In October 1981, Dale Lot, a thirty–seven–year–old veteran working as a fireman in Florida, was forced to quit because of severe congestive heart failure. His case was terminal, doctors told him, and transplant surgery was impossible. The artificial heart was his only hope. He thus began a desperate – and highly publicized – campaign to persuade the University of Utah to let him be their first “human guinea pig.” His appeal, backed by the fire fighters' union and orchestrated by a flamboyant trial attorney, included repeated attacks on the “murdering bureaucrats” at Utah who were blocking his request, a barrage of publicity, and finally a public telegram to the White House. The University of Utah was already trying to broaden patient eligibility, and Lot's campaign probably hastened their efforts. But Lot finally abandoned his personal quest for an artificial heart when it became clear that he would be refused on the grounds that his heart condition was accompanied by diabetes and hypertension, and was linked to long–term alcoholism.

Dr. Barney Clark, the man finally selected as the first recipient, was, by most accounts at the time, an ideal candidate. He was plucky, and he had strong support from his wife and children. He seemed, in the words of a University of Utah social worker, “a classic choice.”

To understand medicine's present predicaments, it is useful to begin with a brief overview of their historical context. The character of medical science has changed dramatically since the turn of the century as a result of increasing public and private investment and expanding scientific capabilities. These changes, in turn, have fundamentally transformed medicine's role in American social and political life. They have created new moral rights on the part of society, and new responsibilities on the part of biomedical science.

There are also countervailing trends. The more esoteric medical advances have become, the more difficult it is for lay citizens and their elected representatives to exercise meaningful influence over policy choices. At the federal level, biomedical and regulatory priorities are increasingly permeated with the government's economic agenda for promoting private technological innovation and development. Opportunities for direct public participation in governmental decisionmaking have been curtailed, and public confidence in traditional forms of representative government has declined sharply in recent decades. Together, these developments lie at the heart of medicine's present dilemmas and represent the central challenge to their resolution.

The nature of modern medical science

Medicine's evolution during this century has altered not only its internal character but also its relation to the larger society. Both types of change have left modern medicine more beholden than ever to the society that supports it and that is affected by its risks and benefits.

Not so long ago, it would have seemed obvious where medical science was headed. Medicine's historic contributions to human health are legendary – vaccines, insulin, anesthesia, electronic heart pacemakers, the heart–lung machine, and many others. Entire diseases, such as smallpox and polio, have been eradicated, or nearly so. Such impressive achievements gave no reason to question clinical progress; indeed, they fueled expectations for still greater accomplishments in the future.

In many ways, medicine has met those expectations, even surpassed them. Modern medicine's technical capabilities are truly extraordinary. Many major organs can now be transplanted. Mechanical devices can partially or substantially replace a person's failing joints, heart, lungs, and kidneys, and there are synthetic substitutes for blood, veins, and skin. Machinery can sustain bodily functions after vital signs have ceased. Researchers are working on developing fully implantable artificial hearts, lungs, eyes, and bladders, and are experimenting with human brain transplants. Through gene splicing, scientists can modify the genetic makeup of living organisms, literally creating new forms of life with desired traits in the laboratory. We are poised on the threshold of applying these techniques to humans – human genetic engineering.

But amid these spectacular accomplishments are new worries and concerns, troubling signs that all is not well in the house of medicine. One of the most visible is the issue of costs. The United States now spends over I billion per day on medical care.

No recent scientific accomplishment has given rise to such wildly enthusiastic expectations – and apocalyptic warnings – as genetic engineering. Emerging from an esoteric area of molecular biology, genetic engineering – known in its early years mainly as recombinant DNA research and, now, as biotechnology or simply genesplicing – has opened up possibilities once relegated to science fiction. These include an essentially unlimited supply of previously scarce vaccines and hormones; the creation of protein–rich foodstuffs from waste products; plentiful new stores of “clean” energy from organic “biomass”; plants bioengineered to be self–fertilizing; animals genetically programmed with the traits of other species – the list is virtually endless. The ultimate and most controversial achievement, of course, is human genetic engineering – actually altering our own genetic makeup.

The implications boggle the mind. Companies of all types are rushing to add specialized new facilities and personnel, and industry analysts spare few superlatives in describing biotechnology's prospects. According to one investment analyst, “We are sitting at the edge of a technological breakthrough that could be as important as electricity, splitting the atom, or going back to the invention of the wheel or discovery of fire.” Noted science writer Lewis Thomas called the current biological revolution “unquestionably the greatest upheaval of biology and medicine ever.”

Even its critics describe gene–splicing in revolutionary terms.

In 1,600 cases scattered across America, people who got swine flu shots in 1976 have sued the United States Government charging that the shots caused Guillain—Barré and other injuries. Total damages claimed exceeded 2.2 billion. More than a decade later, nearly thirty of these suits were still pending in the courts. Of those that had come to trial, the government, represented by the Justice Department, won in more than eight out of ten cases.

DES victims have also turned to the courts for relief, although only a handful have received awards of any magnitude. Many DES daughters have been unable to sue because they cannot identify the particular brand of DES their mothers took. Others have been barred because their injuries came to light after their state's statute of limitations had expired. Those not blocked by legal restrictions face the time, expense, and trauma of bitter court battles, and the likelihood of lengthy appeals by manufacturers. Eli Lilly, the largest manufacturer of DES, has already spent several million dollars fighting DES lawsuits, with no end yet in sight. Although somewhere between 500,000 and six million people were exposed to DES, only about 1,000 DES suits had been filed in the United States as of 1987.

Amid periodic insurance “crises” and publicity aimed at limiting manufacturers' liability, the plight of injured victims often gets lost.

In February 1938, a brief letter appeared in the British scientific journal Nature announcing the synthesis of a nonsteroidal compound with the properties of natural estrogen. The report was by a British scientist–physician, Sir Edward Charles Dodds of the Courtald Institute of Biochemistry in London, and colleagues at Oxford. The researchers named their new compound “stilboestrol”; it later came to be called diethylstilbestrol, or simply DES.

The announcement ran scarcely over a page, yet it sent ripples of excitement throughout the medical community. Here was a substance that could be used instead of the scarce and expensive natural estrogens in treating a wide range of problems thought to be related to imbalances in sex hormones. Clinical investigators around the world lost no time in launching experiments on patients, and most raved about the results. One researcher, speaking at a national medical meeting in the United States in 1939, called DES “the most valuable addition to our therapy in recent years.” A noted gynecologist declared that it had “tremendous clinical possibilities.” By 1940, the response to DES and other synthetic estrogens had been so favorable that Dodds observed that “their immediate practical value is now established.” But he also had grander visions. “It is perhaps not too much to hope,” he mused in 1940, “that just as aspirin marked the beginning of a new era in medicine, so these compounds may be the first steps along a road leading to great advances in the treatment of disease.”;

Medical innovation directly and intimately affects whether and how we live, and when and how we die. Research has unlocked technical capabilities that defy the imagination, and will continue to do so. With genetic engineering, scientists may leave their mark on the genetic heritage of the entire planet. Yet, as the case studies in this book document, we who use medicine's innovations, who bear their risks, and who, both as taxpayers and as consumers, pay for the products and the research that made them possible, have little if anything to say about the nature and pace of medical progress.

This book argues for an end to that exclusion; this chapter suggests the kinds of changes that might bring it about. It draws together many of the themes and issues that have been discussed throughout the book, looking at the lessons that can be learned and how they apply to present and future policies in the United States. These lessons become all the more urgent in light of medicine's deepening dilemmas and the failure of present policies to resolve them.

It is perhaps useful to stress again, as we did at the book's outset, that the four case studies were chosen to illuminate some of the major dilemmas posed by contemporary medicine. Although questions of costs, risks, efficacy, and equity are clearly of widespread concern, we do not intend to suggest that all medical innovations will encounter exactly the same types of problems as occurred in the four cases.