New pathways for innovatives devices in the EU: Breakthrough Devices (BtX) and Orphan devices

Published on: June 2026

Intro

The European regulatory landscape for innovative medical devices has taken an important step forward with the publication of two major guidance documents: the MDCG 2024‑10 guidance on orphan medical devices released in June 2024 and the MDCG 2025‑9 guidance on Breakthrough Devices (BtX) published in December 2025. Together, these documents introduce for the first time in the EU structured regulatory concepts aimed at supporting devices developed for small patient populations or addressing substantial unmet medical needs.

These concepts, orphan devices and Breakthrough Devices, are new under the EU MDR/IVDR framework. Their introduction brings the EU closer to established acceleration pathways seen elsewhere: for example, the US FDA’s Breakthrough Devices Program or the EU’s own Orphan Drug Designation system for medicinal products. Such parallels highlight a longstanding global recognition that certain high‑impact technologies require adapted evidence‑generation models to reach patients without compromising safety.

Their publication also comes at a strategic moment. The European Commission’s MDR/IVDR revision proposal and multiple stakeholder calls have emphasized the need to rebalance the regulatory framework so it remains innovation‑enabling while preserving robustness. In this broader context, MDCG 2024‑10 and MDCG 2025‑9 directly address recurring bottlenecks such as limited clinical data for rare conditions, unpredictable regulatory expectations for novel technologies, and slow access for patients with urgent unmet needs.

Finally, these two guidance documents are notable for signaling new opportunities: earlier and more structured dialogue with regulators, proportionate evidence requirements, clarified interactions with expert panels, and pathways that can meaningfully accelerate access to transformative medical technologies in Europe.

Breakthrough Devices (BtX): scope, benefits and acceleration opportunities

The MDCG 2025‑9 guidance establishes a structured EU framework for identifying highly innovative medical devices and IVDs expected to provide significant clinical benefits for serious or life‑threatening conditions.

BtX designation requires satisfying two cumulative criteria: technological or clinical novelty; and a significant expected positive clinical impact compared to available alternatives and the state of the art, or fulfilling an unmet medical need.

The designation may apply to all devices under MDR and IVDR meeting eligibility criteria, irrespective of risk class or technology, with the exception of custom‑made devices, in‑house devices and Annex XVI non‑medical purpose products.

MDCG 2025‑9 establishes principles for applying existing MDR/IVDR mechanisms more efficiently to accelerate access without lowering safety and performance requirements.

• It introduces opportunities for early and structured interactions between manufacturers, notified bodies, competent authorities, and expert panels, encouraging proactive engagement early in development, streamlining evidence expectations and reducing development uncertainties and time loss.
  
• It supports flexible evidence requirements, allowing reliance on more proportionate early‑stage data, alternative methodologies, and strong post‑market surveillance when scientifically justified. This approach is particularly relevant for novel technologies where for instance conventional clinical investigations may be challenging to obtain.
• Under the guidance principles, scientific‑advice timelines for high‑impact devices would be streamlined with expert panels prioritising BtX cases and aiming to provide scientific opinions within 60 days, especially after positive BtX designation.

Overall, the guidance is intended to accelerate safe patient access to transformative medical technologies while maintaining rigorous MDR/IVDR compliance, offering manufacturers clearer expectations and earlier regulatory alignment.

Orphan Devices: scope, benefits and acceleration opportunities

According to the MDCG 2024‑10 guidance, a device qualifies as an orphan device when it is:

• Specifically intended for the treatment, diagnosis, or prevention of a disease or condition affecting no more than 12,000 individuals per year in the EU.
• Meeting at least one of the following conditions:
  • Existing options are insufficient for the target condition.
  • The device is expected to provide a meaningful clinical benefit compared to the state of the art, considering device‑ and patient‑specific factors.

Provisions for orphan IVD devices remain absent however. Industry groups have called on this gap and provided views on what could be Orphan IVD definition criteria, on the conformity assessment and performance evaluation aspects and on procedure considerations pursuant to such Orphan IVD, in view not to unduly hinder or delay patient access to these important devices [Doc].

The principles of MDCG 2024‑10 may enable faster, more streamlined orphan device development.

• The guidance provides early eligibility clarity with a quantitative prevalence threshold and qualitative criteria related to unmet need and expected clinical benefit. This allows developers to strategically plan evidence generation and regulatory interactions without months of ambiguity, compressing early‑stage timelines.
• It is clarified that all potential sources of non-clinical data should be considered for orphan devices (e.g., laboratory and animal tests; data from computer modelling and simulations, etc.), potentially reducing the burden of pre-market clinical data and helping to justify CE marking with limitations in clinical data where the non-clinical data provide substantial high-quality evidence.
• Limited pre‑market clinical data is accepted when scientifically justified. Acknowledging that rare populations make large, traditional clinical trials impractical, limited pre‑market clinical data may be acceptable, provided the rationale is well‑justified and supplemented by strong non‑clinical data and post‑market plans. This proportionate approach enables earlier CE certification for promising devices, reducing time to market.
• Relying on blended, phased evidence‑generation models where manufacturers may rely on a combination of targeted pre‑market studies, robust PMS/PMCF strategies, and real‑world evidence collection post‑market, is particularly valuable for devices used in very small populations where patient recruitment is slow or unpredictable. Developers can progress more rapidly through conformity assessment while still building the full evidence base over time.
• Mechanisms enabling both manufacturers and notified bodies to seek early input from EMA expert panels on orphan status justification and adequacy of proposed clinical strategies can prevent late‑cycle challenges that often delay MDR assessments and help overcome bottlenecks that historically limited access to orphan devices in the EU.
• Notified bodies may issue certificates with special conditions, such as mandatory PMCF activities when pre‑market evidence is encouraging but incomplete, enabling earlier patient access while ensuring that confirmatory data will be generated post-market.

Together, these mechanisms meaningfully shift the EU regulatory framework toward practical feasibility for rare‑population devices.

The guidance also introduces several broader clarifications that improve predictability and consistency for developers, such as guidance on extrapolation and the use of surrogate populations. 

Summary 

By creating feasible, proportionate, and more transparent regulatory expectations, MDCG 2024‑10 and MDCG 2025-9 address long‑standing challenges faced by developers of rare‑population and highly innovative devices. It enables:

• Reduced regulatory uncertainty, thanks to a unified definition and clear criteria.
• More predictable pathways, supported by early advice and structured evidence expectations.
• Faster market entry, through proportionate evidence requirements and conditional certification.
• Stronger support for SMEs and academic innovators, who are often the primary drivers of rare‑disease device innovation.

Overall, these guidance represent a decisive shift toward a more innovation‑enabling EU ecosystem while maintaining rigorous safety and performance standards.

Despite being a significant step forward, MDCG 2024‑10 and MDCG 2025-9 do not fully resolve the structural obstacles facing orphan and BtX device developers. Key challenges that persist include the absence of formal, distinct or accelerated regulatory pathways; persistently heavy evidence requirements, even with proportionality; Notified Body capacity constraints that can delay assessments; Inconsistent access to expert panels, with no mandated timelines, etc.

On the other hand, Ppart of the MDR/IVDR revision process, some proposed revisions impacting orphan and BtX devices are under consideration, that may further reinforce streamlining and acceleration of the development of innovative devices in more practical ways, notably:

• Possibility for BtX devices and orphan devices to be subject to priority and rolling review.
• Possibility of ‘grandfathering’ of legacy orphan devices that were CE marked under the former Directives and for which an expert panel has confirmed that they meet the criteria of ‘orphan device’.

The MDR revision proposal also includes further evolutions that may support the development of orphan devices:

• The possibility for manufacturers of orphan devices to benefit from notified body fee reductions of at least 50%.
• ‘Grandfathering’ of certain MDD orphan devices, allowing their continued placing on the market beyond the transitional periods.