Value of Gene Therapy and Patient Access White Paper

Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments

Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to optimize patient access. Maximizing patient access to effective gene therapies is one integral part of the overall mission of the American Society of Gene and Cell Therapy, along with maximizing the quality of therapies and minimizing their costs. 

Gene therapy is a radical shift in our approach to disease treatment. By modifying the expression of a patient’s genes or repairing abnormal genes, gene therapy often addresses the root cause of dis- eases. Even though several new gene and cell therapies have received 

U.S. Food and Drug Administration (FDA) approvals over the past 20 years, the field has recently experienced a turning point. Three gene therapies were approved for human medical use in the United States in 2017,1–3 including the first in the country for an inherited condition. Many more approvals are expected in the near future; Mas- sachusetts Institute of Technology’s (MIT’s) New Drug Development Paradigms program estimates FDA approval of three dozen new gene therapies by 2022.4 

The newly approved gene therapies offer substantial benefits to pa- tients who otherwise have little to no hope of cure or even meaningful improvement. They treat two forms of non-Hodgkin’s lymphoma, an acute form of leukemia, and a hereditary genetic defect that nearly always leads to blindness. Each is a potentially one-time treatment, just a single infusion, that may provide long-term, durable efficacy. 

These new and expensive treatments have escalated important discus- sions about how to place a value on gene therapies and how our health care system will pay the upfront costs for these often one-time treat- ments. In essence, payers are being tasked with paying a larger price today versus paying repeatedly for treatments that may be taken at regular intervals for months, years, decades, or even a lifetime. But as- signing value to gene therapies and comparing them with potentially lifelong illness is not an easy or straightforward task. Despite the complexity, this paper identifies unique and relevant aspects that should be considered when assessing the value of gene therapy. A related important discussion is how the costs of these treatments could be reduced, and whether the cost of gene therapy products will be so competitive as to substantially reduce the overall costs of health care for patients. 

The term “gene therapy,” as used in this paper, refers to a set of stra- tegies that modify the expression of an individual’s genes or repair abnormal genes. Specific types of treatments include vector-delivered gene therapy, gene-modified cell therapy, and gene editing. Gene therapy offers new and unique approaches to treating previously intractable diseases. Rather than treating disease symptoms, gene therapy can address the root causes of genetic diseases by modifying expression of a patient’s genes or by repairing or replacing abnormal genes. Many experts believe that gene therapies are “shifting medicine away from a chronic disease management approach toward disease interception and prevention.”5 FDA Commissioner Scott Gottlieb, MD, has stated, “I believe gene therapy will eventually become a mainstay in treating, and maybe curing, many of the most devastating and intractable illnesses.”6 

Successful gene therapies have the potential to prevent years or even decades of morbidity with perhaps just one treatment. In exchange, one-time gene therapies entail a one-time cost, which may seem high until it is compared against many years of expensive, ongoing care. This shift in the timing of health care costs, along with antici- pated new gene therapy approvals, has increased the urgency of dis- cussions about how to determine the “value” of gene therapy.