IRDiRC aims to accelerate progress in this area and ambitiously push the limits of what is currently possible in the longer term with an audacious vision for the field, all with rare disease patients’ lives in mind.
The vision: Enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention.
In order to work towards this bold and ambitious vision, IRDiRC has set three goals for 2017-2027:
Goal 1: All patients coming to medical attention with a suspected rare disease will be diagnosed within one year if their disorder is known in the medical literature; all currently undiagnosable individuals will enter a globally coordinated diagnostic and research pipeline;
Goal 2: 1000 new therapies for rare diseases will be approved, the majority of which will focus on diseases without approved options;
Goal 3: Methodologies will be developed to assess the impact of diagnoses and therapies on rare disease patients;
FCT participates in this International Consortium through its membership in the ERA-NET E-Rare-3 and in the European Joint Programme EJPRD.