According to a new study, patients with a rare immunodeficiency disorder who receive lifelong immunoglobulin replacement therapy (IRT) have a lower risk of premature death than those who receive hematopoietic stem cell transplant (HSCT). Still, they also have a lower quality of life and must shoulder a significant financial burden.
The Most Cost-Effective Immunodeficiency Treatment For U.S. Patients
It was found that the high cost in America makes it less cost-effective than IRT in Europe, where the cost of IRT is a third of what it is here. This finding was based on a computational model that looked at the costs and advantages of IRT and HSCT for agammaglobulinemia patients with similar outcomes.
“The model we developed allowed us to answer questions about cost-effectiveness and outcomes for a patient population that is typically too small to power the robust clinical trials needed to answer these questions,” said first author Di Sun MD MPH, an attending physician in the Division of Allergy and Immunology at CHOP. “Given the results of our modeling, we believe that IRT should remain available to treat patients with agammaglobulinemia in the U.S. But regulatory efforts should focus on reducing its price.”
For many patients, the need for regular intravenous infusions impairs their quality of life, even though this medication has been proven to minimize numerous infections. IRT is also expensive, costing between $30 000 to $90 000 a year in the United States. Though it is not a big number, it can still affect the industry as a whole. Hence the experts favor an option where several treatments can be done simultaneously, and each of them can be monitored individually also to understand the progress and diagnose the situation at a regular interval.
Congenital agammaglobulinemia is a set of primary antibody deficiencies characterized by a lack of B cells or a paucity of B cells which can lead to recurrent infections and possibly life-threatening consequences. A lifelong intravenous infusion of IVIG or a weekly or biweekly subcutaneous infusion of IRT is the major treatment in high-income nations like the United States (SCIG).
Another option for treating agammaglobulinemia is HSCT however, finding a compatible donor can be difficult, and the operation itself carries hazards. However, no one has examined the health hazards outcomes and costs of HSCT with IRT until now.
Computational modeling was used by the researchers to examine the effects of lifetime IRT matched sibling donor HSCT or matching unrelated donor HSCT on a one-year-old kid in the United States with congenital agammaglobulinemia. The researchers utilized their model to simulate over 10 000 patients over 100 years by incorporating expenses probabilities and quality-of-life variables from the literature.
“As a treatment, IRT is an especially good option for older patients and those without suitable donor sources, but our study shows the exorbitant cost for patients in the United States prevents it from being a cost-effective option here,” said Neil D. Romberg MD, an attending physician with the Division of Allergy and Immunology at CHOP and senior author of the paper.
“Until additional treatment options become available such as gene-based therapies, our findings suggest that reducing IRT cost in the U.S. is an immediately actionable intervention that would make IRT the more cost-effective strategy compared with HSCT.” The researchers discovered that while lifelong IRT reduced premature mortality by 37% when compared to HSCT, it was not cost-effective when compared to HSCT because of its high cost. The increase in longevity brought about by IRT was not matched by an enhancement in quality of life. The researchers concluded that lowering the annual cost of IRT by 51% would make it the more cost-effective method
