Real-World Adoption of Orphan Devices: Early Trends and Case Study

Strategic Insights for Early Adoption of the EU Orphan Device Framework

While the orphan device framework in the EU is still in its early stages, there are already emerging trends that highlight how manufacturers are engaging with this pathway.

[Download the EU Orphan Device Clinical and Regulatory Playbook]

Insights from industry discussions, regulatory panels, and recent applications suggest that while orphan device approvals will remain rare, the framework is gaining traction among companies looking for strategic opportunities in niche markets.

Industry Response: Who is Applying for Orphan Device Status?

Since the MDCG guidance was published in mid-2024, only a handful of applications have been submitted for orphan device designation. According to insights from notified bodies and competent authorities:

• The first wave of applicants consists of legacy device manufacturers whose products were previously on the market but were discontinued due to high MDR compliance costs.
• Several companies are revisiting old device lines, exploring whether they qualify for orphan status to re-enter the market under a lower regulatory burden.
• Startups and innovative manufacturers with novel technologies (especially in pediatric cardiology and neurology) are beginning to explore this route as an alternative to full MDR approval.

While notified bodies remain cautious, the expert panel review process has already selected multiple devices for evaluation. This early engagement suggests that interest in orphan device designation is growing steadily.

Case Study: The Pediatric Cardiology Device Shortage

One of the biggest drivers of the orphan device discussion is the shortage of pediatric cardiac devices in the EU. Several manufacturers have already withdrawn critical devices from the market, forcing hospitals to seek exemptions for off-label use or import alternatives from outside Europe.

Example: A European manufacturer that previously produced a specialized catheter for congenital heart defects was forced to discontinue sales due to MDR-related costs. After engaging with regulators, they applied for orphan device status to reintroduce the product.

Key Takeaway: Companies that strategically reposition their devices under orphan status may regain market access while avoiding the full cost of MDR compliance.

This trend underscores the importance of early engagement with expert panels to assess whether previously discontinued products can be brought back under the new orphan device pathway.

The Role of DECODE and Advisory Committees

In parallel with the MDCG guidance, initiatives such as DECODE have emerged to encourage manufacturers to participate in the orphan device program. DECODE is an independent organization promoting the adoption of orphan device pathways, helping companies navigate regulatory requirements and prepare stronger applications.

Key elements of DECODE’s role:

• Providing educational resources on how to justify orphan device status.
• Assisting manufacturers in collecting epidemiological and clinical data for regulatory submissions.
• Advocating for greater alignment between notified bodies and expert panels to streamline approval processes.

With regulatory bodies actively promoting awareness of orphan devices, companies that engage with organizations like DECODE can accelerate their market entry and strengthen their application strategies.

Structured Dialogue: The Importance of Early Regulatory Engagement

A recurring theme in successful orphan device applications is early engagement with regulators. Manufacturers who approach notified bodies and expert panels before submitting full applications benefit from:

• Clarified regulatory expectations – Reducing uncertainty about data requirements.
• Preliminary feedback on clinical evidence – Ensuring that alternative data sources (e.g., in silico modeling) will be considered valid.
• Avoiding costly rework – Companies that fail to engage early risk spending unnecessary resources on studies that may not be needed.

Several manufacturers have reported that by aligning with expert panels early, they were able to refine their evidence strategy and accelerate their approval process.

Future Outlook: What to Expect in the Next Two Years

The EU’s orphan device framework is still evolving, and industry adoption is expected to increase gradually as manufacturers recognize the regulatory and commercial advantages of this pathway. Key predictions for the next 12–24 months include:

• A rise in applications from legacy device manufacturers – Particularly for devices that were previously discontinued due to MDR compliance costs.
• Greater alignment between notified bodies and expert panels – As experience with orphan device applications grows, regulatory inconsistencies will likely be addressed.
• Potential funding or reimbursement incentives – European authorities may introduce financial support mechanisms to encourage orphan device development, particularly in high-need areas like pediatric care.

While orphan device approvals will remain a small fraction of overall MDR certifications, they will play a critical role in maintaining access to life-saving medical technologies for rare conditions.

Why Manufacturers Should Act Now

The first movers in this space will shape the future of orphan device approvals, gaining a competitive advantage in a market where demand is high but supply is limited. Manufacturers that engage now can:

• Benefit from free expert panel reviews before the program ends in 2025.
• Reintroduce previously discontinued products under a more flexible regulatory pathway.
• Position themselves as market leaders in high-need, low-competition areas.

For companies considering orphan device status, the time to act is now. By leveraging early regulatory engagement, aligning with advisory initiatives like DECODE, and strategically navigating the framework, manufacturers can successfully bring their orphan devices to market while addressing urgent patient needs.