United States: Ten-Year Report On Paediatric Regulation Published

Last week, the European Commission published its ten-year report on the implementation of the Paediatric Regulation 1901/2006 (together with a useful Questions & Answers document). The report provides an account of the Regulation's achievements, both in public health and economic terms, and an analysis on the extent to which its objectives and aims have been met.

It concludes that positive advances in the development of medicines for children could not have been achieved without specific EU legislation, and that 260 new medicines (new marketing authorisations and new indications) have been authorised as a result. However, certain problem areas have been identified, in particular in relation to the interplay between the Paediatric Regulation and the Orphan Medicinal Products Regulation 141/2000. The report does not set out any proposals for amendments to the Regulation, but states it will be for the next Commission, after 2019, to implement any necessary changes.

Key conclusions from the report

Published under Article 50(3) of the Regulation, the report seeks to build on the five year report published in 2013, which largely concluded that it was too soon to draw any conclusions on the Regulation. The ten-year report states that the primary aim of increasing the number of products that are authorised and on the market for children, and indeed the number of clinical trials being conducted in children, has been met in many therapeutic areas, most notably in rheumatology and infectious diseases. Indeed, paediatric medicine development is now an integral part of the overall development of medicines.

However, the report also notes that little progress has been made in diseases that only affect children, or where the disease shows biological differences between adults and children, particularly rare diseases such as childhood cancers. In practice, it is still the case that paediatric development follows the manufacturer's plan for development of the adult indication.

The Commission commented on a number of aspects of the Regulation, as follows:

  • Deferrals: Experience shows that the deferral is a widely used instrument. However, the report states that deferrals are sometimes too long, meaning that paediatric development is delayed.
  • PUMA: The paediatric use marketing authorisation (PUMA) was introduced specifically to reward paediatric-only developments. The report notes that this incentive has largely failed, with only three PUMAs having been authorised.
  • Costs: While industry has raised concerns that the Regulation is burdensome and costly (the total R&D costs amount to €18.9 million per PIP, and three additional clinical trials), the report states that the additional costs borne by industry only lead to a limited increase in the total costs of development. It concludes that the Regulation provides overall positive results from a socioeconomic perspective.
  • SPC extensions: The report notes that 55% of completed PIPs benefit from a reward, and only around 40 SPC extensions have been granted. Complications such as the fact that extensions have to be obtained through the national patent offices, and that applications have to be made two years before expiry of the SPC, are cited as difficulties in obtaining the reward, even if the PIP is completed.
  • Access: Availability of new medicines continues to be a concern, and the report states that there has only been an estimated increase in medicines of 5-10%. National pricing and reimbursement decisions, the fact that paediatricians may not immediately switch to newly authorised products, and that companies rely on a staggered roll-out of new products, are cited as possible reasons for the delay.
  • Orphan products: The interplay between the Orphan Medicinal Products Regulation and the Paediatric Regulation is noted as a particular area that needs closer examination. Indeed, in the press release accompanying the report, Vytenis Andriukaitis, Commissioner for Health and Food Safety, stated: "In the next 10 years we must focus on making similar breakthroughs [as have occurred with adult oncology] for children, by combining the incentives under the Orphans and the Paediatric Regulations". When the Paediatric Regulation was introduced, it was thought that many orphan products were not covered by patents, and the reward of an expansion to orphan exclusivity was introduced. However, this is no longer the case, with only around 10% of newly authorised orphan products being off-patent. The report also notes that a number of authorisation holders for orphan products surrendered their orphan exclusivity/ designation in order to obtain the reward of an extension to their SPC.

Next steps

The Commission stated that the report is an essential intermediate step in the debate on the future for paediatric and orphan medicines. The Commission and EMA will now develop an action plan to improve the implementation of the Regulation. The aim is to set out recommendations by 2019, so the next Commission can take decisions about possible policy options.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

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