One of the most challenging moments in the treatment of a child with cancer is when there is a promising drug in an adult trial, but the child is not eligible for the trial. One solution is a compassionate use application, but compassionate use applications can be confusing and time consuming for physicians.
This past year, Kids v Cancer created the Compassionate Use Navigator to help physicians and patients gain access to experimental treatments when approved drugs can’t save a child’s life. Our work will get a little easier if Congress passes the 21st Century Cures Act this week. The bill would require manufacturers of drugs that are being tested for serious and life-threatening conditions to post basic information about how to apply for compassionate use on their websites.
The compassionate use provisions of the 21st Century Cures Act attempt to solve a frustrating problem: Most manufacturers do not publicize their compassionate use policies, and there is no requirement for companies to acknowledge or respond to requests. A survey of 100 drug manufacturers found that only 19 posted basic information about compassionate use policies on their websites, according to a report by Avalere. Physicians and parents are frustrated that the most basic information — where to send a request for compassionate use and the standards used to review an application — is not easily available. People who apply for compassionate use have run out of options, but only 3 of the 100 companies state how long it will take for an acknowledgement of a request.
In addition to requiring manufacturers to make public their policies on evaluating and responding to requests for compassionate use, the compassionate use provisions in the 21st Century Cures Act (Section 561A) require that specific information be posted, including: contact information for making the requests; the general criteria for evaluation of and responding to such requests for individual patients; the time period for acknowledgement of receipt of requests; and a link to the clinical trial record containing information about the expanded access for a drug. The bill does not guarantee that companies will provide access.
The requirement will go into force 60 days after enactment of 21st Century Cures.
Transparency makes it easier to apply for investigational drugs, and saves time for busy physicians. We can’t wait.