The Children’s Tumor Foundation is a US-based not-for-profit organisation dedicated to finding effective treatments for the millions of people worldwide living with a rare genetic disorder, neurofibromatosis (NF). NF is a family of three distinct disorders: NF1, NF2, and schwannomatosis. NF can cause tumours to grow on nerves throughout the body and may lead to blindness, bone abnormalities, cancer, deafness, disfigurement, learning disabilities, and excruciating and disabling pain. The Children’s Tumor Foundation acts as a critical player in getting effective treatments to NF patients faster. In addition to benefiting those who live with NF, CTF will work with the broader rare disease community.

CTF has the following expertise that it is planning to contribute to the project:

• expertise in adaptive trial design and platform trial design;

• build hospital networks and technologies to utilise EHRs;

• design and implementation of disease registries and trial readiness cohorts;

• interact with health authorities;

• connect with NF clinical consortia;

• advocacy to expand leanings of NF experience to other rare disease communities;

• ability to serve as, or to identify, a trial sponsor for implementing the IRP.