摘要
Advances in stem cell science and potential clinical applications have brought clinical medicine closer to the actualization of Regenerative Medicine—an extension of transplantation of organs and cells and implantation of bioprosthetics and biodevices. The goal of such therapeutics will be intervention prior to onset of severe individual disability, enhance organ function and enhance patient performance status without incurring the economic impacts of standard organ transplantation. Regenerative Medicine is already demonstrating proof of principle or efficacy in restora- tion of myocardial contractility, joint mobility and function, immune competence, pulmonary function, immunologic self- tolerance, motor function and normal hemoglobin production with the next targets—diabetes mellitus (type I and type II), neurologic injury, hepatic dysfunction preparing to enter trials. Expenditures on health care needs of an aging U.S. citizenry approximate 20-25% ($3 trillion) of U.S. GDP currently and may to grow to 40% of U.S. GDP by 2025. As the potential of Regenerative Medicine is clinically realized, the societal impact and economic benefits will be disproportionately magnified in the economies of industrialized nations. The experi- ence of the Department of Health and Human Services (HHS), United Network for Organ Sharing (UNOS), the National Bone Marrow Donor Registry (NBMDR), and the National Vaccine Injury Compensation Programs (NVICP) can help ensure that as Regenerative Medicine strives to achieve clinical benefits while avoiding decimation of therapeutic options by product liability and medical malpractice concerns—concerns that crippled the U.S. vaccine manufacturing industry until the creation of the NVICP. The first 50 years of organ/cell/tissue transplantation demonstrates that clinical reality of allogeneic and autologous transplantation can antedate complete understanding of the basic science underlying successful transplantation. Product liability and medical malpractice liability have not impeded the development and growth of organ/cell/tissue transplanta- tion despite increased risks of infection, malignancy and cardiovascular disease in transplant recipients. Currently, human transplantation is only performed using FDA/CBER-approved, non-embryonic stem cells from peripheral blood, bone marrow or umbilical cord blood. Federal legislation passed in 2005 (HR2520 and S1317: The Bone Marrow and Cord Blood Cell Transplantation Program) authorizes the Secretary of Health and Human Services acting through the Director of HRSA to ensure uniform stem cell units distribution and outcomes monitoring via the federally-designated C.W. Bill Young Cell Transplant Program. Historically in the U.S., human biological therapies (vaccines, organ transplant and stem cell transplant) have re- quired federal protections to ensure continued distribution, fair access and avoidance of inhibitory product liability via protections afforded under the “stewardship” of the Secretary of Health and Human Services. The National Childhood Vaccine Injury Act of 1986 established the NVICP to equitably and expeditiously compensate individuals, or families of individuals, who have been declared injured by vaccines, thereby stabilizing a once imperiled vaccine supply by substan-tially reducing the threat of liability for vaccine companies, physicians, and other health care professionals who administer vaccines. Vaccines were the first biologics administered to U.S. citizens en masse and presage stem cell therapeutics (which may similarly be administered to millions) will similarly necessitate that a Stem Cell Injury Compensation Program (SCICP) will also need to be in place to demonstrate an intention to do good, an understanding that industry may do well, but that the health care consumer has a right of protection—all recognized from the outset. The Federal Tort Claims Act (FTCA) addresses liability claims via the Executive, Judicial and Legislative branches of Government, providing an um- brella of liability protection to other participants in the stem cell unit “chain of custody” under the FTCA—similar to the protection from product liability seen in organ and stem cell transplantation for the past 40-50 years. Efficacious development of regenerative medicine capabilities will mandate controlled access must first be provided for individuals with life-threatening diseases without therapeutic options or unable to benefit from or receive proven therapeutic options (ALS, cardiomyopathy and deemed not a candidate for heart transplantation, IDDM with hypoglyce- mic unawareness and no allogeneic source of traditional islet cell replacement available via HRSA) and mandates the prompt adoption of business and legal principles to ensure that the fate of the vaccine manufacturing industry does not become the fate of the stem cell therapeutics industry. If legal and regulatory concerns consume an increasing percentage of health care dollars that could be focused upon innovation, the Regenerative Medicine model will have not realized its full potential. The Diabetes Transplantation/Regenerative Medicine Model is the first organ to cell transplant model outside of oncology to demonstrate the regenerative medicine paradigm. Since all human tissues can be already recapitulated by human stem cells and key patent holders already exist, outlet or distribution of “more-than-minimally-manipulated stem cell units” as an IND approved under FDA/CBER guidelines can be accomplished via the current HHS/HRSA/Dept of Trans- plant methodology. As cardiovascular stem cell researchers develop human therapeutics utilizing more-than-minimally- manipulated stem cell products, they could be afforded protections from product liability historically enjoyed by the transplant community. Extending the Diabetes Transplant/Regenerative Medicine Model to the more than 5 million Americans with chronic heart failure, cell-based therapies to regenerate myocardial contractility could fill an existing void and be delivered in conjunction with and consistent with existing distribution of organs and tissues via HRSA/Department of Transplantation.
