Posted by Michael Wonder on 30 Mar 2019
Statement by FDA Commissioner, Director of FDA’s Center for Drug Evaluation and Research and Director of FDA’s Center for Biologics Evaluation and Research on expanded access–looking forward
29 March 2019 - For more than 30 years, the FDA has supported patients’ access to investigational medical products for treatment, outside of participation in a clinical trial, when appropriate.
The FDA remains deeply committed to this effort. Helping to facilitate access to promising medicines for patients with serious or immediately life-threatening diseases or conditions when no comparable or satisfactory alternative therapy options are available is a high priority for the agency.
We cannot know in advance whether a drug obtained through expanded access (EA) will provide a benefit for these patients who have no other FDA-approved treatment options and cannot enrol in a clinical trial. But such access can often represent a patient’s final hope for a potentially effective treatment. For the mother with breast cancer, it may lead to extra time to attend a daughter’s graduation or offer a father with ALS the chance to be at his son’s wedding. Maybe such access can lessen suffering in the last few months of a patient’s life. Or maybe it enables a child with a rare and fatal disease to live many more years. Fostering these opportunities are among our most critical obligations.
