NORD has published a new 
guide to help people and caregivers living with rare diseases understand expanded access and how to access unapproved medicines. In the guide, NORD answers frequently asked questions including how to determine if you are eligible for expanded access, how to apply, and what you can expect throughout the application period and beyond.
Expanded access (also known as compassionate use) allows people with serious or life-threatening diseases to obtain an experimental medical treatment when there is no treatment alternative. People living with rare diseases often face limited or no treatment options, making this an important topic in our community.
Read NORD’s Expanded Access FAQ here, and feel free to share it with others in your community.
NORD strives to ensure that every patient receives the appropriate treatment to thrive. Let us know if this FAQ is helpful by sending us an email or leaving a note in the comments section, below.
To learn more about expanded access and policy issues that affect the rare disease community, visit the Policy Issues page on our website.
About NORD
The National Organization for Rare Disorders (NORD) is the leading independent charity dedicated to helping the 30 million Americans—1 in 10 people, most of whom are children—with rare diseases, through programs of advocacy, education, research, and patient services.
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