SACHA International
Research type
Research Study
Full title
Secured Access to innovative medicines for CHildren, adolescents and young adults with cAncer (SACHA)
IRAS ID
317855
Contact name
Gerard C Millen
Contact email
Sponsor organisation
Birmingham Women's and Children's Hospital NHS Trust
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Childhood and adolescent cancers comprise a heterogeneous group of rare diseases, mostly distinct to those diagnosed in adults. Survival of children and adolescents with cancer has considerably improved in recent decades, but cancer remains the most common cause of disease-related mortality in developed countries. In the case of disease relapse, the prognosis for paediatric patients is poor with survival rates of <20%.
Better understanding of tumor biology has led to exponentially increased development of novel targeted therapies and immunotherapies in the last two decades. Several molecularly targeted medicines have shown significant activity in specific pediatric cancers in the presence of matching alterations. However, very few have been approved for the treatment of paediatric cancers.
Many children and young adults are currently receiving targeted therapies (usually in the setting of relapse) outside of formal clinical trials, often on a compassionate basis (given freely by the drug company) or "off-label" (paid for but with no license/marketing authorisation for that indication). Data is not routinely collected on the safety and activity of these medications.
The goal of the SACHA international project is to collect real world data on patients being treated with these medications with a particular emphasis on safety and activity. The aim is to share this information so that drugs with poor side effects or low activity levels are not used unnecessarily.REC name
West of Scotland REC 4
REC reference
23/WS/0054
Date of REC Opinion
20 Apr 2023
REC opinion
Further Information Favourable Opinion