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Leading With Science
Leading With Science
The voice of science has been remarkably faint over the past four years. What is science? Why should we trust in science? Who is leading us in science? These are all questions more answerable in the past, and hopefully in the future, than they appear to be today. This is somewhat surprising considering the great discoveries on our immediate horizon and the promise of science for our future. But the combination of the bursting of the technology bubble at the end of the past century, the mixing of science and religion, and the leadership pause in anticipation of breakthroughs associated with genomics have all contributed to several years of scientific silence.
That said, science is quietly rediscovering its voice as clinical decision-making is increasingly grounded in treatment guidelines and consensus statements termed evidence-based medicine. Evidence-based medicine was defined in 1996 by Dr. David Sackett as "the conscientious, explicit, and judicious use of the current best evidence in making decisions about the care of individual patients. The practice of evidence-based medicine means integrating individual clinical expertise with the best available external clinical evidence from systematic research."1
Almost all evidence-based medicine flows from clinical research, or, scientific trials involving human volunteers. Williams Osler, a critical figure in the history of medicine, instructed students to "Observe, record, tabulate, communicate."2 The process to do just that, and to do it in a manner that is reproducible and verifiable, has been slowly evolving over the past 250 years. In 1794, the English physician, William Heberden said, "I please myself with thinking that the method of teaching the art of healing is becoming, everyday, more conformable to what reason and nature require; that the errors introduced by superstition and false philosophy are gradually retreating...."2 Out of the thinking of the day evolved the clinical trial, a carefully planned experiment designed to expose the benefits and risks of a specific intervention in humans.3
While science undeniably has progressed, the process we rely on to separate fact from fiction, the use of randomized, controlled trials that compare a new treatment to a standard treatment or placebo in a matched population, dates back to the 18th century. In 1754, James Lind, a Scottish naval surgeon, studied 12 sailors with scurvy to see if they could be healed by diet. Two were given a quart of cider a day; two sweet oil of vitriol (ether?); two vinegar; two seawater; two an herb paste; and two were given oranges and lemons. Within six days, the fruit's effectiveness was obvious. It took another 40 years for the treatment to effectively eliminate scurvy from British ships, and another century before vitamin C was identified as the critical missing nutrient.4
Science, traditionally, is directed to where there is a need. The clinical trial process approaches patients with need from three different vantage points. Prevention studies examine treatments or behavioral changes that might prevent illness. Diagnostic studies seek new or improved methods of identifying an illness. Treatment studies test a chemical agent for both safety and effectiveness.
Clinical trials require cooperation between government, academia, industry, health professionals and patients, with a clear understanding and commitment to ethical care. Safety should always be a primary concern. A critical question to answer prior to proceeding, "Is the health problem at issue worth any safety risks involved in developing a treatment?" If the answer is yes, a systematic approach has evolved to limit the inherent risk of clinical trials by staging the study into four phases.5,6 First, pre-clinical safety assessment, or testing the treatment in animals prior to human studies. Second, pre-approval safety assessment involving a small number of healthy volunteers. Third, safety assessment in large numbers of patients, with FDA reviews. Fourth, ongoing surveillance, post-FDA approval, for side effects.
The success of science, in the global age of health consumer empowerment, is based on broad public confidence in the integrity and utility of scientific research. What is medical mcience? Medical science is a well-tested, cooperative process of observation, testing and communication, designed to empower patients and caregivers with the best current knowledge to maintain health and prevent and treat disease. Why should we trust science? We should trust science because the process has a strong ethical grounding and a proven process that is transparent and verifiable. Is science perfect? By no means. What we believe we know today may be altered by a new discovery tomorrow. There is much that remains to be learned. That said, science has delivered an increase in the quantity and quality of our lives. Fifty years
ago our physicians barely understood the scientific underpinnings of heart disease, stroke, depression, arthritis, Parkinson's disease, Alzheimer's disease, or the role of cigarettes, diet and exercise in health. And today, this knowledge is commonplace in large segments of our general population. Science created and transferred that knowledge. And with the growing alliance between health consumers, caregivers and scientists, science, with a renewed and stronger voice, could, in the next ten years, deliver quality of life improvement that would make us all proud. William Osler said, "The future belongs to science."2 And indeed it does.
Until next week, for Health Politics, I'm Dr. Mike Magee.
References
1.McNeill WH. Plaques and Peoples. New York, NY: Doubleday; 1989:237.
2.Krentzman BZ. Mayo Clinic Explanation of Clinical Trials.
3.Sackett DL et al. Evidence based medicine: What it is and what it isn't. BMJ: 312, 71-72, 1996.
October 1, 2003
