Weighing the Costs and Benefits of Breakthrough Cell and Gene Therapies

The Rise of Cell and Gene Therapies

In 2024, the landscape of medical treatments saw a significant shift with the approval of 50 novel drug therapies by the FDA. Among these, seven new cell and gene therapies (CGTs) were approved, raising the total to 41. Specialty medications now account for over 75% of all drugs in development. These therapies present a forward-thinking approach to treating complex, chronic, and rare diseases, offering hope and potentially life-changing solutions for patients and healthcare providers.

CGTs are developed to provide treatment options that can lead to curative outcomes and have recently been gaining traction in the areas of oncology, hemophilia and Duchenne muscular dystrophy. For patients suffering from these types of conditions, the promise of gene therapy may mean the end of ongoing therapy requirements. In the case of Zolgensma, a treatment for spinal muscular atrophy, evidence indicates effectiveness may extend up to seven and a half years after administration, showcasing the potential of CGTs to change lives dramatically.

Cell and Gene Therapy Conundrum

These breakthrough treatments do not come without challenges, primarily concerning managing costs. CGTs are among the most expensive drug classes in existence, with therapies costing between $1 million and $4 million or more for a single dose. This places a heavy burden on both payers and patients. Likewise, the juxtaposition of these extraordinarily high costs against the backdrop of potentially life-altering outcomes raises questions about their long-term value and overall impact on healthcare expenditures.

Payers, faced with uncertain case volumes and clinical effectiveness, are starting to examine the economic implications of CGTs and their therapeutic benefits more closely. To assess whether the prices of these innovative therapies are justified, payers are conducting their own clinical and cost-effectiveness analyses, while also turning to reputable third-party organizations for guidance. Organizations such as the Institute for Clinical and Economic Review (ICER), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and the National Institute for Health Care and Excellence (NICE) are crucial in helping to evaluate the economic impact of CGTs.

ICER, which began as a research initiative at Harvard Medical School in 2006, has since evolved into an independent, non-profit organization. It evaluates the clinical and economic value of prescription drugs, medical tests, devices, and healthcare delivery innovations. By collaborating with academic scholars, patient groups, clinical experts, and insurers, ICER publishes comprehensive analyses highlighting various therapies' safety, clinical utility, and economic considerations.

In their clinical and economic assessments of CGTs, ICER often suggests price benchmarks lower than what manufacturers typically charge. For instance, they recommended a price of $2.9 million for Hemgenix, while the actual cost is $3.5 million. Similarly, for Zolgensma, ICER proposed a range of $1.1 to $2.1 million against an actual price of $2.125 million. These disparities underscore the ongoing dialogue regarding the justification of therapy prices in relation to their predicted benefits.

Hurdles Preventing More Widespread Adoption

Despite the immense promise of CGTs, several hurdles remain in achieving their broader application. Critical questions linger regarding the durability and sustained effects of treatment, the identification of suitable patient populations, the possibility of eliminating the need for concurrent or subsequent treatments, and the avoidance of long-term complications. As more long-term clinical data is generated, understanding how these therapies compare to conventional treatments is essential.

As payers and the medical community in general gain access to more robust clinical evidence, it will inevitably become clearer whether these one-time therapies, which may yield lifelong benefits from a health and quality-of-life perspective, represent a more cost-effective option compared to ongoing conventional therapies. The ongoing evolution in CGTs signifies a remarkable shift in medicine—one that holds the potential to reshape healthcare. Yet, it calls for an informed evaluation of their economic viability and clinical efficacy.

Conclusion

The advent of cell and gene therapies can revolutionize treatment paradigms. Understanding the costs and benefits associated with these innovations will be fundamental in bridging the gap between groundbreaking science and practical healthcare implementation. As we progress, collaborative efforts among specialty pharmaceutical stakeholders remain vital in ensuring that patients can benefit from these progressive therapies while maintaining financial sustainability.

Navigating the complex challenges of CGTs—ranging from affordability and access to long-term clinical outcomes—requires specialized expertise and strategic collaboration. Partnering with a trusted organization like AscellaHealth, provides a wealth of experience working with complex, chronic, and rare diseases, access to specialized networks, and proven cost-containment strategies, and can help payers implement effective solutions that enable both sustainability and improved patient outcomes.

To learn how AscellaHealth can help your organization manage specialty pharmaceutical therapies more efficiently, please click here or contact us directly at businessdevelopment@ascellahealth.com.