Ethics and innovation in medicine.

How should one think about innovation in medicine and surgery? Increasingly, the answer to this question has involved reference to what might be called the regulatory ethics paradigm (REP). The regulatory ethics paradigm holds that deviations from standard care involve a degree or kind of experimentation that requires the application of a set of procedures designed to assure the protection of the rights and welfare of the subjects of research. In REP, innovative treatments are regarded as questionable until they are framed in a research protocol with formal mechanisms of informed consent. The protocol must be reviewed and approved by an Institutional Review Board (IRB) or equivalent. The regulatory ethics paradigm in eVect imposes the condition that clinical innovations be conducted according to scientific research methodologies. It creates the presumption that without review by an IRB, innovation cannot be conducted in an ethically defensible fashion. The regulatory ethics paradigm also requires the preparation of investigational protocols according to sound evidentiary and methodological standards. In so doing, it creates a presumption that innovations that are not rigorously validated are ethically dubious. These assumptions have deep roots. The Belmont Report, for example, articulated the orienting intuition that “radically new procedures . . . should . . . be made the object of formal research at an early stage [emphasis added] in order to determine whether they are safe and eVective”. The Belmont Report takes the view that formal research to establish safety and eYcacy of new interventions is usually feasible at an early stage in the development of a novel intervention. It thus establishes a bias that innovative treatments be conducted and evaluated under a research protocol that has passed muster at an IRB review. Given the historical and social context of the movement to protect the subjects of research, it is not surprising that REP tends to assimilate activities that share a family resemblance with scientific research. Clinical experimentation understandably was regarded as suYciently research-like that its participants should be aVorded the protections of REP. The Belmont Report, however, oVered no deep analysis of experimentation in the context of patient care or the development of novel treatments. The regulatory ethics paradigm tends to regard innovative treatment simply as a departure from standard and accepted treatment alternatives. It thus overlooks those clinical situations or fields of medicine in which accepted treatments are ineVective or burdensome. To be sure, some of the core documents forming REP have included an awareness of the need for using unproven procedures, but there has not been much elaboration of the boundaries among research, innovation, and practice. The recent revision of the Declaration of Helsinki importantly recognises that in the treatment of a patient, where proven prophylactic, diagnostic and therapeutic methods do not exist or have been ineffective, the physician, with informed consent from the patient, must be free to use unproven or new prophylactic, diagnostic and therapeutic measures, if in the physician’s judgment they oVer hope of saving life, re-establishing health or alleviating suffering (paragraph 30). Such statements appropriately point to a land outside the pale of REP. This borderland, however, is largely underdeveloped regarding the procedures and standards that should apply. Given the widespread acceptance of REP, bioethicists have not been inclined to examine closely the actual processes by which clinical innovations are developed and the initial conditions prerequisite for the application of the paradigm. This omission is important, because it has permitted the extension of REP into the domain of clinical innovation without an adequate assessment of the advantages and disadvantages of such an expansion. The formal scientific research that REP envisions simply assumes that a degree of standardisation of device, technique, or procedure exists. Before a scientific research protocol is feasible, however, a developmental process must occur. In some cases, a significant and complex developmental eVort is required to bring a procedure to the point at which formal research is possible. In the case of complex surgery, such as coronary artery bypass graft surgery (CABG), the procedures evolved against a background of no or very poor therapeutic options, underwent technical alterations as they were employed, and were dependent upon analogous advances in imaging, anaesthesia, and postoperative care. The developmental eVort involves, among other matters, identifying or clarifying the types of patients who might benefit most from the new intervention, standardising the process or procedures used, and resolving technical problems in Journal of Medical Ethics 2001;27:295–296