Rare diseases (RDs) have been overlooked in the past due to their complexity and rarity of clinical manifestations. In addition, there are obstacles to establishing quick diagnoses for RDs, unpredictability, and problems in the development of treatments. Following the actions of RD patient advocacy groups and the enactment of specific supportive orphan drug regulations aimed at attracting investment in the field, the US National Institutes of Health and the European Commission, along with a vast number of public and private research funders, formed the International Rare Diseases Research Consortium a decade ago (IRDiRC).
IRDiRC was founded in 2011 with two key goals: (1) Contribute to the development of 200 innovative medicines by 2020, and (2) Provide the means to detect the majority of RDs by 2020 . IRDiRC developed a new set of global rare disease goals for 2017-2027, following the…
