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be effective. This dichotomy stems from our imperfect knowledge of the pathophysiology of disease states, such as in the case with gram negative sepsis and shock. Another is the problem of a constantly changing target such as the case with the AIDS virus. Genentech and Amgen have been successful in bringing drugs to the marketplace, but even these companies have struggled to remain viable and maintain adequate cash flow to undertake research and development for the next generation of products. The hundreds, if not thousands, of smaller companies may not fare as well and it is safe to predict that only a small fraction will indeed find a successful product. Besides the discrepancy between the ability to make a compound and its clinical efficacy, there are the problems of corporate capitalization and effective drug development. The mergers of biotech concerns and the established pharmaceutical industry will go beyond improved capitalization and will bring more expertise in drug development and the regulatory approval process to this fledgling industry. But there are further impediments to success. Many of the products of biotechnology synthesis are proteins that are not orally active. A major area of research is going to be to convert the intravenously active compounds to ones with a facilitated means of delivery. Novel drug delivery systems to overcome the problems of lack of oral activity will be crucial. Carrier molecules, topical transport enhancers, and nasal absorption enhancers are but a few of the possible solutions to the drug delivery problem that considerably hampers the biotechnology field. Another approach has been the development of chemical molecules that have similar key structural elements that may permit the chemical compound to act like the protein molecule. If this is possible, we may find ourselves using the tools of biotechnology to enhance the drug discovery process through chemical synthesis. Despite the problems and the inherent limitations, the field of biotechnology will greatly increase the possibilities of compounds available for drug development and, in fact, development in many novel areas that have been very much lacking in therapies. With the initial cost being so great, the pressures for successful development are further heightened and thus the critical elements of the development process will need to be clear if we are not to repeat the mistakes of yesterday. For example, demonstrating the blood-clot lysing capabilities of recombinant tissue plasminogen activator (rTPA) and reversal of an acute MI in development was not enough for commercial viability of the product. Genentech persisted and undertook to perform a comparative study of rTPA with streptokinase to demonstrate the superiority of the rTPA product. The superiority of the rTPA combined with an aggressive marketing strategy permitted Genentech to dominate the thrombolytic market. In the development process of biotechnology products, their value and place in the therapeutic armamentarium may be as important as the demonstration of efficacy in a pivotal trial. The pharmacoeconomic impact of new therapiesthe very great expense of the biotechnology-derived drugs, the benefits |
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