New paper examines patient access to gene therapy

The Arizona Chamber Foundation recently released a new paper, “Advancing Access to Transformative Medicines for Arizonans: A gene therapy primer,” which assesses the progress of gene therapy treatments and how state policymakers and the health care community can expand access to the life-improving treatments to more patients.  

Gene therapies are intended to slow or stop disease progression by addressing the underlying cause of disease at the level of a genetic mutation. The promise of gene therapy and the potentially game-changing effects it could create has been around for decades, and advances occurring each year are quickly moving these treatments from scientific theory to reality. 

Emerging gene therapies hold tremendous promise to dramatically improve and even save the lives of individuals living with conditions such as Sickle Cell Disease, Duchenne Muscular Dystrophy, and Hemophilia. 

The emergence of gene therapy presents a challenge for policymakers, however: How to expand access in the face of costs that might be higher than traditional medications. 

“To ensure that Arizona patients will be able to benefit from these medical advances as they are approved, all stakeholders, including payors, patients, policymakers, and pharmaceutical companies, must work together – quickly – to advance a flexible policy ecosystem that reflects the potentially transformative shift these therapies represent,” the authors write.

The paper explores options for making the therapies more affordable, suggesting that policymakers and health care stakeholders explore new types of payment options. The paper finds that expanding access to gene therapy is likely to put downward pressure on health care costs over the long term, however, by reducing costs associated with chronic diseases, such as medications, interventions, and hospitalization.

“By 2025, it’s anticipated that more than 20 new gene therapies are expected to be approved each year,” Arizona Chamber President and CEO Danny Seiden said. “With innovations emerging more quickly, now is the time for policymakers and the health care community to come together to find ways to get these exciting and life-changing therapies to more patients, with an eye toward not only improving patients’ quality of life, but also toward saving money in the long run. I’m excited that the Arizona Chamber Foundation’s research is advancing this important conversation.” 

Craig Ruiz

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