A number of regulatory provisions, structures and procedures aim at increasing the development of medicines for use in children.
Paediatric Investigation Plan
A Paediatric Investigation Plan is a development plan aimed at ensuring that necessary data are obtained through studies in children, when it is safe to do so and for all subsets of the paediatric population (i.e. from birth to 18 years), to support the marketing of a medicine for children.
PIP in the European Union
According to Regulation (EC) No 1901/2006, the submission of a PIP application is mandatory for medicinal products not yet authorized in the European Economic Area (EEA), as well as for authorized medicinal products with supplementary patent certificate – line extensions, new indications and new formulations – even if the condition does not occur in children. A PIP must be agreed with regulators before submitting a Marketing Authorization Application.
A PIP includes the timing and description of the studies to be conducted in children, as well as the measures to adapt the medicine's formulation to ensure a safe and adapted use within the pediatric population. PIPs are submitted as soon as Phase 1 pharmacokinetics study results are available. PIPs can be modified at a later stage, as knowledge about the product increases or in case the implementation of the agreed plan appears to be unworkable or no longer appropriate.
For specific medicinal products or classes of medicinal products, PIP requirements can be waived. It is also possible to defer some of the studies until after the completion of trials in adults – timing and details of the deferred studies have to be provided and agreed upon at the time of initial PIP submission. Herbals, homeopathy, well-established-use and cellular blood components medicinal products are exempted from PIP requirements.
The European Medicines Agency’s Paediatric Committee (PDCO) is in charge of agreeing on or rejecting PIPs, as well as of ensuring that pharmaceutical companies are in compliance with all agreed requirements at the time of submission of Marketing Authorization Applications.
Pharmaceutical companies fulfilling all pediatric requirements are granted an additional 6-month data protection for both adult and paediatric condition.