This is an old debate and today’s Globe story seems to come down on yes: The story tells the tale of a grandmother who wants access to an experimental new cancer breast cancer drug but has to travel to get it.
Regulators and companies “must make sure a drug that is initially effective does not later prove to be dangerous. That requires years of clinical trials. And to make trials scientifically valid, some patients enrolled in them — no matter how ill — don’t receive the drug being tested….”
Whom to believe?
“There are hundreds or potentially thousands of patients over the next two years who won’t have access to a drug that clearly has benefits for them,’’ said Dr. Ian E. Kropp, assistant professor at Harvard Medical School and a breast cancer specialist at Dana-Farber.
Or:
Dr. Eric Weiner, director of the hospital’s breast oncology center, disagrees with the FDA decision on T-DM1, but he doesn’t support giving it to patients outside of a trial unless the agency indicates approval is imminent.
“It’s a great drug,‘’ he said. "But once the FDA made the decision, patients shouldn’t be able to get the drug except in a legitimate clinical trial where researchers can ask the questions that will lead to the drug approval.’’
Boston doctors also debates this question in a NYTimes story on the use of PLX4032, a potential treatment for melanoma:
The F.D.A. regularly approves such programs, known as “compassionate use,” for promising experimental drugs. But Roche feared a prospective trial candidate might undergo chemotherapy just to qualify for compassionate use and get PLX4032 with no strings attached.
In an emotional moment, Dr. Donald Lawrence of Massachusetts General Hospital e-mailed colleagues about Roche’s decision last spring, under the subject line “moral outrage.”
“Just had yet another conversation with a [patient] with a B-RAF mutation who will die in the next month or so because he can’t get PLX4032,” he wrote. “I feel we need to muster the support of our patients and lobby both Roche and the F.D.A. Compromising the Phase III trial is not justification for withholding an effective drug from dying patients.”
But Dr. Michael Atkins, director of the cancer clinical trials office at Beth Israel Deaconess Cancer Center in Boston, urged him to consider what he thought was the greater good: “Even though it is painful, I think completing a clean Phase III trial and determining if there truly is a survival benefit for PLX would have major value for the field and future patients.”
Some argue there is no harm in trying something that might help. But, all drugs come with side effects and risks. See the story in USA Today about a Vermont woman who suffered serious side effects from an approved drug for breast cancer which, turned out, didn’t work.