ARTICLE SUMMARY:
European policymakers recently mapped out a path under the Medical Device Regulation to certifying devices for rare diseases or subpopulations even with “limitations” in premarket clinical data. In this edition of Consultants Corner, three experts—from a consultancy, a notified body, and a competent authority—offer tips to firms for pursuing the EU orphan device route.
Welcome to Consultants Corner, where we check in with independent experts about questions they are answering or challenges they are solving for clients on the front lines of medtech regulatory, reimbursement, and market access.
There is no definition or clinical leeway provided in the EU Medical Device Regulation for products that target rare conditions or patient subgroups. As a result, so-called orphan devices—specialized stents and catheters used for kids, for example—are increasingly coming off the market as manufacturers realize they won’t be able to enroll the number of patients needed to meet the rigid MDR study expectations.
EU clinicians and lawmakers have cited the fear of shortages in orphan devices as a prime reason for previous delays in MDR transition deadlines and for the need of more direct reforms to the regulation to provide increased leeway for this class of products. The challenge also convinced the Medical Device Coordination Group (MDCG), a consortium including the European Commission and member state authorities, to publish the June 2024 guidance document laying out the conditions for notified bodies to accept a relatively lower quality and quantity of premarket clinical data for orphan devices with assurances of postmarket data collection. Soon after the guidance was issued, the EU launched a pilot program that positions existing MDR expert panels to provide free advice to select companies about whether their product qualifies as an orphan device and what data might be acceptable to support certification.
“We implemented a definition of orphan devices that [is not] in the MDR, but we are still in line with the MDR and have to fulfill all the provisions,” explained Nebojsa Serafimovic, a clinical investigations assessor for the Austrian Medicines and Medical Devices Agency, during a January 21 webinar convened by MedTech Leading Voice and the AKRA TEAM consultancy.
Manufacturers, notified bodies, and other stakeholders are now trying to figure out exactly when it’s appropriate to accept less clinical data, and how much less data, even if it doesn’t meet the letter of the MDR law. Serafimovic was joined on the recent webinar by Matthias Fink, MD, a senior clinical consultant with AKRA TEAM, and Gavin Quigley, MD, a neurosurgery expert with the notified body BSI, to provide advice on leveraging the new EU orphan device pathway.