Revolutionizing rare disease clinical trials: the Children’s Tumor Foundation is shaping the future
The Innovative Health Initiative (IHI), the European Union public-private partnership funding health research and innovation, recently highlighted CTF’s leadership in spearheading a pioneering platform trial for patients with neurofibromatosis or schwannomatosis. Collaborating with the Global Coalition for Adaptive Research (GCAR), CTF is leveraging the groundbreaking framework it developed with EU-PEARL to revolutionize rare disease clinical trials.
By championing a ‘plug and play’ system, CTF is ushering in a new era of cost-effective and time-efficient drug development, with patients at the forefront. This visionary approach offers access to potential life-changing medications and addresses critical challenges in patient recruitment and trial design. Each drug plugged into the trial costs an estimated $2 million per year, offering a cost-effective and time-efficient approach to drug development. Within approximately 36 months, clear signals regarding the efficacy of drug candidates can be obtained, a departure from traditional clinical trial timelines. Patients enrolled in the trial have continuous access to treatments, promising improved outcomes. By addressing the key challenges in patient recruitment and trial design, CTF and its strategic partners are setting a new standard in NF and rare disease research.
As these platform trials gain momentum, pharmaceutical companies are invited to partner with CTF to accelerate the discovery of innovative treatments, making this a pivotal moment in the fight against tumors.
To read the article from the IHI, visit here.
To read the press release announcing the CTF and GCAR strategic alliance, visit here.
To contact the Children’s Tumor Foundation, visit here.
