The European Medicines Agency (EMA) has revamped its application procedure for orphan medicinal product designation by fully integrating the IRIS portal. This move aims to streamline submissions and improve interactions between sponsors and regulatory authorities, ensuring a more efficient review process for treatments targeting rare diseases.
Streamlined Submission via IRIS Portal
All applications for orphan medicinal product designation must now be submitted exclusively through the IRIS | Regulatory & Scientific Information Management Platform. This secure online portal not only manages initial submissions but also oversees post-designation activities, enhancing data management and stakeholder communication. Sponsors are encouraged to request pre-submission meetings via IRIS to discuss their applications in detail, allowing for thorough preparation and quality checks before formal submission.
Guidelines and Appeal Procedures
EMA has outlined comprehensive guidelines to assist sponsors in navigating the designation process. Key criteria include demonstrating the life-threatening or debilitating nature of the condition, the medical plausibility of the treatment, and the rarity of the condition within the EU. In cases where applications receive a negative opinion, sponsors have the right to appeal, provided they adhere to specific timelines and procedural requirements outlined by the agency.
• EMA mandates submissions prior to any marketing authorization applications.
• Pre-submission meetings are free and conducted via TEAMS, fostering proactive dialogue.
• SMEs benefit from waived administrative charges, reducing financial barriers.
Sponsors must ensure their applications are comprehensive and comply with EMA guidelines to avoid delays. The integration of the IRIS portal represents a significant improvement in the designation process, potentially accelerating the availability of critical treatments for rare diseases.
EMA’s updated procedures underscore the agency’s commitment to supporting the development of orphan medicinal products. By providing clear guidelines and facilitating easier submission processes, EMA aims to encourage more sponsors to invest in treatments for rare and underserved conditions. This initiative not only benefits patients with rare diseases but also fosters innovation within the pharmaceutical industry.
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