News on orphan medical devices

The problem of so-called "orphan drugs" is familiar from the pharmaceutical sector: The development of drugs for the diagnosis or treatment of rare diseases is not worthwhile under normal market conditions. In 2000, the EU enacted the "Regulation on Orphan Medicinal Products". On the one hand, this stipulates that approval is granted centrally via the EMA, but that the manufacturer receives fee reductions for scientific advice and the regulatory procedure as well as market exclusivity.

There are also special challenges for medical devices that are to be used in the investigation or treatment of rare diseases. Development is often more demanding, complex and risky. Clinical evaluation is more expensive, as patient recruitment is more complex and time-consuming. All of this can make it uneconomical for manufacturers to launch products for rare diseases on the EU market, as the low sales volumes may not offset the costs of development. This is already leading to bottlenecks, for example in the medical care of young children and infants.

The MDCG document 2024-10 from June 2024 is the first document agreed in the Medical Device Coordination Group that deals with the problem of orphan medical devices. This document is only of a recommendatory nature and has no legal force. However, it is intended to provide assistance for manufacturers and notified bodies, e.g. argumentation aids and explanations on how the scope of the MDR can be used. The document lists criteria for orphan medical devices for the first time; on the one hand, reference is made to the definition of rare diseases (no more than 5 out of 10,000 people in the EU may be affected). Secondly, the product should not be used by more than 12,000 people in the EU per year. It must lack a sufficient alternative or offer an expected clinical benefit compared to the available alternatives.

The scope of the MDCG document does not include custom-made products, in-house manufacturing, products without an intended medical purpose and in vitro diagnostic medical devices. The manufacturer must prove whether these requirements are met. The reason for the low number of persons affected is not relevant here.

Part A of MDCG document 2024-10 contains considerations on clinical evaluation. For example, references are made to permitted restrictions on clinical data or the use of other data sources such as equivalence products, registries or off-label use. The extrapolation of data from other populations is also acceptable under certain circumstances, for example when balancing via post-market activities (PMCF).

Part B contains procedural considerations. For example, the possibility of certification under conditions ("Certificate with Conditions") is mentioned for notified bodies. The role of expert panels in connection with products for rare diseases is also described.

On a positive note, this document is the first to discuss and evaluate aspects of clinical evaluation for orphan medical devices. However, the measures mentioned in this document only simplify the clinical evaluation, but not the entire conformity assessment procedure. It therefore remains questionable whether the necessary simplifications will already be achieved. Nevertheless, the problem of medical devices for rare diseases has been recognized and specified for the first time. It remains to be seen whether further steps will follow, e.g. provision of funding, regulatory support for manufacturers, fee reductions and an overall simplified certification procedure.