EU device package envisages more support for 'orphan' and breakthrough products and flexible access in crises

The Medical Devices Regulation (MDR) and In Vitro Diagnostic Medical Devices Regulation (IVDR) were designed to ensure a high level of patient safety. However, their uniform requirements have proven challenging for devices addressing very small populations or novel technologies with limited commercial prospects.

The targeted evaluation of the regulations launched in 2024 highlighted that adaptive pathways for breakthrough innovation and "orphan" or niche devices do not currently exist within the regulatory framework. This gap has had unintended negative impacts on innovation, competitiveness and patient care.

Stakeholders called for specific regulatory instruments to support devices for rare conditions, facilitate early access to breakthrough technologies under controlled conditions, and allow experimentation via "sandboxes". One of the strongest criticisms of the MDR and IVDR has been that they risked stifling innovation and jeopardising the availability of niche devices serving small or vulnerable patient populations.

The Commission's legislative proposal of 16 December responds by introducing new concepts and pathways for breakthrough and orphan devices, broadening in‑house exemptions, establishing regulatory sandboxes at national and EU level, and refining derogation mechanisms for public health emergencies and crises. These measures seek to preserve high safety standards while offering tailored regulatory solutions for unmet medical needs, rare diseases and crisis situations.

Broadened in house exemption

Both regulations in‑house devices manufactured and used within health institutions from many MDR and IVDR requirements (also called the "health institution exemption"). The exemption is subject to strict conditions, including that there is no equivalent device on the market. This condition has been criticised as too restrictive, particularly where commercial devices exist but are not optimally suited to local needs.

Under the proposal, in‑house devices may now be transferred to another health institution where this is duly justified in the interest of public health, patient health or patient safety, or to prepare for or respond to a public-health emergency, provided that traceability between the sending and receiving institutions is ensured and incidents are reported back to the originating institution. For MDR in‑house devices, the hospital should still show that no equivalent market device meets the target patients’ needs but, once such an equivalent becomes available, it may continue to manufacture and use its own in‑house device for up to 10 years (effectively creating a long transition period before the exemption ceases to apply).

The documentation and accreditation requirements are streamlined, especially under the IVDR. For MDR, the documentation requirement is simplified to what is strictly necessary to allow the competent authority to verify that the relevant general safety and performance requirements (GSPRs) are met, and a separate provision on ensuring manufacture strictly in line with that documentation is deleted. For IVDR, laboratories compliant with EN ISO 15189 (or equivalent national provisions on quality and competence for medical laboratories) can rely on their accreditation. If accredited, they no longer need to justify that no equivalent device is available on the market and the condition that no equivalent market device exists is removed entirely for in‑house IVDs. Where a laboratory is not accredited, specific documentation criteria apply.

Finally, the scope of the IVDR health institution exemption is explicitly extended to certain central laboratories involved in clinical trials of medicinal products. Laboratory developed tests used exclusively in clinical trials under the Clinical Trials Regulation, and not manufactured on an industrial scale or commercialised, are treated in the same way as in house devices manufactured and used within a health institution. This means these tests can also benefit from the upgraded exemption if its (amended) conditions are fulfilled. 

Breakthrough and orphan pathways 

New draft provisions in the MDR and IVDR (both titled "conformity assessment of breakthrough devices and of orphan devices") introduce a dedicated conformity assessment framework for these products.

A breakthrough device is expected to both introduce "a high degree of novelty" in the EU with respect to device technology, clinical procedure or application in clinical practice, and provide a significant positive clinical impact on patients or public health for life‑threatening or irreversibly debilitating diseases or conditions. The positive impact can be either by offering a significant positive clinical or health impact compared to available alternatives and state of the art, or fulfilling an unmet medical need where alternatives are absent or insufficient.

An orphan device is one intended for treatment, diagnosis or prevention of a disease or condition that presents in not more than 12,000 individuals in the EU per year. For a device to qualify as such, there should be insufficient alternatives or the device should be expected to provide a clinical benefit compared to alternatives or state of the art, taking into account device‑specific and population‑specific factors.

Upon a duly substantiated request by a manufacturer or notified body, an expert panel should provide an opinion on whether these criteria are fulfilled. The opinion should be published – without confidential information – and taken into consideration by the manufacturer and notified body.

For confirmed breakthrough or orphan devices, manufacturers may request expert panel advice on clinical development strategies. The proposal provides that notified bodies should then prioritise conformity assessment and may apply a "rolling review" to reduce timelines, giving consideration to expert panel opinions and advice.

Interestingly, notified bodies are allowed under the draft legislation to issue a certificate where pre‑market clinical evidence, "even if based on limited clinical data", is deemed adequate. However, this is only permitted if either the benefit of immediate availability outweighs the risks associated with pending data or if the benefit‑risk ratio is favourable and the manufacturer commits to providing additional data from post‑market clinical follow‑up (PMCF). As additional safeguards, the proposal provides that certificate validity may be limited and that specific PMCF activities may also be required.

'Grandfathering' of legacy orphan devices 

MDR and IVDR orphan devices that were CE marked under the former EU medical devices directives and for which an expert panel has confirmed that they meet the aforementioned criteria may continue to be placed on the market beyond the transitional periods, subject to conditions.

The provision is intended to protect access to existing orphan devices by allowing qualifying products – that are within the MDR or IVDR transitional scope – to be placed on the market or put into service after the transitional dates, even without regulation-compliant CE marking. In addition to expert panel confirmation, there should be no significant changes in the product's design or intended purpose and it should not present an unacceptable risk.

Devices benefiting from this provision should not bear the CE marking if they do not have a valid certificate under the applicable MDR provision. The EU declaration of conformity should state that the device is an orphan device placed on the market under the newly introduced article 120 (14), and manufacturers should inform users, including via summary of safety and clinical performance and (digital) instructions for use or other documentation. In addition, manufacturers should seek an expert panel opinion at least every 10 years to confirm continued fulfilment of orphan criteria.

Regulatory sandboxes 

To facilitate experimentation with innovative devices and regulatory approaches, the proposal introduces regulatory sandboxes in the MDR and the IVDR.

New draft articles allow Member States, on their own initiative or upon request from manufacturers or prospective manufacturers, to establish such sandboxes for devices where application of certain regulatory requirements would not be appropriate. Those requirements can come from chapter V (classification and conformity assessment), chapter VI (clinical evidence, performance evaluation and performance studies) or selected annexes identified in the proposal.

Sandboxes are intended to operate under a specific sandbox plan that identifies which of those requirements are temporarily adapted or waived, justifies why their application is not appropriate, sets out risk control and mitigation measures, defines duration, and lists participants and their roles. Sandboxes may be established in accordance with the draft regulation only where devices are expected to address unmet medical needs or provide significant clinical benefit, and where standard application of requirements would impede or significantly delay development or access.

Sandbox participants should inform the supervising competent authority without undue delay of any harm arising, and authorities should take corrective measures, including suspension or withdrawal of the sandbox if necessary. Under the proposed set-up, manufacturers remain fully liable for damage to third parties.

In addition to national initiatives, the Commission is empowered to establish EU-level regulatory sandboxes by means of implementing acts, for a limited time and pursuant to a specific plan. EU sandboxes cannot involve placing non‑compliant devices on the market, subject to conditions. 

Derogations and crisis authorisations 

The proposal maintains the MDR's article 59 (derogation from the conformity assessment procedures) mechanism, but makes them time‑limited, adds coverage of certain diagnostic or therapeutic services and strengthens transparency and coordination. National competent authorities may authorise, for a limited period, non‑CE‑marked devices or services in the interest of public health, patient safety or patient health, and should inform the Commission, other Member States and the relevant expert panels, among other requirements. Where similar requests are submitted in several Member States, the Commission may, based on an expert panel opinion, extend a national authorisation to the EU or grant a Union‑wide authorisation and may set conditions for device use or service provision.

A new draft article 59a (derogations from certain requirements in the event of a serious cross-border threat to health, disaster or crisis) of the MDR would allow temporary exemptions, in defined emergency situations, from certain procedural requirements for already CE‑marked devices. In the context of a serious cross‑border threat to health, a disaster or a crisis, a manufacturer may request a competent authority to exempt changes to manufacturing, design or intended purpose from specified MDR requirements, provided the device continues to meet the relevant GSPRs. The Commission may extend or grant such exemptions at EU level, including through urgent procedures.

When an EU‑level public health emergency is recognised under regulation 2022/2371, the Commission may, on its own initiative and after consulting the medical device coordination group, authorise the placing on the market or putting into service of devices under article 59 for the duration of that emergency.

The European Medicines Agency (EMA) provides, on behalf of the Commission, scientific, technical and administrative support to national competent authorities to facilitate exchange of experience, cooperation and coordination, including in relation to derogations under the revised articles 59 and 59a of the MDR.

Osborne Clarke comment

The proposed in‑house exemption refinements, breakthrough and orphan-device pathways, regulatory sandboxes and crisis derogations could, if adopted, represent a notable expansion of the MDR-IVDR toolkit. As the text remains subject to negotiation between the European Parliament and the Council, stakeholders should treat these developments as indicative rather than definitive.

For health institutions and central laboratories, the proposed relaxation of in‑house conditions – including the explicit inclusion of trial‑only tests and the removal of the "no equivalent device" requirement for accredited IVDR laboratories – may offer greater flexibility, though robust documentation and quality systems would remain essential.

Manufacturers of niche products may wish to consider whether the proposed orphan-device criteria and associated pathways – including prioritised assessment, rolling review and the "grandfathering" route for legacy devices – could be relevant to their portfolios, bearing in mind that the final legislative text may differ from the current proposal.

The proposed regulatory sandboxes and enhanced crisis derogation tools appear to reflect lessons from recent public health emergencies. If adopted in a form similar to the current proposal, these instruments could facilitate innovation and faster access to critical devices, though their practical operation would depend on implementing measures and coordination between manufacturers, national authorities, EMA and the Commission.