Patient-Centered, Data-Driven Advocacy
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.
If you or your organization are interested in partnering with NORD on a RareInsights study or publication, please contact us at [email protected].
The COVID-19 Community Survey Report reveals the far-reaching impact the pandemic is having on rare patients and families. The findings reflect a community directly affected and overwhelmingly concerned about the COVID-19 crisis.
772 participants responded to the survey conducted by NORD’s research team from April 1 through April 8, 2020. Respondents represent 49 of 50 states and Washington, DC, and multiple disease categories, including genetic conditions, primary immunodeficiencies, neurological, blood, metabolic, movement, eye, skin and other types of rare disorders.Learn more >
- 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!
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 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).