RARE Toolkits

Below is a list of all of our current RARE Toolkits.


What if the medication that could improve or possibly save your life was just out of reach—available to others—but not to you. It’s a reality for many patients with rare and difficult to diagnose conditions who are unable to access the medicines they need. They may have exhausted all available commercial therapies for their disease, they may not have access to a clinical trial, or there may not be any efforts to bring the needed drug to market in the United States. In other cases, a needed medicine may be experiencing a temporary supply shortage or may have been discontinued.

For these patients, access to a medicine outside the clinical trial or commercial setting can represent a new and, in many cases, life-saving treatment option.

The process of obtaining treatments that are not currently available can be confusing for many patients and their families. In this toolkit, we will introduce what access programs are, how they work, and what patients and their families can do to further explore.


What is “Expanded Access” or “Compassionate Use?”

In the United States, expanded access is the Food and Drug Administration (FDA) term used to describe the legal pathways for obtaining access to medicines outside the clinical trial or commercial setting. Also known as compassionate use, expanded access programs allow patients to access medicines that:

• Are still in clinical development
• Are not currently FDA approved or commercially available
• May never be commercially available, but may still offer benefit for a very small population
• Are approved in countries outside the U.S.


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Early Access/Compassionate Use
Dr. Brian Woodfall explains what early access programs are and what they try to achieve.
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“The term compassionate use is often used in place of expanded access, it is appropriate because patients are seeking drugs outside of formal trials and relying on the compassion of physicians, the FDA, and drug makers to grant them access to a potentially life-saving option.”


Shannon Reedy

Director of Chase the Cure