After years of industrys growing influence in the medical research endeavor, the public and the medical community are responding, at times rationally and at times with spasms of righteous indignation. The simple problem is this, the medical profession is based upon knowledge and trustspecialized knowledge of human physiology and disease, and a very special trust to use that information in the best interests of caring for the sick (or promoting health, depending upon your point of view). Industry, in a private enterprise system, has a responsibility of profitability to its investors. Therefore, it is the job of doctors to use their medical knowledge to help people. It is the job of industry to sell product.
Now the story gets a little more complex. In order to bring a medical discovery or body of medical information from the laboratory to the bedside, the idea must frequently be converted into a usable format, which can then be tested for safety and effectiveness, and then introduced into the care of patients. How does this happen? Let us say that careful work in the laboratory had discovered an intracellular mechanism that causes heart cells to fail when presented with certain stimuli. Let us further suppose that the findings were sufficiently compelling to investigate methods of blocking that process and, that when this is accomplished, the heart cells are restored to normal function. There are five million Americans who currently suffer from congestive heart failure; nearly one half million die from it each year. Wouldnt it make sense to see if the development of a medication to achieve this biochemical goal might not help reduce some of the morbidity and mortality of this devastating disease? Who is going to do this? Who is going to undertake the multimillion dollar (sometimes billion dollar) risk of developing, testing, studying, gaining approval for and marketing (i.e. making available to patients) this medication?
Let us not forget – that which works beautifully in a cell culture in the laboratory may fail miserably in the complex multicellular organism of the human being. Even that which is quite successful in animal models may have unpredictable and devastating consequences or side effects when applied to an actual clinical scenario. Unless there is some mechanism to compensate or ensure a return on investment, who has the millions of dollars readily available to drive the research process? The Federal government? Perhaps. We now seem to be entering an age when people are turning in desperation to the government to right all wrongs and cure all ills. However, assuming that our government could somehow find a way to afford it, how well will the people tolerate massive investment in an area where a 10% success rate is considered high? How many failed attempts will the public tolerate before they question whether the government is squandering funds? How long before some Congressman chooses to make a name for himself by investigating the misappropriation of taxpayers hard-earned dollars on medical adventurism? In short, even the stuffiest of purists might admit that there does seem to be a role for industry in the development of medically useful products, be they medications, devices, or even information systems. In fact, it is the very incentive of potential profit that aligns incentives to drive the process toward successful outcomes, thereby facilitating the attraction of necessary creative initiative, talent and dollars.
So here comes the tricky part. Lets say I am Mr. Businessman and I want to invest in a drug to cure heart disease. I want to do that because my friends at the Florida Heart Research Institute have told me that it is the leading killer of men and women in the country. Therefore, I cannot only maximize the good, but I can also guarantee a large market which would be willing to pay a meaningful price. To whom am I going to turn to decide what my wonder drug is going to do? It is only the scientists and physicians in the field who are going to know anything about the disease, its mechanisms and the opportunities for cure.
Change of scene. Now I am Dr. Researcher, and I have just made a discovery which I feel will have a major impact on curing heart disease. I report my findings in the best peer reviewed journals and then what? Its back to lab and the chemicals and cells and mice? Or I find myself a partner who can help me develop this concept into a medication that can actually be delivered to those who need it. And, as the process evolves, I am so convinced that I have a viable and valuable product that I wish to invest my lifes savings in the company which is developing it.
Let us assume for a moment that Dr. Researcher actually reported valid results, based on sound scientific methodology. And that Mr. Businessman made no effort to interfere with conduct or reporting of the research. The story seems to have a happy ending: the drug goes through all the many developmental stages and becomes a remarkably successful treatment that helps to save many lives and relieve much suffering. However, because he actually believed in what he was doing and was willing to put his personal financial life on the line, Dr. Researcher is vilified as a professional pariah for conflict of interest. And, because Mr. Businessman actually had a return on his investment, he is vilified as a greedy wolf preying on the poor innocent lambs who happen to be ill. What is wrong with this picture??
Perhaps as the Director of Research of the Florida Heart Research Institute, a free-standing not-for-profit institution dedicated to stopping heart disease through research, education and prevention, I sit in an ideal position to have a balanced perspective on this situation. The research mission of the Institute involves initiating and developing new and innovative research projects in areas which do not yet or may never have an industry sponsor. However, we certainly recognize that industry has a very important role to play in the translation of scientific finding into clinical reality. It is certainly a path fraught with the potential for corruption and abuse. However, it is the recognition of and transparency regarding these threats, rather than the condemnation of the path itself that will result in the greatest benefit to patients.
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