By Mary Pat Flaherty and Joe Stephens
Washington Post Staff Writers
Friday, February 23, 2001; Page A03

The Food and Drug Administration is weighing whether to approve a Pennsylvania biotechnology company's proposal to conduct an experiment that would leave hundreds of sick, premature infants in Latin America without proven lifesaving medications.

The experiment would be considered unethical in the United States, where researchers are not allowed to use placebos -- or dummy medicines -- on test subjects suffering from potentially fatal illnesses for which there are proven treatments. But Robert J. Capetola, president of Discovery Laboratories of Doylestown, Pa., said its planned tests on newborns with a serious lung disease could shave 18 months off of the development of an important drug.

In some poor Latin American hospitals, Capetola said, infants with the lung illness may not have access to established drugs and would not be left worse off by placebo treatment. He said infants in the study would receive better care than is currently available because Discovery would donate ventilators and antibiotics for their use.

The company wants the FDA to approve the research in case it wants to use the test results to try to market the drug Surfaxin in the United States for treatment of respiratory distress syndrome. The study would divide infants into three groups: those on Discovery's experimental lung medication, those who get another established drug, and a third group that would get only placebo treatment.

That design would enable the company to show whether its unproven product is better than a competitor's but also whether it's better than no treatment at all.

Disclosure of the proposed Latin American experiments comes at a time of mounting concern that drugmakers in the United States and other wealthy nations are increasingly testing new medicines in developing countries where costs are low, patients plentiful and government oversight lax. Placebo trials in particular have generated heated international debate since the mid-1990s, prompted in large part by HIV tests in Thailand and Africa that withheld proven drugs from some pregnant women whose children then were born infected with the virus that causes AIDS.

It would be virtually impossible for a company to include a no-treatment group in a test on the infant lung illness in the United States because standard care in American hospitals -- and in most developed countries -- calls for using established drugs known as surfactants that significantly reduce newborn deaths and complications.

Withholding lifesaving care for respiratory distress syndrome for the sake of a drug experiment is "considered unethical in the USA," according to an FDA document discussing the Discovery Laboratories proposal.

Discovery also plans an experiment on newborns in Europe, Capetola said, where all infants tested would receive some drugs: either Discovery's experimental surfactant or another already approved surfactant.

In the Latin America study, Capetola said that using a placebo group would produce faster comparative results. "We think it would be totally unethical not to conclude it and get it to patients quicker," he said.

But Sidney M. Wolfe, director of the watchdog organization Public Citizen's Health Research Group, yesterday called the test "totally unethical." His group released FDA documents about the proposal and called on both the FDA and U.S. Department of Health and Human Services to halt the research.

"It is the worst kind of race to the ethical bottom," said Peter Lurie, deputy director of Public Citizen's Health Research Group. Surfactant treatment for a premature infant in the United States costs between $1,100 and $2,400. He said Latin American families would be taking part in a test of a product that would ultimately be too expensive for them to afford.

But Capetola said that if Surfaxin proves successful it will be sold at a reduced price to the Latin American countries that hosted the research.

Respiratory distress syndrome is the fourth-largest cause of infant mortality in the United States. In 1990, the FDA approved the first commercially available surfactant, a drug that reduces tension in the infants' tiny lung air sacs. Four surfactants are now marketed in the United States.

HHS spokesman Bill Hall said Secretary Tommy G. Thompson "would not get involved in a regulatory issue. . . . That's for the FDA to decide."

Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said Discovery's proposed experiment remains "under consideration." If the agency concludes the design is unethical, she said, the drug's developers could legally perform the trial outside the United States. But the FDA might refuse to accept the findings as proof the drug is safe enough for Americans, she added.

The Surfaxin proposal was discussed at an FDA ethics round table a month ago specifically because there is a division of opinion inside the agency on the ethical questions it raises, Woodcock said.

The growth in overseas testing was documented in December in a Washington Post series called "The Body Hunters." The articles recounted instances in which American researchers had conducted experiments in impoverished nations that would have been forbidden in the United States.

© 2001 The Washington Post Company

-- Cherri (jessam5@home.com), February 23, 2001