The Children’s Tumor Foundation and the Global Coalition for Adaptive Research are joining forces and using EU-PEARL’s templates for platform trials to treat patients with neurofibromatosis-1 (NF-1) and schwannomatosis (SWN). This collaborative effort leverages the innovative EU-PEARL master protocol to establish a first-of-its-kind clinical trial, marking a significant stride in addressing the high unmet medical needs of those suffering from these rare genetic conditions.
Commenting on the new trial, Anton Ussi (EATRIS Operations & Finance Director) said: “It is so gratifying to see the great work of the IMI-funded EU-PEARL project being used in a game-changing platform trial for patients with neurofibromatosis-1 and schwannomatosis. It is a testament to the power of public-private collaboration and innovation in advancing our understanding of and ability to tackle difficult rare diseases.”
The announcement of this trial builds on a 3-year, 26 million euro investment from the Innovative Health Initiative (IHI). EU-PEARL was a public-private partnership dedicated to transforming clinical trials by making them more efficient and patient-centred. Through this partnership, 36 leading organisations representing patients, clinicians, researchers, health authorities, research infrastructures (including EATRIS) and industry collaborated. A generic framework and a set of tools were developed to conduct patient-centric collaborative platform trials to enhance and expedite the medicine development process, potentially providing quicker access to novel and more effective treatments.
By negotiating clinical trial site agreements upfront and allowing patients to transition between treatments, these trials significantly reduce patient recruitment times and offer individuals a second chance for potentially life-changing medications. This approach can also minimize the use of placebos and can be more time-effective than traditional clinical trials.
The framework established by EU-PEARL provides a “plug and play” system for drug development, offering cost-effectiveness and the ability to assess a drug’s viability within approximately 36 months. The Children’s Tumor Foundation, in partnership with the Global Coalition for Adaptive Research, is actively selecting sites and engaging pharmaceutical companies to participate in the trial, promising an efficient and streamlined process for testing diverse drug candidates.
Dr Annette Bakker, President of the Children’s Tumor Foundation, said: “Without this unique opportunity to bring all stakeholders together within the context of the EU-PEARL project, the design of this platform trial wouldn’t have happened.”
Pharmaceutical companies interested in exploring this opportunity are encouraged to contact The Children’s Tumor Foundation’s Chief Medical Officer (bgalerik@ctf.org). Additionally, the foundation is organising a conference in Brussels, Belgium, from 20-25 June 2024, providing a platform to disseminate information and foster collaboration in the pursuit of groundbreaking treatments for NF-1 and SWN patients. Find out more about the conference here.
- Read the official press release for the announcement here.
- View a news item on the IHI website about this story here.
- Link to the EU-PEARL project page on the EATRIS website.
- Visit the EU-PEARL website here.
