NORD broadens access to unapproved medicines
The guide provides users with answers to frequently asked questions about accessing unapproved medicines. Many people wonder if they’re eligible for this access, how they apply and what they will go through during the application process. They also wonder about what happens after the application process is completed.
Expanded access, also called compassionate use, gives people with life-threatening or serious diseases the chance to use experimental medical treatments when they have no other treatment options. It is common for people who have rare diseases to be challenged with limited or no options for treatments.
The Expanded Access FAQ is available online. NORD wants to use this guide to help every patient receive treatments that could help them.
NORD is welcoming any suggestions, feedback or responses via email. Readers can also leave comments in the comment section, after they read the guidelines.
The Policy Issues page of the NORD website also outlines important information about policy issues and expanded access within the rare disease community. These are important documents to read as people consider using expanded access.
Organizations in this story
National Organization for Rare Disorders (NORD) 55 Kenosia Avenue Danbury, CT 06810