Tuesday, December 09, 2003
RESEARCH TRIAL MEDICATIONS:
From yesterday's Wall Street Journal, a story (sorry, subscription required) about a ethical delima of medical trials: Do you continue to provide the patients with the treatment (chemotherapy in these cases) after the trial is completed?
Twelve-year-old Thomas Tomeny has an aggressive brain tumor. It grew back after surgery last year and didn't respond to either chemotherapy or radiation. Then, in the spring, Thomas was able to join a tiny test of an experimental radioactive drug -- and his tumor shrank by 33% after the first dose.
But despite the improvement, he was told that he wouldn't be given any more of the drug after his third dose in August. He was approved for only three doses -- a condition made clear on the consent form the Tomenys signed in the spring when they agreed to participate.
The story describes other patients in trials and their struggle to continue to receive medications. Two of the patients, one on Erbitux, and one other were dropped because they met exclusion criteria during the course of the study. They had worsening of their symptoms from their malignancy, and were able to be placed on the study drugs again.
Several reasons are given for not allowing study patients to be continued on their medications:
Cost/Supply: One is the cost: even small trials can cost millions and larger ones may run up to $100 million. The majority of drugs tested in clinical trials fail. Many companies make only limited supplies of a new drug, just enough for testing.
Study design: From an analytical standpoint, "it's cleaner and easier to write protocols that say 'this is the beginning' and 'this is the end,' " of a trial, says an FDA spokeswoman
Regulatory/legal: After the trial ends, researchers sometimes have another reason for not allowing patients to continue on the drug. If a person who does well in a trial continues on a medicine and later has a bad side effect, the company is required to report it to the FDA. Companies and researchers worry the FDA could ask questions or even suspend a drug development program if serious concerns about safety arise.
The physician and hospital involved with a trial may be lukewarm in their support of a patient trying to get continued supplies of a drug:
Patients can't always count on hospitals or doctors to go to bat for them. Hospitals compete for clinical trials that bring prestige and money. So hospital boards charged with regulating trials are sometimes reluctant to push too far on patients' behalf for fear companies will take their business elsewhere
An ethical sticky spot, to say the least. The physicians involved in a trial are torn between the very real patient sitting in front of them, and the need to be mindful of the goals and regulations of the trial.
Could not the same argument be made for offering the treatment drug to the control group at the completion of the procedure?
How can a patient make an informed choice (or one at all) when the only hope of extending their life was to enroll in a trial, and hope not to be in the control arm?
When I was a medical student an oncology attending told me that the hope of medical oncology was to make the anecdotal an everyday occurrence. A noble and worthy goal.
Well, got to go and wrap some presents. |
From yesterday's Wall Street Journal, a story (sorry, subscription required) about a ethical delima of medical trials: Do you continue to provide the patients with the treatment (chemotherapy in these cases) after the trial is completed?
Twelve-year-old Thomas Tomeny has an aggressive brain tumor. It grew back after surgery last year and didn't respond to either chemotherapy or radiation. Then, in the spring, Thomas was able to join a tiny test of an experimental radioactive drug -- and his tumor shrank by 33% after the first dose.
But despite the improvement, he was told that he wouldn't be given any more of the drug after his third dose in August. He was approved for only three doses -- a condition made clear on the consent form the Tomenys signed in the spring when they agreed to participate.
The story describes other patients in trials and their struggle to continue to receive medications. Two of the patients, one on Erbitux, and one other were dropped because they met exclusion criteria during the course of the study. They had worsening of their symptoms from their malignancy, and were able to be placed on the study drugs again.
Several reasons are given for not allowing study patients to be continued on their medications:
Cost/Supply: One is the cost: even small trials can cost millions and larger ones may run up to $100 million. The majority of drugs tested in clinical trials fail. Many companies make only limited supplies of a new drug, just enough for testing.
Study design: From an analytical standpoint, "it's cleaner and easier to write protocols that say 'this is the beginning' and 'this is the end,' " of a trial, says an FDA spokeswoman
Regulatory/legal: After the trial ends, researchers sometimes have another reason for not allowing patients to continue on the drug. If a person who does well in a trial continues on a medicine and later has a bad side effect, the company is required to report it to the FDA. Companies and researchers worry the FDA could ask questions or even suspend a drug development program if serious concerns about safety arise.
The physician and hospital involved with a trial may be lukewarm in their support of a patient trying to get continued supplies of a drug:
Patients can't always count on hospitals or doctors to go to bat for them. Hospitals compete for clinical trials that bring prestige and money. So hospital boards charged with regulating trials are sometimes reluctant to push too far on patients' behalf for fear companies will take their business elsewhere
An ethical sticky spot, to say the least. The physicians involved in a trial are torn between the very real patient sitting in front of them, and the need to be mindful of the goals and regulations of the trial.
Could not the same argument be made for offering the treatment drug to the control group at the completion of the procedure?
How can a patient make an informed choice (or one at all) when the only hope of extending their life was to enroll in a trial, and hope not to be in the control arm?
When I was a medical student an oncology attending told me that the hope of medical oncology was to make the anecdotal an everyday occurrence. A noble and worthy goal.
Well, got to go and wrap some presents. |
