Government Reform and Oversight Committee
February 4, 1998 Mr. Arnold Eggers, MDMr. Chairman and Members of the Committee:
I have worked for the past twenty-five years, with some interruptions, on a study of tumor immunology with a view to developing a vaccine treatment for cancer.
I began this project as a medical student at Columbia University, took residency training at New York Hospital, which is Cornell University, and at the Hospital of the University of Pennsylvania, went to NIH in the National Cancer institute for three years, came back to Columbia to finish residency, and have worked as an attending first at Columbia and now at SUNY-Health Science Center at Brooklyn.
The purpose of describing my training is to emphasize that I have good academic credentials and that this is my life's work. It is a long-term commitment to one idea: reproducing the spontaneous remissions which are sometimes seen in cancer patients following bacterial infections. My vaccine is a kind of non-toxic immune stimulation. The final version of the vaccine treatment produced the results which you see before you. Three out of four brain cancer patients who received the final version of the treatment went into remission.
Out of 180 injections of this and previous versions of the vaccine there was only patient with aside-effect--one case of an allergic reaction which was not fatal. Now the man in the street might say that this looks like a promising new treatment which should be supported by our government in the war on cancer, but the man in the street does not know how the system works. Having already received approval from the FDA in 1989 to treat brain cancer patients, I applied in 1994 for permission to treat other kinds of solid tumors as well and was put on clinical hold. It is now almost four years later, the clinical hold is still in effect, and we are still dialoguing. Most of this time has been spent discussing technical minutiae. The [DA inspected my records and issued a warning letter which said "deviations in the conduct of this study appear to be the result of your lack of understanding of the procedures and requirements that govern the use of investigational new drugs."
It is important to emphasize that their citations were in general appropriate and correct from their point of view. They found valid deviations from the rules. Just to give one example, they cited me for incorrect patient consent forms. It turns out there are seventeen elements of informed consent which require one and a half pages of small print just to list. My consent forms, although approved by the local hospital ethics committee, constituted a violation of statutory law. As you know, the FDA has sent at least sixteen people to federal penitentiary in the last ten years for violations of their rules, which have the power of statutory law. I appealed to an ombudsperson at the FDA. She told me that if I wanted to have any hope of meeting regulatory requirements, I needed to hire a professional FDA consultant, one ofthe people drug companies hire to interface with the FDA. This is way beyond my means financially.
In all of this, no one has acted with malice. On one side you have a university scientist approaching the regulatory process with good will and on the other side professional bureaucrats approaching their jobs with goodwill. Yet between us we could not make the system work in four years. The man in the street might want to know what went wrong. I think the problem lies with the system and not with individual bureaucrats, who are only doing their job, and in most cases doing it well.
As everyone knows, the FDA was established in its current form by the Kefauver-Harris Drug Amendment in 1962 as a response to the thalidomide tragedy in Europe, in which 2000-3000 mothers who took this particular sleeping pill gave birth to children with serious birth defects. An FDA employee, Frances Kelsey, became a national hero by blocking legal entry of the drug into the US. It is important to note that the drug was a sleeping pill, and no one dies from insomnia. The mistake in thinking behind Kefauver-Harris is that it fails to distinguish between fatal and non-fatal diseases. In the case of non-fatal diseases like insomnia or acne, you want to protect people from unnecessary side-effects.
This was what thalidomide was about. In the case of fatal diseases like cancer, the situation is more complicated. In deciding if government regulation is worthwhile, you have to compare the number of people who die from toxic side-effects of inadequately-screened new medicines with the number of people who die waiting for the release of successful new medicines. The bureaucratic process saves lives on the one hand by screening for toxicity but takes lives on the other hand by delaying access to treatment.
Cancer kills 500,000 people a year in the U.S. A one-year bureaucratic delay in releasing a cure for cancer would necessarily kill 500,000 people. These are the people who would still be alive if the government hadn't blocked their access to treatment in their lifetime. These days, toxic side effects of drugs are quickly discovered and publicized, or else extremely rare, and it is inconceivable that 500,000 people could be killed by a dangerous new treatment before the alarm was called. I believe this arithmetic or statistical argument shows the error of the current system, which guarantees that there will be a vast unnecessary loss of life if ever cancer is cured.