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Anticancer Fund

ACF

The Anticancer Fund is dedicated to expanding the range of treatment options available to patients, regardless of their commercial value.

Lobbying Activity

Meeting with Sirpa Pietikäinen (Member of the European Parliament)

1 Oct 2025 · Cancer

Meeting with Estelle Ceulemans (Member of the European Parliament, Committee chair)

5 Dec 2024 · Dossier SANT à venir

Meeting with Tilly Metz (Member of the European Parliament) and European Patients' Forum (EPF) and

10 Oct 2023 · Pharma Package

Meeting with Tilly Metz (Member of the European Parliament) and Association of European Cancer Leagues

2 May 2023 · Revision of the Pharma Package

Response to Evaluation and revision of the general pharmaceutical legislation

26 Apr 2021

The European Commission states its intention for its Pharmaceutical Strategy for Europe to be ‘patient-centred’. However, it is crucial that the concept of ‘patient value’ is integrated in the pharmaceutical legislation. It is not sufficient that the EU pharmaceutical system simply ensures quality and safety of medicines, but has in place procedures to ensure that pharmaceuticals bring real added benefit to patients. Assessment of the added clinical benefit (overall survival/quality of life) of a pharmaceutical is currently not within the remit of the European Medicines Agency, so market authorization applicants are therefore not obliged to deliver data (overall survival, quality of life or patient-reported outcomes) that are crucial for informed decision-making by Health Technology Assessors (HTA), payers, treating physicians and ultimately patients. It should therefore be mandatory for HTA to provide input to the design of clinical trials to ensure they address both the patient’s needs and the evidence needs of both regulatory authorities and downstream decision makers. Currently, the European Medicines Agency (EMA) does not require manufacturers to submit individual participant level data from clinical trials to inform its approval decisions. This should become an evidentiary requirement to maximise learnings from this data beyond the specific trial research question. In addition to supporting development of new drugs, these data should also meet the FAIR (Findable, Accessible, Interoperable, Reusable) criteria and be available at no or reasonable cost for comparative effectiveness research by a pre-defined range of third parties (e.g. HTA-organisation networks) to inform health care systems. In order to stimulate innovation and breakthrough therapies in areas of unmet need, third parties having no commercial intent (academia, research foundations, European Reference Networks, collaborative groups) should be incentivized to apply for regulatory approval of medicines not requiring industrial development (combinations of repurposed drugs, autologous cell therapies). In the case of repurposed drugs, public-private partnerships of third parties with drug producers should be supported by a legal system that allows the Market Authorisation Holder (MAH) to rely on data generated by an independent third party to apply for label extension. Scientific advice and regulatory filing should be at minimal cost but will ultimately lead to affordable medicines. Innovative drugs, in particular Advanced Therapy Medicinal Products (ATMPs), often receive regular approval, albeit with incomplete data and uncertain therapeutic benefits. When only incomplete data are available, EMA should routinely use the ‘conditional marketing authorisation’ pathway. EMA, with input from European Health Technology Assessment bodies, should require post-marketing studies that directly address the uncertainties about the drug benefits and harms at the time of approval. Infrastructures to facilitate the collection of real-world evidence and real-world data to confirm proof of efficacy must be prioritised, and investment should be made in data registries which use harmonised standards. It is imperative to allow the transfer of information across borders and as such the General Data Protection Regulation (GDPR) must not be an impediment to collection and sharing of patient data in such a way. The COVID-19 pandemic emphasised the need for the EU’s competences in health to be expanded. Assuming that joint clinical assessment by a European HTA will become a reality soon, an important task could be dealing with false, unproven therapeutic claims. Especially in the domain of cell therapy and oncology, the offer of miracle treatments thrives in Europe, putting at risk patient safety. However, debunking false claims is not currently within the remit of the EU.
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Response to Revision of the EU legislation on medicines for children and rare diseases

5 Jan 2021

The Anticancer fund applauds the awareness of the commission that in oncology common cancers are split into many subsets based on biomarkers, leading to unnecessary multiplication of rare diseases. Therefore, we support the idea that it will no longer be possible to obtain orphan designation for subsets of common diseases. New scientific developments in oncology, such as the successful implementation of immunotherapy (e.g. checkpoint inhibitors), requires accommodation of the regulation since the same medicine can potentially be beneficial for patients with different cancer types, from most common (e.g. lung cancer) to ultrarare (e.g. chordoma). Moreover, depending on the type of disease combination of these products with approved drugs can be effective and add value for patients. Differential pricing of the same drug based on the indication should not be acceptable, not least because it will lead to inappropriate off-label use. Drug producers will preferentially apply for market authorization in the most common indications in order to maximize their financial return in order to serve their stakeholders, who rightly expect return on equity. Dedicated direct funding for independent clinical research by collaborative groups, ERNs, academia, non-profit research foundations (e.g. EORTC) linked to readily available and mandatory free scientific advice from EMA, , could guarantee that patients with rare and paediatric cancers gain equal access to innovative treatments. Private Public Partnerships with the drug producer should be binding for companies to provide drugs for clinical trials in exchange for the clinical study data that they have to submit to EMA in order to obtain label extension (Type II variation). Making this Type II variation free of charge for rare and paediatric diseases could be an important incentive for pharma companies. In this ground-breaking scenario the return on the public investment in terms of societal benefit could be guaranteed. Today access to expensive innovative treatments for patients is often hampered by the delay of HTA approval and reimbursement following market authorization by EMA. This is mainly due to the lack of evidence of added therapeutic value and comparative effectiveness data crucial for HTA approval but not within the requirements for EMA market authorization. If registration trials for rare and paediatric cancers could be designed and executed by collaborative groups, ERNs, academia and non-profit foundations who take into account research questions from patients/health professionals and include relevant comparators and endpoints they will consequently be able to deliver appropriate data for HTA evaluation. Platform trials could be the preferred format since they will allow direct and efficient comparison of different treatments to one control group. The same scheme could also be used to boost the development of possible new uses of old and off-patent products for paediatric and rare diseases. At the same time it would avoid immoral drug repurposing (product hopping) with excessive prices and artificial withdrawals from markets etc. (e.g. Leadiant case) and incentivize clinicians who are using drugs off-label in daily practice to initiate proper market authorization. In blood cancers today there is tsunami of new “orphan drug” development in cell and gene therapy. Despite “orphan” status of the disease indications we notice a multiplication of “conceptual me too” products in development for the same molecular targets (e.g. CD19 CAR-T cells). The definition of “unmet need” does not take into account products in development. Are these redundant developments really serving patients and society? Is the current trial landscape in these diseases not hampering the development of real innovative treatments with different mechanisms of actions? Should a “me too” product not be only conditionally approved requiring added therapeutic value to the product that came to market first?
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Response to Pharmaceutical Strategy - Timely patient access to affordable medicines

7 Jul 2020

Clinical evidence The Roadmap states that ‘new therapies need to be clinically better than existing alternatives. We emphasise the importance of defining ‘clinically better’ and believe robust methodologies for obtaining evidence on efficacy and effectiveness of new treatments are crucial. We recommend: Adequately designed trials (preferably multi-arm) allowing independent comparison between treatments, using the correct comparator. Early involvement of HTAbodies so as to ensure trials provide the necessary data for timely HTA. Consistent definition of patient-centred endpoints and inclusion of PROMs in clinical trials. Fast-tracked conditional approval in the case of unmet need (important in oncology as timely treatments may be life-saving). In oncology conditional approval based on surrogate endpoints can be acceptable in the case of unmet need, but post-authorisation studies or registry-based trials are crucial to prove effectiveness and value for patients and society. Evaluating clinical effectiveness (in oncology a drug is often only part of combined treatment administered to patients, and is rarely given without any other therapy and as such its clinical value in a real-world setting must be demonstrated). While safety and efficacy is an important first step in the evaluation process, it is not sufficient to demonstrate added clinical value for patients and society. Prioritisation of new therapies based on unmet need and aligned with public health and health systems requirements While the Roadmap cites the need for a ‘holistic, patient-centred, forward-looking EU Pharmaceutical Strategy’, we highlight the importance of prioritising new therapies that serve unmet needs versus ‘me-too’ products. ‘Innovation’ should be defined as ‘new approaches to solve a challenge’ and should not be a synonym for high-tech solutions or new products. Finding new uses for ‘old’ drugs should also be considered innovative. Health system preparedness The Roadmap states that ‘health systems may need to be better equipped to ensure deployment and uptake of innovative solutions’. We believe that Health systems should independently ensure deployment and uptake of solutions with proven benefit for patients and society, with emphasis on ‘proven benefit’ rather than innovation Affordability of medicines An overarching goal of the Pharmaceutical Strategy is to ensure Europe’s supply of safe and affordable medicines. There are currently 280 generic drugs that are expected to be beneficial for cancer patients. Also in other disease areas there are numerous repurposed drugs with exploratory evidence and especially for rare diseases these drugs could be an unique and accessible source of treatment options. Drug repurposing offers safe and affordable solutions, yet its potential remains untapped due to lack of funding, incentives and infrastructure. We recommend that these barriers are addressed urgently. EU cooperation on Health Technology Assessment We recommend the following: Common clinical evaluation is an absolute requirement (as proven for COVID-19) The same clinical data from trials cannot be evaluated differently in different Member States. A European HTA body is needed to provide input in the design of the registration clinical trials that provide data to answer both the research questions from EMA and HTA Consultation with stakeholders The Roadmap states that ‘the Strategy will be supported by comprehensive consultation activities with a wide variety of stakeholders, including associations representing patients and consumers, healthcare professionals, the pharmaceutical industry, include SMEs, academia…’ Non-profit research organisations who do not fall under the definition of ‘academia’ (such as the Anticancer Fund) as they are not affiliated with universities, are not listed as stakeholders. This should be addressed urgently and a new category of stakeholders listed which covers such organisations.
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Response to Europe’s Beating Cancer Plan

27 Feb 2020

The Anticancer Fund supports the objective of increasing survival rates and improving quality of life of cancer patients by providing access to the best possible treatments for all. However, studies have shown that the current European system for clinical research, approval and market access of new cancer medicines is failing to achieve this goal. To ensure that the best treatment reaches the patient, Europe’s beating cancer plan should address the following. First, the current regulatory system is focused on approval of a single medical product based on safety and efficacy, NOT on added therapeutic value. The ongoing discussion about suitable endpoints for drug approvals should aim at clinical relevance for patients, and in that context surrogate endpoints could only be accepted to allow fast access if there is a high unmet need and should always be linked to conditional approval and post-authorization effectiveness data collection. To provide the best possible treatment to patients, we recommend strengthened European cooperation in HTA (and preferably joint clinical assessments), earlier dialogue between EMA and HTA organisations, and the requirement for registration studies with robust and meaningful clinical endpoints. Ideally, registration trials should be designed by independent trialists in a patient-centred and efficient way, enabling reliable and timely access to evidence based treatment options. Nowadays companies understandably design their registration trials to maximize their chances of success and financial return. For rare cancers (including paediatric cancers), platform trials managed by collaborative groups could be the preferred format since they will allow direct and efficient comparison of different treatment options. Europe should explore innovative trial designs and could set up a clinical trials transformation initiative (www.CTTI-clinicaltrials.org) in dialogue with the US. Continuous proper registration of cancer treatment outcomes should be enforced by European legislation as these data will be crucial for cost-effectiveness calculations. Second, The Anticancer Fund recognises the need for companies to create value for their shareholders and therefore to develop treatments with high return on investment. However, this should be compensated by non-commercial health stakeholders identifying societal health priorities and investing in areas where there is little or no financial interest, such as drug repurposing and rare cancers including paediatric cancers. A symbiotic relationship between commercial stakeholders and public authorities, foundations, academia, patients and healthcare professionals as non-commercial stakeholders needs to be incentivised. Third, Europe’s Beating Cancer Plan must be developed synergistically with the EU pharmaceutical strategy. The pace at which science is revealing the underlying biology of cancer and how this information is used to develop innovative therapies is unprecedented. As a result, combination treatments are becoming commonplace but the approval system and the price setting framework are not fit for purpose as they are designed to approve one medicine for one indication - something which today applies very rarely in the oncology field. Fourth, Europe must make efforts to ensure a transparent system is in place for provision of information to patients about “the best treatments”. In contrast to FDA, EMA is not policing unproven health claims because this is the legal responsibility of Member States. Unproven health claims and the marketing and selling of cancer treatments over the internet is increasing and is happening across borders. Health fraud scams should be monitored at the European level and actions towards companies and website operators taken in coordination with Member States. Monitoring could be undertaken by the European Cancer Knowledge Centre already suggested in the plan as a possible instrument for connecting data and scientific evidence.
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