The Impact of Gene Therapy

Medicine is constantly evolving, and lately there is buzz around gene therapy. Gene-based therapies inject new, working copies of mutated or missing genes into the patient and gradually restore function.

Gene-based therapies were originally developed to treat ultra-rare genetic diseases that had few, if any, treatment options. However, drug manufacturers are now developing these therapies to treat more common genetic diseases.

The High Costs of Gene Therapy

Specialty drugs have been one of the biggest cost drivers in health care for years, but gene therapies are taking that trend to the extreme.

Greg Gambescia is the director of specialty pharmacy at Independence Blue Cross (Independence). His team focuses on managing specialty drugs and gene therapies. According to Gambescia, “The fiscal impact of these therapies can’t be overstated. Currently, Hemgenix is the most expensive FDA-approved gene therapy on the market at $3.5 million for one dose. And costs for future therapies likely will be higher.”

Further complicating matters, gene therapies have only been studied for a few years. Long-term studies are underway to establish their safety and efficacy, as well as to determine if gene therapy recipients need additional doses to maintain therapeutic response. Gambescia anticipates that results of these studies will add several layers of complexity in a drug class that is already challenging to manage.

The Future of Gene Therapy

Since gene-based therapies were initially used to treat ultra-rare conditions, Independence only saw a few requests for these treatments. However, the future of this treatment class will become significant for two reasons:

A rich pipeline. As many as 12 new gene therapies may be submitted for FDA review in the next few years. And many more are in early stages of research and development.
Higher utilization. The newer gene therapies in the pipeline are designed to treat more common genetic conditions, like sickle cell disease. As a result, more people could be eligible candidates for treatment.

Even as the gene therapy landscape expands, it is important to note that not all people with genetic diseases may be candidates for these treatments. Often, gene therapies are indicated only for individuals within a certain age range or for individuals with a severe level of disease.

How Independence Manages Gene Therapies

Gene therapies are eligible for coverage under the Independence medical benefit, and each gene therapy has its own medical policy. We review these policies regularly to ensure that coverage criteria are comprehensive and current. All gene therapies require precertification approval to confirm that members meet these coverage criteria before receiving treatment.

Gambescia believes Independence will need additional methods to manage these high-cost treatments. He stresses the importance of our strategic platform of “blending innovation, partnerships with in-network facilities, negotiations with manufacturers, and patient education to optimize clinical and financial outcomes.”

As a result, Independence has established an Advanced Network for Gene-Based Therapeutics, which identifies best-in-class facilities for members to receive gene-based therapies. Both the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine are at the forefront of gene therapy research and development. These hospitals have reputations for exceeding quality, safety, and value benchmarks. In addition, they train their staff in recommended protocols established by the manufacturer to get the best possible results. Independence works closely with these hospitals to ensure that they provide complete, coordinated care that centers around the patient.

Independence is also pursuing outcomes-based agreements with certain manufacturers. Under these arrangements, the manufacturer would provide a warranty on cost if the therapy does not produce expected outcomes in members who receive treatment. Gambescia states, “This is a unique opportunity for manufacturers to show confidence in their product.”

Lastly, Independence members receive Wraparound Care Management Services from a dedicated team of Registered Nurse Health Coaches who have experience in genetic disease management. These nurses can answer questions, provide support, coordinate care, and help members navigate the treatment process. They also partner with the Care Management teams at Penn Medicine and Children’s Hospital of Philadelphia to ensure smooth transitions into the network for members who may be receiving care from other health care systems.

Looking Ahead

Gene therapy treatments are innovative, but costly in the short-term and unproven in the long-term. We will continue to monitor results of long-term studies and industry trends in the gene therapy landscape. This will allow us to confidently pursue, implement, and refine strategies that ensure access and manage costs for this important treatment class.

You can read more about the Independence Advanced Network for Gene-Based Therapeutics in our press release.