Orphan medical devices: addressing the regulatory and access gaps in the EU and US.

Background: Orphan medical devices remain the blind spot of rare disease policy. While the United States has recognized them since 1990 through the Humanitarian Use Device (HUD) and Humanitarian Device Exemption (HDE) framework, the European Union only introduced a non-binding definition in 2024 (MDCG 2024-10), revealing a deeper asymmetry: flexibility is codified in the US but interpreted in the EU. Methods: We conducted a document-based policy analysis (1990-2025) and examined legislation, regulatory guidance, and academic literature to compare EU and US frameworks across evidentiary standards, access pathways, economic incentives, and reimbursement-post-market alignment. Three illustrative case studies (Berlin Heart EXCOR, Argus II, and the Medtronic Melody valve) were used to assess real-world consequences for access, sustainability, and patient continuity of care. Results: The EU's Medical Devices Regulation 2017/745 lacks a legal orphan-device designation, dedicated incentives, or a harmonized HTA framework. Access remains fragmented, and recertification under the MDR threatens supply continuity. The US HUD/HDE pathway codifies flexibility through a "probable benefit" standard under IRB supervision and an annual distribution cap, though financial sustainability remains limited by profit restrictions and inconsistent reimbursement. Case comparisons reveal: (i) earlier EU access without post-market safeguards (Argus II); (ii) US capacity to transition from humanitarian to full approval when incentives align (Melody); and (iii) vulnerability of SMEs to compliance costs (EXCOR). Conclusion: Addressing this policy vacuum requires an inflection point equivalent to the 1983 Orphan Drug Act, one that redefines how innovation for the few is valued. Five policy pillars emerge: a legal designation, adaptive evidentiary pathways, sustainable incentives (e.g., pooled procurement), integrated reimbursement mechanisms, and mandatory continuity-of-access or "exit-insurance" schemes. Without such reform, essential technologies for rare diseases will remain trapped between regulatory rigidity and market abandonment. This policy paper highlights structural regulatory gaps and proposes actionable reforms to ensure equitable access to orphan medical devices.