10-year Report to the European Commission

“The Paediatric Regulation had a considerable impact on the development of paediatric medicines in the EU. It ensured that paediatric medicine development became an integral part of the overall development of medicines. This result would have not been achieved without specific legislation and underlines its continued relevance. Moreover, measures taken to improve its implementation have over time strengthened its effectiveness.

In economic terms, the Regulation provides overall positive results from a socioeconomic perspective demonstrating the appropriateness of this direct investment in improving the availability of paediatric medicines. The combination of obligations and rewards seems effective to shift focus to paediatric product development. Still the use of rewards was limited to 55 % of the completed PIPs and there are instances of over- or under compensation pointing to certain limitations of the current system. Additionally, the PUMA concept with its spe – cific reward has failed to deliver.

The increase in paediatric research and the number of new products with specific paediatric indications is encouraging and will ensure that over time the offlabel use of adult medicines in the paediatric population will decrease. Those positive results do however not evenly spread among all therapeutic areas, but concentrate in some, often linked to research priorities in adults rather than children.

This shows that the Regulation works best in areas where the needs of adult and paediatric patients overlap. Especially, in diseases that are rare and/or unique to children and which in many cases are equally supported through the orphan legislation, major therapeutic advances often failed to materialise yet. Why this is the case and why the orphan reward is in some instances not able to drive paediatric development in a simlar way than adult orphan development requires further scrutiny.

Therefore and before proposing any amendments, the Commission intends to take a closer look at the combined effects of the Orphan and Paediatric Regulation through a joined evaluation of those two legal instruments aimed at supporting medicine development in subpopulations of particular need. Given that weaknesses identified in this report often relate to paediatric diseases that qualify as orphan condition, only such combined effort will guarantee to adjust the right parameters, if required.

This report marks not the end, but an essential intermediate step in the de – bate on a joint vision about the future parameters for paediatric and orphan medicines. The further evaluation supporting this process aims at providing results by 2019 so to allow the next Commission to take informed decision about possible policy options. It will also allow the forthcoming results of the SPC evaluation for the future of the Paediatric Regulation to be taken into account.

In the meantime, the Commission is committed to a positive agenda of concrete actions in order to streamline the current application and implementation together with EMA wherever needed. This includes:

  • providing additional transparency of new products authorised with paediatric indications;
  • analysing the experience with use of deferrals and consider changes in practice to ensure speedier com – pletion of PIPs;
  • revisiting processes and expectations in the context of handling of applications for PIPs and if necessary adapt the corresponding Commission guideline; 22. In this context business continuity priorities may need to be taken into account due to the relocation of EMA.
  • exploring opportunities to discuss paediatric needs in an open and transparent dialogue involving all relevant stakeholders like academia, health care providers, patients/care givers, paediatric clinical trial networks, industry and regulators;
  • delivering regular updates about development and trends of the paediatric medicines landscape in the EU; and
  • fostering international cooperation and harmonisation.

Additionally, it will further support highquality healthcare and research for children through projects such as the European Reference Networks, which connect health care providers and centres of expertise. Those networks have the potential of significantly improving access to diagnosis and treatment in the short term and to make a difference in terms of child health”.

The full report is available online.



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