Welcome To IRDiRC
Our 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
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
Latest News
Nomination Call: Therapies Scientific Committee (TSC)
IRDiRC has four Scientific Committees. The Therapies Scientific Committee (TSC) is a multi-stakeholder,…
Join the ISPOR Webinar on “Challenges in Defining Elements of Value in DMD for Decision Making”
An upcoming ISPOR Webinar will explore the challenges in evaluating rare, pediatric, degenerative diseases by…
Newborn Screening by Whole Genome Sequencing pilot project
The US National Institutes of Health (NIH) announces a new collaboratory research opportunity…