Orphan Devices: Why They Matter and What Needs to Change

A Conversation with Former Regulator Tom Melvin on the Overlooked Opportunities in Orphan Medical Devices

We recently connected with Tom Melvin, a regulatory expert and former European regulator, to discuss the critical role of orphan devices, the potential of the Orphan Device Program, and what companies are missing when they dismiss these products as financially unviable.

[Download the EU Orphan Device Clinical and Regulatory Playbook]

 A Moment That Changed Everything

For Tom, his interest in orphan devices wasn’t theoretical—it was personal. It began in October 2021, when he was co-chairing the Clinical Investigation and Evaluation Working Group of EU device regulators. That’s when he started hearing disturbing reports from cardiologists across Europe.

“We started hearing from cardiologists that companies were pulling products they relied on—devices essential to clinical practice were just vanishing,” he recalled. At first, the reasons were unclear. 

However, as the EU MDR increased regulatory costs and complexity, a troubling trend emerged.

These weren’t obscure or outdated products. Many were pediatric cardiology devices, smaller sizes, or highly specialized tools used only a handful of times per year—but when needed, they were irreplaceable. “These are the kinds of devices you might have on a shelf in a cath lab and only need once a year. But when you need them, you need them,” he emphasized.

When these devices disappeared, clinicians were left scrambling for alternatives. It was a moment of realization for Tom. “It was a heart-sink moment. I realized we had unleashed regulatory demons we didn’t fully understand. The MDR made an already fragile market even more challenging.”

The Reality for MedTech Companies

Many companies assume orphan devices aren’t worth pursuing. In Tom’s view, that’s a mistake.

“The common view is: ‘We won’t make any money on orphan devices, so we’re not interested.’ But that’s missing the bigger picture,” he explained. The truth is, orphan devices present real opportunities that most companies overlook.

For one, existing devices could be adapted for niche populations, such as neonates or patients with rare conditions. Many companies already manufacture tools that, with minor modifications, could extend their brand into these underserved markets. Moreover, the clinical evidence burden is often lower for orphan devices compared to standard market entry pathways, which can reduce costs and accelerate regulatory approval.

“If you’re already manufacturing a device, why wouldn’t you explore how it could serve an underserved patient population?” Tom underscored. The regulatory system even recognizes the unique nature of orphan devices, offering pathways with different evidence requirements. Yet, most companies fail to take advantage of this.

The Rashkind Balloon Example: When a Life-Saving Device Disappears

To illustrate what’s at stake, Tom pointed to the Rashkind balloon, a device once manufactured by three companies but now completely unavailable in Europe. This balloon is used in emergency pediatric cardiology cases, yet for years, Europe has relied on temporary national derogations just to keep it accessible.

“For years, Europe has been surviving on bureaucratic workarounds, applying national derogations to keep essential devices available. But that’s not a sustainable strategy,” he said. When companies abandon small-market devices, the consequences extend far beyond business decisions—patients lose access to life-saving interventions.

The DeCODe Project: Mapping the Roadblocks

To address these challenges, Tom has been working on the Developing Child and Orphan Devices, DeCODe Project, a research initiative aimed at understanding the systemic barriers to orphan device development in Europe. The goal is simple: identify where the roadblocks are and how to fix them.

“We’re mapping the ecosystem—looking at what support exists, where the gaps are, and what companies actually need to bring orphan devices to market,” he said. Moreover, DeCODe is currently working with three companies at different stages of development, offering regulatory guidance and innovation coaching.

“Our goal is to pinpoint the real barriers—where companies hit a wall—and identify policy solutions that could make a difference,” Tom emphasized.

One major issue is the cost of EU MDR compliance. As Tom put it bluntly, “Right now, orphan devices can be pushed from ‘barely profitable’ to ‘losing money’ just due to certification costs. That’s a market failure we need to fix.”

What Needs to Change?

There’s no single fix to the orphan device challenge, but Tom believes there are clear ways to improve the system.

For starters, Europe lacks structured support for orphan device development. “In the U.S., we see accelerators dedicated to orphan devices. Europe doesn’t have that kind of framework,” he noted. Without dedicated funding mechanisms and regulatory pathways that account for the realities of niche devices, companies simply walk away from these markets.

Another fundamental issue is the lack of a structured clinical development framework. Currently, there is no clear distinction between devices that should require a full clinical study and those that shouldn’t. “Right now, everything is handled through legal procedures, which lack a solid conceptual foundation,” he said. The result? Unnecessary costs, unnecessary delays, and unnecessary loss of essential products.

A Call to Action

For companies willing to think differently, orphan devices offer real opportunities—but they require a shift in mindset.

“This isn’t just about compliance—it’s about finding ways to keep essential devices available and helping patients who have no alternatives,” Tom said. Companies that dismiss orphan devices outright might be walking away from hidden potential.

His message to MedTech leaders is clear: “If you’re not considering orphan devices, ask yourself why. You might be sitting on an opportunity you haven’t explored.”

Next Steps

For those interested in orphan devices, now is the time to act.

📅 Watch the Webinar Replay on Orphan Devices

📥 Download the Orphan Device Playbook

To hear Tom’s perspective directly, watch the 90-second video clip below, where he explains why this issue is personal to him.

Further Reading on Orphan Devices & Pediatric Medical Devices

For those looking to go deeper into the challenges and policy implications of orphan medical devices, here are two key articles co-authored by Tom Melvin that provide critical insights into the current landscape and potential solutions:

Orphan and Pediatric Medical Devices in Europe: Recommendations to Support Their Availability

This paper, published in Expert Review of Medical Devices, examines the regulatory barriers preventing essential orphan and pediatric devices from staying on the market. It provides policy recommendations to mitigate these risks, including proactive identification of at-risk devices, regulatory flexibility, and financial incentives for manufacturers.

Orphan Medical Devices and Pediatric Cardiology – What Interventionists in Europe Need to Know, and What Needs to Be Done

This Pediatric Cardiology review focuses on the specific challenges faced by pediatric interventionists as orphan devices disappear from the European market. It highlights the impact of the EU MDR on pediatric cardiology and proposes urgent policy interventions to prevent further loss of critical devices.

For regulatory professionals, policymakers, and MedTech innovators, these articles provide essential background on why orphan devices matter—and what can be done to ensure their continued availability.