A Paediatric Investigation Plan (PIP) is a research and development programme for a medicinal product in children and adolescents, agreed upon with the European Medicines Agency. It defines which quality, safety, and efficacy data are to be generated for the paediatric population and when this occurs. The PIP is a core instrument of the European Paediatric Regulation and embeds paediatric aspects early in drug development.<\/p>\n
Regulation (EC) No 1901\/2006 defines the PIP as a research and development programme aimed at generating the data determining the conditions for a marketing authorisation to treat the paediatric population. It does not refer merely to a single clinical trial. Rather, it describes a coherent development programme for the concerned paediatric subpopulations.<\/p>\n
A PIP contains the timeline and the intended measures for assessing quality, safety, and efficacy. These may include clinical and, if applicable, non-clinical investigations, pharmacokinetic concepts, safety measures, as well as the development of an age-appropriate strength, pharmaceutical form, or route of administration. Crucial is the rationale for which subpopulations are investigated, with which methods, and in what chronological sequence.<\/p>\n
This is intended to prevent development from being limited to a retrospective extrapolation of adult data. At the same time, the European framework does not require unnecessary studies in children: the need for data, scientific validity, and the protection of the paediatric participants must be reconciled within the development programme.<\/p>\n
The applicant submits the PIP to the EMA for agreement. The scientific assessment is conducted by the Paediatric Committee (PDCO). This committee specifically evaluates whether the proposed measures and their timing are suitable for obtaining the necessary data for the relevant paediatric subpopulations.<\/p>\n
For certain marketing authorisation applications, the regulation requires the results of all measures included in an agreed PIP or proof of a waiver or deferral. The PIP is thus not the study protocol itself, but the regulatory framework against which the fulfillment of the paediatric requirements is assessed. Individual trial protocols, consent documents, and safety processes are developed separately for the actual conduct.<\/p>\n
Development programmes can change when new data, scientific findings, or practical feasibility issues arise. Therefore, a regulated modification procedure exists for changes to an agreed plan. Early planning is essential because paediatric measures should be integrated into the overall development strategy and not merely just before marketing authorisation.<\/p>\n
A waiver is not a postponed PIP measure, but a full or partial exemption from the obligation to generate paediatric data. The regulation provides for a waiver, for example, if a medicinal product or class of products is likely to be ineffective or unsafe for part or all of the paediatric population, if the disease or condition to be treated occurs only in adults, or if the medicinal product does not represent a significant therapeutic benefit over existing paediatric treatments.<\/p>\n
A deferral, on the other hand, is a postponement. The intended measures remain part of an agreed PIP, but may be completed at a later time. This may be indicated if data from adult studies are required first or if a delay in marketing authorisation for other populations is to be avoided. Waiver and deferral are therefore regulatory decisions in connection with the PIP, but are not to be equated with it.<\/p>\n
The US Pediatric Study Plan, often referred to as the initial Pediatric Study Plan, is also an instrument of paediatric development planning, but is based on US law and submitted to the FDA. It is not a European PIP and replaces neither its agreement with the EMA nor the subsequent EU requirements. EMA and FDA provide joint procedural information for developers using both systems.<\/p>\n
In paediatric studies, the PIP links regulatory targets with the actual conduct of the study. Study protocols must adequately reflect the agreed age groups, endpoints, dosages, pharmaceutical forms, and safety measures. Monitoring and quality management thereby check not only GCP-compliant data collection but also whether the study activities are aligned with the agreed measures and deadlines, and are traceably documented.<\/p>\n
Full-service CROs such as Mediconomics support the translation of a PIP into executable study plans, site management, training and monitoring concepts, data management and pharmacovigilance, as well as documentation for regulatory affairs and medical writing. This allows PIP-related study activities, modification needs, and evidence to be structurally managed across different project teams.<\/p>\n
Is a PIP required for every medicinal product?<\/strong><\/p>\n The obligation depends on the Paediatric Regulation and the respective marketing authorisation application. For certain new medicinal products and certain variations to authorised, protected medicinal products, PIP results or proof of a waiver or deferral must be submitted. For generics and similar biological medicinal products, the relevant requirements do not apply in the same way.<\/p>\n Is a PIP identical to a study protocol?<\/strong><\/p>\n No. The PIP is the overarching paediatric research and development programme. A study protocol, in contrast, describes the detailed planning and execution of a single clinical trial, which may implement a measure of the PIP.<\/p>\n Why can a deferral be useful?<\/strong><\/p>\n A deferral allows the paediatric measures intended in the PIP to be postponed without canceling them. In this way, necessary findings from other development steps can be awaited first, while the obligation for later execution remains in place.<\/p>\n A Paediatric Investigation Plan (PIP) is a research and development programme for a medicinal product in children and adolescents, agreed upon with the European Medicines Agency. It defines which quality, safety, and efficacy data are to be generated for the paediatric population and when this occurs. The PIP is a core instrument of the European […]<\/p>\n","protected":false},"author":10,"featured_media":0,"parent":0,"template":"","meta":{"_acf_changed":false,"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"glossary-cat":[],"class_list":["post-6770","glossary","type-glossary","status-publish","hentry"],"acf":[],"related_terms":"","external_url":"","internal_reference_id":"","_links":{"self":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary\/6770","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary"}],"about":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/types\/glossary"}],"author":[{"embeddable":true,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/users\/10"}],"version-history":[{"count":1,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary\/6770\/revisions"}],"predecessor-version":[{"id":7409,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary\/6770\/revisions\/7409"}],"wp:attachment":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/media?parent=6770"}],"wp:term":[{"taxonomy":"glossary-cat","embeddable":true,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary-cat?post=6770"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Regulatory references<\/h2>\n
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