The Impact of Single Indication Orphan Drugs on the Future of Rare Diseases

What is the impact of rare diseases on individuals and health care systems?

Congressionally funded report details diagnostic challenges and costs of rare diseases in the US

Diagnostics: Moving to the forefront of Global Healthcare

Economic Burden of Rare Diseases Nearly $1 Trillion

CBO Comparison of Brand-Name Drug Prices Among Selected Federal Programs

Orphan drugs account for more than half of all FDA drug approvals in 2020

Rare Disease Stakeholders: Do mRNA COV19 Vaccines Affect Eligibility to Receive Future Gene Therapy?

How do we identify and measure value for patients with rare diseases?

National Burden of Rare Disease Survey