Context of the SACHA Study
France stands as a pioneer in Europe in the field of developing new drugs in pediatric oncology. For example, from 2015 to 2018, over 2,200 patients were enrolled in 34 clinical trials by ITCC (Innovative Therapies for Children with Cancer). Among them, 53% of the patients were recruited in France. However, despite this strong momentum, the therapeutic offerings remain insufficient, and pediatric oncologists and hematologists prescribe innovative drugs either within the framework of compassionate or early access or off-label use for drugs already authorized in adults. For all these prescriptions, safety and efficacy data are rarely collected.
About SACHA
With the aim of securing access to these innovative therapies outside the framework of clinical trials, the New Drugs Committee of SFCE (French Society of Pediatric Oncology) has established an observational prospective cohort study to monitor the use of innovative molecules in oncology and hematology for children, adolescents, and young adults experiencing treatment failure or relapse and who are not eligible for a clinical trial. This is the SACHA program, which is a French observational study involving prospective collection of toxicity and efficacy data of innovative therapies administered off-label and outside of clinical trials.
The data are collected through a validated and secure pharmacovigilance tool (VIGINOM). This study does not modify current clinical practice, whether in diagnosis or in the selection and administration of treatment, its monitoring, and follow-up.
The objectives of the SACHA study are:
- To regulate and secure access to innovative therapies for children, adolescents, and young adults experiencing treatment failures and not eligible for a clinical trial, whether they are targeted therapies, immunotherapies, or chemotherapies.
- To regulate off-label prescriptions and requests for compassionate use or named patient access.
- To secure the use of medications through organized patient follow-up.
- To evaluate the tolerance and efficacy of these treatments under real-world conditions.
- To draft recommendations.
Following the SACHA program, expanded access programs were reformed in July 2021, which led to the creation of Early Access and Compassionate Access programs, strengthening the requirement to collect data.
SACHA does not promote off-label prescribing or compassionate use of innovative therapies but advocates for the systematic discussion of all patients aged 25 or younger experiencing cancer relapse during regional multidisciplinary tumor board meetings and strongly supports their inclusion in timely clinical trials.
Results of the SACHA France Trial:
- All 31 SFCE centers have enrolled at least one patient in the SACHA study.
- 731 patients have been enrolled.
- The main types of tumors recorded are: brain tumors (44%), extra-cerebral solid tumors (41%), and leukemias (15%).
- 63 new monotherapies and 16 combination therapies have been recorded.
- Researchers involved in the SACHA study have worked closely with ANSM (French National Agency for the Safety of Medicines and Health Products).
- HAS (French National Authority for Health) has recognized SACHA as a mobilizable data source.
- Presentation of SACHA France results at the ASCO (American Society of Clinical Oncology) congress in 2022 and 2023.
- Publications: Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study – PubMed (nih.gov) and Chemo-immunotherapy with dinutuximab beta in patients with relapsed/progressive high-risk neuroblastoma: does chemotherapy backbone matter?.
SACHA International:
SACHA International builds on the experience of the SACHA France pilot initiated by the French Society of Pediatric Oncology (SFCE), the success of which confirmed the need for such a registry. SACHA International is an initiative of the ITCC (Innovative Therapies for Children with Cancer) Consortium and plans to enroll 500 patients per year for three years in several European countries, as well as in Australia and New Zealand.
The consortium agreement was signed in May 2023 between France, Spain, the UK, Ireland, Denmark, and Austria (with the first patient enrolled outside of France on June 14, 2023), and the internationalization of SACHA was funded by Fight Kids Cancer. To date, 48 patients have been registered outside of France.
For more information: Interview with Dr. Berlanga
Summary of SACHA France and International:
Promoter: Gustave Roussy Institute
Principal Investigator: Dr. Pablo Berlanga
Program Duration: April 2020 – December 2026
Start of Inclusions: March 2020
End of Inclusions: 2026
Number of Expected Patients: 2,231
Countries Involved: France (all SFCE centers), Italy, Spain, UK, Ireland, Netherlands, Belgium, Austria, Denmark, Australia, and New Zealand
Imagine for Margo Funding: €996,000 (in partnership with SFCE for the France part and Fight Kids Cancer funding for the international part). This large program was co-financed by the Rallyes du Cœur de Versailles in 2019 and 2021 (SACHA France) and the Children Without Cancer Race 2023 (SACHA International – Fight Kids Cancer).
The initial results of this study were presented orally at the American Society of Clinical Oncology (ASCO) on June 6 by Professor Gilles Vassal, pediatric oncologist at Gustave Roussy.
