The SACHA study supports the use of innovative anticancer therapies in children outside clinical trials
Villejuif, 7 june 2022
Although access to novel therapies in clinical trials has grown in France in recent years, there remain uncovered medical needs for the treatment of refractory paediatric cancer. When a child may not participate in a clinical trial or when no clinical trial of their cancer exists, paediatricians prescribe innovative medication outside clinical trials to respond in the best way to each patient’s therapeutic requirements. This becomes a matter of utilisation of agents authorised for the treatment of adults but not children or that still do not have Marketing Authorisation but for whom the French National Agency for Medicines and Health Products Safety (ANSM) authorises access on compassionate grounds or premature access.
The SACHA observational study is routinely collecting prospective data on a national scale on safety and efficacy of innovative therapies administered in this way. SACHA is collecting “real-life data”. This study is protecting safety of access to novel therapies by guaranteeing notification of any serious adverse events. If evidence for efficacy of a drug is observed, then everything is done to initiate a clinical trial to confirm whether or not this early evidence is valid. SACHA is a study launched by the French Society for Childhood and Adolescent Cancer and Leukaemia (SFCE), which is led and sponsored by Gustave Roussy in collaboration with the ANSM. It is open at 32 French paediatric oncology/haematology sites. It was presented at an oral session of the American Society of Clinical Oncology (ASCO) on the 6th June by Professor Gilles Vassal, onco-paediatrician at Gustave Roussy.
Abstract No. 10006 presented orally by Professor Gilles Vassal Monday 6th June 2022 at 11.36 pm
Access to innovative therapies for treatment of paediatric cancer has grown significantly in France in recent years via major research programmes such as AcSé-ESMART, clinical trials or marketing authorisation (e.g. larotrectinib authorised in children and adults simultaneously).
In addition, for a child or adolescent with a cancer relapse or refractory disease, it is now possible to have full sequencing of tumour DNA in the context of the France Génomique plan 2025, the feasibility and benefits of which were demonstrated by the MAPPYACTS (European trial) research programme. The findings have been discussed in inter-regional multidisciplinary meetings involving the 32 SFCE centres. The objective is to direct each child to a clinical trial of the most appropriate therapies, tailored to the abnormalities of their tumour. “The best way to offer access for a child to an innovative medication is to arrange for participation in one of a portfolio of available paediatric clinical trials,” explained Professor Vassal, paediatrician and oncologist at Gustave Roussy.
However, despite the strong motivation to broaden the panel of trials of new drugs, some children and adolescents are ineligible for one or no trial of the medication corresponding to the molecular profile of their tumour exists. The therapeutic offer remains insufficient. In such situations, paediatricians prescribe innovative medication outside clinical trials. This becomes a matter of utilisation of agents authorised for the treatment of adults but not children or that still do not have Marketing Authorisation, but for whom the French National Agency for Medicines and Health Products Safety (ANSM) authorises access on compassionate grounds or premature access. Prior to SACHA, data on safety and efficacy of these prescriptions were not collected or were treated anecdotally with no systematic official notification of serious adverse events.
Analysis of real-life safety and efficacy data
SACHA (Protecting safety of access to innovative oncological and haematological therapies for children, adolescents and young adults) was established in March 2020. It is a prospective, observational study sponsored by Gustave Roussy and conducted at 31 SFCE sites. It is collecting in a thorough manner data on safety and efficacy of innovative therapies administered to children, adolescents and young adults (under the age of 25) with relapse or who are refractory to therapy of a cancer and are not eligible for a clinical trial but have access on compassionate grounds or outside clinical trials.
The objective of the plan is to supervise and protect the safety of these prescriptions by establishing real-life organised monitoring. SACHA is also contributing to knowledge, in particular in pharmacovigilance (tolerance, serious adverse events and toxicity) and it will improve practice. The study findings should thus lead either to justification of the cessation of some individual prescriptions when they are poorly tolerated and/or ineffective or to a recommendation to open a clinical trial when the response rate suggests efficacy.
By April 2022, 340 children, adolescents or young adults had already participated in the study and 52 medicinal products had been evaluated.
A collaborative secure web portal was set up for collection and analysis of relevant data. It is managed by the Gustave Roussy Pharmacovigilance Unit.
The project is supported by the ANSM. SACHA collects data on safety and efficacy of all the innovative agents for which access is afforded by the ANSM on compassionate grounds.
“SACHA is a reliable and useful tool for thorough collection of real-life data. It thus responds to the expectations of administrative health authorities,” pointed out Professor Vassal. The project is also keen to involve other countries in order to establish an international observational cohort.
The SACHA study is financed by Imagine for Margo, Hubert Gouin “Childhood and Cancer” and the LEEM (Medicinal Products Companies) Foundation.
Abstract No. 10006
Securing access to innovative anticancer therapies for children, adolescents, and young adults
outside clinical trials: The SACHA study of the French Society of Pediatric Oncology (SFCE).
Oral abstract session
Monday 6th June 2022 | 23:36 – 23:48 UTC+2