In a significant move to address a critical gap in the healthcare industry, a new pilot program has been launched to support orphan medical devices. On August 2, 2024, the European Medicines Agency (EMA) announced that its effort, as mandated under Regulation (EU) 2022/123 (Regulation – 2022/123 – EN – EUR-Lex (europa.eu)), aims to provide the necessary backing for devices that, despite their potential to improve patient outcomes, struggle to find market traction due to their limited commercial appeal. This development represents a vital step in ensuring that innovations in medical technology are not lost in the shuffle simply because they serve niche or less common conditions.
Orphan medical devices are those intended to diagnose, prevent, or treat rare diseases, which are often characterized by a limited patient population. Historically, these devices have encountered several obstacles, including limited financial incentives, regulatory complexities, and challenges in gaining market access.
The term “orphan” is borrowed from the concept of orphan drugs—medications designed for rare diseases. While orphan drugs have seen some support and incentives, orphan medical devices have historically received less attention, failing to attract sufficient investment or interest from large manufacturers due to their limited market potential, despite their critical importance to specific patient populations.
In June 2024, guidance was released by the European Commission in which it outlined the factors that would determine when a medical device would be considered as orphan and the clinical evaluation criteria of such devices by the Medical Device Coordination Group (MDCG).
The program allows for stakeholders, such as medical device manufacturers and notified bodies, to qualify for free expert advice on product development, specifically in the area of clinical strategies. The EMA will select devices to be included in the pilot using a risk-based approach, giving priority to devices for pediatric applications and those treating life-threatening conditions. As the program progresses, it will be essential to monitor its impact and effectiveness in the hopes that with a positive outcome they will be able to employ the model as a structured pathway to enhance their development and market entry.
By offering targeted guidance and support, the program aims to foster innovation, improve patient care, and ensure that essential medical technologies reach those who need them most.
Participation in the program is available now, by request in writing to the EMA. The EMA held a virtual meeting on September 23, 2024 in cooperation with the EU Commission, MDCG, and panel experts to discuss the program in more depth, including how stakeholders can use the portal for requesting advice, what scientific information is available, how to determine orphan device status, and other key topics.
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