Originally created 02/24/01

Lengthy FDA approval process can be deadly quagmire for cancer patients



MICHAEL J. Clarke wrote a recent letter to The Wall Street Journal in response to my oped piece "Cracking the FDA Code." Clarke has prostate cancer and is afraid he might "not be around to reap the benefits" of new treatments for cancer before they are approved by the Food and Drug Administration. "It is worth the risk," he wrote. "Let me have it." Well said.

Clarke expresses exactly the same view of thousands of patients I have treated over the last 20 years. Many of my patients died waiting for a response to this same request. Patients are willing to take informed risks when they face imminent death; risks that the FDA and our research-regulatory alliance are unwilling to allow them to take. Clarke and patients with life-threatening diseases deserve a response to their plea. Whose life is it anyway?

The research-regulatory alliance in our country consists of the Food and Drug Administration, the pharmaceutical/bio-technology industry, and for cancer, some 25 federally funded Comprehensive Cancer Centers and the American Cancer Society working in concert with cancer specialists responsible for treating patients. These are the "thought leaders" who define the cancer treatment and treating patients. These are the "thought leaders" who define the cancer treatment and research enterprise in this country. This is the alliance that must respond to Clarke's plea.

TOO OFTEN, this alliance emphasizes minimizing risks rather than maximizing opportunity. The Thalidomide story is generally raised by the FDA and the alliance whenever it is suggested patients need earlier access to new treatments. More than 30 years ago, this medication caused terrible birth defects in some children when their mothers took it while pregnant. In a bizarre twist of fate, Thalidomide is now being tested as a cancer drug and appears to be effective in multiple myeloma, a cancer of the bone marrow. This argument has become a mantra for those who would restrict access to new treatments under the guise of "protecting the patient."

The Thalidomide argument is not appropriate for patients with life threatening illnesses, where the issue is not how to avoid the next Thalidomide; the issue is how to save lives of cancer patients today. I believe the patient must be made a voting member of this alliance to make "thought leaders" more responsive to the needs of the very population of individuals the alliance serves.

There are now about 500 drugs being tested in people with cancer. Contrast that to the fact that, as of 2001, fewer than 100 drugs have been "approved" for cancer treatment in the history of medicine and fewer than 30 have actually provided meaningful life extension to patients with advanced cancer. Since 60 percent of all drugs that get into human testing (more than 99 percent never get there) eventually become approved drugs, this means today's patients will have three times as many drugs available to them within the next 2-4 years.

WHAT BETTER time for the alliance to respond to Clarke by accelerating this process and making more opportunities available to cancer patients while they are still alive and willing to take greater risks under informed consent? Will the alliance allow today's patients to die when a few months hence effective treatment is saving the lives of similar patients with cancer?

Consider the case of STI 572, a new drug for chronic myelogenous leukemia. This drug entered clinical testing recently with no more expectations for success than many other drugs at that stage of development. To the surprise of many in the oncology community, it was found immediately and dramatically to be a life saving drug.

Why have we taxpayers spent more than $50 billion on cancer research since President Richard Nixon declared "war on cancer" in 1971 if not for the patient?

We all want to learn more about the biology of cancer, but the taxpayer surely is in agreement with Clarke that the primary objective of all this research is not the acquisition of knowledge per se, but how to improve the plight of the patient with cancer. For the 500,000 Americans who die each year from cancer, for our mothers, our fathers, our sisters and brother and, especially for our children, our thought leaders need to respond affirmatively to Clarke's plea.

(Editor's note: The author, is president of the Augusta-based Cancer Therapeutics Inc.)