Court denies expansion of patients' access to experimental drugs

Physicians say a recent federal court decision prohibiting early access to unapproved prescription drugs protects seriously ill patients and the clinical trial process.

The U.S. Court of Appeals for the District of Columbia Circuit ruled in August that terminally ill patients do not have a constitutional right to use medications before they are cleared by the Food and Drug Administration.

The Abigail Alliance for Better Access to Developmental Drugs, joined by the Washington Legal Foundation, sued the FDA in 2003 over the issue. The organizations say dying patients with no other treatment options should get special approval to use treatments outside the clinical trials process as long as the drugs have made it through phase I testing and shown initial effectiveness and acceptable side effects. The Abigail Alliance -- named after the founder's daughter who died of cancer while seeking access to investigational drugs -- argued that forcing patients to wait violates their right to self-preservation.

But judges, in an 8-2 decision, said the government has a legitimate interest in protecting patients from potentially unsafe drugs.

"Although terminally ill patients desperately need curative treatments ... their deaths can certainly be hastened by the use of a potentially toxic drug with no proven therapeutic benefit," Judge Thomas B. Griffith wrote. He said the right to self-preservation "cannot justify creating a constitutional right to assume any level of risk without regard to the scientific and medical judgment expressed through the clinical testing process."

The ruling overturns a May 2006 decision by a three-judge panel of the court. Griffith suggested that if patients want to change the regulatory system, they seek legislative relief.

The FDA praised the decision. The agency said it is exploring ways to expand existing compassionate-use programs that make experimental drugs available to physicians and seriously ill patients under limited circumstances. But unfettered access is not the answer, the agency said.

FDA spokeswoman Crystal Rice said the decision upholds the "FDA's role in facilitating appropriate treatment access to investigational therapies, while at the same time protecting the public at large by requiring that drugs are proven to be safe and effective before they may be marketed to U.S. consumers."

But Abigail Alliance President Frank Burroughs said the risks are different for dying patients, who are left with no alternatives. The potentially lifesaving benefits of experimental drugs outweigh such danger, he said. The alliance is appealing to the U.S. Supreme Court.

"There is always going to be unforeseen risk, but whose decision should that be?" Burroughs asked. "That should be a decision between a patient and their doctor," he said.

In a dissenting opinion, Judge Judith W. Rogers agreed. "The right to try to save one's life is left out in the cold [by the majority ruling], despite its textual anchor in the right to life," she said. "The court is on even weaker footing when it relies upon the risks entailed in medical procedures to wrest life-and-death decisions that once were vested in patients and their physicians."

Burroughs added that existing FDA programs are open to only a small number of patients who meet specific requirements, making it difficult for most to get in.

Quality of life is crucial

Doctors say they want to help patients get access to lifesaving treatments as early as possible, but without endangering patients' remaining quality of life or the development of promising new drugs.

AMA policy supports expanded access to promising developmental drugs, as long as any changes do not compromise the clinical trials process that gives doctors the necessary efficacy and safety information about a medication.

Allen S. Lichter, MD, executive vice president of the American Society of Clinical Oncology, said only 5% of drugs that make it through phase I clinical trials are approved.

"Frankly, that's because they don't work, or are dangerous, or both," said Dr. Lichter, a retired radiation oncologist. "Exposing patients to these types of agents is not a wise decision."

ASCO, with the Assn. of American Medical Colleges and the National Coalition for Cancer Survivorship, filed a friend-of-the-court brief in support of the FDA.

Dr. Lichter said allowing patients to get around the clinical trials process could undermine the research and hurt physicians' ability to learn from it. He agreed that the FDA's compassionate-use programs, under way for decades, can be cumbersome and exclusive at times. Other times, however, the lack of access is because pharmaceutical companies can't make their drugs available outside of clinical trials because they are expensive to make.

Dr. Lichter said more needs to be done, which is why ASCO has petitioned the FDA to clarify its standards for advanced access to test drugs.

The FDA in December 2006 proposed new rules that would make experimental drugs more widely and easily available to doctors and patients. The regulations also would outline when and how pharmaceutical manufacturers could charge for investigational therapies that might not otherwise be available for treatment use because of their high cost.

The FDA's Rice said the agency is still reviewing comments on the proposed rules.

Abigail Alliance's Burroughs said the proposal "brings about very little change, and as far as the patients we represent, it brings about no change at all." The alliance also is pursuing federal legislation that would give patients with life-threatening illnesses access to drugs in phase I trials.