Introduction

Gene therapy is rapidly transforming the treatment landscape for rare diseases. With innovations like CAR T-cell therapy and CRISPR, patients have new therapeutic options. However, a significant challenge remains: the cost and funding of these treatments. Therefore, the question remains: who will bear the cost of these groundbreaking therapies?

The High Costs of Gene Therapies

Gene therapies, while groundbreaking, come with high price tags. The U.S. Food and Drug Administration (FDA) recently approved two cell-based gene therapies for sickle cell disease (SCD). These treatments, especially those involving CRISPR technology, offer hope to many. However, their cost raises questions about who should bear the financial burden.

Jay Newman, SVP of Commercial Development at Spark Therapeutics, highlighted this issue during the 2024 BIO International Convention. He noted that we are trying to administer 21st-century medicines within a 20th-century payment model. This sentiment resonated with many attendees, including caregivers, patients, and industry experts.

Patient Experiences and Financial Challenges

One caretaker, whose daughter Shelby has beta thalassemia, shared her story at the convention. Shelby participated in clinical trials for ZYNTEGLO, a gene therapy developed by bluebird bio. This one-time treatment significantly improved Shelby’s condition. However, securing insurance coverage was challenging. Initially, their insurer approved only one day of treatment, misunderstanding the therapy’s duration.

Fortunately, the Children’s Hospital of Philadelphia (CHOP) intervened, negotiating with the insurance company. This experience indicates the need for better understanding and coverage of gene therapies by insurers.

Value Over Price

Experts argue that the focus should be on the value of gene therapies, not just their cost. Tom Klima, Chief Operating and Commercial Officer at bluebird bio, emphasised the holistic costs associated with these treatments. One-time curative therapies can eradicate diseases, reducing long-term healthcare costs.

Klima also discussed outcomes-based agreements, which tie therapy costs to meaningful clinical outcomes. For example, bluebird bio offers rebates if their therapies do not achieve transfusion independence for patients. This approach aligns the cost of treatment with its real-world effectiveness.

State Health Plans and Policy Solutions

State health plans face the challenge of covering expensive gene therapies without bankrupting patients or their budgets. Stuart Portman, Executive Director at the Division of Medical Assistance Plans for Georgia, highlighted the need for alternative payment arrangements. These could be based on value or outcomes, but would require changes to existing policies.

Consistency and guarantees from federal entities are crucial. Portman stressed that states need assurance that federal policies around Medicaid will remain stable. Collaboration between federal and state governments, along with industry stakeholders, is essential to develop sustainable solutions.

Conclusion

The cost and funding of gene therapies present significant challenges, but also opportunities for innovation in healthcare financing. By focusing on the value of these treatments and exploring alternative payment models, we can ensure that patients have access to life-changing therapies. Collaboration between insurers, healthcare providers, and policymakers is key to navigating these financial complexities.