Clinical Trials and the Common Good
Testing Innovative Therapies Is More than a Private Affair
It’s a drama that plays out again and again wherever cutting-edge science meets mortal disease. First, researchers test a therapy that works miracles in animals. Expectant patients flock to those researchers. But then regulatory authorities and ethics committees prevent the researchers from offering the new therapy because of concerns about safety or the science behind the animal study.
Some critics view this as a perversion of medical ethics. What happens between dying patients and their physicians, according to these critics, belongs to them and them alone. They further argue that oversight and ethics are now impeding medical progress. We think these critics are wrong on both counts.
Clinical research is not a private act. It is, instead, a public activity. For one, it is aimed primarily at producing a public good: the knowledge communities require to address their unmet health needs. For another, clinical trials are much larger than private transactions between consenting patients and researchers. The development of new therapies is, in the end, a group endeavor: taxpayers support basic research, companies fund trials, academic medical centers provide the space and equipment, and scientists conduct the research. The true measure of a clinical trial’s worth is not whether it provides dying patients access to unproven medications, but rather, whether it produces a bountiful yield of knowledge that empowers future healthcare providers.
Trials of novel interventions often occur at the vanguard of science, and each stage in the process of translating basic research into clinically useful interventions depends critically on the quality of the information generated at prior stages. As a result, physicians and patients will often have a difficult time distinguishing strong studies from weaker ones. Physicians and patient-volunteers thus lack the ability to reward producers of strong studies by joining their trials and avoiding studies of lesser scientific quality. Oversight structures are necessary to ensure that studies are rigorously designed and that they will pass on valuable knowledge to the next link in the chain of discovery. They also help to ensure that participants, physicians, researchers, and investors can pursue their individual interests without compromising the social mission of the research enterprise.
Clinical trials that are unsafe, or that have a poor scientific justification, pose several threats to the kinds of cooperation needed to transform hard-won advances in basic science into improved care at the bedside. A string of disappointing clinical trials can lead sponsors and scientists to prematurely withdraw support from an otherwise promising area. Unexpected and/or mismanaged safety issues can sully the standing of an entire field and interrupt recruitment of talented researchers and investment from academic research centers.
Poorly justified or unsafe clinical trials also threaten a misallocation of scarce social resources. Patient-volunteers, for instance, are in short supply for many types of research, and fewer than 60 percent of National Cancer Institute-funded clinical trials are able to recruit enough volunteers to complete the study. It is a legitimate ethical concern if scientifically unsound studies draw volunteers away from studies that have greater merit. Another way that trials place demands on scarce resources is through personnel: trials require highly specialized medical expertise. Medical scientists who commit their expertise to weak trials are less able to contribute to other, more worthy research endeavors.
Unfortunately, medical research has a long way to go in terms of strengthening the scientific justification for testing novel therapies like stem cells, vaccines, or approaches involving gene transfer. Animal studies are often poorly designed, executed, and reported. In some cases, interventions are introduced into humans in ways that deviate from what was tested in animals. One recent study showed that preclinical researchers often do not publish all their animal studies, leading to significant overestimates of a drug’s effectiveness. It is not at all clear that oversight bodies and ethics committees, which focus on the willingness of researchers and patient-volunteers to undertake a study, do an adequate job promoting the scientific quality of clinical studies.
Policy proposals that would seek to loosen scientific standards so that patients can more easily access unproven therapies miss the point of research. To preserve the power of clinical research as an engine for scientific advancement, we must recognize that successful scientific innovation requires the sustained cooperation of myriad stakeholders and that the knowledge that results from this extended collaboration represents an important social good.
Jonathan Kimmelman is Associate Professor, Biomedical Ethics Unit and Department of Social Studies and Medicine at McGill University. Alex John London is Associate Professor of Philosophy and Director of the Center for the Advancement of Applied Ethics and Political Philosophy at Carnegie Mellon University
This article is based on a commentary appearing in the May 14, 2010 issue of Science.
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