Gene Therapy Innovations and Financial Challenges for the Future of Medicine

Gene therapy represents a revolutionary advance in medicine, offering hope to patients with rare diseases. With innovations such as CAR T-cell therapy and CRISPR, a new era of therapeutic options is emerging, many of which provide alternatives to previously dead-ended diagnoses. Day 1 of the Biotechnology Innovation Organisation (BIO) International Convention saw diverse stakeholders—including caregivers, patients, hospitals, policy experts, manufacturers, and C-suite executives—discuss the potential of gene therapy and the barriers to access. A significant issue is the high cost of these treatments, which can reach millions of dollars.

On Track for Success, Can Payment Plans Keep Up with the Science?

With the fast pace of recent advancements in the field, the future is much closer than we think it is. In December, the Food and Drug Administration (FDA) approved the first cell-based gene therapies for sickle cell disease (SCD) in patients aged 12 and older, one of which uses CRISPR technology. The approval of these therapies marks significant progress, but financial issues pose a significant obstacle to their success. At the end of the day the biggest question remains: who is going to pay for the treatment?

Outdated payment models effectively hamper the process of making this life-changing technology available to all patients that need it. Essentially, the existing payment systems are not equipped to handle the costs of modern gene therapies, necessitating new financial strategies.

Jay Newman, SVP at Spark Therapeutics, Inc., expressed the industry’s frustration: “We’re trying to administer 21st-century medicines within a 20th-century payment model.” During a panel at the 2024 BIO International Convention, “A Perspective from the Trenches: Challenges of Budgeting for Gene Therapies, Innovative Payment Systems That Work for Medicaid Departments and Patients, and What the States Need from CMS”, he highlighted the challenges of financing gene therapies and the need for innovative payment systems for Medicaid and patients alike.

Patients Raise Access and Affordability Concerns

The transformative potential of gene therapies in treating and potentially curing severe genetic diseases is evident. However, transitioning these therapies from clinical trials to real-world applications presents substantial challenges. Unlike chronic treatments administered over a lifetime, gene therapies are typically one-time treatments, demanding a unique approach from all stakeholders, including manufacturers, contract development and manufacturing organizations (CDMOs), health systems, payers, and patients. Effectively addressing these challenges is crucial for unlocking the full potential of gene therapies.

Real-world patient experiences shed light on both the life-changing potential and the complexities of accessing gene therapy. Michelle Campbell, mother of a daughter with beta thalassemia, shared her family’s journey during a panel titled “Lessons from the Oregon Trail: Pioneering Gene Therapy in the Real World.” Initially, managing Shelby’s condition was manageable with regular hospital visits for transfusions and daily medication. However, when her medication became ineffective, the situation turned critical.

The family connected with a team at the Children’s Hospital of Philadelphia (CHOP) and found that Shelby qualified for clinical trials of ZYNTEGLO, a gene therapy recently FDA-approved and developed by bluebird bio, another BIO member. This one-time therapy significantly improved Shelby’s health, demonstrating the transformative potential of gene therapies. However, obtaining insurance coverage for the treatment proved challenging.

“When we approached our insurance for Shelby’s gene therapy coverage, they initially approved it for just one day, thinking it was a single-day treatment,” Campbell recounted. CHOP intervened to clarify that the therapy required an extended treatment period. Fortunately, the hospital’s legal team successfully negotiated with the insurance company, enabling Shelby to receive the necessary care. Campbell’s account highlights the hurdles in securing insurance coverage for gene therapy and the crucial role of advocacy and negotiation in overcoming these obstacles.

Evaluating Gene Therapies: Long-Term Value Over Immediate Costs

Panelists at the BIO convention emphasized the importance of discussing the value rather than the price of gene therapies. This means evaluating gene therapies based on their long-term benefits and potential to reduce overall healthcare costs, rather than solely focusing on their immediate price tags. These therapies involve extensive research and complex manufacturing processes, which drive up costs. Yet, as Tom Klima from bluebird bio noted, their one-time nature can eradicate diseases and reduce long-term healthcare costs.

Klima explained, “One of the common misperceptions is that the cost of manufacturing drives the high cost of therapies. At bluebird bio, we take a very value-based approach where we look at the value of a therapy in a chronic condition that’s both devastating for patients and also very costly to the system.”

Scott McGoohan from Vertex Pharmaceuticals highlighted the necessity of comprehensive patient support and logistical considerations, noting that gene therapies often require travel and involve additional layers of complexity beyond reimbursement.

State health plans face the challenge of covering the cost of gene therapies without overburdening patients or destabilizing budgets. Stuart Portman from the State of Georgia discussed the potential for alternative payment arrangements, such as value-based or outcome-based models. However, Portman emphasized the necessity of federal and state governments working with the industry to create sustainable solutions: “Everyone has to trust that the federal government won’t change its mind.”

While significant obstacles remain in financing gene therapies, their continued integration into healthcare is inevitable. Collaboration between governments and the industry is crucial to ensure that these life-changing treatments are accessible to all patients. Panelists at the BIO convention agreed that addressing these financial challenges is essential for the future of gene therapy. Muscle,

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