Comparative analysis of the scope of European Union paediatric investigation plans with corresponding orphan designations

BackgroundMarket forces may not be sufficient to stimulate research and development of medicines for small patient populations, such as children and patients with rare diseases. Both the European Union Orphan and Paediatric Regulations were introduced to address the unmet public health needs of these smaller patient populations through the use of incentives, rewards and obligations. Developers for new medicines for rare diseases must agree a paediatric investigation plan (PIP) or waiver with the European Medicines Agency’s (EMA) Paediatric Committee (PDCO), and can also apply for an orphan designation (OD) from the EMA’s Committee of Orphan Medicinal Products (COMP). The scope of both the OD and the PIP (or waiver) is defined by the agreed condition.ObjectivesThe aim of this study was to analyse the approach of PDCO and COMP in defining the appropriate condition for a PIP or OD, respectively, in order to investigate potential challenges in the paediatric development of orphan medicines which have to meet the requirements of both legislations.MethodsA comparative analysis of PIP conditions and OD conditions was performed for medicines that have been reviewed by both Committees.ResultsWe found that in the substantial majority of cases there is no divergence between the conclusions of COMP and PDCO with regard to the condition for which a medicine is to be developed.ConclusionThese findings demonstrate that a collaborative approach allows both Regulations to work synergistically to foster pharmaceutical development for rare diseases in childhood.

Better medicines for children: challenges and unmet needs

Better medicines for children: challenges and unmet needs This review deals with the use of medicinal products in children. Many medicinal products administered routinely to paediatric patients have been either not licensed for use in children (unlicensed use) or have been prescribed outside the terms of their product license (off-label prescribing). A lot of surveys have been conducted in the context of identifying the extent of off-label used drugs in children documenting high rates (45-60 %) of such use. Recognising and meeting challenges as development of suitable formulations for very young children and realization of clinical trials involving children present important steps in the enhancement of the present situation. To improve the health of children in Europe, the Regulation (EC) No 1901/2006 addressing the pharmaceutical industry, research teams and member states proposing important incentives and obligations was assigned. Paediatric Investigation Plan and Paediatric Use Marketing Authorisation present the key measures of the EU regulation on medicinal products for paediatric patients.