In an apparent attempt to determine whether the American taxpayer is getting fair benefits from research sponsored by the federal government, the Joint Economic Committee of the U.S. Senate has been considering this question. Historically, basic research has been funded by the NIH and various philanthropic foundations to discover new concepts and mechanisms of bodily function, in addition to training scientists.
The role of industry has been to apply the basic research findings to specific treatments or prevention of disease. This is the appropriate manner in which to proceed. The industry cannot afford to conduct sufficient basic research on new complicated biological processes in addition to discovering new drugs or vaccines. The government does not have the money, time, or required number of experts to discover and develop new drugs.
The process that plays out in real life involves the focus of pharmaceutical industry scientists on desirable biological targets that can be identified in disease states, and to set up the program to discover specific treatments that will show efficacy in human disease.
The compounds that are developed successfully become drugs on which the company holds patents. In this manner, the huge cost of discovering and developing a new drug (estimated at $800 million plus over a period of some 10 years) as noted above can be recouped by the founding company since no competitors can sell the product as long as the patent is in force. Without such a system in place, drug companies simply could not afford to bring new prescription drugs to the market.
In the course of reviewing the matter, the Joint Economic Committee examined a list of 21 major drugs, which was put together apparently as an example of drug products that might justify royalty to the government. One of these agents, captopril (trade name Capoten), was discovered and developed by E.R. Squibb & Sons in the 1970s. At that time, one of Squibb’s academic consultants, Professor Sir John Vane of the Royal College of Surgeons in London brought the idea of opening a new pathway to treat the so-called essential hypertension by inhibiting an enzyme known as the Angiotensin Converting Enzyme (ACE). This biochemical system was certainly known at that time but, in Squibb’s experience in the field of hypertension treatment, was not generally thought to play a major role in the common form of the disease, then known as “essential hypertension.” The company decided to gamble on finding a treatment that was not used at the time and that would be proprietary to the company. Professor Vane (Nobel laureate in medicine in 1982) had discovered a peptide in snake venom that was a potent inhibitor of ACE. Squibb decided to pursue the approach he espoused, resulting in the development of a unique drug for the treatment of this very prevalent and serious disease.
In the first phase of their research, Squibb tested a short-chain peptide isolated from the venom of the viper Bothrops jararaca, with which Vane was working in the laboratory, in human volunteers and showed that it did, indeed, inhibit the conversion of angiotensin I to angiotensin II after intravenous injection. The peptide was also shown to reduce patients’ blood pressure when injected. Since the vast majority of peptides cannot be absorbed from the GI (gastrointestinal) tract, Squibb scientists set out to prepare a nonpeptide compound that could be used orally and manufactured at acceptable cost. The design of a true peptidomimetic that became orally active had not been accomplished at that time. Squibb then carried out a full-blown clinical program on a worldwide basis, which led to FDA approval of Squibb’s drug Capoten (captopril), an ACE inhibitor. Mark also marketed an ACE inhibitor in the same period. This work opened a new area of research that has resulted in a class of new drugs that share this mechanism of action for use as antihypertensive drugs.
In the minds of pharmaceutical researchers and, hopefully, the public at large, the above example illustrates the unique role of pharmaceutical companies in making good use of basic research to discover new treatments for serious and severe diseases. The colossal costs to discover and develop a new drug could not be borne unless the companies knew that, if their gamble worked (which is not the case in the majority of situations), they would be assured of a good financial return (ROI) for their shareholders. This system has served the country well in many fields of endeavor, in and out of the drug arena, and should be retained as such.
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