When does 'compassionate use' help with options?
FDA decides when this is acceptable
What often are called "compassionate use" drugs are investigational new drugs (INDs) in later phase trials where the FDA has allowed the sponsor to apply for an individual treatment IND.
Individual treatment INDs are relatively recent and were the result of the huge research push to find better HIV treatments after the AIDS crisis exploded in the United States in the 1980s and 1990s.
"It started with the AIDS epidemic when AIDS activists asked for more drugs to be made available for use outside of clinical trials, and so the FDA came up with individual treatment INDs," says Mark S. Schreiner, MD, an associate professor of anesthesia in pediatrics at the University of Pennsylvania. Schreiner also is the chair of the committees for the protection of human subjects (IRB) at The Children's Hospital of Philadelphia in Philadelphia, PA.
The more common term of "compassionate use" is not used officially under FDA rules, Schreiner notes.
"The idea of a treatment IND is that there are no other options for somebody or that the patient doesn't qualify for conditions of the clinical trial," Schreiner adds. "As long as the sponsor company agrees and the FDA agrees and the IRB agrees then they can release the product to the individual."
However, the individual treatment IND typically is used when a drug is in phase III trials so there already is evidence of efficacy and safety, Schreiner says.
"You could do this with a phase II drug, but it depends on the strength of evidence and rarity of the condition and the alternatives that are available," he adds.
In the recent court case involving the teenage boy with Duchenne's muscular dystrophy and the investigational drug PTC124, the evidence was not available yet to suggest efficacy, and so the sponsor had not considered making it available for an individual treatment IND.
The court ordered in August, 2008, that the investigational drug be made available to the teenage boy, who had sued for access when he was not enrolled in the phase II trial because of not meeting inclusion/exclusion criteria.
PTC Therapeutics would consider applying to have PTC124 used for individual treatment INDs, but not until the drug has completed phase II trials, says Jane Baj, spokeswoman.
"One could attempt to argue in favor of some humanitarian use exemption," says Ed Gaffney, PhD, executive director of clinical research at the Baptist Health System, Inc., of Birmingham, AL.
"However, since this is a phase II study on an unapproved drug with little or no data to support the efficacy of the drug, it is doubtful that there is any valid basis for this argument," Gaffney adds.
Sometimes an investigation drug in early phases will be made available for an individual treatment IND, but it's never a good idea for a court to make that decision, says Stephanie J. Zafonte, MSN, RN, CCRP, CQA, RAC, director of operations for George Washington University in Rockville, MD. Zafonte also has worked for the National Institutes of Health (NIH) as the senior extramural regulatory analyst in the Office of Clinical Research at the National Heart Lung and Blood Institute.
"I have seen it used even for questionable efficacy for the reason that we have nothing left to offer the people," Zafonte says. "I can understand the whole principle behind compassionate use, but we need to separate that from the drug development process, and if we allow courts to order compassionate use, we need to make sure it doesn't impact the drug development process."
One could argue that as well-meaning as the individual treatment IND process is, it is ultimately is misguided, says LaDale K. George, JD, a health research attorney with Foley & Lardner in Chicago, IL.
"I think finding ways around full-scale clinical trials is ultimately detrimental from a scientific standpoint," George says.
What often are called "compassionate use" drugs are investigational new drugs (INDs) in later phase trials where the FDA has allowed the sponsor to apply for an individual treatment IND.You have reached your article limit for the month. Subscribe now to access this article plus other member-only content.
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