Court Denies Constitutional Right to Experimental Drugs

[October 1, 2007, AJHP News]

Kate Traynor

BETHESDA, MD, 12 September 2007—A legal battle to force FDA to allow terminally ill patients early access to experimental drugs outside of clinical trials was recently decided in the agency's favor, but the fight may not be over yet.

Judges on the U.S. Court of Appeals for the District of Columbia Circuit on August 7 ruled 8 to 2 that dying patients have no fundamental constitutional right to experimental therapy, reversing an earlier decision by a three-judge panel of the court.

The lawsuit and appeal, by the Washington Legal Foundation (WLF) on behalf of the Abigail Alliance for Better Access to Developmental Drugs, were filed against FDA and the Department of Health and Human Services in 2003. The Abigail Alliance is named after Abigail Burroughs, a young woman who sought treatment with investigational drugs for squamous cell carcinoma but died of the disease in 2001 without gaining access to the drugs, both of which have since been approved for the U.S. market.

FDA allows patients limited access to investigational drugs through agency-approved treatment investigational new drug (IND) protocols. According to agency documents, the treatment IND process is available to patients with an immediately life-threatening disease who want to receive investigational drugs when no other treatment alternative exists.

FDA makes investigational drugs available under a treatment IND protocol only after the products complete advanced clinical testing and show preliminary evidence of efficacy. Only patients who are not eligible to receive the investigational drug in clinical trials can receive the therapy under a treatment IND protocol, according to FDA.

But the Abigail Alliance wants to grant access to drugs much earlier in the approval process—as early as the completion of Phase I safety testing.

Scott Ballenger, who argued the Abigail Alliance's circuit court cases, said during a February seminar sponsored by the Food and Drug Law Institute (FDLI) in Washington, D.C., that the issue before the court was whether FDA has a rational basis for denying a patient access to lifesaving medications.

"What's at stake, really, is how good a reason the FDA should have in order to interfere with a patient's own judgment, and their treating physician's judgment, about the best course of treatment for that patient," Ballenger said.

"Our suit raises the question of whether a terminally ill patient who has exhausted all of their approved treatment alternatives and wants to take a drug that is currently in Phase II or Phase III clinical trials—a drug that FDA acknowledges is safe and promising enough for expanded clinical testing," can receive the therapy, he said.

But the appeals court rejected this argument, stating that FDA's policy of limiting access to investigational drugs "is rationally related to the legitimate state interest of protecting patients, including the terminally ill, from potentially unsafe drugs with unknown therapeutic effects."

Ballenger announced after the ruling that he plans to ask the U.S. Supreme Court to hear the case. He noted that additional options include trying the case in another federal circuit court or pursuing a legislative remedy in Congress, according to an FDLI press release.

WLF and the Abigail Alliance also filed a citizen's petition with FDA in 2003 asking the agency to amend its treatment IND process to allow patients access to potentially lifesaving drugs that have completed Phase I testing. The petition states that access would be limited to patients who are ineligible or unable to participate in clinical trials.

FDA has not responded to the petition, but the agency proposed a rule last December to clarify its policies about access to investigational drugs.

During a press conference to announce the proposed rule, FDA deputy commissioner Janet Woodcock said that a "proportional scale" would be used to weigh known risks and benefits of the investigational drug against the seriousness of the patient's condition. Woodcock said some patients might be allowed to use investigational drugs before they are tested in Phase III clinical trials.

Scott Gottlieb, a former FDA deputy commissioner and current resident fellow at the American Enterprise Institute, told FDLI seminar attendees in February that one of FDA's biggest concerns about expanding access to investigational drugs is that fewer patients would participate in clinical trials of a drug if it becomes available through other routes.

"There are very few opportunities in medicine to conduct a perfect experiment," Gottlieb said. "The FDA process provides one of the purest opportunities to conduct that perfect experiment, to conduct a randomized, placebo-controlled trial to look for data about the effectiveness of drugs. And I think FDA believes very strongly in trying to preserve the integrity of that process."

Gottlieb said terminally ill patients who have exhausted traditional medical options should have access to experimental drugs. He said he believes that the clinical trials process could be altered to allow expanded access to investigational drugs while still collecting rigorous data.

Expanding patients' access to experimental therapy also raises the issue of payment for the drugs. FDA allows drug companies to charge patients for the cost of producing, distributing, and administering investigational drugs under a treatment IND protocol. The Abigail Alliance case likewise envisions that companies would be allowed to charge patients to recoup the costs of such therapies.

"If patients are allowed to have access to experimental therapies for treatment purposes for their terminal illnesses, how will they be able to afford this medication?" said Francis Palumbo, executive director of the University of Maryland School of Pharmacy Center on Drugs and Public Policy.

"Newer therapies for cancer are very expensive. They can cost, conservatively, $10,000 for one month" of treatment, Palumbo said, adding that insurers may choose not to cover the drugs.

"Many of these patients are just going to have to pay out of their pocket, and many of them are not going to be able to do that," he said.