The U.S. Court of Appeals for the District of Columbia on March 1 will reconsider a case that "threatens to shake the foundations of the FDA's regulatory authority covering pharmaceuticals and lead to greatly expanded use" of experimental medications, the Newark Star-Ledger reports (Cohen, Newark Star-Ledger, 2/12). In the case, the Abigail Alliance for Better Access to Developmental Drugs and the Washington Legal Foundation in 2003 filed a lawsuit against FDA to obtain access to experimental medications for terminally ill cancer patients. The lawsuit asked FDA to provide a special initial approval of experimental medications that appear effective and allow their sale and distribution to terminally ill patients who have no other approved treatment options. FDA argued that programs currently exist to provide experimental medications to terminally ill patients and that increased access to such treatments would lead to unacceptable risk. In May 2006, a three-judge panel of the court ruled that terminally ill patients have a "right of self-preservation" and should have access to experimental medications that have not reached Phase II clinical trials. According to the 2-1 decision, which reversed the dismissal of the lawsuit by a lower court, "barring a terminally ill patient from the use of a potentially lifesaving treatment impinges on this right of self-preservation." FDA appealed the decision and asked the full court to rehear the case. In November 2006, the court vacated the decision and agreed to rehear the case (Kaiser Daily Health Policy Report, 1/30).

Reaction
Maria Hardin of the National Organization for Rare Disorders said that the group supports "having expanded access to drugs that are in clinical trials" but has concerns about the "pressure that has been put on the FDA to provide drugs to anyone who is in a very serious state and to give it to them when the drug is still in safety trials." Fran Visco, president of the National Breast Cancer Coalition, said, "I understand the emotion that drives the Abigail Alliance, but this isn't about emotion, it's about saving as many lives as possible and not about getting as many drugs out as possible." Visco added, "It's about doing the right research and making sure we have patient protections in place and making drugs available that are truly helpful." Peter Jacobson of the University of Michigan Center for Law, Ethics and Health said that the "future of pharmaceutical regulation is at stake" in the case and that a decision in favor of the plaintiffs could compromise patient safety. The pharmaceutical industry also has raised concerns about the financial, legal and safety implications of expanded use of experimental medications (Newark Star-Ledger, 2/12).

Opinion Piece
Opponents of the lawsuit maintain that "current regulations and processes fully meet the needs" of terminally ill patients -- "doctors merely need to ask the government, on behalf of their patients, for access to any unapproved drug they wish" -- and that change "is therefore unnecessary," Mark Thornton, senior vice president for GenVec, writes in a Wall Street Journal opinion piece. However, according to Thornton, such requests "can sometimes rival receipt of an audit from the IRS" and are "on occasion withdrawn by exasperated doctors or refused by the FDA, leaving patients to fend for themselves." Other critics raise concerns about the safety of expanded use of experimental medications, but the "fact is that all drugs, no matter what stage of development, have the potential to evolve new safety concerns," Thornton writes. He adds that experimental medications "safe enough for the hundreds of patients in the 'Phase II' of human testing" also are "safe enough for a patient whose only option is the terminal progression of his disease" (Thornton, Wall Street Journal, 2/12).

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