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With only a few months left before the European Commission publishes its revision of the EU Paediatric Regulation (Regulation (EC) No 1901/2006), it is paramount to understand what this regulation has brought to the paediatric research landscape, as well as to identify the shortcomings that could be improved during this review.
Though it is undoubtable that the current regulation has supported the development of new therapies for children, with 238 new paediatric medicines or indications being brought to the market since 2007, there remains a number of barriers to address to meet the needs of children.
At Servier, paediatric research has become an integral part of all our development programs and is even a strong driver of many of our oncology and early clinical research programs. As such, Servier dedicates 50% of our development pipeline to oncology, particularly looking at hard to treat cancers and paediatric resistant/relapsing high-risk tumors, some of the highest needs with no current treatments. The EU Paediatric Regulation has been a supportive framework for our ambition, but we believe that it can be further improved to ensure even more new medicines are brought to the market for the children that need them.
To that end, for each of the three avenues aiming at generating medicines for children, we have identified specific barriers, and solutions to tackle these issues in the context of the revision of the regulation.
First-in-child research
While the EU Paediatric Regulation has been instrumental to bringing new paediatric medicines to the market, its mechanism has mainly targeted adult-led research without addressing inherent barriers to first-in-child research such as the limited number of patients and difficulties to design clinical trials. This substantially hinders basic research in the paediatric population. To tackle this issue, it is necessary to identify research priorities by developing a holistic and broad definition of unmet medical needs through a multi-stakeholder dialogue.
Adult-led research
The current regulation has been effective in ensuring that medicines authorized for an adult indication can also be adapted for children if the disease exist in the paediatric population. However, to do so, companies face a complex regulatory process, adding a significative administrative burden. To facilitate the translation of research from adults to children, additional flexibilities should be introduced, such as simplifying the paediatric investigation plan (PIP), allowing a staggered approach and putting in place a framework maximizing the use of data stemming from adult research or from other sources of paediatric data, such as Real World Evidence.
Repurposing of existing medicines
While the current regulation has put in place a specific pathway to encourage the repurposing of off-patent medicines for paediatric indications, namely, the paediatric-use marketing authorization (PUMA), this pathway lacks adequate market and regulatory incentives. Repurposed medicines for a paediatric indication will face competition from both generics and existing medicines with adult indications, thus threatening their economic viability. It is therefore essential to adapt the incentives framework for repurposed products to reward the research efforts, notably through delinking the pricing of new paediatric indications from generic prices.
Servier and the industry stand ready to act to ensure that paediatric unmet medical needs are not left unanswered. To that end, we see the revision of the current legislative framework as an opportunity to improve the current regulation and guarantee the ability to do research in a safe, efficient and conducive framework.
You can find our complete set of proposals in Servier’s position on the revision of the paediatric regulation, accessible here.