A Journal for Industry Executives

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This week, Scott Gottlieb, MD Deputy Commissioner for Medical and Scientific Affairs at the Food and Drug Administration made an interesting speech before the 2006 Conference on Adaptive Trial Design in Washington, DC.Dr Gotlieb commented how today’s clinical trials are highly empirical. Drugs are tested on general populations for a response and a treatment effect that is statistically not likely to be a chance result. This approach is rigorous and focused, but inflexible. According to Dr. Gotlieb, “another problem with the empirical approach is that it yields statistical information about how large populations with the same or similar conditions are likely to respond to a treatment. But doctors don’t treat populations, they treat individual patients. Doctors need information about the characteristics that predict which patients are more likely to respond well, or suffer certain side effect. The empirical approach doesn’t tell doctors how to personalize their care to their individual patients.” This results in a highly empirical approach to the practice of medicine. Gone are the days of: Take two off these and call me in the morning. Gottlieb comments “Doctors prescribe treatments knowing full well that only a certain percentage of their patients will receive a benefit from any given medicine.” This approach is akin to: Take all of these and call me if you have a side-effect. 

But with the demands for personalized medicine and the advent of Pharmaco-genomics, there are potentially better alternatives. By enabling more trials to be adapted based on knowledge about gene and protein markers or patient characteristics, we can help predict whether patients will respond well to a new medicine. “These new approaches to clinical trials can result in trial designs that tell us more about safety and benefits of drugs, in potentially shorter time frames, exposing fewer people to experimental treatments, and resulting in clinical trials that may not only be more efficient but are more attractive to patients and their physicians to enroll in.” said Gottlieb. This is only a first step in the process to develop more adaptive clinical trials. This process will lead to more targeted therapies and in turn, Ii is my hope, more personal and personalized medicine.

During my senior year, I was lucky to have a few job offers to evaluate. The top two were from Johnson & Johnson and Exxon. As part of my research I looked at how each company handled adversity. For me it was an easy choice when I compared J&J’s handling of the Tylenol situation vs. Exxon’s Valdez.  I was drawn by the impact I could make in people’s lives and by the industry’s reputation in general and J&J’s in particular.

But in recent years, our industry has gone though some tough times. We’ve have seen record FDA fines, product recalls and withdrawals as well as the erosion of consumer confidence.  In my opinion executives at some Life-Science companies lost focus on the patient and began to concentrate on profits for Wall Street.

It is in times like these that we must reach for our roots and reflect on the legacy left by our industry’s founders.

In the August 1952 Time magazine interview, George W. Merck said “Medicine is for people, not for profits.” He went on “…if we remembered that, the profits have never failed to appear. The better we remembered, the larger they have been.”

In 1943 General Robert Wood Johnson wrote: “We believe our first responsibility is to the doctors, nurses and patients, to mothers and fathers and all others who use our products and services.” This Credo is still etched in stone at the J&J world Headquarters.
In 1899 Charles Pfizer said, “Our goal, has been and continues to be the same: to find a way to produce the highest-quality products and to perfect the most efficient way to accomplish this, in order to serve our customers. This company has built itself on its reputation and its dedication to these standards, and if we are to celebrate another 50 years, we must always be aware that quality is the keystone.”

In this post Sarbanes-Oxley era, I am heartened to see that our industry is once again putting patients first.

Let us remember to chase after the cures for human ailments, then and only then will fair profits follow.