5 Key Industry Perspectives on Bringing Orphan Devices to Market

Industry Experts Weigh in on the Future of Orphan Devices

Orphan medical devices (OMD) serve critical yet rare patient populations, making their development complex, financially burdensome, and often unfeasible under current regulatory requirements.

[Download the EU Orphan Device Clinical and Regulatory Playbook]

While the European Commission has made strides in addressing regulatory gaps, many challenges remain. We spoke with five industry insiders to understand the regulatory, ethical, and financial hurdles facing orphan devices—and what must change to ensure these devices reach the patients who need them most.

Regulatory Adaptation is Critical

According to Steve Eglem (Former European Commission, Belgium), regulatory incentives, financial support, and clearer guidance are essential to removing market barriers and ensuring manufacturers have a viable pathway for bringing OMDs to patients. He draws a direct parallel to orphan drugs, which have benefited from market exclusivity, fast-track approval, and special designations, allowing them to succeed despite limited patient populations.

However, medical devices lack similar regulatory flexibilities, making investment in these products financially risky for manufacturers—especially SMEs. The DeCODe Project is one initiative working to bridge this gap, fostering collaboration between regulators, industry, and clinicians to create a sustainable framework for orphan device development.

Steve also highlights the importance of balancing early patient access with strong clinical data collection, noting that MDCG guidance clarifies regulatory pathways, but significant challenges remain. One key mechanism that could help is Article 61.10 MDR, which allows manufacturers to justify limited clinical data collection when patient populations are too small to support statistically valid studies. However, more regulatory consistency is needed to ensure that this pathway is usable and predictable for manufacturers across the EU.

Risk-Based Approaches Must Be Strengthened

Marianna Mastroroberto (KIWA ITALIA) emphasizes that when clinical data are not feasible—which is frequently the case for OMDs and pediatric devices—manufacturers must compensate with comprehensive risk assessments and post-market strategies.

Marianna explained that orphan devices often lack the patient volume necessary for traditional clinical trials, making it difficult to meet MDR’s clinical evidence requirements. For pediatric devices, the challenge is different—conducting clinical trials on children often raises ethical concerns, meaning data collection must be approached differently.

In these cases, a tailored approach to risk assessment is essential. Marianna stresses that risk matrices must be customized to account for population-specific variables, including differences in disease progression, physiology, and long-term outcomes.

She sees projects like DeCODe as essential to mapping these challenges and laying the groundwork for harmonized safety and performance standards. Without clear regulatory mechanisms that account for these unique challenges, many manufacturers will abandon orphan and pediatric device development altogether.

Clinical Evaluation of Orphan Medical Devices

Philippe Auclair (PhD, GHWP TC advisor, RAPS Fellow and recipient of Global Leadership Award) called MDCG 2024-10, a very positive development. While it is still fresh in terms of real impact, he recommends applying the same principles to a larger population of innovative devices that are currently missing to cater to unmet medical needs.

“Recent progress in post-market data gathering and analysis (including AI) can enable real-time monitoring of the safety profile in the early stages of the use of a new device while ensuring patients' access to breakthrough technologies.”

Extrapolating Data to Advance Orphan Device Development

Ralf Klein (Radimed GmbH, MDCG TF Orphan Devices, RA Spectaris) believes that one of the most valuable aspects of the MDCG guidance on orphan devices is the ability to extrapolate clinical data from existing products to orphan products.

"The most important point in the guidance is the possibility to extrapolate clinical data from existing products to orphan products—not just orphan products, but also orphan diseases and orphan populations.”

Ralf illustrated this with a real-world example: a company that develops port catheter systems for adults is now facing regulatory barriers when attempting to design smaller ports for neonates.

“They’ve sold thousands of these devices for adults, but now they have a problem when trying to build small ports for babies. The MDCG guidance should make it easier for them to justify extrapolating data from adults, but in practice, they still face barriers.”

Ralf also notes that clinical investigations for certain orphan devices may be unnecessary, particularly for well-understood products where extensive real-world evidence already exists.

“There is no sense in conducting a clinical investigation on these orphan populations if we already have enough experience with adults or other populations.”

The free EMA pilot program could also offer an opportunity for legacy devices to have the validity of the clinical data / clinical strategy confirmed by an expert committee, e.g. with regard to extrapolation. Even if the notified body does not have to follow this expert opinion, it is unlikely that it would be challenged.

His insights highlight a critical issue: without broader regulatory alignment and industry engagement, key programs that could benefit orphan devices will remain underutilized, and many innovative products will never reach the market.

A Public Health Priority, Not Just a Regulatory Hurdle

Professor Stephen Gilbert (Else Kröner Fresenius Center for Digital Health, Germany) warns that current regulatory systems inadvertently exclude the most vulnerable patient groups by applying broad, one-size-fits-all requirements.

“The aim of stringent regulatory frameworks is to ensure safe, high-quality, and effective devices for the majority, but this often disadvantages the few.”

For patients needing orphan devices or pediatric treatments, the cost of development can be prohibitive, effectively making these devices commercially unviable.

Stephen sees DeCODe as a step toward fixing this broken system. The initiative brings together regulatory experts, manufacturers, and clinicians to map out optimal pathways for developing pediatric and orphan medical technologies.

He is optimistic that DeCODe’s findings will lead to improved manufacturer guidance, helping to streamline approvals and reduce regulatory bottlenecks. Without policy intervention and industry-wide alignment, however, Europe risks losing critical medical technologies that serve rare disease populations.

Clinical Evaluation of Orphan Medical Devices

Philippe Auclair (PhD, GHWP TC advisor, RAPS Fellow and recipient of Global Leadership Award) called MDCG 2024-10 a very positive development. While it is still fresh in terms of real impact, he recommends applying the same principles to a larger population of innovative devices that are currently missing to cater to unmet medical needs:

“Recent progress in post-market data gathering and analysis (including AI) can enable real-time monitoring of the safety profile in the early stages of the use of a new device while ensuring patients' access to breakthrough technologies.”

The Bottom Line

The success of orphan devices depends on:

• Regulatory flexibility – Incentives, harmonized pathways, and clearer guidelines for clinical evidence requirements
• Robust post-market follow-up – Strong risk assessment methodologies to compensate for limited pre-market data
• Industry & regulatory collaboration – Greater alignment between regulators, notified bodies, and manufacturers to ensure orphan devices remain viable
• Public health prioritization – Orphan devices should not be commercially abandoned due to excessive regulatory burdens

Europe must adopt a cohesive approach to ensure that innovation serves all patients, not just the majority.