In a great editorial piece in the Wall Street Journal November 27, Darcy Olsen, author of the newly published The Right to Try (HarperCollins), makes the case for supporting what’s known as “right to try” legislation. This legislation, which has now been passed by 24 states, simply says that terminally ill patients have the right to try a promising new drug, even if it has not been approved by the FDA, as long as it has passed basic safety tests.
I liked the editorial because we had a working version of right to try in operation way back in 1976, when I was director of the Division of Cancer Treatment (DCT) at The National Cancer Institute. We called it the Group C program, and it was approved by the FDA and the Pharmaceutical industry, and operated by the NCIThe FDA never liked it. But I had the bully pulpit and I used it.
In the Group C program all cancer drugs in development and supported in any way by the NCI were put into three groups A, B and C. Group A and B were for drugs in early testing, known as phase I and II trials. If a drug showed promise in a minimum of two trials in groups A and B, the NCI could move it into group C. Once in group C, a drug was available for compassionate use for cancer patients who were not in a clinical trial.
The determination of the potential benefit for cancer patients was made by the patient's doctor at the bedside. He or she only had to be registered at the FDA, which required filling out a one page form (Form 1572) only once. The only further paper requirement was that the physician needed to report any adverse event to the NCI, which, in turn, reported them to the FDA. Notice that all the important decisions were made by active cancer doctors. The decision to go into group C by NCI doctors; the decision to use the drug, by the patient's Oncologist.
It was good for doctors, who could try everything for their patients. It was good for patients, who got a shot at a cure or at least longer survival. Even the pharmaceutical industry liked the program, and willingly supplied drugs for distribution to patients in need at no cost. They felt it was good exposure for their programs.
The FDA approved it as part of a master plan submitted by the NCI for cancer drug development. But it was never codified by the FDA, and they took the first opportunity to drop it in1987, shortly before my departure as director of the NCI in favor of the Treatment IND program, which was developed to cover drugs for AIDS patients. The problem is it has never worked for cancer patients. AIDS was one disease with one or two drugs. One treatment protocol could suffice for everyone. Cancer is a hundred different diseases with many drugs and a multitude of protocols. Cancer patients have been denied free access to promising cancer drugs by the FDA ever since.
The FDA claims, as Olsen points out, that patients can still access drugs through their compassionate use program, which is just not true, because the paper burden on a doctor is oppressive and unworkable. And there are other notable difference between the present compassionate use program and NCI's group C program.
In the FDA’s compassionate use program, the determination of whether a drug should be made available at all is made by the FDA, not the NCI, and the determination of whether the drug should be used in an individual patient is made by an FDA bureaucrat thousands of miles away--not the patient's doctor, who is actually at the bedside.
And another factor was added by the FDA. They had to determine that giving out the drug would not be harmful to NCI's clinical trials program. There is no other way to determine this except to ask the person running the trials in question, who invariably protect their turf by saying (guessing) that it will harm their trial.
But it’s not true. I ran the clinical trials program from 1976 until 1987, and there was no evidencethat making drugs available to patients, many of whom weren't even candidates for clinical trials, had any negative effect on NCI’s trials.
The right to try legislation differs in some ways from the group C program, too. In right to try, a drug has to have completed phase I testing for safety. This is not unreasonable, but in some circumstances, drugs already show great promise in Phase I trials.
The most recent example is the agent brentuximab vedotin, the hottest new treatment for advanced Hodgkin's disease. This drug produced complete remissions in heavily pretreated patients (i.e. patients you might expect to be resistant to drug treatment) in its phase I trial, the results of which were published in the prestigious New England Journal of Medicine.
The oldest example is the drug vincristine, which is part of almost all curative programs for childhood leukemia, andwas clearly active in the earliest of clinical trials. Drug companies are now able to recover the cost of drugs (but make no profit) in right to try legislation.
The FDA is now panicking over loss of control as a consequence of right to try legislation, and it is promising to speed up their compassionate use program, which is really a tacit admission of the failure of the program. Nothing promising has been forthcoming and none is likely.
The NCI, which should be on the bully pulpit, urging passage of right to try legislation, has been silent. So have organizations that purport to be the voice of cancer patients like ASCO (The American Society of Clinical Oncology), the AACR (American Association for Cancer Research), and the American Cancer Society. They’re silent because they believe they are defending the integrity of clinical trials--based on zero evidence. Meanwhile, patients who want—and deserve—a shot at survival, aren’t getting it. Patients, I might add, who have paid for the war on cancer with their tax dollars. What they need is a for the director of the NCI to rise to the occasion, take the lead, and speak up for them.