Christopher’s Smile is actively campaigning to make more new targeted drugs available earlier to children who desperately need them.
While we are confident that our funded research will make a real difference to children with cancer we increasingly believe that research in isolation will not achieve the step change needed in timely introduction of new agents. The time lag between agents being initially available for adults and then subsequently available for children is unacceptable. Unless more agents are made available sooner, the harsh protocols that can leave children with a legacy of issues will remain with us for decades rather than years.
Enable access for academic paediatric researchers to all agents following a successful Phase 1 trial in adults.
PIP waivers can only be sought if the agent’s mechanism of action does not occur in a known paediatric condition.
Following a successful Phase 2 trial in adults, allow academic paediatric researchers to test an agent in combination with any agent from any manufacturer (conditional on the same permissions from all parties).
The earlier development of treatments for some children who presently cannot be saved.
The earlier development of treatments for some childhood cancers where the current treatments leave an unacceptable legacy of health issues.
An increase in the number of approved oncology drugs for children.
Work to start on combination therapies at a much earlier stage.