EU’s Pediatric Regulation: European Commission Seeks Feedback for New Report

Regulatory NewsRegulatory News | 15 November 2016 |  By 

The European Commission (EC) on Tuesday launched a public consultation to obtain feedback for its second report on the Pediatric Regulation, ten years after its implementation.

The comments on the consultation, which is open until 20 February 2017, will help inform the EC's report, which is expected to be published in 2017 and will assess the regulation's impact on public health and the pharmaceutical industry.


The Pediatric Regulation, known in the EU as the Paediatric Regulation, came into force on 26 January 2007 and has led to the authorization of 68 new medicines and indications for pediatric populations between 2012 and 2014, which is more than double the number of approvals between 2004 and 2006, prior to the regulation's implementation.

“For example, medicines to treat certain rheumatology conditions in children, infectious diseases such as chronic hepatitis C and HIV infection, hypertension and paediatric cancers like acute lymphoblastic leukaemia are now available on the basis of studies conducted in children,” EMA said.

In 2013, the Commission published its first report on the Pediatric Regulation and while it revealed some signs of progress, it also found that, due to the lengthy time it takes to develop a new drug, it would take at least 10 years to gain a full understanding of the situation.

In addition, EMA’s pediatric committee (PDCO) by the end of 2015 had adopted 860 opinions for pediatric investigation plans (PIP) and will soon pass 1,000. PIPs are the main tool of the regulation to ensure that previously unmet therapeutic needs in children are researched and appropriate medicines are developed.

Clinical trials in children initiated as part of an agreed PIP now represent about 30% of pediatric trials recorded in the EU Clinical Trials database (EudraCT), EMA says.

In addition, the European Network for Pediatric Research at the EMA (Enpr-EMA), a network of 38 national and international networks recognized for their pediatric research experience, has been set up to facilitate clinical studies in children and share best practices.


The consultation, which will be presented to the European Parliament and the Council as part of a plan to further inform EU decision-makers about the experience so far, includes 17 questions ranging from those on the availability of pediatric medicines in the EU, to the cost for pharmaceutical companies to comply with PIPs.

In terms of company costs, an EC evaluation of PIPs agreed to over the last ten years shows that the total R&D costs per plan on average amount to €18.9 million ($20.3 million), with each plan including an average of three clinical studies.

On top of this, EC figures that companies incur administrative costs of around €720,000 ($771,000) for the initial PIP filing and subsequent modifications, bringing the total estimated average incurred costs per PIP to about €20 million ($21.4 million).

Based on the average number of new plans agreed to per year (107 in 2008-2015), this amounts to total annual costs of €2.1 billion ($2.3 billion) incurred by the industry.

“At the same time, this may be an overestimate given that not all agreed paediatric investigation plans will be completed as some are discontinued, for example if the company decides to shelve the adult development programme,” EMA says, adding that the figures “suggest that the additional costs incurred by industry as a consequence of the Paediatric Regulation are reasonable and that they lead to only a limited increase in the total cost of medicine development.”

The consultation also discusses the reward system whereby companies can recuperate these additional costs, though an analysis of the PIPs completed so far “shows that not all companies were able to obtain a reward.”

However, at least 55% of the completed PIPs benefitted from a reward, most of which were for a prolonged of the supplementary protection certificate (SPC), which is basically an extension of a drug’s patent life.

And in four cases, the market exclusivity period of an orphan medicinal product was extended, while in several other instances, companies waived the product’s orphan status shortly before marketing authorization in order to make the product eligible for the SPC reward rather than the orphan reward, as the former is often considered to be economically more attractive.



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