Open Call: Orphan Medical Devices Targeting Paediatric Patients

Medical devices and in vitro diagnostics have a fundamental role in saving lives by providing innovative healthcare solutions for the diagnosis, prevention, monitoring, prediction, prognosis, treatment or alleviation of many diseases, including noncommunicable diseases such as cancer, cardiovascular diseases or diabetes.

Medical devices are subject to Regulation (EU) 2017/74519, while in vitro diagnostic medical devices are subject to Regulation (EU) 2017/74620.

For the purpose of this action, orphan devices are medical devices, including in vitro diagnostic medical devices, that benefit a relatively small group of patients in the treatment or diagnosis of a disease or condition and where no or only insufficient suitable alternative therapeutic or diagnostic options with expected similar clinical benefit and safety exist. Criteria for orphan devices are specified in guidance MDCG 2024-10 Clinical evaluation of orphan medical devices.

The need to consider the specificities of orphan devices has been repeatedly highlighted as an area of priority by the Commission as well as by the Member States and the European Parliament.

At the Union level, no specific legislation exists regarding the development and/or the market access of orphan devices which are in a large part intended for paediatric patients.

The level of clinical evidence that is required to place medical devices on the market has been increased by the MDR, including an increased need for pre-market clinical investigations for certain higher risk devices to verify their safety and clinical performance. These increased clinical evidence requirements present a challenge for devices specifically intended for use in rare diseases/conditions, or in specific indications for rare cohorts of patients with an otherwise non-rare disease/condition. In many cases, orphan devices are intended for use solely or predominantly in minors and paediatric populations, and/or in emergency situations. Proactively generating clinical data within an appropriate time in small patient populations is particularly challenging, as is the case for vulnerable populations in light of the ethical and regulatory requirements to appropriately protect these populations, as well as greater practical challenges of performing clinical studies in certain cohorts such as infants and children.

Paediatric patients usually differ from adults in terms of their size, growth, development, body chemistry, and disease propensity, adding to the challenges of paediatric device development. Costs related to market access, in particular clinical evaluation and conformity assessment, often render the development of paediatric devices economically not interesting. Innovation for paediatric patients therefore lags behind the advances made for adult devices.

This action implements the EU4Health Programme’s general objective of improving the availability, accessibility and affordability of medicinal products, medical devices, and crisis relevant products (Article 3, point (c), of Regulation (EU) 2021/522) through the specific objectives defined in Article 4, points (a), (c) and (h), of Regulation (EU) 2021/522.

The focus areas include supporting platforms for academic bodies, scientific societies, developers of devices, especially SMEs, and NGOs to foster and guide orphan device development in areas of unmet medical needs and paediatric populations. It seeks activities spanning development, design, production, distribution, intellectual property advising, prototyping, engineering, laboratory and animal testing, clinical investigation design, mapping unmet needs, encouraging innovation, managing projects through development stages, connecting developers to financing, assessing scientific merit, gathering pre-clinical data, business development support, training, regulatory advising to achieve CE marking, and leveraging scientific advice procedures. The call highlights the special challenges related to orphan devices due to small patient populations, ethical and regulatory requirements, paediatric patient differences, and increased clinical evidence needs under current regulations. It emphasizes promoting paediatric device innovation which currently lags behind adult device advances.

The programme is designed to enhance availability, accessibility, and affordability of orphan medical devices primarily targeting paediatric populations, aiming to address unmet medical needs and improve health outcomes. Eligible applicants include non-profit entities or consortia comprising academic bodies, scientific societies, small and medium enterprises, and NGOs with a special interest in innovative medical devices.

This initiative builds on successful experience with previous EU4Health calls, taking inspiration from the US FDA Paediatric Device Consortia Grants Program. It encourages managing clinical data collection and evaluation projects, fostering conformity assessment to EU regulations, supporting prototype development, and assisting in post-marketing needs to accelerate orphan device market access.

The estimated EU contribution for this topic is € 1,200,000.

For more information, visit EC.