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Posted on May 31, 2019 at 5:27 PM
This blog post made available by the American Journal of Bioethics. You can read this editorial and accompanying articles here.

by George J. Agich, Ph.D.

The broad sweep of medicine’s history since the middle of the 20th century has been one of improvement in the care of patients, but that progress has not been uniform. The very processes that lead to important advances in medical care also fail; some of these failures are catastrophic. Patient welfare and improvement in health or functional status comprise the central goal of medical care, yet harm to patients is an ever-present risk, especially in the case of employing novel interventions. Of the processes that have improved medical care, formal clinical research is best known in bioethics circles since it has the most robust mechanisms for the protection of subjects. Other processes involve modifications to existing treatments or wholly new interventions or approaches within the course of patient care. Here the mechanisms for ethical and safety oversight are less well defined.

While medical centers tout their offering of innovative diagnostic and treatment procedures for patients, their motives in doing so are frequently driven by goals beyond patient welfare: competition, profit, and self-aggrandizement. These goals complicate analysis of innovative treatments. So, the questions of “Should innovation in medicine be controlled or regulated? If so, how and what standards should guide oversight?” are not only important, they are complicated by a broad set of competing values and interests beyond patient welfare. Yet even before one begins to answer these questions, one needs to attain some clarity about the very nature of innovation: “What is it? How should innovation in medicine be conceptualized?”

Clarity of definition is not easy to attain since the contexts and types of alterations in diagnostic and treatment approaches are so varied across the many domains of clinical practice. Innovation and the practice of innovation need to be understood as a multifaceted set of activities that share, at best, a family resemblance, but not a universal set of features. In reflecting on the ethical challenges, the failure to refine the focus of attention to the specific and sometimes quite narrow context of clinical practice can be the Achilles heel of bioethical discussions. Generalized conclusions based on abstract principles of ethics or idealized mechanisms of oversight provide little help for those practitioners earnestly striving to responsibly improve patient outcomes.

The article in this issue, Jake Earl’s “Innovative Practice, Clinical Research, and the Ethical Advancement of Medicine”,and the peer commentaries that follow address some of these questions. As a set, they touch on a cluster of important issues that involve not only bioethical concerns, but also philosophical, historical, and social policy, and institutional concerns that are integral to sound treatments of innovation in medical care. There is no dispute about whether innovation should be undertaken responsibly; rather, differences emerge about how to conceptualize innovation in medicine and regarding what mechanisms and standards should be normative. At bottom are different visions about how to assure a responsible practice of innovation in medicine.

In the target article, Jake Earl argues against a proscriptive approach to the problem of innovation and critically examines some proposals to restrict innovations to the context of formal research studies. Instead of such arbitrary restriction, he endorses “a permissive approach to management and oversight” by professional societies, health care organizations, and governmental entities. He asserts that there is an emerging consensus among professional societies that there is a research standard for ethical medical practice and stipulates the requirement that clinicians who propose innovative interventions also “justify engaging in innovative practice rather than clinical research”. This approach is permissive in establishing a path for oversight of innovation outside the well-known mechanisms of formal research review—but at the same time acknowledging their importance in their own context—while leaving open the form that this oversight should take. He thus defends a permissive dual-deviation standard and argues that because innovative practice deviates from both the idealized expert-consensus standard and the research standard, both deviations should be justified in a prospective review.

Earl is particularly concerned that enthusiasm for a novel intervention can cause a widespread, runaway diffusion. The case of percutaneous coronary intervention (PCI) for stable angina illustrates this well. PCI was widely adopted into practice even though subsequent research showed it to be no better than placebo for chest pain, and the current runaway diffusion in the use of autologous stem cells for a broad range of orthopedic conditions in spite of a lack of evidence supporting this use has resulted from a permissive regulatory environment. Here, again profit appears to be a driving force that shoulders out the commitment to increase clinical knowledge by scientific methods. Hunbyrd and Wynia suggest that when there is a strong financial interest in providing an innovative treatment, greater oversight is required.

Some of the most likely mechanisms for overseeing the introduction of innovative procedures into clinical practice, such as institutional and peer professional review, have failed, some in obvious ways. Macchiarrini’s work on transplanting a tissue-engineered trachea led to a series of monumental failures. Of the eight patients transplanted, seven died. Scientific misconduct was involved as several institutions failed to oversee this “star” clinician. Social oversight, however, has failed to constrain the proliferation of various kinds of stem-cell-based “treatments.” One way around regulatory oversight has been to move these interventions into lax jurisdictions, promoting an industry of medical tourism for dubious interventions. Holzer and Mastroleo point out that this industry has crowded out support for sound clinical research in Latin America. Again, financial interests seem to trump the primary duty to promote patient welfare.

Because innovations in medical devices and procedures can undergo refinement or modification in ways that make a rigid insistence on formal research trials at an early stage premature and counterproductive, there are still questions about “How, under what conditions, and what form should oversight, of innovations in medical care occur?” Chapman and colleagues point out that the current Food and Drug Administration (FDA) policy of expanded access (EA) does meet Earl’s dual-deviation standard, but that so-called right to treat (RRT) advocated by libertarian groups does not since it does not require a third-party review.

Some authors contrariwise contend or imply that all innovation in medicine should be conducted as a matter of research and so should be subject to the review by institutional review boards (IRBs) charged with protecting the rights and welfare of research subjects. There are several reasons why this is infeasible. First, innovations occur in the very midst of patient care as physicians using their professional judgment and experience to dynamically shape treatments to respond to the needs of patients. Surgical and other procedural interventions also are embedded in practice settings that can be modified to adjust to the care needs of individual patients. Similarly, patients who do not match the admission criteria in a successful randomized clinical trial may in practice benefit from the interventions when they become available even though their clinical characteristics differ from the study patient population. When interventions are used for these patients, the interventions are conceptually an innovation in clinical care. Such “innovations” are ubiquitous in everyday medical practice and any thorough reflection on innovation should accommodate this practice. The problem here is one of definition. For the practice of surgery this is especially difficult. Identifying or defining innovative practices is a problem that is at least as challenging as whether and how they should be reviewed. Besides these questions, one must be open to ask, as do Kahn and Mastroianni, once undertaken, when should special oversight of an emerging medical technologies or clinical practices end?

Calling for institutional, regulatory, professional oversight is justified when we are clear about the practices that need oversight, but only if we recognize that such oversight is not a panacea. Formal review can fail just as does individual or peer professional judgment when financial, prestige, or other interests overshadow the commitment to patient welfare—even when interests competing with patient welfare are removed. Conceptually sound policies that establish oversight of innovative interventions will have to be sensitive to the context of practice. In the field of procedural surgical innovation, Das and McKneally report a nuanced policy developed in Toronto that charges the surgeon-in-chief and an innovation committee with the responsibility to oversee innovations in surgical practice, rather than an IRB or research ethics committee. Since innovation in surgical procedures is often iterative in the sense that each case is an “informative test for the next”, oversight in this context is best assured by a policy that is driven by a defined goal rather than a protocol.

Although the benefits of advances in medical care are undeniable, they do not follow predictable paths. New treatments emerge in an uneven and sometimes serendipitous fashion. Progress is not uniform or certain. Failure and harm to patients and the health care system are constant dangers and occur with a frequency that cannot be responsibly ignored. The conclusion, tentative though it be, is that while unbridled enthusiasm about innovation in medicine is unwarranted, so is a conservative acceptance of the status quo.

Taken as a set, the article and commentaries lead to the conclusion that responsibly conducted innovation in medicine will have to be sensitive to the specific clinical and institutional contexts in which it occurs; it will need to address the robustness of administration, collegial, and social support, and the adequacy of the regulatory framework for fostering sound ethical oversight of innovation from its first appearance through the process of development and deployment.

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