RAPS recognizes that the current situation in Ukraine impacts our members and customers on many levels. If you are directly impacted by the current situation in the region and are challenged to meet your deadlines or obligations to RAPS, please reach out to raps@raps.org so that we can defer those challenges. Your health and safety are paramount to us.

Regulatory Focus™ > News Articles > EU Pediatric Regulation’s 10-Year Anniversary Report Highlights Shortcomings

EU Pediatric Regulation’s 10-Year Anniversary Report Highlights Shortcomings

Posted 26 October 2017 | By Zachary Brennan 

EU Pediatric Regulation’s 10-Year Anniversary Report Highlights Shortcomings

Ten years ago, the European Commission created a new regulation as part of an effort to try to encourage more pediatric research.

But in the years since, companies have been slow to increase their development of treatments for pediatric populations, particularly in oncology, where many of the medicines used were developed in the 1990s, “if they exist at all,” an EC report released Thursday said.

The report highlighted that the number of completed pediatric investigation plans (PIPs) is clearly trending upward, though estimates say the increase in available medicines for children is in the range of 5-10% and the proportion of clinical trials that include children increased from 8.25% to 12.4% between 2007 and 2016.

The report follows the release of comments from regulators and governments saying the pediatric-use marketing authorization (PUMA) has not lived up to expectations.

German regulator BfArM said PUMA "certainly did not meet” expectations, while the Dutch government said, "the effect of the PUMA-concept is rather falling short" and the UK government described the PUMA concept as "a failure."

The new EC report singles out the surge in innovative adult cancer treatments, with some first-in-class products hitting the market in the last decade, though “this pace of advances observed in adult therapies is so far not mirrored in pediatric patients.”

Vytenis Andriukaitis, European Commissioner for Health and Food Safety, said in a statement Thursday: "When we consider the advances in adult oncology, it upsets me deeply that we have not made the same progress in treating the cancers that affect children. In the next 10 years we must focus on making similar breakthroughs for children, by combining the incentives under the Orphan and Pediatric Regulations, and by ensuring that the European ReferenceNetworks - in particular on pediatric cancer, reach their full capacity.”


The Pediatric Regulation has three main objectives: To encourage high-quality research on medicines for children, to ensure that most pediatric medicines are specifically authorized with age-appropriate forms and formulations, and to increase the availability of high-quality information about medicines used by children.

To meet these objectives, the regulation sets up a system linking the obligation of conducting the additional pediatric research with two types of rewards (an orphan reward and a supplementary protection certificate reward ) to allow companies to recuperate the additional upfront costs by effectively delaying the market entry of competitor products.

The EC report notes that the obligation and reward aspects of the EU regulation differ from the US system, where pediatric requirements imposed by the US Food and Drug Administration (FDA) mean a company can voluntarily engage in additional research following a corresponding “Written Request” from FDA.

In the EU, the report adds: “The reward becomes available once the PIP [pediatric investigation plan] is completed and its results are reflected in a corresponding marketing authorization. The company is entitled to the reward even if the results of pediatric studies finally fail to support a pediatric use of the compound, as it is meant to compensate for the research as such, not a particular outcome.”

Expenses vs. Rewards

Based on an external study, EC estimates the total costs of the regulation for industry to be €2.1 billion ($2.5 billion) per year, which is a figure derived from an extrapolation based on 85 PIPs.

The total R&D costs, based on an average of eight examples, amount to €18.9 million ($22 million) per PIP, with each plan including an average of three clinical studies. In addition, EC said companies incur overhead costs of about €720,000 ($843,000) for filing an initial PIP submission and subsequent modifications.

In terms of rewards, the report noted that it’s “not possible to estimate the economic value of the orphan reward,” but the supplementary protection certificate (SPC) reward, which is under review and may be modified “in the coming months,” the patent extension “is often considered as the most precious reward.”

Overall, the report said the adjusted economic value of the SPC reward for the eight products amounted to €926 million ($1.1 billion), or about $116 million on average, though there are “significant differences” between the eight products and member states.

“In addition, the Regulation may generate economic spill-over effects due to additional R&D investment towards new and improved medicines that triggers further investment and contributes to the creation of jobs, growth and innovative activity across sectors,” the report said. “A more conservative estimated rate of return from an annual EUR 2.1 billion investment in pediatric R&D could after 10 years, yield a total societal return of around EUR 6 billion. This estimated societal return is significantly higher than the economic value of the SPC extension, suggesting that in monetary terms, the benefits of the Regulation for society outweigh the costs of the additional monopoly rent.”

State of Paediatric Medicines in the EU - 10 years of the EU Paediatric Regulation


© 2022 Regulatory Affairs Professionals Society.