SACHA: a Pediatric Cancer Registry
SACHA (Securing and Accelerating access to innovative therapies for CHildren and Adolescents with cancer) is an international observational study coordinated by Gustave Roussy. It aims to secure access to innovative anticancer medicines for children, adolescents and young adults (AYA) outside of clinical trials, while collecting real-world evidence (RWE) of safety and efficacy to inform future research and regulatory decisions.
Launched in France in 2020, SACHA extended across multiple countries from 2023 thanks to Fight Kids Cancer.
Rationale & objectives of the SACHA Study
SACHA was developed to address the urgent need of adequate collection of safety and efficacy data of compassionate and off-label use of innovative therapies in pediatric oncology.
The project has several key goals:
- Securing early access to innovative anticancer treatments outside of trials
- Collecting structured real-world data (RWD) on safety and potential efficacy
- Identifying early efficacy signals to support future clinical trials
- Supporting regulatory and ethical frameworks for compassionate use programs
- Promoting international collaboration and data sharing in pediatric oncology
Methodology of a Real-World registry-based study
SACHA is a prospective, non-interventional, multicenter cohort study. It includes patients aged 0–25 years treated with innovative anticancer therapies used outside of clinical trials. Inclusion is based on a compassionate or off-label use of an innovative anticancer therapy validated by a multidisciplinary tumor board.
The SACHA study does not provide any treatment recommendations, but collects the safety and efficacy data of those prescriptions
For each therapy interest clinical data is analyzed by each country national coordinator, and the pharmacovigilance Team at Gustave Roussy reviews remotely all related adverse drug reactions including CTCAE ≥2 clinical or ≥3 biological. For hematological events only transfusion-requiring thrombocytopenia/anemia or grade 4 neutropenia ≥7 days. Generated CIOMS forms are then sent to the national coordinating center.
SACHA is recognized as a real-world data source by the French Health Technology Assessment institution (French National Authority for Health, HAS) and strongly supported by the French National Agency for the Safety of Medicines and Health Products (ANSM). SACHA is listed in the HMA-EMA catalogue of real-world data sources and supported by the French Society of Pediatric Oncology (SFCE) and the Innovative Therapies for Children and Adolescents with Cancer Consortium (ITCC).
SACHA eligibility criteria
SACHA Patients
- Patients are ≤ 25 years-old with a recurrent/ refractory pediatric malignancy
- Patients are not eligible in a clinical trial or with no therapeutic option available in an ongoing trial
- Patients are treated with a new anticancer medicine through either off-label or compassionate use
- SACHA treatments are recommended by a multidisciplinary tumor board.
SACHA therapies of interest
- Any innovative drug through compassionate use
- Any anticancer medicine with a first European Marketing Authorisation after 2007 used off-label
Progress to Date
Initial implementation of the SACHA observational study (RIPH3) at Gustave Roussy
Inclusion of the first patient in Austria
Inclusion of the first patient in Spain
Publication of the landmark SACHA-France study
Inclusion of the first patient in the UK
Inclusion of the first patient in the Denmark
Publication of the first ancillary study linked to the SACHA study
Inclusion of the first patient in the Italy
Launch of the SACHA International website
SACHA France proof-of-concept
Prescriptions number per year (France)
114 prescriptions
187 prescriptions
193 prescriptions
57 prescriptions
as of July 2025
monitored
