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Archives for: March 18th, 2019

Orphan Drug Act Resolution Introduced in the House of Representatives

Written by Christina Jensen on March 18, 2019

Today, Representatives GK Butterfield and Gus Bilirakis introduced a resolution that celebrates the success of the Orphan Drug Act (ODA) and calls for continued support of the legislation.

Now in its 36th year, the ODA has successfully encouraged the pharmaceutical industry to develop therapies for those with rare diseases. Prior to… Read More

NORD Welcomes Rachel Sher as Vice President of Regulatory and Government Affairs, Lesli Nordstrom as Director of Marketing and Communications

Written by Christina Jensen on January 17, 2019

Washington, DC, January 17, 2019–The National Organization for Rare Disorders (NORD)Ⓡ, the leading independent nonprofit organization representing the 25-30 million Americans living with rare diseases, has announced two additions to its management team, effective immediately.

Rachel Sher joins NORD as Vice President of Regulatory and Government Affairs,… Read More

Leveraging Real-World Treatment Experience from Expanded Access Protocols

Written by Christina Jensen on November 19, 2018

Building on the Reagan-Udall Foundation for the FDA’s work with FDA and other stakeholders to develop the Expanded Access Navigator, the Reagan-Udall Foundation is convening stakeholders from government, industry, academia and patient groups to discuss process issues that include:

  • Increase understanding of the expanded access program
    • Identify and discuss strengths, challenges… Read More

Updated Study Analyzes Use and Cost of Orphan Drugs

Written by Laura Mullen on October 18, 2018

Washington, D.C., October 18, 2018— 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, according to a new study released today by the IQVIA Institute. The study… Read More

Five Years of Patient Registry Success: NORD’s IAMRARE™ Registry Program Celebrates New Partnerships and Models of Engagement  

Written by Laura Mullen on October 3, 2018

Washington, D.C., October 3, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today is celebrating five years of patient registry success with the announcement of a new multi-stakeholder model of engagement in collaboration with the Foundation for Prader-Willi… Read More