CCI Europe
CCI Europe
The Vision of CCI Europe is for children and adolescents with childhood cancer to be cured with no, or as few as possible, long-term health problems and late-effects.
ID: 682126441400-50
Lobbying Activity
7 Nov 2025
Childhood Cancer International Europe (CCI Europe; CCI-E), the biggest pan-European childhood cancer parents and survivors organisation, welcomes this consultation on the role of civil society in the next MFF. As a network of parent and survivor groups, CCI-E ensures that the voices of children with cancer, their families, and survivors are heard across Europe. Our experience shows that civil society is indispensable in delivering EU health, research and social objectives. Childhood cancer remains the leading cause of death by disease in children over one year old in Europe, with over 6,000 deaths and 35,000 diagnoses each year. EU support through Horizon Europe, EU4Health, Europes Beating Cancer Plan and the EU Cancer Mission has enabled major progress: improving access to care, supporting collaborative research, and building survivorship initiatives. These programmes have directly empowered civil society organisations and professional networks to implement EU priorities on the ground. Examples include: - The TREL Project, twinning paediatric oncology centres to exchange expertise and reduce inequalities in care - EU-CAYAS-NET, improving survivorship and mental health for young survivors - UNICA4EU, supporting the uptake of clinical AI tools - ITCC-P4, fostering publicprivate partnerships in paediatric oncology drug development. - ERN PaedCan, improving access to standard of care across borders - and many more EU-funded initiatives building a European childhood cancer ecosystem These results show that EU civil society funding delivers clear added value and tangible European impact. However, without sustained EU support, the contribution of patient organisations and learned societies risks being weakened. These organisations bridge policy and practice by shaping care, raising awareness, guiding research priorities, and providing essential psychosocial and educational support to families. They embody the EUs values of solidarity, inclusion and participation. 1. Guarantee operating grants for health civil society. Operating grants under EU4Health are essential to maintain stable and independent participation of patient organisations in EU policymaking and project delivery. They enable long-term strategic work, coordination with national groups, and evidence-based contributions to EU initiatives. The next MFF must ensure the continuity and accessibility of these grants for healthcare civil society, including childhood cancer organisations. 2. Enable independent and meaningful participation in EU projects. Civil society must continue to be eligible as full and equal partners in EU-funded projects. Their expertise improves project relevance, outreach and sustainability. Future programmes should include simplified procedures and dedicated calls for civil society-led consortia in health and social innovation. 3. Support the evidence-generating role of civil society. Patient organisations and learned societies collect and translate unique data on patients needs, service gaps and long-term outcomes. EU programmes should fund these evidence-building activities, which strengthen the knowledge base for EU policy and enhance transparency and accountability. 4. Promote cross-border collaboration. Civil society networks like CCI-E foster alignment in paediatric cancer care not only within the EU but also with candidate and neighbouring countries, by developing and promoting evidence-based recommendations such as the European Standards of Care for Children with Cancer. Continued EU funding for cooperation, awareness and education projects will help sustain this solidarity, strengthen European identity, and empower young survivors as advocates for change. In conclusion, civil society is aa partner in delivering Europes values and priorities. Stable, predictable funding for organisations such as CCI-E is essential to ensure that EU investment continues to save lives, reduce inequalities, and foster solidarity across borders.
Read full responseResponse to EU’s next long-term budget (MFF) – performance of the EU budget
7 Nov 2025
Childhood Cancer International Europe (CCI Europe), the biggest pan-European childhood cancer parents and survivors organisation, welcomes the Commissions efforts to simplify and strengthen the EU budget performance framework. Simplification is necessary, but it must not come at the cost of visibility for high-impact areas that represent the EUs greatest added value. The future framework should measure not only efficiency, but impact - on people, on health, and on the resilience of European society. 1. Simplification must not erase health and social impact. Reducing the number of performance indicators is understandable, but the new framework must still capture what matters for citizens: better health outcomes, reduced inequalities, and improved quality of life. Many of the EUs most valuable achievements - such as those in rare diseases and childhood cancer - are not visible through financial or economic metrics alone. Indicators must therefore include outcomes related to survival, access to care, research participation, and quality of life for patients and survivors. Streamlining should clarify, not narrow, what the EU measures as success. 2. Measure European added value, not just efficiency. The true performance of the EU budget lies in what can only be achieved collectively at European level. In fields like childhood cancer, no single Member State can deliver equal access to care or innovation on its own. EU programmes such as Horizon Europe, EU4Health and Europes Beating Cancer Plan have proven how cross-border research, data sharing and collaboration can save lives and strengthen Europes innovation base. The new performance framework must recognise this European added value through indicators that measure cooperation, research leverage, and capacity building across borders - not just national outputs. 3. Simplification should strengthen, not weaken, accountability. A single, consistent performance framework can improve transparency and reduce administrative burden. However, it must be built with input from those delivering and benefiting from EU programmes, including health stakeholders and civil society. Indicators that are too generic risk disconnecting the framework from real-world results. Including health and research actors in the design of common set of indicators will help ensure that accountability is meaningful, measurable and relevant to citizens. Simpler reporting must still reflect complex societal goals like innovation, equity, and long-term health resilience. 4. Transparency and access to information empower citizens. Improving access to performance data is essential for public trust and democratic accountability. Civil society, including patient and survivor organisations, can play a constructive role in analysing, communicating, and improving the EUs performance results. The new framework should therefore ensure that data on EU-funded health and research outcomes is accessible, comparable, and understandable. Transparent reporting on indicators related to health impact, equality, and research collaboration will enable citizens and policymakers alike to see how EU investment improves lives.
Read full responseResponse to EU’s next long-term budget (MFF) – EU funding for competitiveness
7 Nov 2025
Childhood Cancer International - Europe (CCI Europe), the biggest pan-European childhood cancer parents and survivors organisation, welcomes the opportunity to contribute to this consultation. Europes competitiveness must be underpinned by a healthy, skilled and resilient population, and by an innovation ecosystem that addresses areas of unmet need. Investment in childhood cancer achieves both: it builds Europes leadership in health innovation while safeguarding the future of its children and workforce. 1. Health innovation as a competitiveness driver. The next MFF must place health innovation at the heart of Europes competitiveness agenda. Paediatric oncology is a high-impact test case: it combines frontier science, cutting-edge technologies, and cross-border collaboration. Yet, with only 16 paediatric oncology drugs approved in the past decade versus 150 for adults, Europe risks falling behind in innovation precisely where it can deliver unique global value. Boosting EU funding for paediatric oncology drug development and digital research infrastructures will reinforce Europes life sciences competitiveness and Europes biotech ecosystem, while addressing an area of high social return and low commercial incentive. 2. Build Europes research powerhouse. EU-funded initiatives such as the EU Cancer Mission, Horizon Europe projects (ITCC-P4, UNICA4EU) and the European Reference Network for Paediatric Cancer (ERN PaedCan) already demonstrate that public investment can drive competitiveness. These cross-border networks transform fragmented capacities into collective excellence - a hallmark of European value added. The EU should continue to fund such important frameworks. The next MFF should scale this model by creating Comprehensive Childhood Cancer Infrastructures within ERN PaedCan to integrate research, care, innovation and training. Europe can become the global centre of excellence in paediatric oncology if it chooses to invest boldly and strategically. 3. Lead the world in data and AI for health. Europes strength lies in its ability to turn data into innovation. Paediatric oncology projects (e.g., UNCAN.eu & UNICA4EU) already show how AI and federated data systems can accelerate discovery and personalise care. The next MFF should make data-driven health innovation a flagship of EU competitiveness by investing in secure, interoperable infrastructures under the EHDS - turning Europes digital sovereignty into global leadership in health innovation. 4. Mobilise public - private partnerships. Europes competitiveness depends on aligning public missions with private innovation. Paediatric oncology, with limited commercial incentives, requires EU leadership to de-risk R&D and crowd in private investment. Dedicated Horizon Europe calls, innovative finance tools and regulatory flexibility can stimulate SME and biotech engagement. Turning unmet medical need into an innovation opportunity will strengthen Europes life sciences sector while delivering breakthrough treatments to patients. 5. Invest in human capital and skills. Every child cured from cancer is a citizen who can study, work, and contribute to Europes prosperity. Over 500,000 survivors in Europe live with long-term health or psychosocial effects that limit participation in education and work. EU investment in survivorship research, rehabilitation and healthcare integration is therefore an economic and moral imperative. In addition, training clinicians, researchers and data scientists through ERN PaedCan and EU Cancer Mission Hubs builds the high-end skills that power Europes innovation economy. 6. Empower civil society as a competitiveness asset. Patient organisations amplify EU investment by bridging policy, research and citizens. They generate data, shape priorities, and translate innovation into impact. Sustained EU4Health operating grants and project-based funding are essential to keep them active within Europes innovation ecosystem.
Read full responseResponse to European Research Area (ERA) Act
10 Sept 2025
Childhood Cancer International Europe (CCI Europe, CCI-E) is the largest pan-European childhood cancer parents and survivors organisation. We represent childhood cancer parents and survivors groups and other childhood cancer organisations. Paediatric cancer is the leading cause of death by disease in Europe for children over 1 year, and two-thirds of survivors suffer from late effects caused by toxic treatments. We welcome the opportunity to respond to this Call for Evidence, as science and innovation hold the promise of more effective and safer therapies for children and young patients with cancer. Multi-country clinical trials (MCCTs) are essential to accelerate drug discovery and development. Cross-border data sharing, scientific collaboration, and comprehensive as well as structural funding schemes are crucial to support high-quality paediatric oncology trials. Harmonised digital tools and infrastructuressuch as AI, the EHDS, Real World Evidence (RWE) platforms, and data consistency mechanismswill further strengthen MCCTs and speed up drug development. CCI-E underlines the competitive advantages of a EU Single Market R&I ecosystem for researchers, scientific knowledge and technology. Europe has the potential to become a global leader in fair, patient-centred innovation for children with cancer, attracting investment and talent. Yet paediatric oncology is currently a near non-existent field within the health R&I landscape, with persistent barriers slowing drug development. Paediatric cancers are so rare that commercial research cannot offer any return on investment. Academic centres are a driving force in bridging this market failure in paediatric cancer research and clinical trials. Global leadership in this field requires collective, cross-border efforts to: 1) support fundamental and translational academic research to discover treatments specifically targeting paediatric cancers (first-in-child); 2) build an clinical trial-prone infrastructure to speed up access to these innovative treatments; and 3) contribute to the creation of alternative business routes to market for ultra-rare diseases (such as joint procurement contracts). The research and development should speed up access to the most effective medicines with reduced toxicity, improving survivors long-term health and reducing treatment side effects. Advanced Therapy Medicinal Products (ATMPs), such as CAR-T (chimeric antigen receptor) and omics-based therapies, hold great promise in paediatric oncology. Academic centres increasingly take on a direct role in their development. Explicit recognition in the ERA Act of academias role alongside industry in ATMP development would corroborate the EUs objective to support all key actors in the R&I ecosystem and speed childrens access to cutting-edge therapies. To ensure sustainable access to child-centred therapies, Europe must create an investment-friendly environment combining public and private funding. This is vital to secure capital and reduce financial risk of innovative research, particularly in ultra-rare fields like paediatric oncology, which do not attract traditional venture capital. Investing in paediatric oncology R&I would yield widespread benefitsnot just for patients, but for European society and the economy as a whole. It would reduce childhood cancer mortality, increase workforce productivity and scientific knowledge, attract highly skilled talent, and cement Europes leadership in patient-centred innovation. Paediatric oncology clearly illustrates the need for a strong European Research Area. Only a single, borderless R&I market can deliver the scale required for cross-border trials, genomic data sharing, and researcher mobility. By prioritising investment, boosting the uptake of innovations, and ensuring equitable access to excellence, the ERA Act can transform Europe into a global leader in paediatric cancer research and deliver life-saving therapies to children across all Member States.
Read full responseResponse to EU Civil Society Strategy
3 Sept 2025
Childhood Cancer International Europe (CCI-E) is the biggest pan-European childhood cancer parents and survivors organisation. We welcome the opportunity to respond to the European Commissions Call for Evidence and support the EU Civil Society Strategy. For decades, civil society organisations (CSOs) have filled the gaps between national systems and societys needs. CSOs are driving forces for local as well as (inter)national support, representation and empowerment of society, including awareness-raising, education, provision of services, policy monitoring and advocacy. CSOs mostly rely on limited, if any, national or European funding, private donations, and are heavily supported by volunteers work and commitment. Safeguarding the independent, uninfluenced representation of CSOs in national and European political decision-making is vital to ensure all voices and expertise are heard and considered; addressing the actual needs, priorities and challenges of society in public policy. The unique position of CCI-E is a challenge: as a pan-European organisation, representing our members in Europe comes with high responsibility. Being the beneficiary of several EU4Health Operating Grants allowed us to work independently and to broaden the reach and activities to include more training for patient advocates across Europe. However, the absence of operating grants in the 2025 EU4Health Work Programme poses a serious risk to the work and impact CCI-E brings to its members and other actors within the childhood cancer community. Umbrella or pan-European CSOs often are ineligible for national and local grants. We expect the EU Civil Society Strategy to fill this gap with a more sustainable, long-term funding programme. Monitoring of use of funding should be transparent, equal and fair across fields of engagement. This will enhance the public reputation and credibility of both NGOs and the EU, demonstrating a shared commitment to transparency and positive impact. Simultaneously, it is vital to consider the limited resources (funding and personnel) of CSOs: intensive and burdensome bureaucracy are significant constraints that discourage applicants from engaging in EU-funded activities. The lack of financial compensation for volunteer work the foundation of many CSOs is further deterrent. It not only undermines the valuable contributions of CSOs but also risks straining their relationship with EU institutions. We therefore propose the EU Civil Society Strategy to include: a guarantee of long-term funding, easier access and simplified bureaucracy for civil societies, and support of voluntary work. We endorse the creation of a Civil Society Platform to strengthen the dialogue between public authorities and CSOs. We encourage for the platform to be co-created with CSOs to bolster effective dialogue based on transparency, equity and accountability. Through our advocacy work, we represent minorities and vulnerable groups: safeguarding their rights is a high priority. As a pan-European organisation, close collaboration with the EU institutions and decision-making entities is a key component of our work. We are the link between the EU and our local national organisations: actions and activities promoted and organised by CCI-E have the potential of faster and direct effect in Member States. A significant part of this work is continued exchange with public authorities: the EU Civil Society Strategy has the unique opportunity to further promote and support these connections and dialogues. CCI-E is committed to building bridges between local, national and European policymaking, implementation and sustainability, working with and for European citizens. We are dedicated to continuing to do so, stressing that sustained EU funding and support, including systemic, full-circle involvement of CSOs in decision-making processes are crucial to ensure effective work and services independently from commercial actors, for the benefit of the European citizens.
Read full responseResponse to Critical Medicines Act
4 Jul 2025
Childhood Cancer International - Europe (CCI-E) welcomes the European Commissions proposal for a Critical Medicines Act (CMA) and fully supports its objective to strengthen the EUs supply resilience for essential medicines. Representing Children, Adolescent, and Young Adult (CAYA) cancer patients, along with survivors and parents, CCI Europe highlights the need of addressing childhood cancer, a therapeutic area of (ultra) rare and life-threatening diseases with high unmet medical needs (UMN). It remains the leading cause of death by disease in children >1 year old in Europe, with 35,000 cases diagnosed and 6,000 deaths annually. Inequalities in access to treatment, care and research protocols across Europe contribute to an estimated 20% gap in childhood cancer survival rates, disproportionally affecting Eastern European countries. The core challenge is the lack of commercial interest in developing drugs for rare paediatric cancers, namely drugs targeting a specific paediatric cancer type, also known as first-in-child drugs. In the past decade, only 16 anti-cancer medicines were approved for children, compared to over 150 for adults. For the most lethal childhood cancers, only a few new treatments have emerged in 30 years. Through this Act and other EU initiatives, we see major opportunities for the childhood cancer community provided key conditions are met. We therefore call for: Clear definition of market failures and of medicines of common interest. The inclusion of medicines for rare diseases within medicines for common interest. Strategic projects specifically targeting rare diseases. The inclusion of patient representatives in the Critical Medicines Alliance Coordination Group to ensure healthcare decisions reflect real patient needs. The application of this new regulation to already authorised medicines - many of which remain unavailable in parts of the EU. Targeting only novel common interest medicines put onto the market after assessment through the new HTA process risks maintaining or deepening access inequalities. CCI-E strongly supports EU-level joint procurement (JP), including voluntary tenders beyond crisis use. We believe JP can increase timely and equitable access to medicines, especially when those medicines address rare diseases and other unmet needs. However, CCI-E recommends lowering the minimum number of Member States required to start the JP procedure to less than 9 Member States. This would guarantee that Member States with smaller markets have more bargaining power and access to medicines for a lower cost, thereby creating more equal access to critical medicines across Europe. We stress that addressing supply chain vulnerabilities will be insufficient to ensure robust healthcare systems without improving innovative research and equal access to treatment and care across Europe. We therefore highlight the importance of CMA alignment with other health-related legislation, including the Pharma Package revision, new Health Technology Assessment (HTA) Regulation, Biotech Act, and the Multiannual Financial Framework (MFF). We also urge the Commission to include dedicated EU funding for medicines security in the 2028-2034 MFF, including for rare disease and cancer medicines. Medicines security is an essential part of EU competitiveness, defense and security, which should be matched accordingly by secured investments in its resilience.
Read full responseResponse to Biotech Act
11 Jun 2025
Paediatric oncology is a field that has yet to reach its full potential. The creation of a legal framework for biotechs, within a truly Single Market, holds the potential to support transformative research, improve treatments, and ultimately help cure more children affected by cancer. The biopharmaceutical market includes the development of highly promising novel paediatric medicines and omics-based therapies, including Advanced Therapy Medicinal Products (ATMPs) such as CAR-T (chimeric antigen receptor) cell therapy. Within the EU biotech sector, Europe has the potential to become a global frontrunner in innovation and competitiveness in health biotech. Achieving global leadership in equitable, patient-centred innovation for childhood cancers demands a collective, cross-border effort. This means encouraging earlier and dedicated paediatric drug development, stimulating first-in-child research and innovation, and incentivising treatments that target the unique genetic mutations found only in paediatric cancers. The development of such biopharmaceutical products should aim to create the most effective medicines for children with the least possible toxicity. This would ultimately improve long-term health outcomes for survivors and reduce the mental and physical side effects of treatment. In paediatric oncology, multi-country clinical trials (MCCTs) are essential for accelerating drug discovery and development, ensuring timely access to innovative, more effective, and less toxic therapies. Because paediatric cancers are often ultra-rare, cross-border data sharing and scientific collaboration are crucial to support high-quality MCCTs. A harmonised implementation of digital infrastructuressuch as the European Health Data Space (EHDS), Real World Evidence (RWE) platforms, and data consistency mechanismswill strengthen MCCT research and speed up the development of biopharmaceutical products. Innovative, targeted, and paediatric-centred therapies must be brought to market at a reasonable cost to safeguard the long-term sustainability of Europes social security systems. To achieve this, we must create an investment-friendly environment that combines public and private funding. This is essential to secure access to capital and minimise the financial risk of innovative research, particularly in ultra-rare fields such as paediatric oncology, which do not attract traditional venture capital. The biotech sector holds the promise of creating a sustainable model for delivering affordable paediatric cancer medicines and therapies. Doing so will require lean management structures, efficient supply chains, and optimised biomanufacturing to keep development costs low. Such a low-cost, high-value model would yield widespread benefitsnot just for patients, but for European society and the economy as a whole. It would reduce childhood cancer mortality, increase workforce productivity, and help attract a highly skilled talent pool. It would also cement Europes leadership in meaningful, patient-centred biomedical innovation.
Read full responseMeeting with Olivér Várhelyi (Commissioner) and
28 May 2025 · Childhood cancer
Meeting with Victor Negrescu (Member of the European Parliament)
26 Mar 2025 · EU annual budget and EU health policies
Response to Critical Medicines Act
27 Feb 2025
Childhood Cancer International - Europe (CCI-E, or CCI Europe) is the biggest pan-European childhood cancer parents and survivors organisation. It represents childhood cancer parent and survivor groups as well as other childhood cancer organisations in Europe: 64 organisations in more than 30 European countries are members of CCI-E. CCI Europe works together with all relevant stakeholders for the same aim: help children and adolescents with cancer to be cured, with no - or as few as possible - long term health problems/late effects. Cancer remains the first cause of death by disease in Europe in children older than 1 year - more than 35,000 cases are diagnosed every year and over 6,000 young patients die annually. Ensuring equitable access to essential medicines for paediatric cancer patients remains a major challenge in Europe. The relative lack of fast market-driven therapeutic innovation in paediatric cancers is among the reasons for stagnating cure rates and persisting adverse long-term side effects of using older medicines in survivors. 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. Medicine shortages and disparities in availability continue to significantly impact treatment outcomes. To address these challenges, the European Society for Paediatric Haematology (SIOPE), which represents all professionals working in the field of childhood cancers launched the Essential Anticancer Medicines Project to systematically identify and provide a list of essential anticancer medicines for paediatric cancers in Europe. Of the 66 essential medicines identified, 35% of all medicines were prescribed off-label in at least one country and 44% were always available in >90% of countries. The main determinant of unavailability was shortages, reported for 72% of medicines in at least one country. This initiative contributed to the successful addition of medicines to the WHO Essential Medicines List for Children. CCI Europe, in collaboration with SIOPE, also provided six recommendations regarding access to medicines within the framework of the revision of the EU Pharmaceutical legislation: 1. aligning regulations with science and unmet needs, 2. ensuring child-specific drug innovation, 3. strengthening multi-stakeholder cooperation, 4. securing sustainable funding for paediatric and orphan medicines, 5. improving coordination between global agencies to streamline paediatric drug development, 6. and ensuring equal access to medicines. Request for inclusion of paediatric cancer essential medicines in the Critical Medicines List: Given the severe consequences of medicine shortages for childhood cancer treatment, CCI Europe calls for the inclusion of essential paediatric oncology medicines in the Critical Medicines List. Paediatric cancer patients face unique vulnerabilities, often lacking alternative treatments, making the availability of these medicines crucial. Many of these essential drugs are off-patent and produced by a limited number of manufacturers, heightening the risk of supply disruptions. Their inclusion in the Critical Medicines List would establish a regulatory framework to help secure their supply. We urge the European Commission to include the expertise of the paediatric oncology community and the patient community when updating the Critical Medicines List within the framework of the Critical Medicines Act. It is essential that patients be actively involved in the development of this legislation to ensure that their needs and challenges are fully considered. This step will reinforce the EUs commitment to equitable healthcare access and contribute to improving survival rates for children and adolescents with cancer across Europe.
Read full responseMeeting with Vlad Vasile-Voiculescu (Member of the European Parliament)
5 Feb 2025 · Follow-Up CCI
Meeting with Olivier Chastel (Member of the European Parliament) and KickCancer
28 Jan 2025 · Politique de santé de l'Union européenne
Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament) and KickCancer
20 Nov 2024 · Introductory Meeting
Response to Health technology assessment – Joint scientific consultations on medicinal products for human use
29 Oct 2024
Childhood Cancer International Europe (CCI-E), the largest pan-European organisation representing childhood cancer patients, parents and survivors, welcomes the opportunity to give feedback on the public consultation Implementing Regulation on joint scientific consultations on medicinal products for human use at Union level. Childhood cancer is a rare and life-threatening disease that affects over 35,000 children per year in Europe. Among the 500 000 survivors, 2/3 of them experience acute and/or long-term side effects. Our patient community, composed of parents and survivors, is looking forward to work with the EU Commission, EMA, HTA/Payers, clinicians and industry to facilitate transparent, efficient and timely scientific consultations which contribute to the development of innovative life-saving treatments for children. However, we would like to raise some points of concern relating to the draft of the implementing regulation: - Participation of caregivers: our community includes caregivers parents or survivors of childhood/young adults cancer. In our particular disease area, caregivers are fully-fledged patient experts. When assessing new childhood cancer medicines, parents will be called to voice the patient journey of their children, and specific challenges related to this condition. The input of survivors of childhood cancer can also be key for HTA assessments; their lived experience is paramount to assess the benefits of a new medicine, influence on quality of life and means of administration. - Identification of patient experts: CCI Europe encourages the early identification of experts, as per recital 9. As childhood cancers are ultra rare diseases, it is challenging to find the most suitable patient or survivor to take part in JSC's, as their experience should in principle cover several Member States, in the disease or the therapeutic area of the JSC, according to article 5; 5. Regarding sources for identifying and gathering patient experts, 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, as per article 11. - Capacity building amongst patient experts: HTA training programs available on the long-term are of an inestimable value for our patient community. We advocate for continued funding from the European Union forprogrammes such as EUCAPA. With the new HTA regulation set for implementation in January 2025, our community will need ongoing support to train and equip our patients with the best skills and expertise to represent the patients' voice at the table of HTA negotiations. - Streamlining of HTA & EMA Processes: the efforts made to streamline the timelines, processes and outcomes of the HTA JSCs and EMA early scientific advice, as per recital 7,8 & 12, is a positive aspect. Such efforts are crucial in supporting the generation of sound and supportive early guidance that drive the development of innovative life-saving treatments. - Timeline of JSC: Understanding the timeframe in which individual experts are expected to provide input, as per Article 10, would be valuable information, helping organizations to prepare their expert members for expected workloads and processes. - Engagement of JSCs and Paediatric Investigation Plans (PIPs): Concerning the specific needs of paediatric medicines, especially for rare diseases like childhood cancer, early JSC during Paediatric Investigation Plan (PIP) development should be considered. Paediatric cancer medicine development has been driven largely by the adult market, with only 16 medicines authorised since 2007, and few first-in-class indications (e.g., Larotrectinib, Dinutuximab, Kymriah). Many future compounds for paediatric malignancies, first developed in adults, may not qualify for JSC despite addressing unmet medical needs.
Read full responseMeeting with Stella Kyriakides (Commissioner) and
30 Jan 2024 · Meeting of Commissioner Kyriakides with Childhood Cancer International Europe on Europe’s Beating Cancer Plan
Response to Interim evaluation of the EU4Health Programme 2021-2027
4 Jan 2024
Childhood Cancer International Europe (CCI-E), the biggest pan-European childhood cancer patients, parents and survivors organisation, welcomes the EU4Health programme as one of the main instruments to pave the way to a European Health Union, and to improve the health of EU citizens, especially children and adolescents with cancer. Childhood cancer is a rare and life-threatening disease that affects over 35,000 children per year in Europe, and therefore requires specific attention on several issues addressed by the EU4Health programme 2021-2027. As a funding instrument of the Europes Beating Cancer Plan (EBCP), EU4Health delivered on some of the flagship initiatives of the EBCP related to paediatric cancer. We are indeed grateful that the EU decided to put childhood cancer under the spotlight in the EBCP. In the framework of the Helping Children with Cancer Initiative, the EU Network of Youth Cancer Survivors (EU-CAYAS-NET), coordinated by CCI-E, was established to improve the quality of life of childhood, adolescent and young adult cancer survivors through improved social networking. We hope the network will continue to foster social networking, peer-support, knowledge-exchange, and will improve the quality of life of survivors, the care for adolescents and young adults (AYA) with cancer, upholding Equality, Diversity and Inclusion along the whole treatment and survivorship trajectory. With the aim of reducing inequalities across the EU, CCI-E supports the development of the European Cancer Inequalities Registry (ECIR) in 2021 and the inclusion of a section dedicated to childhood cancer. CCI-E, in collaboration with the European Society for Paediatric Oncology (SIOP Europe), advocated the introduction of data on inequalities in the field of paediatric cancer. CCI-E welcomes the support offered by the programme for the establishment and development of European Reference Networks (ERN) on specific cancer types which aim at fostering equal access to the best healthcare for rare diseases. In 2017, the European Reference Network on Paediatric Cancer (ERN PaedCan) was launched to reduce inequalities in childhood cancer survival by providing high-quality, accessible and cost-effective cross-border healthcare to children and adolescents with cancer, regardless of where they live. CCI-E hopes that the programme will leverage the full potential of existing ERNs by providing better financial support (e.g., remuneration for the time invested by doctors). CCI-E is involved in the UNCAN.eu project to represent childhood cancer patients. The aim of this project, implemented by both the Mission Cancer and the EBCP, is to advance the understanding of cancer mechanisms to improve cancer prevention, early diagnosis and treatment, providing a basis for saving millions of European citizens lives. CCI-E hopes continued support will be provided to generate a blueprint, namely the establishment of a European Federated Cancer Research data hub where challenges in cancer research will be addressed. Finally, the co-funding scheme of the EU4Health programme is a major challenge for small NGOs. The work for the project consumes most of their capacity and it is almost impossible to raise funds for co-funding at the same time. As a result, there is almost always "underspending" accompanied by a very high workload to be able to fulfill the project tasks as expected. In conclusion, CCI-E supports the work carried out so far under the EU4Health programme, both as a funding instrument for the EBCP and as a complementary instrument to other Research & Innovation EU spending programmes (e.g., Horizon Europe). However, better results could be achieved if financial resources were made available to ensure the follow-up of the projects. Moreover, the workload of beneficiaries could be reduced if EU4Health provided more budget for certain tasks. CCI-E hopes the EU4Health programme 2021-2027 will continue to deliver on topics related to paediatric cancer.
Read full responseResponse to Evaluation and revision of the general pharmaceutical legislation
6 Nov 2023
Childhood Cancer International Europe (CCI-E), the largest European patients organisation in paediatric cancer, has been involved in the review of the EU Paediatric and Orphan Regulations since 2012. We welcome the proposal by the Commission for revision of the EU Pharmaceutical legislation as it covers some areas of concern of the paediatric cancer community. First, we are very satisfied with the introduction of paediatric medicine development based on the molecular target of a new medicine (art. 75 Regulation). Under the current framework, there is no obligation for a medicine developer to submit a Paediatric Investigation Plan (PIP) if the medicine originally developed for an adult cancer does not exist in children, even when the medicine has a relevant mechanism of action for a given type of paediatric cancer. The use of the concept is aligned with the US RACE for Children Act and meets our expectation to harmonise the global regulatory environment in the field of drug development in paediatric oncology. We support the proposal in article 48 of the Regulation to facilitate the repositioning of medicines, shelved or developed for other conditions, for the treatment of paediatric diseases and the role of non-for-profit entities (academia) in generating data for repurposed medicines through fit-for-filing trials. EMAs role in ensuring that the label of those medicines, as repositioned, is extended, is crucial to ensure access for young cancer patients. However, this measure requires adequate resources for academic-led research and increased capacity within the EMA to improve access without delay to lifesaving drugs for patients who must otherwise resort to drugs prescribed off-label. A mechanism is therefore needed to ensure industry provides academia with new drugs and funding for trials. We commend the introduction of the stepwise PIP (art. 76 Regulation) which aims to accelerate and simplify paediatric medicine development and support developments based on needs and robust science. However, we advocate a clear obligation to submit PIPs upon completion of phase I of clinical studies in adults rather than prior to the initiation of the phase II, as well as penalties to deter companies from delaying the submission of PIPs. We support the cap applied to the PIP deferral to the extent that the total duration of the deferral, including any prolongation granted, may not exceed 5 years. We support the introduction of Unmet Medical Need (UMN) and High UMN (HUMN) concepts. However, their definition (art. 83 Directive & art. 70 Regulation) should allow a medicine to be considered as addressing a UMN or HUMN when it contributes to reduce acute or long-term toxicity. Indeed, children with cancer suffer from a vast amount of side effects due to the treatments they receive, usually designed for adults. Reduction of these side effects can only be achieved through the development of new treatments specifically designed for children, adolescents and young adults with cancer. In line with this last comment, the proposal does not provide for a framework or specific incentives for first-in-child development and marketing authorisation of medicines. Currently, the development of first-in-child anticancer drugs is overlooked as the complexities related to paediatric cancer (e.g., challenges in designing clinical trials and heterogeneity of the paediatric population in need of age-specific formulations) make investments in the development of drugs for a small, high-risk population economically unattractive for pharma companies. We suggest including in the Regulation (art. 71) a 12-year market exclusivity period for orphan drugs whose first authorised indication addresses a HUMN in a paediatric population to increase commercial interest in the development of medicines specific to paediatric cancers. Besides, we call for programmes allocating public funds to research projects addressing UMN in paediatric indications.
Read full responseMeeting with Sara Cerdas (Member of the European Parliament)
26 Oct 2023 · Childhood Cancer International Europe
Response to A comprehensive approach to mental health
15 Feb 2023
Childhood Cancer International Europe (CCI Europe; CCI-E), the biggest pan-European childhood cancer patients, parents and survivors organisation, welcomes the European Commissions initiative to establish a comprehensive approach to mental health. Childhood cancer is a rare and life-threatening disease that affects over 35,000 children per year in Europe. During their treatment and beyond, many patients experience mental disorders such as depression, anxiety and fear. In addition, adolescents and young adults (AYAs) with cancer experience specific additional psychosocial issues given their development stage (e.g., social isolation, distress about life choices and establishing identity). Unfortunately, psychosocial care for children and their family is not everywhere provided in a preventive, systematic and consistent manner within paediatric cancer programs. The following are recommendations formulated by CCI Europes Psychosocial Care Pillar that could improve childhood and AYA cancer patients mental health: - Firstly, the healthcare team caring for childhood cancer patients should be well-trained and aware about their specific psychosocial care needs. A childs cancer diagnosis and treatment plan requires psychological adjustment and an understanding of its impact on mental health. - Secondly, patients and affected families should be informed at the earliest possible stage by the healthcare team of the potential psychosocial issues related to the cancer diagnosis, treatment and even those arising after recovery, and existing support and measures to lessen the effects. - Thirdly, as psychosocial consequences of cancer diagnosis can be equally impacting the patients everyday life as can physical late effects of the disease and its treatment, psychosocial care needs of patients should be an integral part of the entire treatment pathway. This includes the integration of the psychosocial care in long-term follow-up care plan for survivors (example are recommendations from the International Late Effects of Childhood Cancer Guideline Harmonization Group, 2022, https://pubmed.ncbi.nlm.nih.gov/35358467/). Indeed, survivors encounter events related to their cancer, even after recovery, that affect their mental health. Hence, support should be provided to manage any psychological, psychosocial or socioeconomic consequences of their illness and treatment. - Fourthly, affected families of children and AYA with cancer should receive systematic assessments of their psychosocial care needs and support to manage any mental consequence of the illness and treatment. The provision of psychosocial care, and quality of life, could be improved through measures implemented through the integrated care pathway: from single-point of contact for psychosocial care throughout treatment and follow-up, a multidisciplinary teams with a psychologist, systematic assessments of psychosocial care needs, facilitating return to school, patient-centered follow-up care to peer support. For all these recommendations to become reality, investment is necessary in psychosocial care research in paediatric cancer, training (inter-disciplinary) for the health workforce and measures to support patients and their families. Improved exchanges and networking among mental health professionals, researchers and patient organisations focusing on the specific needs of this vulnerable group is essential to improve patients and families mental health. Finally, we would like to mention the Standards for the Psychosocial Care of Children With Cancer and Their Families (2015 and 2019), established by a group of paediatric oncology psychosocial professionals, that would benefit our community if considered (https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6397048/). CCI Europe hopes these orientations will be considered to address the specific psychosocial care needs of childhood patients, survivors and their family and stands ready to support the EU with its endeavours.
Read full responseResponse to Interim evaluation of Horizon Europe
29 Jul 2022
Childhood Cancer International – Europe (CCI Europe, CCI-E) is the biggest pan-European childhood cancer parent and survivor organisation representing 67 member organisations at local and national levels in 34 countries across Europe.
CCI-E participated in several EU projects under the Horizon Europe programme of relevance to paediatric cancer, including UNCAN.eu and EUPPC3 (project application only - rejected).
DESIGN & IMPLEMENTATION
The patient role in larger consortia has been well acknowledged and reflected in all Work Packages of the rolled out calls of relevance to paediatric cancer. Recognised and fully utilised role of patient advocates in a multistakeholder setting is a positive and welcoming sign that should remain and be encouraged for the benefit of patients. In some of the EU projects with rather wide-ranging scope of themes, a dedicated chapter on paediatric cancer was highly welcomed. It has been shown that cooperation between the adult cancer community and paediatric cancer community can bring advantage to both sides only if synergies are well mapped out at the early start of project design and that findings from the paediatric cancer research may serve as a blueprint for some adult cancer approaches. Nevertheless, it is clear that paediatric cancers have distinct needs that go beyond merely extrapolation of adult cancer services.
RESULTS, IMPACTS AND SUSTAINABILITY
Inclusiveness is of the essence, hence multidisciplinary and interdisciplinary collaboration and education should be encouraged in all EU projects in order to create tangible results that would build on and help create future goals. Patient organisations should participate and give their contributions and expertise in all project Work Packages, beyond merely dissemination. A defined patient role can enrich the consortia, bring in fresh perspectives from the consumer angle, further guarantee to meet the needs of patients and assist in formulating subsequent projects and actions.
In situations where a project application has been denied (especially in calls of relevance to all cancers e.g. palliative care), there is a strong need for having a distinct childhood cancer call that would be more compatible and realistic to address the specific needs of this population.
PROJECT DISSEMINATION & EXPLOITATION OF RESULTS
Open access policy is very important for the patient community as it helps in revealing new information to a broader audience whilst educating them. Patients are very keen to share their data with appropriate safeguards to ensure proper research progress can be achieved across all of Europe.
COSTS AND BENEFITS FOR APPLICANTS
Currently about 80% of EU funding is managed under programmes jointly administered by the European Commission and national authorities in the EU countries. However, sometimes only 60% of EU funding is administered by the EU and that is imposing an additional hurdle to NGOs and patient entities who require resourced actions to operate. In some of the EU projects, budget allocation was evenly shared between the partners. Nevertheless, there are situations where NGOs require more resources (e.g. for translation services) or are stripped of funding, thus they cannot engage to the expected level through volunteering solely. A compensation scheme that would reflect the work performed by volunteers and NGOs is highly welcome so that organisations can effectively manage the resources allocated to a project.
FINAL REMARKS
Regular public funding is required to sustain the operations of patient organisations and empower them to convey the urgent needs and speak for this underserved population. The EU can empower patients, survivors and their families all across Europe by involving parent, patient and survivor representatives in the development of cancer and health policy instruments and projects, from development to implementation, monitoring, and evaluation.
Read full responseResponse to Ex-post evaluation of Horizon 2020
29 Jul 2022
Childhood Cancer International – Europe (CCI Europe, CCI-E) is the biggest pan-European childhood cancer parent and survivor organisation representing 67 member organisations at local and national levels in 34 countries across Europe.
CCI-E participated in several EU projects under the H2020 programme of relevance to paediatric cancer, including PanCareFollowUp, PanCareSurPass and HARMONY.
DESIGN & IMPLEMENTATION
In some of the projects’ pathways, patient community would have benefited more from the early involvement, including from the project design to its implementation and dissemination. Patient entities should be encouraged to set joint project expectations with the rest of the consortia to improve later coordination and communication.
Healthcare professionals (HCPs) must be enabled to share project data and NGOs and learned societies should be adequately resourced to lead and represent respective stakeholder groups/organisations and institutions/universities. In rare disease settings, such as paediatric cancer, it is desirable to have distinct calls to childhood cancer that would allow the community to comprehensively engage and conceptualise the project plan according to the needs of children and adolescents with cancer, together with NGOs representing HCPs and patient, parents and survivors of childhood cancer. This approach would also help in avoiding the dilution of the needs of this population and ensure the delivery of promised plans and actions.
Participation in projects of relevance to childhood cancer survivorship requires the early engagement of skilled and educated survivors who would be on equal footing involved in the project design and implementation as HCPs, but for that the resources must be enabled to bring new survivors that will be educated, empowered, and heeded by all the stakeholders.
RESULTS, IMPACTS AND SUSTAINABILITY
There is a need to invest more in the sustainability of all projects and that is difficult to achieve if common aims and expectations are not realistic and set on time (early enough) with all respective partners, which was sometimes the case in projects with overarching themes that were broad scoping diverse disease entities. In addition, adequate resourcing for institutions that are supposed to implement the project results should be a prerequisite and must be cultivated before the project commences.
Global emergencies (e.g. COVID-19 pandemic) have decreased the engagement of patient representatives. Whilst in some cases, high level technical and administrative burden deteriorated their participation as they were ought to contribute voluntarily.
PROJECT DISSEMINATION & EXPLOITATION OF RESULTS
Project dissemination has been very successful for most projects, hence everyone usually felt supported by the responsible partner or agency. However, it would be important to dedicate in future calls more resources for patient and healthcare NGOs so they could purposefully engage in the project conduction.
COSTS AND BENEFITS FOR APPLICANTS
In many projects budget allocation was fairly divided between all the partners. Nevertheless, there is an urgent need for continuous and adequate budget allocation to patient organisations in all EU projects to efficiently contribute, as to date their volunteering is unsustainable to reach a complete lifecycle of patient engagement. Besides, EU projects with broader themes could impose an additional challenge for patient organisations to participate as they tend to be under-resourced.
FINAL REMARKS
Inclusive and transparent multi-stakeholder consortia is a clear necessity in all EU projects. Regular public funding is required to sustain the operations of patient organisations and empower them to convey the urgent needs and speak for this underserved population. Thus, paediatric cancer patient advocates and survivor groups should be appropriately resourced to efficiently engage in EU projects & policy dialogues of relevance to childhood cancer.
Read full responseResponse to Proposal for a Council Recommendation on long-term care
29 Mar 2022
CCI-E Response to the Revision of Barcelona Targets on Childcare
Cancers in children truly differ from cancers in adults and their causes are not well understood to be prevented to this day. Still, children of all ages, races and economic backgrounds get cancer, but not all of them have equal access to care regimens across Europe.
When a child has cancer, every family member needs support and families often feel shocked & overwhelmed following their child’s cancer diagnosis, with mothers experiencing poorer quality of life (QoL). This difference between the genders is also present in the occurrence of post-traumatic stress disorders among parents. For this reason there is a need to recognise individual family dynamics and officially acknowledge pivotal role of mothers & evolving role of fathers, as informal carers.
Schooling & Education Opportunities
As a child begins cancer treatment, their daily activities change with attendance at school being disrupted because of the illness. This can contribute to longer-term socioeconomic impacts stretching into adulthood, including financial hardship & difficulties in accessing health insurance and securing work.
Children with cancer may experience a wide range of vulnerabilities and lower academic achievement than peers without cancer, whilst adult childhood cancer survivors can have worse health related QoL than the general population. This could affect their subsequent school performance and educational attainment, as it has been found that survivors are at higher risk of not going on to further education after comprehensive school.
While re-entry into formal education can be seen as a positive milestone, it is not without difficulties. Re-entry to school has been found to be very challenging for most children with cancer due to treatment side-effects, prolonged absences, disrupted peer relationships, lack of preparation and lack of communication between schools, families and healthcare professionals.
Further social & socioeconomic impacts affecting cancer survivors in adult life have been identified with some survivors reporting difficulties in accessing healthcare or maintaining secure employment.
Minorities & Undeserved Groups
In addition to the factors described above, there are factors affecting the ability of different groups of children to access and benefit from treatments. Migrant & refugee children with cancer face specific challenges when using health services in their new countries. These challenges can sometimes span multiple generations. Cultural, religious and ethical concerns can affect the ability of patients and families to access care. Roma people and other traveller communities are also known to experience barriers to accessing health care. These include lacking the required documentation, facing discrimination, lack of cultural awareness, linguistic and health literacy barriers, economic barriers relating to healthcare costs, and travelling to appointments.
Cancer stigma in some cultures can limit the ability of patients & their families to seek social support, hence talking to other affected families is a potential solution. Where cancer is linked with guilt, self-blame and stigma, there must be ensured social support for families to determine mutually agreed approaches for medical discussions to increase collaboration between family & medical team.
Conclusions
Often, one parent must put its professional life on hold to care for its sick child while the other must free up the significant time to look after family. For this reason our community ask for a formal recognition of informal carers with sustainable plan to save all families from the harm of poverty, which calls for a specific financial compensation for families who need to seek treatment (within & between countries) & ensuring education facilities to support childhood cancer inpatients and children & adolescents who are receiving care out of hospital but are too ill to attend school.
Read full responseMeeting with Stella Kyriakides (Commissioner) and European Society for Paediatric Oncology 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
CCI-Europe is thankful for the opportunity to give feedback on the revision of the EU General Pharmaceutical Legislation.
CCI-Europe is the biggest pan- European childhood cancer parents’, survivors’ and patient representatives’ organisation (66 organisations in 33 European countries) that advocates patient-centred treatments and research via inclusion, collaboration and transparency.
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 because most standard treatments still include old cyto-toxic drugs. There are over 60 individual paediatric cancer types, each further sub-divided by stage, biology…
As a result, young patients (children, adolescents and young adults) facing cancer experience specific issues that differ from other age groups and diseases. Young patients need a specific protection with respect to the availability and affordability of drugs to cure them and prevent adverse side-effects.
The main issues faced by our community (save for the lack of access to innovation, discussed separately in the framework of the revision of the Paediatric and Orphan Medicines Regulations) are:
1. Unequal access to affordable drugs in all Member States (MS)
Some drugs, whether curative or supportive, are not available in all MS, despite the fact that they are identified as “standard of care” in international treatment protocols. This can relate to basic drugs as pain management.
This issue, among other causes, causes inequalities between MS where survival chances for young patients can differ by up to 20%.
We want the pharmaceutical legislation to include provisions that incentivise companies that market their drugs in all MS and penalise those that do not.
2. Risks of shortages
Economic drivers lead to shortages of cheap decades-old drugs despite the fact that (i) they are standard-of-care in many paediatric cancer protocols and (ii) there is no alternative to those drugs.
We plead for the identification at a European level of those essential drugs and for a coordinated, end-to-end approach to prevent drugs shortages.
3. Reimbursement of standard of care drugs
Many (sometimes old) drugs are used off-label in paediatrics and in oncology. In several MS, drugs are not reimbursed when they are used off-label, which creates an access issue for the patients even though those off-label drugs’ safety and efficacy are well demonstrated and regarded by specialists as standard-of-care.
We want essential drugs used off-label, including supportive care, to be reimbursed in all MS.
4. Affordability and availability of innovative drugs
New drugs in paediatric oncology are expensive, which can lead to: (i) great delays in their availability in many countries (longer evaluation procedure prior to their reimbursement) and (ii) a plain unavailability in some countries.
We are advocating new regulations (i) to create a more transparent environment around the pricing of new drugs and (ii) to curb the pricing of innovative drugs. Likewise, we want the General Pharmaceutical Legislation to support the availability of new drugs in all the MS (no “pick and choose” of “interesting” MS).
5. Need for international harmonisation
We already mentioned that paediatric cancers are all individually rare, which means that the drug development process requires a collaborative environment among countries, beyond the European Union.
This means that clinical trials often need to be conducted simultaneously with other industrialised countries. The diverging regulatory requirements to authorise new drugs is a barrier to their development in rare indications.
We request a greater harmonisation of the pharmaceutical regulation at an international level.
6. Alignment with new technologies
The Commission’s intent to align the Legislation with the new technologies, such as genomic sequencing or artificial intelligence, is pertinent
Read full response