What is the Paediatric Regulation?

The Paediatric Regulation was adopted in 2007 to address some key issues regarding the development of children's medicines.

Before the regulation was developed, many medicines were prescribed to children based on experience of the clinician treating the patient, as opposed to evidence from clinical research into the drugs use in children. The Paediatric Regulation works to increase the number of drugs that are being investigated for use in children and provides obligations and rewards for drug companies to ensure children's medicines are regularly being investigated. 10 years on, drugs companies now see paediatric investigation as an integral part of their product development, and the Paediatric Regulation has been successful in its aims.

The core principle of the Paediatric Regulation is that every time a drug company introduces a product to be authorised for use in Europe to treat an adult condition, it should be evaluated for its benefit in children where there is currently an unmet therapeutic need.

However, there is agreement that there is a need for a revision of this principle.

How does this relate to childhood cancers?

Each year, approximately 15,000 children under 15 years of age and 20,000 teenagers and young adults (aged 15 to 24) are newly diagnosed with cancer in the EU alone. Leading to around 6,000 deaths in the EU each year of children with cancer.

Many childhood cancers are in isolation to adult cancers and therefore (following the current core principle of the regulation) would not be investigated by drug companies in many cases. The Bone Cancer Research Trust and many other organisations hope that a revision of the Paediatric Regulation can be made to increase the investigation of cancer drugs specifically for children.

Cancer Research UK, SIOPE (The European Society for Paediatric Oncology) and Unite2Cure have published a position statement calling on European institutions to support the urgent revision of the Paediatric Medicines Regulation. Together they have developed mutually agreed recommendations for such a revision, which include:

  • Ensuring drug companies have an obligation to investigate paediatric medicines based on how a drug works, rather than this condition occurring in adults
  • Adding more effective and flexible rewards for drug companies who are undertaking paediatric investigations and researching therapies specifically for childhood cancers.

Their position statement can be read in full here.

The Bone Cancer Research Trust's response

The Bone Cancer Research Trust are in agreement that the consequence of the current approach lies with progress in paediatric medicines relying on the company's adult product pipeline and advances required in adult populations.

Adult conditions do not necessarily overlap with paediatric conditions, leaving a considerable amount of paediatric conditions that are not investigated to their full potential and therefore receive no benefit from the paediatric regulation.

We are very much in support of these recommendations and have provided an official response to the consultation, highlighting these recommendations along with expansion to include our own comments and recommendations.

Our full consultation paper can be viewed here