Abstract
Objectives: In this paper, we investigated the effects of the European Paediatric Regulation (EC) N° 1901/2006 with respect to satisfying the paediatric therapeutic needs, assessed in terms of the increased number of paediatric medicinal products, new therapeutic indications in specific high-need conditions (neonates, oncology, rare disease, etc.) and increased number of paediatric clinical studies supporting the marketing authorisation.Methods: We analysed the paediatric medicinal products approved by the European Medicines Agency in the period January 2007-December 2019, by collecting the following data: year of approval, active substance, legal basis for the marketing authorisation, type of medicinal product (i.e., chemical, biological, or ATMP), orphan drug status, paediatric indication, Anatomical Therapeutic Chemical code (first-level), number and type of paediatric studies. Data were compared with similar data collected in the period 1996–2006.Results: In the period January 1996–December 2019, in a total of 1,190 medicinal products and 843 active substances, 34 and 38%, respectively, were paediatric. In the two periods, before and after the Paediatric Regulation implementation, the paediatric/total medicinal products ratio was constant while the paediatric/total active substances ratio decreased. Moreover, excluding generics and biosimilars, a total of 106 and 175 paediatric medicines were granted a new paediatric indication, dosage or age group in the two periods; out of 175, 128 paediatric medicines had an approved Paediatric Investigational Plan. The remaining 47 were approved without an approved Paediatric Investigational Plan, following the provisions of Directive 2001/83/EC and repurposing an off-patent drug. The analysis of the clinical studies revealed that drugs with a Paediatric Investigational Plan were supported by 3.5 studies/drug while drugs without a Paediatric Investigational Plan were supported by only 1.6 studies/drug.Discussion: This report confirms that the expectations of the European Paediatric Regulation (EC) N° 1901/2006 have been mainly satisfied. However, the reasons for the limited development of paediatric medicines in Europe, should be further discussed, taking advantage of recent initiatives in the regulatory field, such as the Action Plan on Paediatrics, and the open consultation on EU Pharmaceutical Strategy.
Highlights
In Europe, children represent more than 20% of the population, with about 100 million people aged < 19 years
During the period 2007–2019, 296 medicinal products (MPs) (34%) and 216 active substance (AS) (36%) were approved as paediatric medicines, demonstrating that the paediatric MPs (p-MPs)/MPs ratio remained stable while the p-ASs/ASs ratio decreased compared to the previous period (1996–2006) (Table 1)
Increase Rate of Approved MPs/ASs The annual trend for increase is shown in Figure 2 and demonstrates a relevant increase of MPs approved by the EMA since the set-up of the Agency, while the average annual increase of both p-MPs and p-ASs is significantly lower
Summary
In Europe, children represent more than 20% of the population, with about 100 million people aged < 19 years. With the aim of handling these concerns and assuring that children have safe access to both old and new medicinal products (MPs), the European Paediatric Regulation (EC) N◦ 1901/2006 [5] (Paediatric Regulation) entered into force on 26th January 2007. The Paediatric Regulation established the European Medicines Agency-Paediatric Committee (EMA-PDCO) and made a Paediatric Investigational Plan (PIP) mandatory, prescribing studies in the paediatric population whose results have to be included in the Marketing Authorisation (MA) documentation unless a waiver is granted. It is possible to grant a deferral in order to delay the results of some studies. These provisions apply to any new or in patent drug for which a MA or a MA variation is requested (articles 7 and 8 of the Paediatric Regulation). To compensate for the burden of this requirement, incentives are available to the industry, including a 6-month extension of the supplementary protection certificate and an additional 2 years of market exclusivity for paediatric orphan medicinal products (p-OMPs)
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