EU medical devices regulation

Proposals to simplify the EU Medical Device Regulation (MDR) and the EU In Vitro Diagnostic Medical Device Regulation (IVDR) include the introduction of statutory orphan and breakthrough designations for certain categories of medical device and in vitro diagnostic medical device (IVD) that serve unmet medical needs in the EU.

One of the most striking aspects of the simplification proposal is the introduction of these designations in binding legislation. Devices that obtain orphan or breakthrough designation would, under the proposal, be entitled to a range of procedural and substantive benefits before and during the conformity assessment process, with the stated objective of facilitating faster access to the EU market.

This article explains the orphan and breakthrough designations set out in the simplification proposal and considers how impactful the proposal is likely to be in practice.

Breakthrough designation

Under the proposal, a device will be entitled to breakthrough designation if it meets the following criteria:

• it is expected to introduce in the Union a high degree of novelty with respect to the device technology, the related clinical procedure, or the application of the device in clinical practice; and

• it is expected to provide a significant positive clinical impact on patients or public health for a life-threatening or irreversibly debilitating disease or condition, by either:

  • offering a significant positive clinical or health impact compared to available alternatives and the state of the art; or

  • fulfilling an unmet medical need where there is an absence or insufficiency of available alternative options for that purpose.

In simplified terms, a device will qualify for breakthrough designation where it is both highly novel and capable of delivering a significant positive health impact for a serious disease or condition, either by improving on existing alternatives or by addressing a medical need for which alternatives are non-existent or inadequate.

Further guidance on these concepts is provided in the Medical Device Coordination Group (MDCG) guidance on breakthrough devices, which was issued alongside the proposal in December 2025.

To obtain breakthrough designation, either the manufacturer or the Notified Body responsible for the conformity assessment must submit a substantiated request to an expert panel convened by the European Medicines Agency (EMA). The expert panel will issue an opinion on whether the breakthrough criteria are met, and that opinion will be published on a dedicated website.

While the opinion of the expert panel will not be legally binding, the Notified Body will be required to give it due consideration and to provide a reasoned justification for any departure from the panel’s conclusions.

Orphan designation

Under the proposal, a device will be entitled to orphan designation if it meets the following criteria:

• it is intended for the treatment, diagnosis, or prevention of a disease or condition that presents in not more than 12,000 individuals in the Union per year; and

• at least one of the following conditions is met:

  • there are insufficient available alternatives; or

  • the device is expected to provide a clinical benefit compared to available alternatives or the state of the art, taking into account both device-specific factors and patient population-specific factors.

The criteria for orphan designation are narrower and simpler than those for breakthrough designation. Orphan designation is directed at devices intended to address rare diseases or conditions, where existing options are limited or where the device offers a meaningful improvement over current alternatives.

As with breakthrough designation, a substantiated request must be submitted by either the manufacturer or the relevant Notified Body to an expert panel, which will issue an opinion on whether the orphan criteria are met. The Notified Body will again be required to give the opinion due consideration and to justify any departure from it.

Notably, guidance on orphan devices already exists, with an MDCG document published in June 2024. The MDCG guidance on breakthrough devices confirms that the earlier orphan guidance remains relevant to the new statutory scheme introduced by the simplification proposal.

Benefits of orphan or breakthrough designation

Manufacturers of devices that obtain orphan or breakthrough designation will be entitled to a number of procedural advantages, including:

• access to scientific advice from an expert panel convened by the EMA on clinical development strategy and the appropriate preclinical or clinical data required for the clinical evaluation of the device;

• priority rolling review by the Notified Body during the conformity assessment process, meaning that the device should be prioritised in planning and resource allocation;

• the possibility for the Notified Body to grant a Certificate of Conformity on the basis of limited clinical data where long-term evidence is lacking, provided that the benefit of immediate market availability outweighs the risks associated with data limitations, or where the benefit-risk ratio is favourable and the manufacturer commits to generating additional data through post-market clinical follow-up (PMCF); and

• an obligation on the Notified Body to have due regard to any opinion issued by an expert panel when assessing whether the benefits of market availability outweigh limitations in the clinical evidence.

Taken together, these measures are intended to facilitate earlier market access for orphan and breakthrough devices, including in circumstances where the available clinical evidence is necessarily limited. In such cases, PMCF studies are expected to play a central role in generating additional data post-certification.

The MDCG guidance on orphan and breakthrough devices indicates that Notified Bodies should impose conditions on certificate validity to mitigate the risks associated with limited clinical data. These may include requirements to disclose the device’s orphan or breakthrough status on the label, to highlight areas where the risk profile is not yet fully understood, such as long-term use, and to ensure that mechanisms for reporting safety incidents are clearly communicated to users.

The proposal also introduces a significant derogation for legacy devices, defined as devices holding a valid certificate issued under the former medical device directives. Where such a device obtains orphan designation, its existing Certificate of Conformity would remain valid indefinitely, notwithstanding the ordinary transition periods under the MDR and IVDR, provided that the device undergoes no significant changes to its design or intended purpose and continues to meet the criteria for orphan designation.

A brief regulatory history

Although the Commission now proposes to codify orphan and breakthrough designations in the MDR and IVDR, regulatory guidance in this area is not new. MDCG guidance on orphan devices has been in place since 2024, and references to that guidance in the new breakthrough guidance suggest that it remains relevant under the proposed statutory regime, albeit potentially subject to future updates.

Even earlier, a MEDDEV guidance document on the clinical evaluation of so-called breakthrough devices was issued under the former medical device directives in 2019. That guidance adopted a broader conception of a breakthrough device, focusing on whether the device addressed a life-threatening or debilitating disease or condition and whether existing alternatives were adequate. There was no requirement for technological novelty or rarity of the disease or condition.

As a result, many devices that would now fall within the proposed orphan or breakthrough categories would previously have been considered breakthrough devices under the MEDDEV framework.

The regulatory approach endorsed by both the MDCG orphan guidance and the earlier MEDDEV breakthrough guidance closely resembles the approach now proposed in legislation. Where available evidence indicated that a device addressed an unmet medical need, Notified Bodies were encouraged to accept a lower standard of clinical data, such as evidence derived from smaller patient populations or with limited long-term follow-up. To offset this reduced evidentiary threshold, certificates were to be granted subject to conditions, including PMCF requirements, enhanced warnings to users, and intensified post-market surveillance.

In practice, therefore, Notified Bodies have been able to grant Certificates of Conformity for orphan and breakthrough devices on the basis of limited clinical evidence for several years. From the perspective of legal certainty alone, codifying these practices in binding legislation is a welcome development. The more difficult question is what, in practical terms, will change under the simplification proposal.

Assessing the impact of orphan and breakthrough designation

The orphan and breakthrough designations form part of a longer-term policy trajectory aimed at facilitating market access for devices addressing unmet medical needs. The proposal goes further than previous guidance by embedding these mechanisms in legislation and recalibrating institutional roles.

Several aspects are particularly notable.

First, responsibility for determining whether a device qualifies as orphan or breakthrough would shift from Notified Bodies to expert panels convened by the EMA. While expert panels already exist under the MDR and IVDR and can advise on clinical evidence and regulatory classification, the proposal would allow manufacturers to apply directly to an expert panel for designation and scientific advice at an earlier stage of the conformity assessment process.

Second, the proposal materially alters the position of Notified Bodies. At present, a Notified Body considering whether to grant a conditional Certificate of Conformity for a device supported by limited clinical evidence must determine for itself whether doing so is appropriate, bearing the associated regulatory and liability risks. Under the proposal, Notified Bodies would be under a statutory obligation to grant a Certificate of Conformity for orphan or breakthrough devices where the benefits of immediate market availability outweigh the risks arising from evidentiary limitations.

Expert panel opinions would play a central role in this assessment. While Notified Bodies would retain formal decision-making authority, they would be required to justify any departure from an expert panel’s opinion. In practice, this is likely to encourage greater deference to expert panels, particularly where opinions are clearly reasoned and evidence-based, although Notified Bodies would remain entitled to seek clarification where necessary.

Third, the introduction of priority rolling review is new and, in principle, should accelerate conformity assessments for designated devices. However, the precise legal content of priority remains under-defined. The MDCG breakthrough guidance refers to priority only briefly and does not specify what, if anything, manufacturers may legitimately demand by way of accelerated timelines.

Finally, the indefinite validity of certificates for legacy orphan devices represents a significant departure from the existing transition framework. Provided that the relevant conditions are met, this measure should ensure continued market access for certain rare-disease devices without the need for recertification under the MDR or IVDR.

Overall, the orphan and breakthrough designation framework should be of considerable interest to manufacturers and their advisers. It signals a willingness on the part of EU legislators to tolerate a higher degree of regulatory risk in order to ensure that innovative devices and devices addressing unmet medical needs are not discouraged from early entry into the EU market.

That said, caution remains appropriate. The practical impact of the proposal will depend heavily on how expert panels interpret the designation criteria, the extent to which Notified Bodies rely on expert opinions, and how risk-mitigation conditions are applied in practice. Nevertheless, the proposal represents a meaningful step towards a more flexible and predictable regulatory pathway for high-need medical devices, and its progress through the EU legislative process will warrant close attention from regulatory and life sciences practitioners.

Implications for practitioners

The proposal enables manufacturers to seek orphan or breakthrough designation and associated scientific advice at an earlier stage in the conformity assessment process. Practitioners advising life sciences clients should consider front loading regulatory strategy, particularly where clinical evidence is necessarily limited or patient populations are small.

Shift in risk allocation

By placing statutory obligations on Notified Bodies to grant certification where benefit outweighs evidentiary risk, the proposal partially reallocates regulatory risk away from manufacturers. Advisers should closely scrutinise how expert panel opinions are framed, as these are likely to carry decisive weight in certification decisions.

Greater importance of expert panel submissions

Designation requests and scientific advice submissions to EMA convened expert panels will become critical regulatory documents. Legal input will be increasingly important in shaping designation arguments, managing evidentiary presentation, and anticipating how Notified Bodies may interpret or rely on panel opinions.

Conditional certification and compliance planning

Practitioners should expect greater use of conditional Certificates of Conformity, with enhanced post market obligations. Advising on PMCF design, labelling disclosures, surveillance systems and risk communication will become central to compliance strategies for designated devices.

Legacy device opportunities

For clients holding certificates under the former directives, orphan designation may offer a route to indefinite market access without MDR or IVDR recertification. This presents both an opportunity and a compliance risk, requiring careful assessment of whether design changes or expanded indications could jeopardise legacy status.

Regulatory uncertainty remains 

While the proposal increases legal certainty at a structural level, key concepts including priority rolling review and expert panel practice remain under defined. Practitioners should advise clients to plan conservatively until implementation patterns emerge.

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