Thursday, September 25, 2003

When an untested drug is a patient's last chance.

An article in the Boston Globe this week explored the problems faced vby patients with terminal illnesses who have run out of drugs to help them fight their disease. In the view of some, "the current ways to get experimental drugs -- through clinical trials, 'compassionate-use' or 'expanded-access' programs -- are tragically inadequate." In response, some patients' groups are suing the FDA "to create a new level of review called Tier 1 Initial Approval. Under this plan, patients would be able to get an experimental drug if it has passed Phase I trials, if the patient has been rejected from clinical trials of the drug, and if nothing else has worked. Perhaps most controversially, they also want to allow patients to pay manufacturers for the drugs, an issue that opens a Pandora's box of ethical and liability questions." Opposition to the proposed new classification is mounting. "Nancy Roach, a director of the Marti Nelson Cancer Foundation based in Vacaville, Calif., . . . opposes the Tier 1 idea, saying, 'It would rip the heart out of clinical research.' . . . At the end of a Phase I (safety trial), a drug may have been tested in only a few dozen people. (It is not until Phase II and Phase III trials that a drug is tested in more people and researchers study dosages and efficacy.) 'You don't give drugs to people unless there's a good reason to, you know how to give them, and the person has some chance of benefiting,' she said." The widely respected physician-ethicist, Marcia Angell is also against the idea: "'New drugs are far more likely to fail than to succeed,' she said, 'so the chances are that a patient will be hurt by a drug rather than helped.' . . . She said she also is appalled that patients might end up paying manufacturers for experimental drugs. Drug companies already 'are profitable beyond any industry,' she said. 'They are protected by the government. They have monopoly rights, patents, tremendous tax breaks.' And the opposition doesn't stop there. Alan Goldhammer, associate vice president for regulatory affairs for the Pharmaceutical Research and Manufacturers of America, said it would be "potentially reckless" for manufacturers to release drugs after only Phase I trials because at that point there is "no proof of efficacy at all."
posted by tommayo, 6:17 PM

Health care law (including public health law, medical ethics, and life sciences), with digressions into constitutional law, poetry, and other things that matter