Assessment of US Pathway for Approving Medical Devices for Rare Conditions

The humanitarian device exemption (HDE), established by the Safe Medical Devices Act of 1990 for devices treating diseases affecting fewer than 4,000 US patients annually, does not require manufacturers to demonstrate effectiveness, only safety and a favorable benefit-risk balance. Restrictions on for-profit sales were also removed for pediatric devices in 2007 and all HDE devices in 2012. The authors examine the scientific and regulatory characteristics of HDE-approved devices since the pathway’s inception, considering benefits and risks of this reduced evidentiary standard and drawing lessons for ongoing regulatory reform efforts in both the US and EU.