Orphan and paediatric medical devices in Europe: recommendations to support their availability for on-label and off-label clinical indications.

Introduction: The Medical Device Regulation (EU)745/2017, increased the regulatory requirements and thus the time and the cost associated with marketing medical devices. For a majority of medical device manufacturers, this has lead to reconsiderations of their product portfolio. The risk of important or essential devices being withdrawn is particularly relevant for pediatric patients and other rare disease patients where limited numbers of devices can be sold and hence the investment needed may not be recovered.

Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.

Rare diseases are life-threatening or chronically debilitating low-prevalent disorders caused by pathogenic mutations or particular environmental insults. Due to their high complexity and low frequency, important gaps still exist in their prevention, diagnosis, and treatment. Since new drug discovery is a very costly and time-consuming process, leading pharmaceutical companies show relatively low interest in orphan drug research and development due to the high cost of investments compared to the low market return of the product.

Orphan devices: yesterday is history; tomorrow is mystery: towards a European orphan device directive?

Background: Regulatory and economic frameworks stimulated the research and development of orphan drugs, but very little has been done for devices necessary for the in-vivo diagnosis, prevention and treatment of life-threatening conditions with a low prevalence/incidence.

Discussion: A general public consultation in Europe has shown a positive attitude towards an "orphan device" directive. The United States of America have a Humanitarian Use Device exemption, but Europe is still waiting for such a stimulating framework.

Rare diseases and orphan drugs: 500 years ago.

In 1581 Rembert Dodoens wrote "Medicinalium observationum exempla rara, recognita et aucta" a Latin book about the diagnosis and treatment of disorders with a low prevalence.