On the basis of the Joint Action on Rare Cancers (JARC) survey and its conclusions, SIOP Europe mobilised the pan-European community of clinicians, researchers, and parents to develop a list of essential medicines for childhood malignancies based on most recent evidence.
The aim is to present the list to all relevant stakeholders, including the World Health Organization (WHO) for the next revisions of the WHO Essential Medicines List for Children (EMLc) and to foster access to these medicines for all children and adolescents who need them across Europe.
Another aim of the project is to explore the Health Technology Assessment (HTA) evaluation for newly approved expensive medicines.
The JARC survey (2018) demonstrated that children and adolescents with cancer across Europe faced barriers to accessing anti-cancer medicines defined as essential by the community:
- Shortages concerned over 70% of the surveyed medicines
- Financial barriers applied in lower-income countries and to newly approved expensive medicines across all settings, with out-of-pocket costs reported in 8 out of 30 countries
- 27% of the orally administered medicines were never available in child-friendly formulations, causing concerns over alignment with the prescribed protocol and parental distress
- There were marked differences between countries in access to pain control for children undergoing invasive procedures.
The survey led to the publication of a manuscript in a peer-reviewed journal in 2021.
The findings made clear the need for urgent action to ensure equal access to essential medicines used to treat paediatric cancers in Europe and prompted SIOP Europe to initiate the Access to Medicines Project.
SIOP Europe Access to Medicines Project
The project was launched in August 2019. With the coordination by SIOP Europe and Young SIOPE, all European Clinical Trial Groups (ECTGs) contributed to a systematic evaluation of essential anti-cancer medicines for the treatment of children and adolescents with cancer in Europe.
- To provide an updated evidence-based reference list of essential medicines for all paediatric cancer diseases that should be always (24/7) accessible to children and adolescents who require them across Europe, and to feed biannually into revisions of the WHO Model List of Essential Medicines for Children (EMLc).
- To initiate a dialogue with Health Technology Assessment (HTA) bodies regarding the evaluation of newly approved expensive anti-cancer medicines for children and adolescents.
In total, 22 mentors from the different ECTGs and 35 Young SIOPE members screened 63 treatment protocols and defined a preliminary list of essential anti-cancer medicines. In addition, 16 external senior professionals further reviewed the findings.
During the course of the project, collaboration with the ERN PaedCan – SIOP Europe European Standard Clinical Practice (ESCP) Project was also established, with Young SIOPE members contributing to both projects and supporting the exchange of information.
A second part of the project, initiated in collaboration with Young SIOPE and two HTA experts, was conducted to evaluate the process of HTA evaluations for 3 selected anti-cancer medicines for children and adolescents with cancer (Blinatumomab, CAR-T and Dinutuximab).
In addition, SIOP Europe will work with ACCELERATE with the goal to facilitate access to newly approved effective anti-cancer medicines for paediatric cancer and a more aligned and accelerated evaluation by HTA bodies across Europe.
A selection of the identified medicines was presented to the WHO Essential Medicines Committee and in September 2021, 16 new paediatric cancer medicines and indications were included under the updated WHO Essential Medicines List for Children (2021).
Gilles Vassal, Reineke Schoot, Maria Otth, Eva Brack, Marko Ocokoljić, Olga Kozhaeva.