Access to essential anticancer medicines for children and adolescents in Europe

Abstract

Background: Essential anticancer medicines are an indispensable component of multidisciplinary treatment of paediatric malignancies. A European Society for Medical Oncology (ESMO) study reported inequalities in the availability of anticancer medicines for adult solid tumours and provided a model for the present survey. The aim of this survey was to assess the accessibility of essential medicines used in paediatric cancer patients aged 0 to 18 years across Europe from 2016 to 2018. Methods: A list of medicines was drawn with input from the European Society for Paediatric Oncology (SIOP Europe) Clinical Research Council referring to the World Health Organization Model List of Essential Medicines for Children (WHO EMLc) 2017. A survey was sent to nominated national clinician and pharmacist rapporteurs and parent associations in up to 37 countries; answers were obtained from 34 countries. Results: The full survey list contained 68 medicines, including 24 on the WHO EMLc 2017. Health professionals reported that 35% of all medicines were prescribed off-label in at least one country and that 44% were always available in >90% of countries. Only 63% of the EMLc 2017 medicines were reported as always available. The main determinant of unavailability was shortages, reported for 72% of medicines in at least one country. Out-of-pocket costs were reported in eight countries. Twenty-seven percent of orally administered medicines were never available in childfriendly formulations. Parents detailed individual efforts and challenges of facilitating ingestion of oral medicines as prescribed. Inequalities in access to pain control during procedures were reported by parents across Europe. Conclusions: Children and adolescents with cancer in Europe experience lack of access to essential medicines. Urgent actions are needed to address shortages, financial accessibility, availability of safe age-appropriate oral formulations, and pain management across Europe.

Off‐label and compassionate use of targeted anticancer therapies: The experience of an Italian pediatric cancer center

“…In this context, a better understanding of the biology of tumors has led to an exponential increase in the development of new targeted therapies and immunotherapies 10 ; however, access to these treatments remains very limited 3 . Currently, pediatric onco‐hematologists, given the lack of approved treatments and the scarce number of clinical trials available for the pediatric population diagnosed with neoplastic disease, still regularly prescribe off‐patent medicines for an off‐label indication 5 . In this context, it is crucial to collect real‐world data on safety and efficacy generated from these unauthorized uses of innovative therapies in children and adolescents with cancer.…”

“…3 Currently, pediatric onco-hematologists, given the lack of approved treatments and the scarce number of clinical trials available for the pediatric population diagnosed with neoplastic disease, still regularly prescribe off-patent medicines for an off-label indication. 5 In this context, it is crucial to collect real-world data on safety and efficacy generated from these unauthorized uses of innovative therapies in children and adolescents with cancer. These data, once available for the scientific community, age did not correspond with the SmPC.…”

“…In this framework, most of the drugs currently used to treat childhood cancers are off‐patent adult medicines, prescribed off‐label in the pediatric population. Over the past 50 years, safety and efficacy data of these drugs in children and adolescents have been evaluated through prospective academic studies; however, these did not lead to approval of new pediatric indications 5 . Due to the unavailability of adequate authorized treatments and the insufficient number of clinical trials, pediatric onco‐hematologists in Europe often resort to off‐label and compassionate uses.…”

“…Over the past 50 years, safety and efficacy data of these drugs in children and adolescents have been evaluated through prospective academic studies; however, these did not lead to approval of new pediatric indications. 5 Due to the unavailability of adequate authorized treatments and the insufficient number of clinical trials, pediatric onco-hematologists in Europe often resort to off-label and compassionate uses. There is therefore an urgent need to collect data from compassionate and off-label uses of innovative treatments.…”

The threat of the COVID-19 pandemic on reversing global life-saving gains in the survival of childhood cancer: a call for collaborative action from SIOP, IPSO, PROS, WCC, CCI, St Jude Global, UICC and WHPCA

“…Efforts to ensure access to drugs on the WHO Essential Medicines List for children and those proposed in the paediatric review of the Essential Medicines List for cancer [24,25] are vital to ensure curative care can be delivered to as many children in as many clinical settings as possible.…”