Beyond the budget impact model: A case study for glucarpidase and the real cost of managing curable toxicities

The greatest challenge in healthcare today lies in managing limited resources to deliver high-quality care to the patients who need it the most. Payers heavily rely on budget impact models to assess the net costs or potential savings associated with adding a new intervention and to inform their formulary decision. Drugs developed for rare conditions are often prohibitively expensive due to the complex manufacturing processes and investments required to undertake clinical trials. These interventions often play a critical role in organ preservation and can be lifesaving when no alternative therapies are available. The significant cost of these treatments has to be weighed against the potential cost consequences of not using the treatment including downstream complications of an avoidable event. Over the past few decades, oncologic treatments have seen significant advancements. Despite these innovations, radiation therapy and chemotherapy remain the backbone of treatment for many types of cancers. These therapies often damage healthy cells alongside cancer cells, leading to a range of side effects that can affect multiple organ systems. While some side effects, such as those from radiation therapy, may be resolved within weeks or months after treatment ends, others may persist or emerge months to years later. Worse yet is the impact of these side effects on patients' ability to continue with their cancer treatment regimen. Here we discuss a case of glucarpidase in managing high dose methotrexate toxicity and consideration of full impact, not only on the budget but also the patient.