The Children’s Tumor Foundation co-leads the NF work package in EU-PEARL (EU Patient-Centric Clinical Trial Platforms), a €26 million (Innovative Medicines Initiative) IMI-funded collaboration among European academic and industry leaders in NF. This exciting multi-stakeholder project that will provide the framework for designing and executing cross-company Integrated Research Platforms, and importantly, faster and better clinical trials to benefit patients.
CTF Europe collaborates with CTF United States on an IMI Joint Undertaking through EU-PEARL, providing in-kind contribution. The project receives support from the European Union’s (EU) funding program, Horizon Europe, 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 42-month project aims to shape future clinical trials that will be more patient-friendly by design and patient-focused by outcome in four separate disease areas: Major Depressive Disorder, Tuberculosis, Non-Alcoholic Steatohepatitis and Neurofibromatosis. 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 utilised 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. For the purposes of the project, a network of clinicians needed to be set up in order to exchange information between these specialized clinical care centres for the treatment of NF patients.