In December 2016, the European Parliament adopted a Resolution on the Regulation on Paediatric Medicines. TEDDY has contributed to it in providing some suggestions and comments in the drafting phase. The document acknowledges that relevant results have been achieved following the entry into force of the Paediatric Regulation. In the meantime, the burdens still remaining are mentioned, with a particular reference to the topics of neonatology, oncology and life-threatening disease. A special attention is paid to the theme of off-label medicines use, that is still very relevant even after 10 years the Regulation came into force. Despite recognising the change of mind that the Regulation has introduced in the pharma plans, the regulatory approach is considered still unsatisfactory in the case of childhood-specific diseases. Moreover, the extensive/systematic use of deferral means, multiple modification to the agreed PIPs and the quite low percentage of completed PIPs have been mentioned as factors impairing the availability of paediatric medicines.
Most of the points addressed by this document appear reasonable and well-documented. The reference to the issue of paediatric off-label use is particularly appreciated, since it demonstrates that this matter is entering in the policy makers’ agenda. Moreover, this Resolution pushes for several aspects that have been proposed by the TEDDY Network in several occasions, for example the need to critically review and renew the funding provisions developed to support high-quality paediatric clinical research in the H2020 programme. Another relevant achievement is related to the call for the EU Commission to strengthen the role of European networking for paediatric clinical research, and to ensure that Member States enact measures to support research into and the development and availability of medicinal products for paediatric use.
On the other side, the Resolution shows a redundant and not justified reference to the theme of oncology. In fact, despite recognising the low availability of duly-developed anticancer drugs and the devastating consequence for the children’s health and well being, oncology is not the only neglected therapeutic area in paediatrics. Just to mention some examples, this is also the case for inborn errors of metabolism, neonatal and rare diseases.
Aside from the specific contents of the Resolution, we welcome the point that to date progresses in the paediatric setting cannot be satisfactory: the 10-year report has shown us the light and the shadows of the Regulation, but an in-depth and shared analysis is needed to focus the efforts in several fields.
The full-text of the document is available here.