifecycle.
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Response to Interim evaluation of Horizon Europe

29 Jul 2022

The European Society for Paediatric Oncology (SIOP Europe, or SIOPE) is the only pan-European organisation representing all professionals working in the field of childhood cancers. PROGRAMME DESIGN, IMPLEMENTATION AND FIRST RESULTS The rarity and specificity of childhood cancers combined with their high overall burden in Europe require funding opportunities specifically addressing this disease area. In this light, SIOPE welcomes ring-fencing funding for projects fully addressing childhood, adolescent and young adult cancer. This approach is implemented in two calls of the Horizon Europe Work Programme (HE WP) 2021-2022 under the Cancer Mission, namely: "Pragmatic clinical trials to optimise treatments for patients with refractory cancers (HORIZON-MISS-2022-CANCER-01-03) and "Improving and upscaling primary prevention of cancer through implementation research (HORIZON-MISS-2022-CANCER-01-01). Although favourable to fostering funding opportunities in this manner, SIOPE would like to point out that the topic of primary prevention (HORIZON-MISS-2022-CANCER-01-01) is of limited relevance for tackling childhood cancers. Indeed, no external carcinogenic factors, e.g healthy lifestyles, have been identified that would help prevent cancer in children. The cause of up to 90% of paediatric malignancies is unknown, while 10% to 15% are driven by genetic predisposition. Research needs to be intensified on the causes of childhood cancers. Other calls under the Cancer Mission and the overall Health pillar of HE retain a general thematic nature that may disadvantage projects in specialised rare disease areas, such as paediatric oncology. However, the emphasis on synergies between the Cancer Mission and Europe’s Beating Cancer Plan (EBCP) in the HE WP is encouraging. As the EBCP allocates a spotlight section to childhood cancer, we trust that projects funded under the Cancer Mission calls will likewise assign appropriate funding and work streams to childhood cancer. One example is the UNCAN.eu project. Tasks specific to paediatric cancers emerged during the project set-up stage and will be driven by SIOPE. UNCAN is set to pave the way for stronger European-wide research networks in paediatric oncology. It will also seek to reduce inequalities in paediatric cancer research capacity across Europe, address the needs of childhood cancer survivors, and explore the application of Big Data and Artificial Intelligence across the paediatric cancer patient pathway. Another important need in paediatric cancer research is human and social sciences, incl. to explore socio-economic factors contributing to inequalities in access to standard care and innovation across Europe. In conclusion, SIOPE favours the approach to award a specified number of projects to applications that specifically address childhood cancer and remains committed to carve opportunities for childhood cancer under broader health and cancer topics. COSTS AND BENEFITS FOR APPLICANTS Project applicants in the SIOPE community express the need for simplified administrative requirements to facilitate the call application process. Continued specific funding opportunities are needed for childhood cancer. Calls with overlapping topics should be avoided to streamline applications from the paediatric cancer community. For this reason, continued communication with relevant stakeholders remains important throughout the programme cycle. RELEVANCE, COHERENCE, EFFICIENCY, EFFECTIVENESS There is a strong EU value added for research projects in paediatric cancer as a rare disease area, and results can be relevant for other illnesses. Through the years, paediatric cancer research received important support from EU Framework Programmes for Research and Innovation. The community is inspired by the opportunity to build on prior achievements, ensure continuity, and advance strong and sustainable cross-border research networks in paediatric oncology with the support of Horizon Europe.
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Response to A New European Innovation Agenda

10 May 2022

Lack of access to innovation is a principal problem behind the persisting burden of childhood cancers in Europe. The European Innovation Agenda focuses on several key aspects of relevance to childhood cancer: issues in society, pro-innovation regulation, synergistic approach between programmes, and addressing inequalities. SOCIETAL ISSUE Paediatric cancers are individually rare but together represent the leading cause of children’s death by disease over the age of one in Europe: over 6,000 young lives lost are lost each year. Most of the estimated 500,000 childhood cancer survivors in Europe are affected by adverse late complications, which limit their socio-economic activity and participation. PRO-INNOVATION REGULATION Due to their rarity, childhood cancers have seen limited market-driven innovation. Progress has relied on academic-driven research using older medicines originally meant for adults. In the absence of sufficient and sustainable funding and disruptive innovation, several hard-to-treat malignancies still drive high childhood cancer mortality today. In survivors, the use of older medicines is consistently associated with acute and long-term adverse side effects. To address these barriers, the European paediatric cancer community calls for a targeted revision of the EU Paediatric Regulation to enable: • Paediatric medicine development driven by mechanism of action, disease biology, and patient needs • Reduced delays in starting the development of paediatric medicines, incl. by means of better-tailored incentives • Repositioning of medicines failing in adults towards the treatment of paediatric diseases when there is a scientific and preclinical rationale • Effective incentives for ‘first-in-child’ development and marketing authorisation of medicines against specific paediatric biological targets • Alignment in the global regulatory environment for paediatric medicine development, such as in relation to the RACE for Children Act (US) • Comprehensive public investment into research & development by academia that can scale up existing collaborative platforms by ensuring sustainability and enabling to capitalise on Big Data and Artificial Intelligence. Furthermore, there is a need for: • Better incentives and investments into specific new medicine development for children and adolescents with cancer that will encourage and attract small companies and biotech, thus creating economic activity and innovation. • Building a new innovation ecosystem including both academic researchers/institutions and small companies and biotech. • Bridging between academia and private research. Disruptive innovation will emerge from cross-fertilisation of the academic and industry worlds, especially in the field of rare diseases, incl. childhood cancers, supported and funded by significant public investments. SYNERGISTIC APPROACH Important political commitments were made to childhood cancer in the EU, incl. the anticipated revision of the Paediatric Regulation as part of the Pharmaceutical Strategy for Europe and Europe’s Beating Cancer Plan as well as the potential for investment into childhood cancer research in the Horizon Europe Cancer Mission. Turning these commitments into life-saving gains requires their rapid, smart and coordinated implementation. The European childhood cancer community calls for the creation of an inclusive EU Childhood Cancer Advisory Board: a multi-stakeholder group that would support cross-talk among EU initiatives of relevance to childhood cancer, foster a comprehensive and inclusive approach, avoid the risk of fragmentation, and accelerate progress. ADDRESSING INEQUALITIES Inequalities in childhood cancer survival amount to an estimated 20% across Europe. Up to 40% of all paediatric cancer patients are treated within clinical studies. Some member states may lack the structural capacity to take part in clinical research platforms. Comprehensive investment is therefore needed in this area.
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Response to Proposal for a Council Recommendation on long-term care

29 Mar 2022

The SIOP Europe response to the call for evidence focuses on the needs of children with cancer, their carers, and childhood cancer survivors. Children with cancer and childhood cancer survivors are specifically mentioned in the EU Strategy on the Rights of the Child, which we trust represents important synergies with the future European Care Strategy. We welcome the focus of the announced European Care Strategy on “affordable care services for children and people who need long-term care” and “addressing care in a comprehensive way, from childcare to long-term care in old age.” Paediatric cancers are all rare yet together represent a leading health burden in Europe, with 35,000 new cases diagnosed and 6,000 young lives lost each year. Most of the estimated 500,000 childhood cancer survivors in Europe experience long-term complications, and many of them lack appropriate support structures and face discrimination. The rarity of individual paediatric cancer types and their leading collective health burden across Europe make coordinated EU-level approaches crucial, with particular focus on the following areas. Access to the Best Possible Care for Children with Cancer and Survivors: There are substantial inequalities in access to the best available standard care and expertise across Europe causing up to a 20% difference in children’s survival. The European Reference Network on Paediatric Cancer (ERN PaedCan) aims to reduce these inequalities by linking specialist centres across Europe. It can also optimise long-term follow-up (LTFU) care for an increasingly mobile population of (young) adult childhood cancer survivors who move around Europe for work and education. The sustainability of the ERN PaedCan should be supported by specific EU funding allocations and their integration into national health systems should be fostered. In addition, there is a need to fully realize the patients’ rights to cross-border care, including by providing clarity on the rules governing reimbursement of treatment and follow-up care received outside of one’s home country. Right to Parental Presence and Support during the Child’s Illness: The right of the hospitalised child to constant and continuous parental involvement is embedded in the European Standards of Care for Children with Cancer. Socio-economic provisions that reflect the needs of parents of children with cancer and other life-threatening diseases are necessary. The EU is well-positioned to foster targeted provisions on employment and income security for these families in highly demanding circumstances. These measures should be defined, implemented, and evaluated in cooperation with parents and their advocacy groups as well as healthcare professional associations. Survivors - Long-Term Follow-Up Care and Transition from Childhood to Adulthood: A substantial proportion of childhood cancer survivors experience worse health-related quality of life (QoL) compared to the general population. They may be “lost” in the healthcare system once they become adults if transition from active treatment to LTFU and from pediatric to adult care is not organised. Healthcare providers in adult settings need to be informed about the need for LTFU and the importance of supporting survivors to take ownership of their care. LTFU guidelines for specific childhood cancers exist. Their utilisation across Europe, along with the implementation of portable personalised survivorship care plans (e.g. Survivorship Passport), provide a clear pathway for high-quality survivorship care. Non-Discrimination of Survivors: Initiatives like the Right to be Forgotten (implemented in 5 EU Member States to date) can counter discrimination faced by childhood cancer survivors and should be explored for further implementation across Europe. Conclusion: Coordinated action is needed across Europe to ensure that high-quality care is in place for children with cancer, their families and carers, and childhood cancer survivors.
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Meeting with Irena Joveva (Member of the European Parliament)

13 Sept 2021 · Paediatric oncology in the BECA report

Meeting with Stella Kyriakides (Commissioner) and CCI Europe and PanCare

18 May 2021 · Exchange of views on the implementation of Europe’s Beating Cancer Plan in paediatric oncology

Response to Evaluation and revision of the general pharmaceutical legislation

27 Apr 2021

SIOPE welcome evaluation and revision of General Pharmaceutical Legislation hoping that it can deliver urgently needed improvements for over 6000 youngest citizens dying of cancer each year in Europe by addressing: 1.Revise Legislation to Unlock Innovation Concluded by Paediatric and Orphan Regulations (OPR) evaluation “neither regulation has proven effective in boosting development of innovative medicines for children with rare diseases, such as paediatric cancers”. OPR revision can address unmet medical needs (UMN) of paediatric cancers & boost innovation. Identifying UMN will be crucial after OPR revision. Fixed set of criteria established in the legislation would be detrimental and counterproductive. UMN definition should be dynamic and defined in multi-stakeholder setting. The legislation should provide opportunity to identify and evaluate needs & prioritise products for development. It is crucial to incentivise and accelerate science-driven development of innovative drugs for PAEDIATRIC CANCERS and to support academic research and SME involvement. A more tailored system of incentives to increase access and innovation in PAEDIATRIC CANCERS will promote private engagement where the low market does not attract investment. To boost innovation in PAEDIATRIC CANCERS proposed measures include (but are not limited to): -Reduce delays in starting drug development based on mechanism of action -Facilitate repositioning of unlicensed products failing in adults -Ensure child specific & First-In-Child innovation -Introduce tailored and flexible incentives awarded incrementally -Align regulations with other international jurisdictions on global level -Allocate funding to academia and SMEs For more info. read our 6 Key Recommendations in the attachment. In addition, AMR threatens modern cancer care and may have devastating consequences. Hence, PAEDIATRIC CANCERS should be considered across all initiatives on AMR 2.Leverage Digitalisation to Simplify Procedures In PAEDIATRIC CANCERS community a wealth of untapped data is not accessed/fully utilised for patients’ benefit. Multiple datasets need to be shared, integrated & analysed as one. EU initiatives are instrumental to gather data where machine and deep learning methods could be applied in novel drug development & repurposing. We need to scope potential applications of AI in PAEDIATRIC CANCERS. Potential data-driven technologies could include (but are not limited to): - Point of care AI applications based on real-world data - Innovation in drug development (in silico etc) - The use of real-world data and 'smart trials' - AI approaches to improve understanding of PAEDIATRIC CANCERS - Patient empowerment via better personalised care, incl. use of digital medicine The goal is for multi-dimensional data to meet FAIR principles to improve treatments & outcomes for all patients & survivors. 3.Prevent Shortages & Assure Equal Access to Improve Cure Rate The findings of Joint Action on Rare Cancers (JARC) paediatric survey demonstrate need for coordinated European initiatives to ensure access to essential medicines (EM) for treatment and care of children and adolescents with cancer. Focus areas are shortages, financial accessibility to new medicines, child-friendly formulations and equal access to pain control. It takes different length of time for same medicine to be marketed in all EU countries. A more harmonised HTA assessment across EU would enable faster and fairer access to innovative medicines, which can be critical for PAEDIATRIC CANCERS. SIOPE EM Project aims to: 1) establish a list of EM that should be available at all times across whole Europe 2) formulate recommendations for harmonisation of HTA evaluations to assure accessibility to new medicines in time Our recommendations to improve access to medicines: - JARC Report 9.1: https://bit.ly/3aIVoJE - AoO Paper, Access to Essential Anticancer Medicines for Children and Adolescents in Europe: https://bit.ly/3tVtVfu
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Response to Evaluation of patient rights in cross-border healthcare

11 Feb 2021

SIOP Europe welcomes the European Commission’s initiative to evaluate the functioning of the EU Cross-Border Healthcare Directive (CBHD). Whereas the Directive enabled developments of major value for the European paediatric cancer sector, there remain important bottlenecks and areas of improvement for the community to fully benefit. Paediatric cancers are all individually rare but together represent the leading cause of children’s death by disease and an important contributor to morbidity in survivors in Europe. Disparities in access to the best possible diagnostics, standard treatment, innovation, and research lead to an estimated 20% difference in survival across Europe. Cross-border healthcare collaboration is required to share expertise on rare and complex cases and ensure the best possible standard care to all children and adolescents with cancer no matter where in the EU they live. Over the past 50 years, paediatric cancer treatment centres and academia across Europe cooperated actively with each other, leading to important scientific and clinical advances. Today these cross-border networks are united by the SIOP-E governance. Further progress requires more enabling cross-border rules and sustainable funding. Originating from the CBHD, the European Reference on Paediatric Cancers (ERN PaedCan) links specialist healthcare providers across Europe to reduce inequalities in children’s survival. The ERN PaedCan particularly emphasises expertise-sharing in virtual tumour boards as opposed to patient travel to minimize stress on the affected families. The ERN PaedCan also fulfils other important functions, e.g. twinning to raise standards of care across Europe and working with SIOP-E to produce up-to-date clinical practice guidelines across paediatric cancer entities. The ERN PaedCan has great potential but faces challenges such as in relation to compensation of virtual consultations, resourcing twinning programmes, integration into national systems, and effective reimbursement of patient travel when this is required. Innovative therapies delivered in early clinical trials can be lifesaving for children with relapsed or refractory non-curable malignancies. The Innovative Therapies for Children Cancer (ITCC) network of excellence unites centres running such trials across Europe. Many ITCC centres are also members of ERN PaedCan. Yet access to innovative therapies in clinical trials is not currently considered as part of the cross-border healthcare reimbursement programmes, which is a major concern. In this framework, the recommendations from the European paediatric cancers professionals on improving the policy environment for cross-border healthcare are summarised below. • The sustainability of the ERN PaedCan and its ability to perform all functions as per CBHD delegated acts should be ensured through non-competitive funding calls. • Better integration and formal acknowledgement of the ERN PaedCan by national healthcare systems should be encouraged. • Solutions are urgently needed to ensure seamless access to information on cross-border healthcare reimbursement and its effective application for paediatric patients. • Current cross-border healthcare reimbursement rules should foresee the needs of the child to be accompanied by family members or carers. • Current cross-border healthcare reimbursement rules should be adapted to the needs of an eHealth based virtual cross-border care, such as by modifying the current mechanisms to compensate also for virtual care time provided by experts through teleconsultations. • Cross-border healthcare reimbursement should cover participation in early phase clinical trials for children with relapsed or refractory non-curable malignancies. • Official recognition of the ERN PaedCan – SIOP-E guidelines by national health authorities should become the backbone of decision-making agreed upon in the setting of cross-border healthcare.
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Response to A European Health Data Space

3 Feb 2021

SIOP Europe welcomes the European Health Data Space Roadmap with its focus on eradicating inequalities, facilitating research, and empowering patients. The roadmap objective 1: ‘Legal and governance framework to cover the access to and exchange of health data’ is particularly relevant for addressing the challenges and opportunities in the paediatric sector. Secure data sharing is vital to facilitate progress in childhood cancer: a collection of rare diseases with distinct age-related features that also represent a major shared burden in Europe. Cross-border collaboration is required to overcome small sample sizes in research and to share expertise on rare and complex cases to ensure the best possible treatment and care. Today, childhood cancers are still the leading cause of children’s death by disease in Europe, an important contributor to morbidity in survivors, and marked by inequalities in survival rates and care across the EU. An enabling legal framework for health data sharing can play a fundamental role for childhood cancer patients and survivors including in relation to the below initiatives: - The European Reference Network on Paediatric Cancers (ERN PaedCan) links specialist healthcare providers across Europe to reduce inequalities in children’s survival as well as in their continued care both during and after treatment. It emphasises expertise-sharing in virtual tumour boards as opposed to patient travel to minimize stress on the affected families. The ERN PaedCan would benefit greatly from increased interoperability and provides an ideal setting to foster application of eHealth solutions in childhood cancers. - The Survivorship Passport model, developed with the EU framework programme support, empowers childhood cancer survivors to have control of their data and to allow access to medical/ research professionals. Its wide application for the growing and increasingly mobile population of childhood cancer survivors in Europe requires interoperability and secure data transfer. - Outcome data collection and long-term health surveillance are needed to adequately monitor the optimal use of therapies in both paediatric and adult cancers. it is critical to develop outcome research, real life data collection on utilised therapies, and make use of big data in health such as by linking long-term observational studies (registries). - The concept of a EUPID - European unique patient identifier (EUPID) should be explored to underpin the full functionality of the European Reference Network model and reflect the interconnectedness of a united Europe. Resourcing considerations underpin the capacity of the European paediatric cancer sector to benefit from the European Health Data Space developments: - Sustainable public funding is instrumental to secure continuity of existing eHealth networks and platforms and enable their full functionality. - The costs involved in accessing paediatric cancer data for research, quality improvement, and provision of better information should not be excessive as most of such activities are funded by voluntary sector donations or patient efforts. - There are major opportunities to translate Artificial Intelligence and big data into clinical practice but sustainable funding of cross-border research is a clear pre-requisite To conclude, the rarity of paediatric cancers combined with their major shared burden place the highest importance on secure health data sharing to address inequalities and advance research. We look forward to being involved in the elaboration of the legislative proposals to ensure that the specific issues of the childhood cancer sector are fully reflected.
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Response to Revision of the EU legislation on medicines for children and rare diseases

6 Jan 2021

SIOPE and CCI-E welcome the proposed revision of EU regulations on medicines for children and rare diseases. All paediatric cancers individually are rare, but jointly are the leading cause of death by disease in children above one year of age in Europe. Most survivors experience long-term side-effects due to older medicines. There are over 60 individual paediatric cancer types, each further sub-divided by stage, biology, and other factors. As concluded by the evaluation of the Paediatric and Orphan regulations (OPR) “neither regulation has proven effective in boosting the development of innovative medicines for children with rare diseases, such as paediatric malignancies”. A concurrent revision of the OPR has the potential to provide solutions to address unmet medical needs (UMN) of children with cancer and greatly enhance innovation. We support the overarching objectives, in particular: - Fostering research and development of medicines devised for adult cancer in the paediatric population and prioritizing products that address UMN - Revising conditional exemptions from the obligation to study new medicines in children e.g. those with a relevant mechanism of action (MOA) - An improved rewards system that would better help patients with UMN and ensure equal access to medicines for children with cancer across Europe - Dedicated funding to academia and SMEs. To accelerate innovation for children with cancer, we are strongly advocating for additional measures to achieve the following goals: - Reduce delays in starting MOA-driven paediatric development of medicinal products - Facilitate repositioning of unlicensed products failing in adults for the treatment of paediatric diseases, when there is a scientific and preclinical rationale. - Incentivise ‘First-in-Child’ development and marketing authorisation for medicines against specific paediatric biological targets - Introduce tailored and flexible incentives awarded incrementally and not only at the end of Supplementary Protection Certificate (SPC) - Align regulations with other international jurisdictions, because drug development for rare paediatric diseases is global. Identifying UMN will be paramount in the implementation of the revised regulations. Defining UMN in paediatric malignancies is complex as in other therapeutic areas. A fixed set of criteria established in the legislation would be detrimental and counterproductive. The definition of UMN should be dynamic and established in a multi-stakeholder setting. The legislation should provide a structure and framework where the needs can be continually identified and evaluated and the products prioritised. MEDICINES FOR CHILDREN: SIOPE & CCI-E favour Option 3 (building on Option 1). We propose to incentivise starting paediatric development early by introducing changes to the timing and nature of rewards. A segmented approach with part delivered on completion of an interim deliverable would be a significant advance, while the remainder would be given on completion of the full PIP, as currently defined. For products addressing UMN, we support a novel reward that would complement or replace the SPC prolongation. To this extent transferable vouchers are particularly attractive to incentivise First-in-Child development and development of medicinal products specific to paediatric diseases. MEDICINES FOR RARE DISEASES: SIOPE & CCI-E favour Options 2 and 3. We fully support the use of incidence to qualify rare, including paediatric, cancers. There are many epidemiological registries that can provide relevant information on cancer incidence. Novel incentives would probably boost the development of products addressing UMN of rare paediatric diseases. CONCLUSION: The European childhood cancer community is eager to contribute further and proposes the attached 6 Recommendations for Paediatric Cancers on access to medicines and innovation. These are also relevant to other paediatric life-threatening rare diseases.
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Response to Delivering for children: an EU strategy on the rights of the child

5 Aug 2020

SIOP Europe welcomes the European Commission’s Strategy on Children’s Rights and the emphasis on mainstreaming this critical aspect in all EU policy areas, including the new initiatives proposed for 2019-2024. We would like to highlight the rights of CHILDREN AND ADOLESCENTS WITH CANCER – an area of large unmet need and persisting inequalities. Paediatric cancers are all rare yet together represent the first cause of children’s death by disease above the age of one in Europe: 6,000 young lives are lost each year. Lack of novel therapies, particularly when compared to the market-driven adult cancer sector, and unequal access to the best possible care across Europe are among the drivers of this high mortality. After the disease, the over 500,000 survivors of childhood cancer in Europe lack appropriate support structures and face discrimination. The affected families are confronted with financial hardship when trying to reconcile their role as carers with gainful activities. Despite this high health and socio-economic burden, childhood cancers tend to be left behind from overarching health and inclusion programmes. - The EU CHILDREN’S RIGHTS STRATEGY should include the urgent need for EU action to uphold the rights of children and adolescents with cancer and survivors to health, parental support, and socio-economic participation. There is also critical need for synergies with EU initiatives in health, research, and social protection, including but not limited to the following: - Europe’s Beating Cancer Plan - Horizon Europe Cancer Mission Area - EU4HEALTH Programme - EU Pharmaceutical Strategy Below are more specific considerations; please refer to enclosed position paper for further details. RIGHT TO HEALTH Member states commitments enshrined in the UN Convention on the Rights of the Child are to diminish child mortality, ensure provision of necessary medical assistance and health care to all children, and to combat disease. It is increasingly recognised that childhood cancer patients require tailored approaches different from adult cancer services (Joint Action on Rare Cancers, 2019; WHO Global Initiative on Childhood Cancer, 2018). Europe’s Beating Cancer Plan, Cancer Research Mission, Pharmaceutical Strategy, and EU4Health are among the initiatives with great potential as they can dedicate funding to childhood cancers - an area of relative market failure - and call on all stakeholders to allocate specific efforts to these underserved patients. More specifically, there is an urgent need to: - Boost targeted innovation for young cancer patients to the level seen in adult cancers: in the past 10 years, only 9 new anti-cancer medicines were approved for a paediatric indication in contrast to over 150 in the adult cancer area. - Redress inequalities in access to the best possible standard care and the resulting difference in childhood cancer survival of up to 20% across Europe. - Foster coordinated support structures and organized transition from paediatric to adult follow up care for childhood cancer survivors. RIGHT TO PARENTAL PRESENCE AND SUPPORT The right of the hospitalised child to “constant and continuous parental involvement” is embedded in the European Standards of Care for Children with Cancer. - EU action can be instrumental in raising awareness and mobilising the necessary action: targeted provisions on employment and income security for families in these highly demanding circumstances. NON-DISCRIMINATION - Initiatives like the Right to Be Forgotten law (eg in France) can counter discrimination faced by childhood cancer survivors. This type of legislation should be explored for implementation across Europe. CONCLUSION Childhood cancer patients, survivors and their families stand to benefit greatly from inclusion in the EU Children's Right Strategy and across synergistic EU policy areas. - PLEASE REFER TO ENCLOSED PAPER FOR DETAILS
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Meeting with Mariya Gabriel (Commissioner)

9 Jun 2020 · EU Paediatric Cancer Research agenda and how the European Paediatric Cancer Community could best partake in and support relevant initiatives

Meeting with Stella Kyriakides (Commissioner)

26 May 2020 · VC meeting to discuss the EU's Cancer Plan and its objectives, cancer care and treatment during the COVID-19 pandemic

Response to Europe’s Beating Cancer Plan

3 Mar 2020

SIOPE welcomes Europe’s Beating Cancer Plan and its links with research, global agenda, and prior EU projects. -Neglected area: PAEDIATRIC CANCERS should not be overlooked: all individually rare, they are jointly the leading cause of death by disease in children older than 1 year in Europe, contribute to up to 60% life-long morbidity in survivors, and are marked by inequalities. As recognised in the Joint Action on Rare Cancers (JARC), PAEDIATRIC CANCERS specificities require tailored approaches beyond simple extrapolation of adult services. -Best practices, evidence: The WHO Global Initiative on Childhood Cancer as well as the JARC Book with dedicated sections on PAEDIATRIC CANCERS and the SIOPE EUROPEAN CANCER PLAN FOR CHILDREN & ADOLESCENTS, both created with EU support, are valuable resources. -Prevention: 10% of PAEDIATRIC CANCERS are currently known to be linked to predisposition syndromes based on somatic genetic mutations. Here, surveillance and genetic counselling for early family guidance are needed. In contrast, 40% of adult cancers are potentially preventable through external exposure modification. More dedicated research on oncogenic drivers, big data integration, and artificial intelligence (AI) are needed to find new prevention avenues for PAEDIATRIC CANCERS. -Early detection, diagnosis, treatment: Professional education and public information may improve early detection of PAEDIATRIC CANCERS. Investment into advanced diagnostics is imperative. There are major inequalities in access to this resource but also to standard treatment, innovation, care, and research, resulting in inferior survival especially in ‘widening’ EU countries. The European Reference Network for PAEDICATRIC CANCERS can address this but requires enabling cross-border governance and sustainable funding. In contrast to the market-driven adult cancer sector with over 150 new medicines in the last decade, only 9 of these were seen for a truly PAEDIATRIC CANCER indication. Most PAEDIATRIC CANCER drugs are older and used off-label. Access to essential medicines and innovation across the PAEDIATRIC CANCERS spectrum (>100 molecular subtypes) is urgently needed. Dedicated international academic research platforms, cross-linked and informed by adult cancer and industry-driven research, are vital to foster therapeutic innovation in PAEDIATRIC CANCERS. -Quality of life: A child’s right to parental presence during illness requires social protection and employment security for carers. There are around 500,000 PAEDIATRIC CANCER SURVIVORS in Europe and this number may grow. The Cancer Control Joint Action identified this group as in need of specific measures to improve their quality of life and reduce the socio-economic impact of long-term morbidities. Health infrastructures supported by digital tools must enable surveillance of long-term side effects and follow-up care delivery, incl. transition to adult care settings and psychosocial dimension. Measures to enable full integration of PAEDIATRIC CANCER SURVIVORS into society and the economy are also needed, such as the ‘right to be forgotten’ legislation. -Knowledge, data, evidence: Population-based standardised registry data collection across the patient pathway is needed. Parents, patients, survivors want to share data to advance research. E-health, AI, and European data integration can drive substantial progress. -Areas for EU level cooperation: A comprehensive EU effort is ideally positioned to steer progress in PAEDIATRIC CANCERS due to their individual rarity and high overall burden. -Conclusion: Europe’s Beating Cancer Plan must acknowledge that children and adolescents are not small adults and deserve specific recommendations as a vulnerable group with high underserved needs. A synergetic Horizon Europe Mission focused on the complex needs of PAEDIATRIC CANCERS can be transformative, generating evidence to advance diagnosis, innovate treatment, and improve care standards. SOURCES: ATTACHED
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Meeting with Stella Kyriakides (Commissioner) and

21 Jan 2020 · Discussion on cancer

Response to European Partnership for innovative health

27 Aug 2019

SIOP Europe – the European Society for Paediatric Oncology representing paediatric cancer clinicians and academia welcomes Innovative Health as a candidate for a European Partnership in Horizon Europe. We particularly identify with the objective to address high unmet health needs where market-driven innovation may be limited. Concerted multi-stakeholder cooperation at the EU level provides unique opportunities to make progress in such disease areas. Important examples of unmet health needs are cited in the Inception Impact Assessment document. We would like to add the case of paediatric cancers: a collection of rare diseases with specific biological characteristics that represent a major shared burden across Europe. All paediatric cancers are rare but commonly diagnosed in children and adolescents. The overall incidence in Europe is over 35,000 new cases per year while annual mortality reaches 6,000 (IARC 2018). The latter makes paediatric cancer the leading cause of deaths by disease in children above one year of age in the EU. Furthermore, most patients who survive childhood cancer and become adults are affected by adverse side-effects following treatment which limit their socio-economic activity and participation. In terms of new medicines for children with cancer, regulations lead the field and industry investment is driven by specific obligations and incentives therein. The regulatory environment is changing in the US and may be changing in Europe in the near future as well, requiring companies to consider the mechanism of action of their assets in relation to the biology of paediatric malignancies and patients’ needs and to develop their medicines in children. More than ever, public-private partnership projects will be essential to improve and optimise the development of new paediatric oncology medicines and ensure that industry meets arising regulatory obligations in the best way to meet patient needs. IMI2 started to address the needs through the development of a (limited) preclinical paediatric oncology platform: the only paediatric oncology project in IMI2. More is needed, specifically: expansion of the preclinical paediatric oncology platform to address the needs in a comprehensive manner; a dedicated platform to generate long-term follow-up data for new anticancer medicines, as required by regulations; generation of real-world data through artificial intelligence to provide additional evidence for the use of new (expensive) biomarker-driven medicines for children with cancer; an initiative to facilitate repositioning of medicines that are abandoned by large pharmaceutical companies that may have a strong rationale for development in paediatric malignancies; a project open to start-ups and small biotech companies (also beyond EFPIA members) to facilitate working with academia and thus incentivise the development of specific paediatric oncology medicines. SIOPE is partner of the other paediatric IMI2 project, Connect for Children, and is sharing approaches to multi-stakeholder collaboration and working closely with other paediatric fields. Our view on the policy options presented is that several funding and collaborative modalities are highly relevant for the paediatric cancer field given the magnitude, number, and specificity of issues faced in the sector. Partnership with industry and other stakeholders is certainly a key pillar of what we would envisage as a comprehensive EU level approach to countering this life-threatening disease.
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Response to European Electronic Health Record (EHR) Exchange Format

19 Dec 2018

SIOPE RESPONSE: Despite progress in the last decades, childhood cancer remains the leading cause of disease-related death in children above one year of age in Europe. Moreover, the majority of childhood cancer survivors face long-term side effects that negatively affect their quality of life and participation in society. Thus, whereas individual types of cancers in children and adolescents may be rare, taken together childhood cancer represents a leading public health issue in Europe. As a result of these two characteristics - rarity combined with an important shared burden - paediatric cancer patients and survivors stand to benefit significantly from health data sharing in research, diagnosis, treatment, and follow up. An overarching consideration is that the costs involved in accessing data for these purposes should not be excessive, as activities are largely funded by voluntary sector donations or patient efforts. - Research Successful approaches are about multi-stakeholder dialogue and collaboration, balancing data privacy and the need for research progress. A “broad one-time consent” is a basic requirement in childhood cancer research, underpinned by right of withdrawal, privacy safeguards, and involvement of trusted third parties. Depending on the child’s age, parents’ consent may be applicable. Survivors may wish to take over responsibility of their data when reaching adulthood and give a second broad consent at this point, as well. Fostering interconnection is the only way to arrive at a sustainable base for future joint data analysis and research including clinical trials and biobanks in rare diseases. Outcome data collection and long-term health surveillance is another critical dimension: to adequately monitor the optimal use of therapies, it is critical to invest in and develop outcome research, real life data collection on utilised therapies, and big data in health, such as by linking long-term observational studies (registries). The possible implications of the EU GDPR, such as increasingly working with pseudo-anonymised or anonymised datasets, may somewhat hinder retrospective data linkage for research purposes. In rare diseases such as childhood cancer, digestion of Big Data based on international collaboration to overcome small volume sample sizes is required in order to investigate disease mechanisms and promote functional studies. Many areas of health ICT innovation require greater capacity – both trained workforce and analytical platforms. - Diagnosis and Treatment There are still large disparities in Europe in access to standards of care for childhood cancer, including highly specialised interventions. There is an urgent need to implement mechanisms enhancing diagnosis and treatment of these patients and sharing knowledge across Europe. The Cross-border healthcare paediatric network ERN PaedCan aims to implement cross-border virtual advice, treatment and care through virtual advisory board functions. Fostering interconnection is instrumental to enable fast transfer of data and aid in the clinical decision-making process. Further functionality is possible with eHealth tools, incl. linking to research platforms. - Long-term follow up Cross-border exchange of health data is important to ensure seamless long-term follow up of childhood cancer survivors as they transition to adult care settings and /or move cross-border. As survival from childhood cancer has been rising, an estimated 300-500,000 citizens in Europe are currently former childhood cancer patients. Proper exchange of follow-up data is instrumental to foster the long-term well-being and empower survivors to proactively manage their life-style and health care needs for a healthier adulthood. The Survivorship Passport is an innovative model that can facilitate long-term follow up of childhood cancer survivors accordingly: https://www.siope.eu/activities/joint-projects/survivorship-passport/.
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Meeting with Robert Schröder (Cabinet of Commissioner Carlos Moedas)

13 Dec 2018 · Health research and innovation

Response to Evaluation of the legislation on medicines for children and rare diseases (medicines for special populations)

8 Jan 2018

Dear, Please find enclosed the feedback from SIOPE - the European Society for Paediatric Oncology, entitled: "Paediatric Cancers and the 2 Regulations*: a catch-22 situation?" Please also find a summary of the SIOPE recommendations here below: • Explore why pharmaceutical companies use the Orphan Regulation for adult anticancer medicine development but not for paediatric oncology programmes; • Understand why the Orphan Regulation has been effective focusing on rare diseases but not on paediatric malignancies; • Consider how both Regulations could better incentivise science-driven paediatric development plans based on the disease’s biology and the drug’s mechanism of action; • Critically assess the European regulatory environment compared to the US environment; • Conduct the evaluation as a truly multi-stakeholder initiative. --- (*The Paediatric Regulation (EC) No 1901/2006 and Orphan Regulation (EC) No 141/2000) --- With best regards, Olga Kozhaeva Sr. Policy and Public Affairs Coordinator European Society for Paediatric Oncology - SIOPE Tel. +32 2 775 29 34 - www.siope.eu
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Meeting with Marika Lautso-Mousnier (Cabinet of Vice-President Jyrki Katainen)

19 Sept 2017 · Paediatric Regulation evaluation report

Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis)

12 Jun 2017 · Paediatric medicines