Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

Figures

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  Figure 1. Overview of current paediatric investigation plan (PIP) application process. The flow diagram was based on the processes outlined in the standard operating procedure for PIP approvals available at European Medicines Agency (EMA) website [13] and information from the document “Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation” [14]. PUMA is a paediatric use marketing authorization granted to a product that has been developed specifically for the paediatric population.

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  Figure 2. Total number of PIPs agreed by the PDCO, subdivided by decision type (see Table 1), since the EUPR was initiated and until 20 October 2019. Data taken from the EMA repository [20]. See methods for details on data mining. PIP: paediatric investigation plan; PDCO: Paediatric Committee; EUPR: European Union Paediatric Regulation; P: decision agreeing on an investigation plan with or without partial waiver(s) and or deferral(s); PM: decision on the application for modification of an agreed PIP; RP: decision refers to a refusal on a proposed paediatric investigation plan; RPM: decision refers to a refusal on the application for modification of an agreed PIP; RW: decision refers to a refusal on a request for waiver in all age groups for the listed conditions; W: decision granting a waiver in all age groups for all conditions/indications.

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  Figure 3. Number of positive (P/PM) PIP decisions and waivers (W) granted per year by the PDCO, since the EUPR was established in 2007. Data from 2007 and 2019 correspond to incomplete years (February to December 2007 and January to October 2019). Data taken from EMA repository [20]. See methods for details on data mining. P: decision agreeing on an investigation plan with or without partial waiver(s) and or deferral(s); PM: decision on the application for modification of an agreed PIP; W: decision granting a waiver in all age groups for all conditions/indications.

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  Figure 4. Total number of PIPs (all decision types combined) granted by the PDCO since establishment of the EUPR in 2007, subdivided by EMA therapeutic areas. Data taken from EMA repository [20]. See methods for details on data mining. PIP: paediatric investigation plan; PDCO: Paediatric Committee; EUPR: European Union Paediatric Regulation.

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  Figure 5. Number of positive decisions (P or PM) granted by the PDCO since the establishment of the EUPR in 2007 for the EMA therapeutic areas. Data taken from EMA repository [20]. See methods for details on data mining. PIP: paediatric investigation plan; PDCO: Paediatric Committee; EUPR: European Union Paediatric Regulation; P: decision agreeing on an investigation plan with or without partial waiver(s) and or deferral(s); PM: decision on the application for modification of an agreed PIP.

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  Figure 6. Number of active substances on the APN list per EMA therapeutic area that require additional development for paediatrics, as outlined by the Paediatric Working Party in 2007. Data taken from [16]. See methods for details on data mining.