BioCentury
ARTICLE | Market Access

How patients are repurposing compassionate use 

Requests seek to bridge gap between off-label indications payers cover and those physicians believe patients need

July 19, 2022 11:50 PM UTC

Patients and their families are repurposing FDA’s expanded access pathway, turning a tool for providing access to unapproved drugs into a mechanism for overcoming payers’ refusals to cover off-label uses of approved drugs.

The phenomenon is a response to gaps between the off-label indications private and public insurers are willing to cover, and those physicians believe their patients need, especially to treat cancer. Because drug companies usually do not charge for expanded access, it can be a lifeline to patients who are unable to pay tens or hundreds of thousands of dollars to purchase drugs that represent their only hope. ...