Court to rule on dying patients' access to experimental drugs

Doctors worry that permitting early use could compromise the very process that helps them learn more about promising medications.

By Amy Lynn Sorrel — Posted Dec. 18, 2006

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Should dying patients have special access to prescription drugs after just the first stage of clinical testing? The full U.S. Court of Appeals for the District of Columbia plans to take up that thorny question next year.

The Abigail Alliance for Better Access to Developmental Drugs argues that terminal patients should have the ability to take medications that have cleared phase I clinical trials and shown initial effectiveness and acceptable side effects.

But physicians say early access to these drugs poses ethical problems and could hurt the quality of the remainder of patients' lives.

The Abigail Alliance, with the Washington Legal Foundation, in 2003 sued the federal government to get initial access for certain patients.

The patient advocacy group, named after a 21-year-old who died of cancer, asks that the Food and Drug Administration allow pharmaceutical companies to sell experimental drugs to terminally ill patients who have no other approved treatment options.

A three-judge panel of the appeals court in May ruled that barring seriously ill patients from using potentially life-saving therapies impinges on their constitutional rights to self-preservation and to decide whether to seek medical treatment.

But in November the court accepted the FDA's appeal of the decision, and the full 10-judge bench is expected to rehear the case in March 2007.

"People who are fighting for their lives deserve every chance to live just like people now getting approved drugs," said Abigail Alliance President Frank Burroughs. "There is a way to see which drugs early on are extending and saving lives, but the FDA approval process is very cumbersome." Pharmaceutical companies have no incentive to participate in expanded access or compassionate use programs, he added.

Existing programs accommodate only a small number of patients who meet specific criteria, making it difficult for the vast majority of patients with serious illnesses to get in, Burroughs said.

The FDA's pre-approval process, even for fast-track status, also is too slow for terminal patients to be able to use promising new drugs before they die, he added.

Looking for balance

But the FDA argues that opening up investigational new drugs too quickly could create unnecessary risks that could further diminish patients' remaining quality of life.

"Access to life-saving treatments is essential, but we need to find the balance between immediate access and evaluation of safety and efficacy," said FDA spokeswoman Crystal Rice.

The agency said it was exploring new ways to broaden investigational new drug use. It has proposed clarifying how doctors and pharmaceutical makers can make developmental treatments available to patients with serious or immediately life-threatening conditions and no alternative therapies. It also has proposed outlining how drug companies would charge for the medications, which are typically offered for free in such cases.

Current FDA regulations say investigational new drugs not yet approved for marketing can be made available for treatment in seriously ill patients during phase III clinical trials, which test new treatments against standard therapies, but no earlier than phase II, which tests how well the medicine works on a disease and tells more about side effects.

Doctors say they want patients to get life-saving drugs as soon as possible but also want to make sure they are protected from further harm.

AMA policy supports expanded access to promising developmental drugs, as long as it does not compromise the clinical trials process that gives doctors the necessary efficacy and safety information about a medication to get it approved for the market.

Massachusetts oncologist Therese M. Mulvey, MD, said, "We want to be sensitive to patients' needs, but simply offering them untried or unproven therapy may not be fair or ethical to the patient.

"The problem with opening up phase I trials is you don't know what the adverse effects are, and most of the drugs we're dealing with are very toxic," said Dr. Mulvey, who is a member of the American Society of Clinical Oncology.

ASCO, with the National Coalition for Cancer Survivorship, in March filed a petition with the FDA asking the agency to clarify its standards for initiating expanded access to unapproved drugs.

Doctors say it is through the clinical trials process that they are able to get promising new drugs for dying patients. "If you just offer access to drugs not shown to be effective, you not only subject people to potential harm, but you don't learn anything in the process," said Porter Storey, MD, executive vice president of the American Academy of Hospice and Palliative Medicine.

But more can be done, he said.

"It's very important that we do more research in the care of the terminally ill because we know very little about what works," Dr. Porter said. "I have seen many patients sign up for experimental treatments that made them much worse and die sooner than they should have."

Dr. Porter said he would like to see the National Institutes of Health, for example, devote more of its budget to studying end-stage diseases, such as heart, lung or liver failure. Doctors say several clinical trials are out there, often through research institutions, such as universities or the NIH.

"The hard part is for the busy physician to find the time to sort through the morass, but I would not abandon a system that is set up for the safety of patients," Dr. Mulvey said.

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Case at a glance

Abigail Alliance for Better Access to Developmental Drugs v. Andrew C. von Eschenbach, MD, in his official capacity as acting commissioner, Food and Drug Administration

Venue: U.S. Court of Appeals for the District of Columbia
At issue: Whether terminally ill patients with no other standard treatment options should have access to investigational new drugs that have gone through phase I clinical trials.
Potential impact: Doctors say early access to such drugs before they are adequately tested could expose patients to unknown risks that could cause them to die more quickly than without the drugs. Patients say that when they have no other choices available, they have the right to access new therapies once they have shown initial effectiveness.

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