In 2007, the EU Paediatric Medicine Regulation was launched and it has benefited many children with diseases but not with cancer, because:
- The paediatric development of oncology drugs is often waived because the therapy being developed is for an adult disease which does not occur in children.
Example: crizotinib, ALK inhibitor, in lung cancer; olaparib, PARP inhibitor, in ovarian cancer
Indeed, many cancers in adults do not occur in children, or extremely rarely.
In the Class waiver list, all adult conditions occurring only in adults (80% of class waived conditions are malignancies) are listed and pharmaceutical companies receive a waiver without the need to provide scientific and medical justifications.
Furthermore, the way the drug works (its Mechanism of Action, MOA) in an adult type of cancer may be relevant to a cancer type that predominantly occurs in children;
Example : ALK in anaplastic large cell lymphoma, inflammatory myofibroblastic tumour and neuroblastoma in children and adolescents
- There are major delays in starting clinical trials of oncology drugs for children while waiting for the drug to show promise in adult cancer patients without regard to its potential benefit in children. Indeed, deferrals are granted for the vast majority of paediatric investigation plans (PIP). Financial incentives to develop drugs for a cancer which only occurs in childhood often come too late for pharmaceutical companies to consider investing in research and development in the paediatric oncology space;
- Several PIPs have proved to be unfeasible, in particular when they focus on the rare occurrence of an adult cancer in a child, such as evaluation of vemurafenib, a B-RAF inhibitor, in metastatic B-RAF melanoma in adolescents.
In October 2017, the European Commission published the 10 years scientific and medico-economic report of the EU paediatric regulation.
This report showed that the Regulation had a considerable impact on the development of paediatric medicines in the EU. Some considerable progress has been made in the availability of medicines in certain therapeutic areas, such as rheumatology and infectious diseases.
The report recognized that “paediatric oncology is often used as a case study for insufficient advances in area of high unmet needs”. The report detailed the issue of class waiver in oncology and MoA of medicinal products. Organizations representing patients and academia have advocated for a stronger reliance on the MoA principle and legislative changes to the waiver concept (suppression of article 11.b). However, no legislative changes have been proposed by the Commission to the European Council and Parliament.
In July 2018, EMA launched the Revised Class waiver list and this will result in a substantial reduction in the number of malignant conditions where a waiver can be simply granted
The EU commission concluded that the regulation works best in areas where the needs of adult and paediatric patients overlap.