The European Medicines Agency (EMA) provided detailed revised guidance "Procedural Advice for Orphan Medicinal Product Designation" for sponsors seeking orphan medicinal product designation. This guidance aims to streamline the designation process, ensuring compliance with legislative requirements while promoting the development of treatments for rare conditions.
Orphan Medicinal Product: A drug intended for the diagnosis, prevention, or treatment of rare diseases, affecting ≤5 in 10,000 individuals in the EU. Orphan medicinal products address critical unmet medical needs, particularly for rare and debilitating diseases. EMA’s streamlined process ensures sponsors can navigate regulatory requirements efficiently, fostering innovation and improving patient outcomes.
The main goals of orphan designation are:
Addressing life-threatening or debilitating conditions.
Ensuring medical plausibility for the proposed use.
Verifying that the prevalence in the EU is ≤5 in 10,000 or demonstrating insufficient market return without incentives.
Highlighting significant benefits over existing treatments.
The general principles outlined in the EMA guidance for orphan medicinal product designation provide a structured framework for sponsors to navigate the process effectively and some of the key highlights are
The EMA highly recommends pre-submission meetings as an opportunity for sponsors to align their orphan designation application with regulatory expectations. These meetings serve as a forum to clarify application requirements, discuss potential challenges, and ensure completeness before formal submission.
The EMA assigns rapporteurs from the Committee for Orphan Medicinal Products (COMP) to evaluate each application. Rapporteurs are tasked with thoroughly reviewing the scientific rationale, prevalence data, and significant benefit claims provided by the sponsor.
Sponsors must use EMA's IRIS Portal for all submissions and communications. Key steps include:
Pre-submission meetings: Recommended to address potential issues.
Rapporteur appointments: EMA assigns experts to evaluate applications.
Submission and validation: Applications are rigorously checked for compliance with procedural requirements and completeness.
While most fees are waived for orphan designation applications, administrative charges apply in specific scenarios, such as withdrawn or rejected applications. SMEs (Small and Medium Enterprises) benefit from full fee waivers, reinforcing EMA’s commitment to innovation.
The Committee for Orphan Medicinal Products (COMP) conducts evaluations. Sponsors receive either:
A negative opinion, with options to appeal or resubmit with additional data
A positive opinion, advancing the application to the European Commission for final approval.
The sponsor receives official notification and benefits such as market exclusivity and fee reductions if approved. The decision is published in the EU’s Community Register of Orphan Medicinal Products, ensuring transparency.
From pre-submission meetings to final decisions, each step is designed to support sponsors in bringing innovative treatments for rare diseases to market. For more detailed insights, download the complete guidance here.
Comments