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EMA Guidance: Applying for Orphan Designation, IRIS Guide & Orphan Des Procedural Guidance & Forms

The IRIS (Information Repository and Information Submission) platform is the EMA's secure online portal for the submission and management of regulatory and scientific information. The IRIS Guide for Applicants serves as a comprehensive manual that outlines the procedures, technical requirements, and best practices for using the IRIS platform to submit various types of applications, including marketing authorization applications (MAAs), variations, and renewals.

The purpose of this guide is to inform applicants how to prepare, submit, and manage applications and/or data on the IRIS platform, whether it is for orphan designations, scientific advice, requests for ITF briefing meetings, PRIME, marketing status reports, inspections, or applications for Parallel Distributions.

By understanding the requirements and processes outlined in this guide, applicants can ensure that their submissions are accurate, complete, and compliant with the EMA's standards, thereby facilitating a smooth and efficient review process.

A rare disease, also known as an orphan disease, is defined in the EU as a disease affecting not more than 5 in 10,000 persons. Orphan designation is a status granted to a medicinal product intended for the diagnosis, prevention, or treatment of a rare disease. Orphan designation aims to encourage the development of treatments for rare diseases by providing regulatory and financial incentives to sponsors.

By following the guidance provided by the EMA and seeking early engagement with regulatory authorities, sponsors can navigate the orphan designation process effectively and increase the chances of success for their orphan medicinal products. See the Sponsor's guide to an orphan designation for clear visualization of the flow chart.

RPM for PLM refers to the regulatory procedures and processes involved in managing the lifecycle of a medicinal product from its initial development to its post-authorization phases. It encompasses activities such as submission, assessment, approval, and post-marketing surveillance of medicinal products to ensure their safety, efficacy, and quality throughout their lifecycle.

This Q&A details some of the important questions and answers that guide the applicant related to Product Lifecycle Management.


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