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The FDA ruled that those trials were insufficient. AIDS activists, though, argued that the hormone had been used safely in children for more than a decade -- and that FDA approval before Serono had finished conducting more tests would be appropriate.
"We felt that while we were disappointed that the data presented were not better and that Serono had not done as great a job as might be wished, we had to balance that against what we felt was the need for the community for this therapy, which we feel is promising," Baker says.
At stake in the FDA approval for the drug is more than just access to it. Under current law, any doctor can prescribe human growth hormone -- Genentech's, Eli Lilly's, or any other company's approved product -- for any use, on a case-by-case basis, according to FDA Public Affairs Specialist Janet McDonald. That is to say, even though the FDA has only approved the hormone for growth deficiencies in children and adults, doctors are free to use their judgment to prescribe it for other maladies where they think it might be effective. But unless the FDA approves the specific use of the drug, then public and private insurance companies will most likely not pay for it.
"Under federal law any drug that is specifically for AIDS or cancer is automatically added to the medical formulary," August explains. "Boom -- it goes on and it's medically approved."
In addition, if the drug does gain FDA approval, then other publicly funded AIDS drug programs, such as the state-and-federally-funded AIDS Drug Assistance Program (ADAP), will be expected to provide it, activists say. And with the cost of the hormone as high as it is, that has led some to speculate that the reason for the FDA's balking is more budgetary than scientific.
When human growth hormone was first introduced, it was not genetically engineered but instead came from the pituitary glands of human cadavers. That made the hormone very rare and expensive -- and, as it turned out, dangerous. The early natural versions of the hormone resulted in the infection of three people with Creutzfeldt-Jakob disease, the human equivalent of bovine spongiform encephalopathy, or mad cow disease.
The Genentech hormone posed no such danger, as it was bioengineered. That hormone, rather than being taken from corpses, was made in vats with E. coli bacteria, rather like the process of fermenting beer. Much easier, some observers argued, than pillaging the brains of corpses, and yet the price of the hormone remained high. Genentech won a specialty patent for its human growth hormone, which gave it exclusive marketing rights for seven years. Complaints about the price of the hormone, along with other so-called "orphan" drugs, so named because they were developed to address diseases that affected fewer than 200,000 people in the U.S., prompted a review of the exclusivity laws. But no changes were made.
Right now, Serono's hormone is available to some 1,300 people nationwide through an expanded-access drug program, a federal program that allows experimental drugs to be provided to people with AIDS. Historically, these drugs have been provided free of charge. But Serono has asked for, and received, permission to charge for participation in the drug access program, the first time that that has ever happened. Public and private insurance programs, as well as individuals who can afford it, have been charged the approximately $54,000 a year for the hormone. But just last week, AIDS activists say they negotiated a cap on the amount of money Serono would charge for the drug, bringing the price down to $36,000 a year.
That's still outrageously high, AIDS activists say. "Their view of something like growth hormone is clearly to charge all the market will bear," says Stephen Le Blanc, of ACT UP Golden Gate. "That has got to stop."
"Two years ago you didn't hear AIDS activists talking too much about price," he says. "This year every activist both in America and really across the world is talking about price and trying to convince drug companies to change their marketing strategy."
That will be one of the focuses in Vancouver, where the XI International AIDS Conference convenes July 7. There, activists promise to be focusing on both AIDS wasting and on drug company prices. Instead of selling the drug at high cost to a few, Le Blanc argues, why not sell it to many at a lower price?
"I think all of these companies are going to hear this message very loudly," Le Blanc says.
But, Cella says, Serono hasn't decided what price it will charge if the hormone does win FDA approval. If the approval, in fact, does occur.
"It could be a couple of weeks," Cella says. "It could be a month.