AAMC Reporter: September 2007

Court Rejects Claim to Experimental Drug Access

Last month, a Washington, D.C., appellate court ruled against the Abigail Alliance, a patient advocacy group that had sought to compel the U.S. Food and Drug Administration (FDA) to allow incurable patients access to experimental drugs after phase I clinical trials.

The 8-2 ruling by the full U.S. Court of Appeals for the District of Columbia Circuit reverses a May 2006 decision by a three-judge panel on the same court, which sided with the alliance in Abigail Alliance for Better Access to Developmental Drugs, et al., v. Andrew C. Von Eschenbach, et al. At that time, the judges ruled in a 2-1 decision that patients have a constitutional right to experimental therapies outside clinical trial environs.

"We were very pleased by the decision of the full court and the decisive margin under which it was issued," said David Korn, M.D., senior vice president of the AAMC's division of biomedical and health science research. The AAMC, the American Society of Clinical Oncology (ASCO), and the National Coalition of Cancer Survivors (NCCS) filed amicus briefs in the suit supporting the FDA and its current clinical trials process.

Abigail Alliance officials said they will continue to push for earlier drug access.

"Obviously, we are disappointed. But we will be appealing the case to the Supreme Court," said alliance co-founder Steve Walker. "We still believe we have good arguments."

The Abigail Alliance views this litigation as a system too restrictive for patients out of traditional options. "If we win, the FDA will have to alter their processes and regulations with regards to investigational drugs, so that patients who have a terminal disease and who can't get into a clinical trial are not barred from then going to a drug company that is running it and saying ‘I would like to have your drugs under the same condition,'" said Walker, whose wife died of colon cancer.

Alliance volunteer John Rowe, whose daughter Kianna Karnes died at age 44 from advanced kidney cancer, agreed that the current testing system is too limiting. Himself a cancer survivor who said he owes his life to a clinical trial, Rowe became an advocate for easier access to unapproved therapies after they were denied to his daughter in 2004.

New therapies at phase I testing are not guaranteed to effectively treat a specific condition. Rather, this point in the process is focused on determining the side effects of drugs at different dosage levels.Many drugs that enter the clinical trials process do not emerge as FDA-approved products.

"I realize there are questions to be hashed out...but, gosh, if we could do anything so other families don't have to go through the grief that we went through, I would sure like to do it," Rowe said.

Early access to investigational drugs, however, concerns many members of the academic medical community, said Korn, because of its potential effect on patient safety and the integrity of the clinical trials process.

"One sympathizes with people with incurable illness, but on the other hand, these products have not been sufficiently tested. It is unknown if these medicines are effective in the first place, and they could have very bad toxicity," said Korn.

Questions existed over the potential effects on the system's ability to quickly produce high-quality drugs.

"We became concerned that an effort to make drugs available while they were still in early phase testing would slow down the development of drugs. New drugs are essential to the advance on cancer, and to the advance in every discipline," said ASCO Executive Vice President and CEO Allen S. Lichter, M.D. "Patients may choose not to enter clinical trials if they can actually take drugs that they would previously have had to enter the trial to receive. If that happens in large numbers, progress is slowed tremendously."

Defenders of the current FDA trials system said there are other federal laws that allow patients to obtain investigational therapies: the "compassionate use" exemption, the treatment investigational new drug (IND) mechanism, and the expanded access program. These programs allow people to obtain unapproved drugs, provided that specific criteria are met. The "compassionate use" exemption allows terminally sick patients to obtain unapproved medicines with a physician's guidance and approval from a local institutional review board.

In the treatment IND process, people can gain access to an experimental drug that is close to approval if there are life-threatening conditions involved and the medication has displayed preliminary evidence of efficacy. During the Abigail Alliance litigation, the FDA made another effort to provide experimental drugs via its expanded access program. Unlike the other two programs, the FDA did not develop a new patient access mechanism but reorganized its regulations to encourage pharmaceutical sponsors to make more unapproved drugs accessible. Abigail Alliance said they still believe that even with these regulations many people are denied experimental therapies.

According to Lichter, there is no need to fix a system that, from a procedural standpoint, does not appear to be broken.

"High-quality clinical science is one of the greatest achievements of oncology today," Lichter said. "It is why death rates have started to decrease. It is why five-year survival has increased from 50 percent to nearly two-thirds over the last 25 years. It is why there are over 10 million cancer survivors. We do not see a reason to make a wholesale, radical change like this."

—By Elissa Fuchs

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