Clayton EW, Smith M, Fullerton SM et al.; eMERGE Consortium Consent and Community Consultation Working Group. Address ethical, legal and social issues in real time within the Electronic Medical Records and Genomics (eMERGE) consortium. Genet Med 2010;12:616â620. While translational research for drug development can provide patients with valuable therapeutic resources, it is not without risk, especially in early-stage studies that present the highest degree of uncertainty. With the extraordinary development of biomedical technologies, an increasing number of innovative products based on human cells and gene therapy are being tested and used as medicines. Their use on humans poses several challenges. The first and most important principle of the Nuremberg Code, informed consent, has become the cornerstone of the ethical practice of medicine and human research. In research and clinical settings, the goal is a relationship of information exchange and trust between physician and patient. Translational ethics, like translational research, uses research codes for the protection of human subjects as standards of behavior for clinical practice. Other important concepts of translational ethics include autonomy and the ability to withdraw from treatment or research protocol. There are also important forms of policy-making that are rarely codified in specific documents or by certain organizations.
At the societal level, for example, investments in genomics research, including the allocation of research funds and capital investments, can have a significant impact on genomics research and its application.21 Federal granting agency initiatives, such as the NHGRI`s launch of a research program to evaluate the outcomes of genomic sequencing in health care,22 contribute significantly to the evidence that decision-makers need to be available. Pharmaceutical companies, biotech industries and private investors are influencing how genomics is transferred from the lab to potential healthcare applications. By deciding which tests and procedures to reimburse at which level, healthcare payers influence both investment decisions and clinical implementation potential, thereby influencing the translational process. The doctrine of informed consent is to inform the patient about (1) the diagnosis for which the intervention is proposed, (2) the recommended intervention with the associated risks and benefits, (3) the expected outcomes or prognosis if no intervention is attempted, and (4) all significant alternatives and associated risks and benefits. In this thesis, we discuss some ethical issues related to translational research in gene and cell therapy. We focus on preliminary studies that analyze the regulatory approach of Europe and the United States. We report on the current recommendations and guidelines of international scientific societies as well as European and American regulatory authorities. ELSI research offers a range of methodological approaches that provide normative analysis and empirical data that influence the policy-making process.
This thesis addresses all phases of the policy-making process in a wide range of policy-making sites. In addition, the ELSI research community created by the NHGRI ELSI research commitment provides expertise as a participant in advisory and policy bodies, as well as in expert testimony. Our experience in finding ways to increase the policy relevance of our work through CEER has been incorporated into both innovative dissemination approaches (Table 1) and recommendations to enhance the translational impact of ELSI research (Table 2). Recommendation 4. Research teams should explore a wide range of strategies and funders should promote a wide range of strategies to improve interdisciplinary communication and collaboration among ELSI researchers and between these disciplines and genomic research. We focus on translational research in early gene and cell therapy trials, which first analyze its general significance and related ethical considerations. We then look at the regulatory approach of Europe and the United States, as well as current guidelines and recommendations from the European Medicines Agency (EMA) and the Food and Drug Administration (FDA), respectively. We also report on the positions of international scientific societies, which dealt in particular with the criteria for the correct and timely transfer of stem cell research to clinics.
“ELSI research,” as it is commonly known, encompasses a wide range of disciplinary perspectives, including bioethics, history, law, medicine, genetics, economics, philosophy, and behavioral and social sciences. Unlike related work supported by other federal agencies such as the National Endowment for the Humanities or the National Science Foundation, a common challenge for ELSI research has been to examine policy-relevant issues and communicate their findings in places that help inform the policy-making process. Just as basic genomics scientists are encouraged to engage in “translational” research that enables the creation of medically useful tools, ELSI researchers have a “translational” mandate to conduct studies that help solve practical policy problems related to human genomics.2 Another World War II product with implications for medical ethics was the principled idealism that led to the creation of the United Nations. Its charter, declarations and works contain codes for the protection of the individual.