It’s probably helpful to describe the decision-making process that led to our position regarding the use of a placebo. We are a diverse group of GIST patients, families and friends in about 30 countries. Some of us interact with one another solely through the Internet. Some of us belong to groups that meet face-to-face in such places as Chicago, the Netherlands, New York, Germany, Los Angeles, Switzerland, Detroit and Texas.

We created a 12-person clinical trials advisory group that has met for many hours of deliberation in person, by teleconference and through the Internet. This group of 10 GIST patients and two caregivers includes an accountant, lawyer, medical doctor, executive, scientist, newspaper editor, MIS director, Marine Corps colonel, public health professional and others. This group unanimously opposes the use of a placebo.

We listened attentively to the explanations offered for a placebo. At our third meeting with Pfizer, we naively accepted in good faith their offer to work with us to construct a database that could be considered as an alternative to a placebo group. At our last meeting we discovered Pfizer considered this to be a “waste of time,” to quote the senior Pfizer representative present. We have yet to see a scintilla of evidence supporting the need for a placebo, nor any assumption of the burden of proof incumbent upon anyone pursuing such a research protocol.

We have yet to see any analysis of the risk a placebo poses to patients, nor any risk benefit determination for using such a placebo.

We have heard many carefully scripted statements to the effect that Pfizer must “document measurable clinical benefit,” and that “historical data lacks scientific validity and does not exist,” (although a Pfizer vice president acknowledged he had not looked at the historical data, deeming it “a waste of his time”).

We’ve been told the United States Food and Drug Administration supports the use of a placebo. Let’s see what the FDA actually has to say: Historical data “is particularly useful when the disease being treated has high and predictable death or illness rates … Is it ethical to give patients a placebo when effective treatment is available? … the generally accepted practice … is that fully informed patients can consent to take part in a (placebo) … trial, even when effective therapy exists, so long as they are not denied therapy that could alter survival or prevent irreversible injury …”

We submit that GIST has a high and predictable death rate. We submit that GIST patients on the placebo will experience irreversible progression of their tumors, causing injury, and possibly death.

It is also striking that the only consent form given study patients in this trial that we have seen fails to include a single statement addressing dangers to the placebo patient.

Since it seems abundantly clear Pfizer has no intention of reconsidering the use of a placebo in this trial, what is the point of going on record as opposing it?

The Life Raft Group is a tiny patient organization that is less than two years old. Pfizer is the largest pharmaceutical company in the world, with huge financial and political resources. There is a disconnect between our patient group and those behind this study and, frankly, we are somewhat afraid and intimidated by the consequences of opposing them. It could discourage Pfizer from holding clinical trials needed to develop drugs for GIST and other cancers, and it will likely eliminate the Life Raft Group from any possible grant support from Pfizer for our educational and outreach activities. To quote our Pfizer colleagues, the train has “certainly left the station.”

The voice of our group is nonetheless focused and clear.

It is wrong to give a sugar pill to a cancer patient for whom there is no documented treatment other than the trial drug.

It is wrong to give a sugar pill to a cancer patient whose almost inevitable disease progression may not be reversed by a too-late switch to the actual drug.

It is wrong to trade irreversible, life-threatening damage to cancer patients for an undocumented rationale focused upon bringing a drug to market faster.

We oppose the use of a placebo in this trial simply because it is wrong.

Jim Hughes
Author: Jim Hughes