To ensure that the information resulting from research is relevant to patients, the Patient‐Centered Outcomes Research Institute eschews the “traditional health research” paradigm, in which investigators drive all aspects of research, in favor of one in which patients assume the role of research partner. If we accept the premise that patient engagement can offer fresh perspectives that shape research in valuable ways, then at least two important sets of questions present themselves. First, how are patients being engaged—and how should they (...) be engaged? Second, which patients are being engaged—and which patients should be? This set has received relatively less attention, and the neglect is surprising, given that the “who” question is conceptually prior to the question of “how.”This article focuses attention on the “who” of patient engagement in research. First, we provide background on the rationale for patient engagement, underscoring the importance of ensuring the representativeness of engaged patients. Second, we present what little is known about patients engaged in PCORI‐funded research. Third, we identify and discuss the ethical implications of ways in which current practices of patient identification and recruitment may lead to a lack of representativeness. These practices include reliance on the well‐connected and well‐informed, reliance on patients who are not well trained or well‐informed, and reliance on patient advocacy organizations. Finally, we consider several strategies for addressing these pitfalls in order to maximize the positive goals of patient engagement. Patient engagement is intended to address the inability of researchers, funders, and others to fully represent patient views and priorities, but without sufficient attention, the patients selected for this role may still leave important gaps. (shrink)

Stem cells derived from pluripotent cells offer the hope of new treatments for diseases for which current therapy is inadequate. Clinical trials are essential in developing effective and safe stem cell therapies and fulfilling this promise. However, such clinical trials raise ethical issues that are more complex than those raised in clinical trials using drugs, cord blood stem cells, or adult stem cells. Several clinical trials are now being carried out with stem cells derived from pluripotent cells, and many more (...) can be expected in light of the rapid scientific progress in the field.Degenerative neurological diseases are desirable targets for stem cell clinical trials. The FDA has approved Phase 1 clinical trials of neural stem cell transplantation for Batten Disease, Pelizaeus-Merzbacher Disease, and spinal cord injury. In Parkinson Disease, stem cell transplantation could correct the primary pathophysiological defect — inadequate levels of the neurotransmitter dopamine. Current treatment is unsatisfactory in late-stage PD. (shrink)

This essay analyzes the concept of public trust in science and offers some guidance for ethicists, scientists, and policymakers who use this idea defend ethical rules or policies pertaining to the conduct of research. While the notion that public trusts science makes sense in the abstract, it may not be sufficiently focused to support the various rules and policies that authors have tried to derive from it, because the public is not a uniform body with a common set of interests. (...) Well-focused arguments that use public trust to support rules or policies for the conduct of research should specify (a) which public is being referred to (e.g. the general public or a specific public, such as a particular community or group); (b) what this public expects from scientists; (c) how the rule or policy will ensure that these expectations are met; and (d) why is it important to meet these expectations. (shrink)

Although the therapeutic misconception (TM) has been well described over a period of approximately 20 years, there has been disagreement about its implications for informed consent to research. In this paper we review some of the history and debate over the ethical implications of TM but also bring a new perspective to those debates. Drawing upon our experience of working in the context of translational research for rare childhood diseases such as Duchenne muscular dystrophy, we consider the ethical and legal (...) implications of the TM for parental consent to research. In this situation, it is potentially the parent who is vulnerable to TM. In our analysis we not only consider the context of informed consent for research but also the wider environment in which the value of research is promoted, more broadly through the media but also more specifically through the communication strategies of patient organizations. All dissemination about developments in research for health runs the risk of portraying an overly optimistic view of the promise of biotechnological solutions and has the potential to encourage a ‘collective’ TM. In this paper we consider the challenge that TM presents to parents as well as explore the ethical and legal responsibilities of researchers to ensure an appropriately informed consent: compatible with a hopeful disposition of parents who consent for the their children whilst avoiding a blind and misleading optimism. (shrink)

In March 2000, President William Clinton signed Executive Order 13,147, establishing the White House Commission on Complementary and Alternative Medicine, to develop public policy proposals geared toward maximizing “the benefits to Americans of complementary and alternative medicine.” Disconcertingly, the Commission's charge presumed the safety and efficacy of complementary and alternative medicine. In so doing, it placed the proverbial cart before the horse by setting the Commission on a mission to “address education and training of health care practitioners in CAM; [coordinate] (...) research to increase knowledge about CAM products; [provide] reliable and useful information on CAM to health care professions, and [provide] guidance on the appropriate access to and delivery of CAM.”The Commission's final report, issued in March 2002, similarly skirts the fundamental question of whether evidence exists that CAM interventions are safe or offer sufficient benefit to justify their proliferation. (shrink)

Human clinical research has become a huge economic enterprise (Morin et al. 2002; Noah 2002). Because the human subject at the center can be so easily marginalized, many commentators recommend spec...

Openness is one of the most important principles in scientifi c inquiry, but there are many good reasons for maintaining secrecy in research, ranging from the desire to protect priority, credit, and intellectual property, to the need to safeguard the privacy of research participants or minimize threats to national or international security. This article examines the clash between openness and secrecy in science in light of some recent developments in information technology, business, and politics, and makes some practical suggestions for (...) resolving confl icts between openness and secrecy. (shrink)

Post-9/11, concern about bioterrorism has transformed public health from unappreciated to a central component of national security. Within the War on Terror, bioterrorism preparedness has taken a back seat only to direct military action in terms of funding. Domestically, homelessness, joblessness, crime, education, and race relations are just a few of a litany of pressing issues requiring government attention. Even within the biomedical sciences and healthcare, issues surrounding the fact that more than 40 million Americans lack health insurance, the rising (...) cost of prescription medications, and the use of government funds for research using embryonic stem cells remain unresolved. Should we prioritize a hypothetical threat (bioterrorism), or existing conditions that have implications for identifiable individuals? Even more fundamentally, should we prioritize research aimed at defense from bioterrorism (or even terrorism in general) when there are so many pressing social problems that affect the U.S. population? (shrink)

I welcome the efforts described by Neal Dickert and his colleagues in “Partnering with Patients to Bridge Gaps in Consent for Acute Care Research” (Dickert et al. 2020). I am a long-time supporter...

Academic medical centers and drug manufacturers have traditionally occupied very distinct positions with regard to public trust. As collaborations among medical researchers and pharmaceutical companies expand, however, worries about the aggressive pursuit of profit that has tarnished the reputation of the pharmaceutical industry may be transferred to medical institutions and clinical investigators, suggesting to some that biomedical research is more about increasing profit than promoting public health. Consequently, when medical institutions forge research collaborations with industry they should be mindful of (...) the potential for these relationships to erode public confidence in the integrity of clinical research.Recent events have heightened concerns about the financing of clinical research. These include the widely publicized deaths of several research volunteers, scandals at the nation's largest funder of biomedical research, and evidence of fabricated research findings in prominent medical journals. These unfortunate events have prompted many to re-examine strategies for managing industry relationships and the financial conflicts of interest they may create. (shrink)

As everybody knows, advances in medicine and medical technology have brought enormous benefits to, and created vexing choices for, us all – choices that can, and occasionally do, test the very limits of thinking itself. As everyone also knows, we live in the age of consultants, i.e., of professional experts who are ready, willing, and able to give us advice on any and every conceivable question. One such consultant is the medical ethics consultant, or the medical ethicist who consults.Medical ethics (...) consultants involve themselves in just about every aspect of health care decision making. They help legislators and judges determine law, hospitals formulate policies, medical schools develop curricula, etc. In addition to educating physicians, nurses, and lawyers, amongst others, including medical, nursing, and law students, they participate in clinical decision making at the bedside. (shrink)

Social scientists have begun elucidating the variables that influence public trust in science, yet little is known about hype in biotechnology and its effects on public trust. Many scholars claim that hyping biotechnology results in a loss of public trust, and possibly public enthusiasm or support for science, because public expectations of the biotechnological promises will be unmet. We argue for the need for empirical research that examines the relationships between hype, public trust, and public enthusiasm/support. We discuss the complexities (...) in designing empirical studies that provide evidence for a causal link between hype, public trust, and public enthusiasm/support, but also illustrate how this may be remedied. Further empirical research on hype and public trust is needed in order to improve public communication of science and to design evidence-based education on the responsible conduct of research for scientists. We conclude that conceptual arguments made on hype and public trust must be nuanced to reflect our current understanding of this relationship. (shrink)

This article addresses the impact of the potential conflict between the roles of physicians who are both clinicians and researchers on the recruitment of persons into research trials. It has been proposedthat a physician breaches inter-role confidentiality when he or she uses information gathered in his or her clinical role to inform patients about trials for which they may be eligible and that clinician-researchers should adopt a model of preliminary consent to be approached about research prior to commencing a clinical (...) relationship. This article argues that even if we grant the legitimacy of inter-role confidentiality (which is open to question), there are circumstances in which other obligations physicians bear override the obligation of inter-role confidentiality. Moreover, it is argued that the practice of preliminary consent is morally suspect and that such consent cannot be deemed valid. The article concludes with a series of recommendations of ways in which the legitimate concern regarding the conflicting roles of clinician-researchers can be addressed in the recruitment stage of research. (shrink)

The government-sponsored Tuskegee syphilis study had a huge impact on U.S. research ethics and policy. Study investigators regarded subjects as “mere means” to their research ends, which led to a variety of ethical violations. Investigators used deception so that subjects would see participation as therapeutic — researchers promoted the therapeutic misconception because this advanced study objectives. The research would produce important information, and this justified lying to research subjects.Today we see this sort of intentional deception as unjustified no matter how (...) important a study might be. But what do we make of the claim that the syphilis study had value? As James Jones reported, its objectives were to test the prevailing views that syphilis affected blacks and whites differently, and that the disease was less harmful to blacks than whites. U.S. Public Health Service researchers and officials assumed the study had sufficient value to justify not only deceiving subjects, but also depriving them of safe and effective treatment. (shrink)

When scientific research collides with social values, science's right to self-governance becomes an issue of paramount concern. In this article, I develop an account of scientific autonomy within a framework of public oversight. I argue that scientific autonomy is justified because it promotes the progress of science, which benefits society, but that restrictions on autonomy can also be justified to prevent harm to people, society, or the environment, and to encourage beneficial research. I also distinguish between different ways of limiting (...) scientific autonomy, and I argue that government involvement in scientific decision-making should usually occur through policies that control the process of science, rather than policies that control the content of science. (shrink)

The use of foster children as subjects in the pediatric HIV/AIDS clinical trials has been the subject of media controversy, raising a range of ethical and social dimensions. Several unsettled issues and debates in research ethics underlie the controversy and the lack of consensus among professional researchers on these issues was neither adequately appreciated nor presented in media reports. These issues include (1) the tension between protecting subjects from research risk while allowing them access to the possible benefits of research; (...) (2) the blurring of the potentially conflicting roles of investigator and physician and the boundaries between research and therapy; (3) the adequacy of Institutional Review Board oversight; and (4) trust and the relationships among physicians, investigators and industry. The media controversy about the pediatric HIV/AIDS clinical trials can be seen as a means of “manufacturing mistrust” in health care, research and social services that have not always met the needs and expectations of the public. In an era of emerging infections, it is critical to the public’s health that people understand the role of rigorous and ethical research in the development of safe and effective care. Investigators, journalists and the public need to become knowledgeable about major ethical issues in the conduct of research in order to engage in dialogue about balancing research risks and benefits and to be able to distinguish fact from distortion in an era of multiple and rapid transmission of information. (shrink)

We commend Mergenthaler and colleagues for bringing the topic of patient recruitment and consent in intracranial electrophysiology research to the attention of the neuroethics community. Mergenthal...

Stories of rare disease advocacy can elicit strong but ambivalent responses. It’s moving to read about the heroic efforts of parents “becoming drug developers to find a cure for their children’s ra...