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After nearly thirty years of biological discovery underpinning gene therapy vectors and more than twenty years of clinical gene therapy trials, cases with clear evidence of clinical efficacy have now been demonstrated. Examples include restoration of immune function in children with X-linked severe combined immune deficiency (X-SCID) and chronic granulomatous disease (CGD), restoration of vision in Leber Congenital Amaurosis (LCA), and marked improvement in motor function of Parkinson disease (PD). Likewise, cell-based therapies beyond the well-established bone marrow transplant techniques are emerging, including the use of mesenchymal (MSC) and hematopoietic (HSC) bone marrow stem cells, umbilical cord cells, human embryonic stem (hES) cells and induced pluripotent stem (IPS) cells. Applications for these new forms of cell-based therapies include a variety of neurodegenerative disorders (such as amyotrophic lateral sclerosis [ALS]) and ophthalmologic disorders, such as age-related macular degeneration. At the same time U.S. spending for health care has been on an unyielding upward path—reaching $2.5 trillion in the aggregate, $8,100 per person, and 17.6 percent of gross domestic product (GDP) in 2009 (Schoenman and Chockley, 2011). The ever-rising cost of health care has serious consequences for individuals and the country. For example, from 1999–2009 health care costs cancelled out real income gains for an average U.S. family (Auerbach and Kellerman, 2011), and the 2011 Medicare Trustees report projects that Medicare has an unfunded liability of over $38 trillion (www .cms.gov/ReportsTrustFunds/downloads/tr2011.pdf ). The unfunded liability is the difference between the benefits that have been promised to current and future retirees and what will be collected in dedicated taxes and Medicare premiums. These facts have led to proposals that radically redesign the funding of health care in the United States, with the goals of providing access to care for all Americans, improving quality, and reducing costs. Ironically, the emergence of all the potential promise of these innovative molecular therapies is coinciding with a future in which the costs of new therapies may be the major determinate of whether they will be available to patients. Is there a future state that will embrace the equally noble goals of using cutting-edge science to devise advanced biological therapeutics for previously untreatable illnesses and of using systems engineering to provide all Americans with the lowest cost care possible? The answer is far from certain. What is clear is that if cost-effectiveness is to be addressed in the context of gene and cell therapy, it will have to include a long time-horizon in which the therapy may avoid or ameliorate complications of the disease and accurate monetization of the gains in quality of life and function for the individuals who are treated. Incorporating these elements into an economic model of the value of gene and cell therapy is challenging but not unprecedented. What is even more problematic is the uncertainty about what the new payment models will include, and whether such models are truly suited to look at global lifelong benefits to patients.

The Role of Gene and Cell Therapy in the Era of Health Care Reform