The technical feasibility of gene therapy “has been established in multiple diseases and with different technology platforms,” says Dr. James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, in his commentary “It’s Time for Gene Therapy to Get Disruptive!” Wilson predicts that “2012 will usher in an era of commercial development of gene therapy that, although likely to begin slowly, will quickly gather momentum.”
The critical path to the development of gene therapy for diseases like hemophilia may reside in the creation of new business models that reimburse for the long-term efficacy afforded by a single gene therapy injection/treatment. One approach is to charge the insurance company an annual fee for gene therapy as long as it continues to work. This may be difficult to implement in countries in which health care is covered by private insurance companies and patients frequently change their health insurance affiliations. For example, it is unlikely that insurance company A will continue to pay the annual fee if the patient moves to insurance company B, which, in the United States, occurs quite frequently. This leaves one to conclude that reimbursement for a gene therapy product may be substantially less than that provided under current protein replacement therapies, leaving companies with traditional franchises in these diseases little incentive to invest in gene therapy. Those who lead in this scenario are often new to the business area and not concerned about compromising competing internal programs. The other important point to consider is that many early candidates for gene therapy are associated with significant disability and early mortality and, unlike hemophilia, have no effective treatments. Truly effective therapies in these disorders will likely be rewarded in terms of reimbursement in proportion to the value that they bring, which may be enormous if they address a substantial unmet need.
Wilson says we need to focus on discovery of new business models to support these new therapies. Gene therapy should not be though of as just a new model of car, rather its like the Model T which fundamentally changed personal transportation. Gene therapies are not just another bottle that sits on the shelf next to aspirin. Gene therapy may not fit in the established medial/health care business model. Instead, we need to develop entirely new models of reimbursement for these entirely new technologies. Those working on the business side of biotechnology need to be as creative as the scientists in the lab.