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Children's Tumor Foundation Europe

CTF Europe

The Children’s Tumor Foundation Europe (CTF Europe) is a Belgian Foundation of Public Utility, a non-for-profit medical foundation dedicated to revolutionize the field of neurofibromatosis (NF) research with the aim to accelerate treatments for NF Patients.

Lobbying Activity

Meeting with Pascal Arimont (Member of the European Parliament)

18 Feb 2025 · Rare disease policy

Meeting with Tomislav Sokol (Member of the European Parliament) and Eli Lilly and Company and European Federation of Associations of Health Product Manufacturers

16 Oct 2024 · Health policy

Response to Revision of the EU legislation on medicines for children and rare diseases

18 Dec 2020

The Children’s Tumor Foundation (CTF) Europe welcomes the European Commission’s efforts to address shortcomings in the development of pediatric and orphan medicines through the evaluation of the EU legislative framework. CTF Europe is concerned with neurofibromatosis (NF), three rare genetic tumor suppression syndromes (NF1, NF2, Schwannomatosis), mostly starting as pediatric conditions with an overall incidence of 1 in 3000. We strongly welcome the Roadmap’s option outlined to improve orphan drugs availability and accessibility, where if a company loses commercial interest to a product it should be encouraged to offer or transfer it to another company rather than withdrawing it. We assess that: 1) there has to be particular attention to off-patent medicines and to compounds that become shelved due to the lack of economic incentive linked to their further development and 2) there needs to be a multi-stakeholder endorsed definition of ‘unmet medical need’ to avoid that a too narrow definition would disincentivize orphan drug development, with dire consequences for patients. As a Research Foundation, we face difficulties to access promising drug candidates for neurofibromatosis that have been shelved by pharmaceutical companies. Pharmaceutical companies often rather opt to shelve compounds instead of making them available for repositioning to address unmet needs in pediatric and rare diseases. Also, the preclinical expertise and rare diseases models often lie in academia, which further complicates the development of pediatric and orphan medicinal products. Foundations could have an important role in exiting shelved assets by developing a framework to efficiently exit these assets from companies and streamline the process. There are a few spot examples of foundations that have managed to reach that goal in the current context but EU-led policies are needed to incentivize such a process on a larger scale. While we appreciate that some amendments to the legislative framework may be necessary, particularly on the Orphan Medicines Regulation, we consider that policy actions would be more effective. We encourage policy action to: - Optimize a system of incentives for pharmaceutical companies to 1) release all data on off-patent compounds and 2. release shelved assets (and their associated data) that have the potential to become effective treatments for pediatric and rare diseases. The latter will allow third parties to reposition them and cost effectively develop treatments for these indications. - Within the system of incentives, the industry should also open their drug pipelines to academia to break the silos and assist in the development of orphan medicinal products. Additionally, policies should be developed to ensure that industry can easily access the preclinical models that are currently silo-ed within academia. We recommend that the construction of a truly multi-stakeholder (industry, research and healthcare communities, regulators) community including the nonprofit sector will be crucial to expedite the development and approval of more efficient treatments for patients. - Pediatric research infrastructure and additional clinical trials support would be important to drive research in areas of unmet medical need, within the remit of a multi-stakeholder aligned definition. - Leveraging from international collaboration on pediatric and orphan medicines will be of critical importance, to ensure a faster, fair, efficient and predictable outcome for patients. - Critical investment in the development of creative/ innovative research business models through the EU funding Programs such as Horizon Europe and the future Innovative Health Initiative could contribute towards a faster process for drug development. Ultimately, providing incentives for the above issues could drive investment and bring more concrete outcomes for the treatment of pediatric and rare diseases.
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