Should innovative therapy occur only within a research paradigm and under institutional review board oversight? The health risks from current human embryonic stem cell clinical applications have raised again a fundamental question addressed first in papers submitted to inform the writing of the Belmont Report. Revisiting the thinking underlying the Belmont Report, together with examining changed circumstances since then, leads to a new model for overseeing innovative therapy based on its unique risks and context, important changes since the Belmont Report, (...) and new opportunities for addressing risks through safety and quality systems in health care. (shrink)

Innovative therapy is the name we give to novel medical interventions, radically different from the standard of care, provided in order to benefit a patient, rather than to acquire new knowledge. They are paradigmshifting, not incremental, responses to serious patient problems that standard medical care inadequately addresses. Innovative therapies are often devised by clinicians, not basic science researchers; they do not follow the linear model of basic research, to translation, to clinical research, to application. Instead, they come from thinking backwards (...) from the patient’s circumstances, and forward from deep knowledge of how the body functions, to challenge the limits of current mechanisms for effecting cures. (shrink)

To be a functional alternative to government regulation, self-regulation of science must be credible to both scientists and the public, accountable, ethical, and effective. According to some, serious problems continue in research ethics in the United States despite a rich history of proposed self-regulatory standards and oversight devices. Successful efforts at self-regulation in stem cell research contrast with unsuccessful efforts in research ethics, particularly conflicts of interest. Part of the cause for a lack of success in self-regulation is fragmented, disconnected (...) oversight, and failure to embody genuine scientific and public consensus. To be accountable, credible and effective, self-regulation must be inclusive and multidisciplinary, publicly engaged, sufficiently disinterested, operationally integrated with institutional goals, and must implement a genuine consensus among scientists and the public. The mechanisms of self-regulation must be sufficiently broad in their oversight, and interconnected with other institutional forces and actors, that they do not create fragmented solutions. (shrink)

Key ethical issues arise in association with the conduct of stem cell research by research institutions in the United States. These ethical issues, summarized in detail, receive no adequate translation into federal laws or regulations, also described in this article. U.S. Federal policy takes a passive approach to these ethical issues, translating them simply into limitations on taxpayer funding, and foregoes scientific and ethical leadership while protecting intellectual property interests through a laissez faire approach to stem cell patents and licenses. (...) Those patents and licenses, far from being scientifically and ethically neutral in effect, virtually prohibit commercially sponsored research that could otherwise be a realistic alternative to the federal funding gap. The lack of federal funding and related data-sharing principles, combined with the effect of U.S. patent policy, the lack of key agency guidance, and the proliferation of divergent state laws arising from the lack of Federal leadership, significantly impede ethical stem cell research in the United States, without coherently supporting any consensus ethical vision. Research institutions must themselves implement steps, described in the article, to integrate addressing ethical review with the many legal compliance issues U.S. federal and state laws create. (shrink)