A post-authorization efficacy study, or PAES, addresses scientifically substantiated uncertainties regarding the efficacy of an already authorized medicinal product. It is imposed by a competent authority when relevant efficacy questions can or should only be answered after authorization. The results can influence the benefit-risk assessment and the product information.<\/p>\n
The obligation for a PAES may arise upon granting authorization if certain aspects of efficacy have not yet been conclusively clarified and need to be investigated after marketing. It can also be ordered later if new insights into disease understanding, clinical methodology, or real-world application suggest a significant re-evaluation of previous efficacy assessments.<\/p>\n
Delegated Regulation (EU) No. 357\/2014 describes situations in which such a study may be imposed. These include, for example, ambiguities regarding efficacy in clinically relevant subgroups, long-term efficacy, or efficacy when used under routine conditions. A PAES is therefore not an arbitrary post-marketing study, but a targeted regulatory requirement to close a substantiated evidence gap.<\/p>\n
The requirement integrates the study into the framework of the authorization decision. For centrally authorized medicinal products, imposed post-authorization efficacy studies are documented in Annex II of the authorization decision. The study concept must answer the specific regulatory question and provide a comprehensible justification for endpoints, population, comparison, and evaluation. Depending on the question, the investigation can be designed as a clinical trial or in another suitable study format.<\/p>\n
The data obtained must be submitted to the authority. They are not read in isolation but evaluated in conjunction with all available efficacy and safety information. Possible consequences range from an update of the product information to further obligations or measures if the benefit-risk ratio changes. A PAES therefore belongs in a controlled lifecycle management, not at the end of development planning.<\/p>\n
An authority-imposed PAES must be distinguished from voluntary health services research. Companies can also collect data on treatment practice or efficacy without a specific requirement. However, this does not make the investigation a PAES in the regulatory sense. Characteristic is the specific requirement of the competent authority, triggered by scientific uncertainty. It defines the relationship between the open question, the medicinal product, and the evidence to be provided.<\/p>\n
The design of the study does not follow a rigid universal design. It is crucial that it reliably answers the underlying question. This may focus on efficacy in a specific population, the duration of an effect, suitable endpoints, or the transferability of authorization data. The sponsor must plan the implementation in such a way that the result is regulatively interpretable and does not merely generate additional data without decision-making value.<\/p>\n
A PAES should be managed concurrently with the other authorization obligations. Responsibilities for study leadership, data evaluation, safety assessment, and regulatory communication must be clear. When results become available, not only the study report is relevant: it must also be assessed whether they affect the authorized indication, instructions for use, or further requirements. The scientific question thus remains the benchmark throughout the entire study cycle.<\/p>\n
The PAES concerns efficacy questions. Distinct from this is the post-authorization safety study, or PASS. A PASS serves to identify, characterize, or quantify a safety risk, confirm the safety profile, or measure the effectiveness of risk minimization measures. Its research interest is thus pharmacovigilance-related.<\/p>\n
Efficacy and safety can be considered together in daily study practice, but regulatively, PAES and PASS remain distinct instruments. The existing related entry ‘post-authorisation-safety-study’ specifically refers to this safety instrument. A study should therefore not be classified as a PAES or PASS solely because of its timing after authorization; the objective and regulatory basis are decisive.<\/p>\n
A PAES requires an early translation of the requirement into a methodologically sound protocol. Recruitment, population, comparison strategy, endpoints, data sources, and governance must be tailored to the specific efficacy uncertainty. Unclear questions, unsuitable endpoints, or delayed study startups not only jeopardize the validity but also the fulfillment of the authorization requirement.<\/p>\n
Full-service CROs like Mediconomics support protocol development, feasibility assessment, study management, monitoring, data management, biostatistics, and medical writing. They also help align the results with regulatory obligations, safety processes, and planned updates to the product information.<\/p>\n
Is every post-marketing study a PAES?<\/strong><\/p>\n No. A PAES is a study focused on efficacy uncertainties and imposed by an authority. Other post-authorization studies may pursue different purposes.<\/p>\n Can a PAES change the product information?<\/strong><\/p>\n Yes. The data can become relevant for the benefit-risk assessment and thus for the product information or further regulatory measures.<\/p>\n Is a PAES different from a PASS?<\/strong><\/p>\n Yes. The PAES addresses efficacy, while the PASS focuses on the safety and safety profile of an authorized medicinal product.<\/p>\n A post-authorization efficacy study, or PAES, addresses scientifically substantiated uncertainties regarding the efficacy of an already authorized medicinal product. It is imposed by a competent authority when relevant efficacy questions can or should only be answered after authorization. The results can influence the benefit-risk assessment and the product information. When a PAES is Imposed The […]<\/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":[20],"class_list":["post-8056","glossary","type-glossary","status-publish","hentry","glossary-cat-regulatory-affairs-zulassung"],"acf":[],"related_terms":"","external_url":"","internal_reference_id":"","_links":{"self":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary\/8056","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":0,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary\/8056\/revisions"}],"wp:attachment":[{"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/media?parent=8056"}],"wp:term":[{"taxonomy":"glossary-cat","embeddable":true,"href":"https:\/\/mediconomics.com\/en\/wp-json\/wp\/v2\/glossary-cat?post=8056"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Regulatory References<\/h2>\n
\n