Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under investigation to ensure safety and efficacy. Furthermore, research in this field is particularly challenging given the inherent nature of rare diseases and genetic disorders. Approximately 30 million individuals in the United States are living with a rare disease, which by definition have a prevalence of 200,000 or fewer cases in the United States. Disease heterogeneity and geographic dispersion add to the difficulty of completing robust studies in these small populations. Given these challenges and a limited amount of resources available, gene therapy is currently in clinical trials only for diseases that have no other cures.
Genetic disorders and other rare diseases present unique challenges for health insurers in terms of requisite clinical intervention, disease management, and economic burden. The treatment landscape in US health care has already evolved significantly over the past several decades and appears to be on the precipice of its most groundbreaking innovation to date: gene therapy. This program is designed to address the educational needs for payers and managed care professionals regarding the solutions necessary to address the unique clinical and financial considerations surrounding gene therapies that will ensure quality care and appropriate patient access.
At the conclusion of this activity, participants should be able to demonstrate improved ability to:
- Explain the molecular and physiologic principles of gene therapy in the treatment of rare diseases
- Review outcomes measures for clinical trials in gene therapy and the pertinent clinical trial data for investigational treatments
- Evaluate the financial implications of gene therapy in terms of acquisition costs reconciled with the potential for improved outcomes and reduced health care service utilization
- Assess current and proposed payment models aligned with appropriate use for high-cost therapies