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Patient-Centered, Data-Driven Advocacy

RareInsights® is a NORD initiative to expand public knowledge of rare diseases and translate that knowledge into real-world solutions for patients and families.

Through this initiative, NORD is commissioning and undertaking a broad range of projects to collect and analyze empirical data for next-generation advocacy that is patient-centered and data-driven. Information is shared with the community in a variety of accessible formats, including reports, white papers, infographics, fact sheets, and more.

Patient-Centered, Data-Driven Advocacy


If you or your organization are interested in partnering with NORD on a RareInsights® study or publication, please contact us at [email protected].

RareInsights® Spotlight

As part of an ongoing mission to generate evidence and data to advance our collective understanding of the rare disease patient experience, NORD conducted two studies aimed at identifying trends in rare disease diagnosis, care and treatment in the United States. The findings are outlined in this new report that looks comparatively at survey data from 1989 and 2019.

Download the report

A new report from the IQVIA Institute, Orphan Drugs in the United States: Exclusivity, Pricing and Treated Populations, provides:

  • A new perspective on the sequence of orphan and non-orphan indications approved and their associated patent and market exclusivities
  • An examination of orphan drug pricing relative to patient numbers and how those prices change over time
  • A comparison of current disease epidemiology to the number of treated patients to demonstrate the challenges in bringing orphan drugs to patients even after they are approved

This report is a companion analysis to the examination of the orphan drug market published by the IQVIA Institute in October 2018, Orphan Drugs in the United States:
Growth Trends in Rare Disease Treatments.
Download Latest Release

The Orphan Drug Act (ODA) of 1983 is a federal law that incentivizes biopharmaceutical companies to develop drugs and biologics, know as “orphan drugs,” for individuals with rare diseases. Prior to the ODA, there were only 34 therapies indicated to treat a rare disease. Now, there are over 700 and counting!

Learn more about the Orphan Drug Act in this NORD RareInsights® Orphan Drug Fact Sheet.

Today, there are still many misconceptions about orphan drugs and about the Orphan Drug Act.  Download the newly released study by the IQVIA Institute, “Orphan Drugs in the United States: Growth Trends in Rare Disease Treatments,” which finds that despite a record-breaking number of new approvals, orphan drugs remain a nominal part of overall drug spending, accounting for only 9.6% of total sales in the U.S. in 2017-2018. Click here for a one-page information sheet highlighting the study’s key findings.

Download Full Report from IQVIA Institute

Download the 5 Myths About the Orphan Drug Act and Orphan Drugs infographic.

Download Trends in Orphan Drug Costs and Expenditures Do Not Support Revisions in the Orphan Drug Act: Background and History. (2017)

Download Orphan Drugs in the United States: Providing Context for Use and Cost. (2017)

Download Impact of Orphan Drug Tax Credit on Treatments for Rare Diseases. Published by NORD and BIO (2015).

DownloadQuantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders. Published by Frank J. Sasinowski, M.S., M.P.H., J.D., former Chairman of the Board for NORD (2011).