More effective graft-versus-host prevention strategies could yield “real cost savings for the healthcare system.”

Richard T. Maziarz, MD, and a team of researchers evaluated the average medical cost of allo-HCT throughout a patient’s lifetime, as well as the net financial savings and value associated with reducing complications.

pharmacy hours® interviewed Richard T. Maziarz, MD, Professor of Medicine at the Knight Cancer Institute at Oregon Health & Science University, about the poster presentation entitled Estimating the Lifetime Medical Cost Burden of an Allogeneic Hematopoetic Cell Transplantation Patient and the Value of Addressing the Unmet Need” at the 64th Annual Meeting and Exposition of the American Society of Hematology (ASH).

With the advancement of allogeneic hematopoietic cell transplant management (allo-HCT), molecular detection assays and therapies have emerged that can treat post-hematopoietic cell transplant complications such as graft-versus-host disease (GVHD). With these advances, however, the subsequent economic burden on healthcare systems has remained unclear, as only treating complications that arise after the initial transplant can prove costly. In addition, it is possible that alternative therapies using newly developed donor allografts that replace standard allo-HCT and prevent late complications could improve outcomes and address a larger unmet need in patients.

To evaluate this hypothesis, Maziarz and a team of researchers attempted to estimate the average medical cost of allo-HCT over a patient’s lifetime and estimate the net financial savings and value associated with reducing complications.

pharmacy hours®: What is the current standard practice for managing allogeneic hematopoietic cell transplant complications such as graft-versus-host disease?

Richard Maziarz: There are several interventions to prevent and treat GVHD. For prevention, several treatment regimens are accepted as standard of care (SOC) and continue to be the subject of research. Although effective, 30% to 60% of patients still suffer from acute and/or chronic GVHD.

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Regarding treatment, steroids were SOC for acute and mild chronic GVHD. For moderate to severe cases of chronic GVHD, there have recently been multiple approvals of drugs such as ruxolitinib, ibrutinib and belumosudil for the treatment of chronic GVHD. Ruxolitinib is also approved for advanced or steroid-resistant acute GVHD.

pharmacy hours®: How could this current approach put a significant strain on the healthcare system?

Richard Maziarz: The economic burden of acute GVHD is influenced by hospitalization and resource consumption. Chronic GVHD is associated with increased costs to the healthcare system due to increased use with the admission of additional agents for treatment. Chronic GVHD is also associated with negative impacts on quality of life, as many patients require ongoing medical care and are unable to return to normal life, including returning to work.

pharmacy hours®: What could be an approach to address this economic burden more effectively?

Richard Maziarz: A real potential to reduce this economic burden is to pay more attention to the development of products and interventions that can prevent GVHD without losing the graft vs. malignancy effect.

pharmacy hours®: Would this improvement to the current approach primarily address the economic burden on the healthcare system, or would it also help address issues related to financial toxicity for patients?

Richard Maziarz: I think there is potential for both. If you’re able to provide better prevention, these costly complications could be drastically reduced, meaning fewer hospitalizations, ICU stays, and costly treatments like oral medications that have high patient costs. In addition, reducing the need for long-term medical care will limit these financial toxicities.

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pharmacy hours®: How was the financial burden assessed in the study?

Richard Maziarz: In this detailed analysis, a short-term decision tree and a long-term partitioned survival model were developed that estimated the average lifetime cost per patient, expected life years, and quality-adjusted life years (QALYs) for an allo-HCT patient from a US healthcare system perspective. The patient population modeled an average 53-year-old patient undergoing transplantation for the most common indications for allo-HCT: acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes.

In addition, the short-term decision tree calculated the costs and consequences of allo-HCT hospitalization up to 100 days, during which patients could experience GVHD, underlying disease recurrence, GRFS, or death. After the first 100 days, the patient cohort was assigned to three mutually exclusive health conditions in a segmented survival model for the rest of their lives: (1) graft-vs-host-free (GRFS), recurrence-free survival; (2) advanced and/or GVHD; and (3) death. The costs of allo-HCT, acute GVHD, chronic GVHD, relapse, infection, maintenance therapy and end of life were included in the model.

pharmacy hours®: What did the study results show in relation to the average total lifetime medical costs of a patient who underwent allogeneic hematopoietic cell transplantation?

Richard Maziarz: Cost results were presented as a range based on variation in the percentage of patients with chronic GVHD remaining on treatment at 2 years (15% or 39%). In the lifetime base case analysis, the average medical cost per patient for allo-HCT was estimated to be $942,373 to $1,247,917.

pharmacy hours®: What did the study results show in terms of net cash savings and the value associated with reducing late complications?

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Richard Maziarz: In the scenario analyses, the net monetary savings (via medical cost recovery and QALYs gained) achieved by improving GRFS outcomes were associated with a net monetary value ranging from $695,709 to $911,062.

pharmacy hours®: What are the implications of these results for the health system?

Richard Maziarz: If we can develop more effective GVHD prevention strategies, there should be real cost savings to the healthcare system. This outcome would benefit individuals and healthcare systems financially while improving the quality of life for their patients.

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