June 15th, 2015

Almost exclusively, a new drug or device is not available for public consumption or use until the FDA gives its approval.  This approval is usually based off of years of data collected through clinical trials attesting to the safety and efficacy of the drug or device.  In theory, this would mean that a treatment would not be available to the public outside of a clinical trial; however, in rare cases, expanded usage (or “compassionate use”) is granted for patients with no other treatment option or for patients in life-threatening situations who do not meet criteria to enter a clinical trial to gain access to the investigational drug or device.

The topic of compassionate use has recently peppered the media.  Last year, the case of a 7-year-old boy attracted national attention when a North Carolina pharmaceutical company denied his family’s application for compassionate use.  The antiviral drug they were seeking for his treatment had not yet been approved by the FDA, but was one of the only options left to possibly successfully treat the child.  After an increase in social media support and petition, the pharmaceutical company worked with the FDA to establish a new open-label trial so that the child, along with 20 other patients with struggling immune systems, would have access to the drug.

Also, last summer, the media revealed that the two American missionaries that had contracted the Ebola virus while serving in Liberia had been given access to an experimental drug.  This drug had not been used in humans before, but it was successful in treating several monkeys with the virus.  The details are still not fully known by the public, but CNN reports that “it may have been given under the [FDA’s] ‘compassionate use’ regulation.”

Compassionate use is rare, but not uncommon  in healthcare.  In 2013, the FDA approved 974 instances of patients wanting to receive treatment via compassionate use.  Several such cases in recent years will have come through OCRI, as we have a few recent instances of patients under the age of 18 needing access to non-FDA approved treatment.

OCRI is currently enrolling a hip replacement study for adult patients using ceramic components.  Since this study is only approved by the FDA for those patients that are considered “skeletally mature”, the study does not allow for patients under the age of 18 to participate. But because ceramic is such a durable material, it can last longer and prolong the need for a subsequent revision to the hip replacement.  For these reasons, it can be ideal for younger patients who may have to face a revision to their replacement in the future, and compassionate use approval from the FDA is requested by their physician.  These patients are followed for the duration of the trial like other study patients, but their data may or may not be used in the final analysis.