Expert: More Effective Graft-versus-host Disease Prevention Strategies Could Provide ‘Real Cost Savings to the Health Care System’


Richard T. Maziarz, MD, and a team of investigators assessed average medical costs of allo-HCT throughout a patient’s lifetime and the net monetary savings and value associated with reducing complications.

Pharmacy Times® interviewed Richard T. Maziarz, MD, professor of medicine at the Oregon Health & Science University Knight Cancer Institute, on the poster presentation titled “Estimating the Lifetime Medical Cost Burden of an Allogeneic Hematopoietic Cell Transplantation Patient and the Value of Addressing the Unmet Need” at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition.

As allogeneic hematopoietic cell transplantation (allo-HCT) management has evolved, there has been an emergence of molecular detection assays and therapies that can treat post-hematopoietic cell transplantation complications such as graft-versus-host disease (GVHD). However, with these advances, the subsequent economic burden on health care systems has remained unclear, since only managing complications that occur following initial transplantation can prove costly. Further, it is possible that alternative therapies involving novel engineered donor allografts that would replace standard allo-HCT and prevent late complications could improve outcomes and address a greater unmet need among patients.

To assess this hypothesis, Maziarz and a team of investigators looked to estimate the average medical costs of allo-HCT throughout a patient’s lifetime and to estimate the net monetary savings and value associated with reducing complications.

Pharmacy Times®: What is the current standard practice for managing allogeneic hematopoietic cell transplantation complications such as graft-versus-host disease?

Richard Maziarz: There are several interventions used to both prevent and treat GVHD. For prevention, multiple regimens are accepted as standard of care (SOC) and continue to be subjects of research. While they are effective, there are still 30% to 60% patients who experience acute and/or chronic GVHD.

Regarding treatment, steroids have been SOC for acute and mild chronic GVHD. For moderate to severe cases of chronic GVHD, there have been several recent approvals of drugs like ruxolitinib, ibrutinib, and belumosudil to treat chronic GVHD. For advanced or steroid resistant acute GVHD, ruxolitinib is also approved.

Pharmacy Times®: How might this current approach be placing significant economic burden on the health care system?

Richard Maziarz: The economic burden of acute GVHD is influenced by hospitalizations and resource utilization. Chronic GVHD is associated with increased cost to the health care system due to increased utilization with the approval of additional agents for treatment. Chronic GVHD is also linked to a negative impact on quality of life with many patients requiring ongoing medical care and an inability to return to a normal life, including going back to work.

Pharmacy Times®: What may be an approach that could address this economic burden more effectively?

Richard Maziarz: A real potential for lowering this economic burden is to focus more attention to developing products and interventions that can prevent GVHD without loss of the graft vs malignancy effect.

Pharmacy Times®: Would this improvement to the current approach primarily address economic burden on the health care system or would it also help to address issues around financial toxicity for patients?

Richard Maziarz: I believe there is potential to do both. If you are able to provide better prevention, this could dramatically reduce these costly complications, which means less hospitalizations, ICU stays and costly treatments such as oral medications that have a high patient out of pocket cost. Additionally, reducing the need for long-term medical care will limit these financial toxicities.

Pharmacy Times®: How was 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 per-patient lifetime cost, expected life years, and quality-adjusted life years (QALYs) for an allo-HCT patient from a United States health care system perspective. The patient population modeled an average 53-year-old patient who was undergoing transplant for the most common indications for allo-HCT: acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes.

Additionally, the short-term decision tree calculated the costs and consequences from the allo-HCT hospital admission up to 100-days in which patients could experience GVHD, relapse of the primary disease, GRFS, or death. Following the first 100 days, the patient cohort was assigned to 3 mutually exclusive health states in a partitioned survival model for the remainder of their lifetime: (1) graft-vs-host free (GRFS), relapse-free survival; (2) progressed and/or GVHD; and (3) death. The cost of allo-HCT, acute GVHD, chronic GVHD, relapse, infection, maintenance therapy, and end-of-life were included in the model.

Pharmacy Times®: What did the study results show regarding the average total lifetime medical costs of a patient undergoing allogeneic hematopoietic cell transplantation?

Richard Maziarz: Cost results were reported as a range based on varying the percent of chronic GVHD patients that remained on treatment after 2 years (15% or 39%). In the base case analysis over a lifetime, the average per-patient medical cost of allo-HCT was estimated to range from $942,373 to $1,247,917.

Pharmacy Times®: What did the study results show regarding the net monetary savings and value associated with reducing late complications?

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

Pharmacy Times®: What are the implications of these results for the health care system?

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

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