In recognition of the tremendous opportunities to develop and advance neurofibromatosis (NF) research in Europe, the Children’s Tumor Foundation started engaging more actively in Europe since 2017. As we celebrate today the second anniversary of the European partner of CTF, the Children’s Tumor Foundation Europe, we are delighted to share a success story that demonstrates the results of CTF’s efforts in Europe and its dedication to End NF globally.
While the European Union has prioritized policies around rare diseases in the past few years, especially through the creation of the European Reference Networks, there has been limited funding attributed to NF research. The European Union dedicates a large amount of funding to Research and Innovation either through research and development projects, regional collaborations or through public private partnerships.
Persisting in the mission to End NF, CTF Europe has advocated successfully to include NF among other disease areas (namely Major Depressive Disorder, Tuberculosis and Non-Alcoholic Steatohepatitis) in a €26 million project under the auspices of the Innovative Medicines Initiative (IMI), entitled EU-PEARL (EU Patient-Centric Clinical Trial Platforms). The 42-month project is the largest European funding project that has been dedicated to NF. The project receives support from the European Union’s (EU) funding program, Horizon 2020, in-kind contribution by pharmaceutical and biotech companies that are members of the European Federation of Pharmaceutical Industries and Associations (EFPIA), and the IMI Associated Partners.
The multi-stakeholder project expects to provide the framework for designing and executing cross-company Integrated Research Platforms that can be used in these four and other diseases, such as rare diseases, in which NF is being utilized as a prototype example. Alongside the Erasmus University Medical Centre in Rotterdam, CTF is co-leading the work package on establishing an Integrated Research Platform on NF. Specialized clinical care centres for the treatment of NF in Europe are benefiting from the European funding to advance research for NF patients.
