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May. 29, 2018

TOPIC: Advocacy, Featured News, Press Releases

NORD Launches RareInsights, New Initiative to Support Data-Driven Advocacy, and Presents 5 Myths About Orphan Drugs and the Orphan Drug Act

Posted by Laura Mullen

Washington, D.C., May 30, 2018—The National Organization for Rare Disorders (NORD) today launched RareInsights™, its new initiative to expand public knowledge of rare diseases and translate that knowledge into real-world solutions for patients and families, and kicked off the program with the  infographic 5 Myths About Orphan Drugs and the Orphan Drug Act.

“At NORD, we believe that it is critical for next generation advocacy to include empirical data in order to affect change,” said Pamela Gavin, Chief Strategy Officer for NORD. “RareInsights is the program through which NORD will release data-driven insights that support rare disease advocacy priorities. It will feature presentations of data-based information in a variety of accessible formats, including white papers, infographics, fact sheets, and more.”

The first offering from RareInsights, 5 Myths About Orphan Drugs and the Orphan Drug Act, is an infographic that addresses some of the most common misperceptions as identified in a recent study commissioned by NORD and published by the QuintilesIMS Institute (now IVQIA). The study analyzed the role of the Orphan Drug Act and orphan drug usage and costs in the context of public policy and health care financing issues in the rare disease community.

The 5 Myths touch on hot button topics including the limited role that orphan drug costs contribute to overall health care costs, distinguishing between specialty vs orphan drugs, and the benefits of the Orphan Drug Act.

According to NORD President and CEO Peter L. Saltonstall, there are many misconceptions about orphan drug costs and about the benefits of the Orphan Drug Act.  “The QuintilesIMS study provided data reinforcing the basic value of the Orphan Drug Act and quantifying orphan drug spending within the broader context of total drug sales. Simply put, orphan drug spending is not as substantial as has been reported. As NORD marks its 35th anniversary and the 35th year of the Orphan Drug Act, we look forward to continuing to provide the voice of the rare disease patient community and playing a vital role in these discussions,” he said.

About 7,000 rare diseases have been identified, affecting 25 to 30 million Americans. Of the 7,000 that exist, fewer than 500 rare diseases have FDA-approved treatments. Many affect only a few hundred or a few thousand individuals. Rare diseases tend to be chronic, serious and life-threatening.  More than 80% are believed to be genetic. The Orphan Drug Act is a catalyst for incentivizing interest in developing treatments for rare diseases and is critical for continuing to close the gap for the 95% of patients who are still without any approved treatment options.

Editor’s note:  Peter L. Saltonstall, President and CEO of NORD, is available for interviews on RareInsights and the 5 Myths About Orphan Drugs and the Orphan Drug Act.  Contact Laura Mullen at


One Response to “NORD Launches RareInsights, New Initiative to Support Data-Driven Advocacy, and Presents 5 Myths About Orphan Drugs and the Orphan Drug Act”

  1. Kathye Bergin says:

    Rare Diseases: Shifting the Paradigm

    As a patient with multiple, debilitating autoimmune diseases in my early life and now spending most days in bed when I should be enjoying my grandchildren and hobbies, I continue to be shocked by the cost ($7,000.00+/week) of the main infusion drug for my Stiff Person Syndrome with Parkinsonism traits, Polymiositis with organ involvement, Multiple Organ Atrophy Syndrome, advanced spinal/vertebral deterioration and severe limb neuropathic pain and weakness.

    I await the day when our legislators can negotiate with big pharmaceutical companies to research more effective treatments that do more than slow the progress of the rare diseases.

    I also await the day when Medical Schools require ALL residents to become familiar with NORD and Orphan Disorders related to each resident’s preferred specialty. Today, MOST OF MY SPECIALISTS (other than my neurologist) know little to nothing about Stiff Person Syndrome and dismiss the severity of extreme spasms and recurring falls. Some of them–even “Pain Specialists”— insist that spasms cannot cause nerve pain or damage nerves.. As a result of their incomplete education, they have no basis for understanding the interactions of their treatments and medications on my central autoimmune disorders.

    Thus, my message begs the question: How can the United States, known for its outstanding medical services, continue to underestimate or dismiss deeper research into rare diseases.

    The body is an integrated masterpiece…when it works as it should. Might research into more effective treatments for rare disorders also lead to breakthroughs in treatments for more common diseases?

    I urge legislators, pharmaceutical companies, and medical schools to reassess not only the great need for advances in both the education about rare diseases such as mine but the possible effect of newly discovered treatments on medicine in general.