The SACHA study
SACHA is a prospective observational cohort study that monitors the use of innovative drugs in oncology and haematology for children, adolescents and young adults who are in a situation of therapeutic failure or relapse and who are not eligible for a clinical trial.
For children and adolescents with relapsing cancer in France, clinical trials provide the main means of access to therapeutic innovation. This is an area that attracts major research programmes including studies such as MAPPYACTS and AcSe-ESMART, which allow for secure access to molecular profiling of relapsing tumours and to innovative targeted therapies through in-depth molecular analysis of their tumour. Nevertheless, despite considerable efforts to broaden the range of trials on new medicinal products in France, the therapeutic options available remain limited. As a result, paediatric oncologists and haematologists prescribe innovative medicines either in the context of an early access authorisation programme and compassionate access programme * or in off-label use (i.e. outside the indication for which the medicine is approved).
What is SACHA and what is its main objective?
SACHA is an observational (non-interventional) study that prospectively collects data on the toxicity and efficacy of innovative therapies administered in the context of an early access authorisation programme and compassionate access programme or in off-label use in children, adolescents and young adults under 25 years of age with cancer who are in a situation of therapeutic failure or relapse and who are not eligible for a clinical trial.
The objective of the SACHA project is to oversee and to ensure the safety of these prescriptions by providing organised follow-up on patients.
By collecting data on the prescriptions, the project will enhance our knowledge and improve our practices in multidisciplinary discussions of cases involving relapse and inclusion in clinical trials. It will also provide justification for stopping certain individual prescriptions when they are shown to be ineffective our particularly poorly tolerated.
What is the procedure for including patients?
The SACHA study is being conducted in collaboration with the network of interregional hospital organisations that provide care in paediatric oncology (OIR), which is a forum for interregional multidisciplinary consultative meetings in paediatrics (RCPPI), bringing together the 31 centres of the French Society for Cancer and Leukaemia in Children and Adolescents (SFCE). The files on patients in relapse or in treatment failure are discussed in consultative meetings to propose potential inclusion in the SACHA project. The aim is not under any circumstances to take the place of clinical trials but, quite the contrary, to ensure that patients are not eligible to participate before resorting to off-label use.
What data are collected and how?
VIGINOM, a secure collaborative Web portal, authorised by the French data protection agency CNIL, has been set up to collect and analyse the data of interest. The portal is managed by the Pharmacovigilance Unit (UFPV) of Gustave Roussy and has been validated in a number of pharmacoepidemiological studies, in particular in oncology. VIGINOM was set up to collect and analyse data on the efficacy and safety of treatments and to allow for preparation of drug safety reports.
The study is supported by the French Society for Cancer and Leukaemia in Children and Adolescents (SFCE) and funded by the LEEM Foundation, as well as the associations “Imagine for Margo” and “Hubert Gouin- Childhood & Cancer”.
Contact:
Project Leader: Dr Lee Aymar Ndounga-Diakou
Leeaymar.NDOUNGA-DIAKOU@gustaveroussy.fr
* An early-access procedure to enable certain categories of patients to have access to medicines that have not yet been placed on the market but that constitute a major therapeutic advance or correspond to an unmet medical need.
