White House Respond to Refael Elisha

OFFICIAL WHITE HOUSE RESPONSE TOauthorize the FDA to grant a compassionate use exemption to Refael Elisha Cohen for Antineoplaston therapy.

What We Have to Say About Your Petition and Expanded Access

Thank you for your petition. The situation faced by the Cohens -- and all families facing childhood cancer -- is heartbreaking.

As you may know, the Food and Drug Administration (FDA) is responsible for ensuring that drugs and other medical products sold in the United States are safe and effective. Drugs that haven't yet been approved for marketing are called "investigational new drugs." When those drugs show promise, the FDA may allow patients to enroll in clinical trials of those drugs or -- in cases when joining a trial isn't possible and there are no comparable or satisfactory treatments -- to be able to use those drugs through expanded access.

This is often referred to as "compassionate use," but it's not without risk. Since the treatments are still being investigated, it isn’t known if they are safe or effective. Indeed, there's the risk that the treatment could be not only ineffective, but make a patient's condition worse. 

In cases like Refael Elisha's, the drug company that produces the treatment in question works together with the patient and, in some cases, with the patient’s doctor, to request approval from the FDA to treat the patient with the investigational drug. The same standards that prevent the White House from interfering with the outcome of an agency's independent adjudications or research also apply in cases like Refael Elisha's. The decision whether to allow compassionate use depends on a number of factors, including what the agency knows about the safety and effectiveness of the proposed treatment. The patient his illness, his treating physician, and the drug company are all involved in that decision.

We are confident that FDA will rely on its expertise and scientific judgment as it evaluates Refael Elisha’s case.

The FDA provides a variety of resources should you want to learn more about compassionate use or want to request approval from FDA:

• Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access)

• Investigational New Drug (IND) Application

You may also contact the FDA's Office of Health and Constituent Affairs for information and assistance.