At the European Health Workshop entitled ‘Are children still missing out on potentially life-saving treatments?’ held on the 16th June 2015 held at the European Parliament in Brussels, Kevin and Karen spoke on their experiences of Christopher’s illness and the need for a change to the implementation of the EU Paediatric Regulation. The Working Group was co-chaired by Glenis Willmott MEP (UK) and Alojz Peterle MEP (Slovenia). The speakers were:
· European Commission – Olga Solomon
· European Federation of Pharmaceutical Industry Associations (EFPIA) – Magda Chelbus
· Parents – Kevin and Karen
· Clinical Expert – Professor Andrea Biondi
The focus of Kevin and Karen’s presentation was the need for more drugs to be made available earlier in their lifecycle for children with cancer. The EU Paediatric Regulation currently allows pharmaceutical companies to gain a waiver for developing a drug in children because it has been produced to treat an adult disease. This allows many drugs that have potential paediatric use to be denied to children.
Kevin and Karen discovered that the implementation of Paediatric Regulation Article 11; the article that states that waivers can be based upon the disease or condition for which the drug is intended to be used in the adult population. If the adult disease or condition does not occur in children the drug’s manufacturer is granted a waiver to develop the drug in children. However the Paediatric Regulation does not define disease or condition. Disease or condition for the purposes of the regulation are defined by a separate document, a document few people are aware of, an EMA document – Policy on the determination of the condition(s) for a Paediatric Investigation Plan/Waiver (scope of the PIP/waiver).
This document is not a legislative document but a policy document produced by the EMA and the EMA’s Paediatric Committee the PDCO. What is interesting is that this document holds the key to why waivers are given to pharmaceutical companies for producing drugs with a potential paediatric use. You can view the document here. Karen and Kevin have asked for an urgent review of this document to change the level on the classification scale upon which a waiver is based.
The agreed actions from the Working Group were proposed by Kevin and Karen:
· The European Commission to instruct the EMA & the PDCO to implement Article 11 (b) to issue waivers based on Condition where Condition is defined by biological or genetic abnormality (MedDRA HLT vs PT)
· A full review of Class Waiver list & removal of all diseases where biological or genetic mutation occurs in the paediatric oncology population (not just a cosmetic change currently under consideration)
· MEPS to work with the Commission to ensure these changes are made at earliest opportunity.
Below are links to documents associated with the Working Group discussion