Pharmaceutical Greed and Redundancy

Mr. Fenstermacher at the start of a trial of an experimental drug, which has since shown signs of helping fight his cancer. Credit George Etheredge for The brand-new York Times

Pharmaceutical Greed and Redundancy

"I think there is a lot of exuberant rush to market."
"And we are squandering our most precious resource -- patients."
"That [research trials with few patients] leaves some of us evidence geeks wondering if it works."
Dr. Peter Back, Memorial Sloan Kettering Cancer Center, New York

"We used to have trials not long ago that had 700 patients per arm."
"That's almost undoable now. Trials can be [comprised of] eight patients."
Dr. Norman Sharpless, University of North Carolina

What concerns both Dr. Sharpless and Dr. Bach is that those few numbers of patients taking part in research trials results in studies so small and of short duration that their outcomes often resemble the result of chance. Reliably hard data is difficult to come by with these trials. And according to Dr. Scott Ramsay of the Cancer Research Center in Seattle, serious side effects may not be detected with new methods since the treatments are of such brief duration.

As for the patient end of the enterprise, when new drugs and their accompanying regimens turn out to be promising, he also points out that with success there is a clamour among patients to have access to them, "but you wonder if you are doing them any favours", taking into account their expense, usually sky-high, and the imponderable of potential side effects going undetected.

What accounts for so many trials of experimental new drugs and their therapeutic value? Ah, back to the sky-high pricing. There is money to be made from treatment patented to give people another chance at life when grim diagnoses attached to a killer-disease like cancer is involved. Which translates to pharmaceutical enterprises viewing a breakthrough achieved by a competitor wanting a piece of that remunerative action for themselves.

Take for example the new leukemia treatment recently approved by the U.S. Food and Drug Administration and hailed as a true breakthrough in the treatment of child and young adult leukemia that fails to respond to conventionally successful treatment. Other pharmaceutical companies than Novartis are anxious to develop their own immunotherapy. Leading to a situation where over one thousand immunotherapy trials are currently underway.

Many of which are similar, reflecting one another, representing no particular breakthrough of their own, and as such representing a redundancy. "It's hard to imagine we can support more than 1,000 studies", observed Dr. Daniel Chen of Genentech biotechnology company. And in 'supporting' that number the search is on for participating patients, and they're not all that numerous, since each of the trials looks for patients with mutations in their cancers.

In the United States alone, over 85,000 cases annually of melanoma present themselves, and while most melanomas are handled by surgery, roughly 10,000 of the total are patients who suffer relapses and thus may be potential candidates for experimental treatments. And these are the patients that pharmaceutical companies must test their experimental treatments on. Without enough patients enrolled in a study it simply becomes difficult to determine whether a treatment works reliably.

Many pharmaceutical companies want to develop their very own anti-PD-1, a protein that immunotherapy drugs attack, and approved for treatment of lung, renal cell and bladder cancers, and Hodgkins's disease. The idea is once they succeed in formulating their own anti-PD-1 drug, it can be used along with their own formulae of secondary drugs. This is a multibillion dollar industry, after all, that all the competitors want a part of.

"How many PD-1 antibodies does Planet Earth need?", Dr. Roy Baynes of Merck wondered of the drugs that attack mutations required by tumours to grow and thrive and which targeted therapies hope to cure by blocking the mutations so the tumours will perish. Since mutations are relatively rare and the patients with cancers and the mutation do not themselves know, groups of cancer patients must undergo genetic testing of their tumours to determine whether they have the mutation.

The Food and Drug Administration is comfortable with small control groups for trial purposes; however, they're on the lookout for new drugs proving so effective it is a foregone conclusion that they succeed; where patients go into remission with the new treatment, even though their condition was so dire they were informed to prepare for death.

Pfizer experienced some difficulties in locating 50 lung cancer patients carrying a rare aberration called ROSI which merely one percent of patients carry. It took them three years to recruit that number of patients for their study.

Memorial Sloan Kettering is actually testing a drug which attacks a tumor using a mutation found in just 1 percent of cancer patients.  Credit  George Etheredge for The brand-new York Times