Friday, November 27, 2015

In a great editorial piece in the Wall Street Journal on 27 November , Darcy Olsen author of " The Right to Try", out this month from Harper/ Collins, makes the case for support of right to try legislation now passed by 24 states. This legislation simply says that terminally ill patients have a right to try a promising new drug, even if it is not 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, approved by the FDA and the Pharmeceutical industry and  operated by the NCI all the way back in 1976. It was called the Group C program. It was a rough slug fest to get it operational, as the FDA never liked it. But the NCI director has the use of the bully pulpit to defend its programs and cancer patients and I used it.  The FDA never liked being out it bright sunshine so they approved it as part of a master plan submitted by NCI for cancer drug development. But it was never codified by the FDA so they took the first opportunity to drop it in  1987 shortly before my departure as NCI Director  in favor of the treatment IND program . The latter was developed to cover drugs for AIDS patients and has never worked for cancer patients who have been denied free access to promising cancer drugs by the FDA ever since. This is all described in detail in my book "The Death of Cancer ", written with Elizabeth DeVita -Raeburn, in a chapter called " The Francis Kelsey syndrome" also out this month from Ferrar Strauss Giroux.
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. A and B were for drugs in early testing called  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 cancer patients who were not in a clinical trial for compassionate use. 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 1573 only once. The only paper requirements 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. The FDA claims, as Olsen points out, that patients can still access drugs through their compassionate use program which is pure and utter nonsense 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. 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 to the authority of 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 it would be. This is a straw man used to suppress access to drugs by cancer patients by organizations that should know better including the NCI . I ran the Clincal trials program in the period from 1976 until 1987 and there was no evidence  that making drugs available to patients, many of whom weren't  even candidates for clinical trials, had any negative effect on NCIs trials. The Pharmeceutical industry by the way liked the program and willingly supplied drugs to NCI at no cost . They felt it was good exposure for their programs.
The right to try legislation differs in some ways from the group C program. 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 , which was already producing complete remissions in heavily pretreated patients in its phase I trial which was 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 which was clearly active in the earliest of clinical trials. And drug companies are able to recover the cost of drugs in right to try legislation.
Two more points.  The FDA is panicking over loss of control as a consequence of right to try legislation and is promising to speed up their compassionate use program which is really a tacit admission of the failure of he program . Nothing promising has been forthcoming and none is likely.
 The final point is that the NCI which  should be on the bully pulpit urging passage of the legislation has been silent. So have organizations who purport to be the voice of cancer patients like ASCO ( The American Society of Clincal Oncology ) and even the AACR ( American Association for Cancer Research) and the American Cancer Society all in defense of the integrity of clinical trials based on zero evidence. We need an NCI director who will rise to the occasion and  take the lead again and speak up for cancer patients everywhere.

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