It was Thanksgiving when a 75-year old physician — we'll call him Mark Hall for the sake of privacy — suspected that his stomach pains were more than just an ulcer. A week later, the diagnosis of cancer of the pancreas was confirmed.
Pancreatic cancer, especially after it has metastasized, as had Dr. Hall's, is one of the most dangerous of all cancers. Only 4% of patients are alive five years after diagnosis, compared to 64% for other cancers. With much difficulty, Dr. Hall was signed up for a clinical trial of a new drug. His goal: attending his grandson's bar mitzvah in the spring.
But there were glitches in Dr. Hall's getting started with the clinical trial. He went three weeks without any treatment at all because he had to wait for the new drug trial to start. A condition of participating was that he forgo any prior treatment. Then, at the last moment, he was not permitted in the trial due to an additional hurdle imposed by the Food and Drug Administration. His electrocardiogram was a fraction off the FDA standard. He's now on the standard treatment, Gemzar, approved by the FDA a decade ago.
To begin with, the odds are stacked against terminally ill patients. Then, in addition to their problems, they find their access to new drugs severely restricted. Even though exceptions have been made for cancer and AIDS, doctors and pharmaceutical companies are concerned that they will get into trouble with the FDA if they give patients certain drugs before approval.
A University of Chicago professor, Richard Epstein, discusses this problem, along with many others, in his illuminating new book, "Overdose: How Excessive Government Regulation Stifles Pharmaceutical Innovation," published by Yale University Press. According to Mr. Epstein, "There are huge numbers of requests for compassionate exemptions to FDA safety regulations, often brought by sophisticated consumers before drugs reach the market. Statutes that are designed to protect everyone give differential advantages to the rich, although, ironically, we would all be better off without them."
On December 11, the FDA issued new proposed rules to try to increase access to new drugs. In a press statement accompanying these rules, the FDA notes, "One of the major criticisms about access to investigational drugs is that the criteria for authorizing access are unclear and that there is not broad knowledge among affected, or potentially affected, parties about the mechanisms or procedures to obtain access."
The FDA's goal is to update existing regulations to clarify when doctors can prescribe new drugs. The proposed rules concern individual patients in both non-emergency and emergency settings; small groups of patients, and larger groups of patients who are being treated with investigational new drugs.
These rules are clearly a step in the right direction. But they do not go far enough. They are still complex enough to deter doctors and patients from accessing the drugs, and they place too much of a burden on the FDA to approve individual situations. Just as is the case now, only educated or lucky consumers will have access to the newest drugs.
Indeed, the FDA expects the number of individual patients — those not in group trials — who receive investigational new drugs to rise from the current 659 sick people to 1,054 over three years and to remain constant thereafter. This is a tiny fraction of sick people.
The new rules stipulate that there must be no other approved therapy for the seriously ill patients. But patients differ in their response to drugs. For some patients, an approved therapy may not work. For others, it might work just fine. And even approved therapies can be improved.
Benefits to the patient should be greater than the risks, according to the new FDA rules. However, benefits and costs also differ by patient, and, being subjective, are best assessed by patients themselves. Some people may have significant side effects from a drug that others tolerate well.
A resident scholar at the American Enterprise Institute, John Calfee, has a better suggestion: "Let patients and doctors choose whether to use new drugs." Since patients have very specific needs, and doctors have the most information about these needs, it makes sense for patients and doctors to make the decision.
To the contrary, Dr. Marcia Angell, senior lecturer at Harvard Medical School, writing in the New York Review of Books earlier this year, says, "We have an FDA precisely because we know that drug companies, given their inherent conflict of interest, should not be left to decide on their own whether their products are safe. Caveat emptor may be a reasonable approach for many consumer products, but not for prescription drugs."
But the process of matching cures with individuals' diseases is practically impossible for the government to get right because patients have different preferences for risk. Although we save some lives through higher safety standards, the delay in drug approval is causing others to die.
Dr. Scott Gottlieb, a physician who is the FDA's deputy commissioner for medical and scientific affairs, in a speech earlier this month said, "In the end, it is very hard for those of us here in Washington to anticipate and understand everything that goes on in a doctor's office, when patients are confronted with hard and uncertain choices. Many decisions are guided by tiny bits of pertinent evidence, not because people prefer it that way, but because peoples' problems don't fit the clean categories that may be studied in a clinical trial."
New Year is almost upon us, with its traditional wishes for health and happiness. In our great universities and laboratories, scientists have been making discoveries that can keep Dr. Hall alive until his grandson's bar mitzvah and that can keep others alive for the equally important events in their lives. Let's pray that our regulatory system will allow these discoveries to be used in time. Time is short, and it's all that we have.
This article originally appeared in the December 29, 2006, New York Sun.