ESMO - SIOPE E-Learning: Improving AYA Access To Innovative Therapies by Breaking the 18 Years Dogma

Learning Objectives

  1. To raise awareness about needs for new drugs for haemato-oncology treatment in adolescents and young adults
  2. To provide an overview of the European regulation and current landscape in the drug development and early drug access in the adolescent and young adult population
  3. To provide an overview of proposed changes in trial strategy for adolescents and young adults, from early drug development to accelerate AYA access to the therapeutic innovation, by the FAIR (Fostering Age Inclusive Research) trial group of the multistakeholder European Paediatric Platform ACCELERATE
Title Duration Content CME Points CME Test
Improving AYA Access To Innovative Therapies by Breaking the 18 Years Dogma 26 min. 32 slides 1 Take Test
Nathalie Gaspar
Nathalie Gaspar
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This E-Learning module represents a further commitment of the joint ESMO/SIOPE Working Group on Cancer in Adolescents and Young Adults (AYA) to educate oncology professionals on different aspects related to cancers in this patient population and build a repository of teaching modules covering a range of different cancer types and cancer-related issues in this setting.

Cancer is the third cause of death in adolescents and young adults. By showing lower survival gains over years that paralleled the under-representation of this population in therapeutic trials, the author underlines that access to innovative therapies might improve AYA survival. The author also emphasizes that the impressive progress, recently observed in adult cancers through the introduction of new drugs, has not yet been translated to adolescents.

The current drug development landscape separates adult and paediatric drug development. Adolescents are grouped with children, leading to a mismatch with a lack of trials for adolescents with relapsed cancer and delayed access to new, effective drugs already available for adults. The author underlines that AYA inclusion rate is higher in AYA diseases where joint paediatric/adult phase III trials are available.

In some cancer types with identical drug targets in the paediatric and adult populations, adult phase II trials have demonstrated efficacy, but paediatric clinical development commenced much later. Such situation has led to a significantly delayed introduction of beneficial drugs to adolescents.  

Furthermore, the author discusses about non-viable adolescent specific trials in adult disease that are rarely present in adolescents and off-label use in adolescents of new efficient drugs approved in adult indications. Young adults might benefit from disease-specific paediatric phase I to III trials
when they suffer from paediatric cancer.

In this E-Learning module, the author reviews the European regulation and current landscape in the drug development for adolescents and critically discusses how to change the current status. In that regard, she proposes to include adolescents in « adults » trials from early phases, when appropriate (same disease or same target in paediatric and adult population), with no opposition from the industry, no breaks from the European health legislation, and the importance of patient and parents support, as long as AYA’s care take place in an AYA adapted structure.

Proposed changes of the FAIR (Fostering Age Inclusive Research) trial group of the multistakeholder European paediatric platform ACCELERATE consider an increased collaboration between paediatric and « adult » oncologists from early trial conception to the implementation of the trial in an AYA appropriate care, and an increased collaboration among all multistakeholders involved in the new drug development.

Last update: 10 April 2019

The author has reported no conflict of interest.