The European Medicines Agency (EMA) has issued comprehensive guidance "European Medicines Agency Pre-Authorisation Procedural Advice for Users of the Centralised Procedure" offering procedural advice for sponsors and applicants preparing for the centralised authorisation procedure. This guidance provides insights into navigating the pre-authorization phase, ensuring compliance with regulatory requirements, and facilitating successful submissions for marketing authorisation applications (MAAs).
The centralised procedure allows applicants to obtain a single marketing authorisation valid across all EU Member States, Iceland, Liechtenstein, and Norway. This pathway is essential for certain product categories, including innovative medicines, biotechnology-derived products, orphan medicines, and those addressing critical public health needs.
The guidance outlines essential processes, clarifies roles, and provides procedural templates for applicants, reducing uncertainties during the pre-authorisation phase.
1. Overview of the Centralised Procedure
Eligibility: Specific products, including those for rare diseases (orphan drugs), advanced therapy medicinal products (ATMPs), and medicines containing new active substances, are required or eligible for this pathway.
Scope: The procedure applies to human and veterinary medicinal products seeking EU-wide marketing authorisation.
Benefits: A harmonised regulatory decision that ensures equal access to medicines across all EU markets.
2. Pre-Submission Activities
To ensure a seamless submission process, applicants are advised to engage in robust pre-submission activities, including:
Eligibility Requests: Confirming eligibility for the centralised procedure early in the development process.
Pre-Submission Meetings: Engaging with EMA to clarify expectations regarding dossier content, procedural timelines, and regulatory requirements.
Product Classification: Confirming whether the product falls under specific categories like orphan designation or ATMPs.
3. Marketing Authorisation Application (MAA) Requirements
The guidance provides detailed instructions on preparing a high-quality dossier, adhering to the Common Technical Document (CTD) format:
Administrative Information: Includes legal status, product name, and manufacturing details.
Scientific Data: Comprising non-clinical and clinical study results, quality assessments, and risk management plans (RMPs).
Good Manufacturing Practice (GMP): Certification for all manufacturing sites involved in the production of the medicinal product.
4. Timelines and Procedural Milestones
The centralised procedure follows a structured timeline:
Submission Validation: EMA verifies dossier completeness within 30 days.
Assessment Phase: Involves evaluation by rapporteurs and co-rapporteurs appointed by the Committee for Medicinal Products for Human Use (CHMP).
Opinion Issuance: CHMP provides its opinion on the MAA within 210 days, excluding clock stops for applicant responses.
5. Handling Orphan Medicines
Products intended for the treatment of rare diseases must hold orphan designation before the MAA submission. Key considerations include:
Demonstrating the medicine’s significant benefit over existing therapies.
Providing data to justify orphan status under Regulation (EC) No 141/2000.
6. Other Key Features
Scientific Advice: Sponsors can request scientific advice or protocol assistance from EMA to optimise development programs.
Accelerated Assessment: Available for medicines addressing unmet medical needs, reducing the review timeline from 210 to 150 days.
Pediatric Investigation Plan (PIP): Mandatory for medicines intended for pediatric use, except for certain exemptions.
This guidance serves as a critical resource for ensuring regulatory preparedness and fostering successful centralised authorisation submissions. By aligning with EMA’s expectations, applicants can:
Expedite the evaluation process.
Minimise the risk of delays or rejection.
Deliver high-quality medicines to the EU market efficiently.
For more details, access the full guidance here: EMA Pre-Authorisation Procedural Advice.
Also, check out this Guidance "European Medicines Agency Procedural Advice for Users of the Centralised Procedure for Similar Biological Medicinal Products Applications" that addresses a number of questions that users of the Centralised Procedure may have about Biosimilars. It provides an overview of the EMA position on issues, which are typically addressed during Pre-Submission interactions/meetings.
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