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IMI Launches Significant Neurofibromatosis Call for Patient-Centric Drug Development

The Children’s Tumor Foundation is an Associated Partner of the European-based Innovative Medicines Initiative


UPDATE: The recording of the “Integrated Research Platforms Enabling Patient-Centric Drug Development” presentation can be viewed here and the presentation can be downloaded here. In this presentation, neurofibromatosis (NF) is presented as the case study for rare diseases, with Children’s Tumor Foundation President Annette Bakker, PhD as the package lead for NF. Watch the presentation to learn more about the objectives of this transformative project, which include the development of best practices, tools and platforms for multi-company platform trials, and the development of platform trial designs with clinical and patient data networks. Read below for additional details on this major advancement for NF research.

The Innovative Medicines Initiative (IMI), a jointly funded partnership between the European Union (EU) and the European Federation of Pharmaceutical Industries and Associations (EFPIA), will soon issue a call for an integrated research platform enabling patient-centric drug development. The Children’s Tumor Foundation (CTF) has strongly advocated for the inclusion of neurofibromatosis (NF) as a rare disease in the call, and we are thrilled to announce that not only is CTF an IMI Associated Partner, but that NF is included in the upcoming IMI call.  CTF and IMI will work together to establish integrated research platforms that enable more effective and accelerated drug development in areas of unmet medical need.

As the world’s largest private/public partnership in the life sciences, the IMI brings the best minds together to turn knowledge into treatments by speeding up vital research in order to overcome serious medical challenges faced by patients. Funded by residents of the European Union (represented by the European Commission), as well as leaders in the pharmaceutical industry (represented by EFPIA), the IMI facilitates open collaboration, cross-sector coordination, and accelerated patient access to medicine. The organization currently has an overall €3.3 billion budget ($3.8 billion), as well as the support of major global pharmaceutical companies for its initiatives.

The U.S.-based Children’s Tumor Foundation, the largest nongovernmental funder of NF research in the world, has developed an innovative research model aimed at translating discoveries into treatments, and then bringing those treatments to trials more quickly and at a lower cost. This unique research model, called Synodos, is driven by a collaborative open science platform consisting of multidisciplinary experts from across the globe, real-time data sharing, and patient-driven insights at every step of the process.  This collaboration with the IMI represents a significant extension of a coordinated global effort to tackle the complications of NF, and also demonstrates the impact of NF research as a prototype for other rare diseases.

“NF knows no geographic boundaries,” said Annette Bakker, PhD, President of the Children’s Tumor Foundation. “We have committed to expanding the focus of NF so that we increase knowledge about the disease at all levels, attract the involvement of the world’s best researchers and pharmaceutical companies, and provide answers, and hopefully soon effective treatments, for NF patients wherever they may be.”

The IMI is conducted an online webinar, open to all researchers, that provided information about this call, entitled “Integrated Research Platforms Enabling Patient-Centric Drug Development”, on July 5, 2018. The presentation can be viewed here and the presentation can be downloaded here.

To learn more about the specifics of the IMI call, click here.

To learn more about the NF Conference, the world’s largest gathering of NF experts, taking place in Paris in November 2018, click here.