Dying Patients Fight the FDA

A lawsuit could make access to experimental medicines a constitutional right, and alter how drugs for life-threatening diseases are developed

by Catherine Arnst

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Everett Davis had been living with a rare, deadly blood disease for more than a decade. By 2005 he needed a new liver to survive, but doctors would not do the transplant unless they could figure out how to keep the disease from destroying the new organ. His one chance: an experimental drug called Soliris from Alexion Pharmaceuticals Inc.. Yet he didn't qualify for the clinical trial. His doctors, friends, and family members all searched for a way to get him the drug, finally calling his senator, Arlen Specter (R-Pa.), who intervened. "I was informed on Thanksgiving of 2006 that I would get the drug," says Davis, 32. "It's the best news I ever got."

Some 97% of the sickest patients are ineligible for or don't have access to the clinical trials that are testing experimental medicines. They're getting help from patient advocates who are aggressively lobbying Congress, other patients, and the Food and Drug Administration to either speed up approval or relax rules restricting access to clinical drugs.

Landmark Case?

Before long, the results of that effort may prove more than anecdotal. A lawsuit by a group of patient advocates could make access to experimental medicines a constitutional right, resulting in a dramatic shift in the way medicines for life-threatening diseases are developed and regulated.

The Abigail Alliance for Better Access to Developmental Drugs filed suit against the FDA in 2003 seeking expanded access to experimental drugs for terminally ill people. The nonprofit group was founded by Frank Burroughs in 2001 after his 21-year-old daughter and only child, Abigail, died of head and neck cancer. She had been turned down for clinical trials for ImClone Systems' (IMCL) Erbitux and AstraZeneca's (AZN) Iressa, both of which went on to win FDA approval. After a long legal battle, a three-judge panel from the District of Columbia Circuit Court of Appeals ruled last year that the case could be heard in district court.

The FDA requested that the court's full, 10-judge panel consider the case, and its decision is widely expected this summer. Whichever side loses is sure to appeal to the Supreme Court. "This could be a landmark civil rights case," Burroughs says. That right, if the Abigail Alliance prevails, would let a terminally ill patient try any drug that has proven safe in early-stage human studies, even if there is no evidence of its efficacy.

Cost Concerns

The prospect of such a right is disquieting to drug companies that say they can ill afford to make experimental drugs more widely available. There are currently more than 600 experimental cancer drugs in development. All must go through years of testing in costly clinical trials. Most will fail at some point.

Very sick patients who can't get into a clinical trial can petition the company and FDA to try some drug on a so-called compassionate use basis, but few companies offer such an option because the drugs are usually provided free of charge (insurers typically will not pay for experimental treatments).

"Very small companies like ourselves don't have the resources to give away drugs free on an ongoing basis," says Timothy Walbert, chief executive officer of IDM Pharma (IDMI), which is seeking FDA approval of a drug for a rare form of childhood bone cancer called osteosarcoma—a disease that has its own advocacy group pushing for approval of the IDM drug. "There are only about 900 kids a year in the U.S. who get this disease," Walbert says. "We can't afford to provide it free to all the kids who need it."