Health Economics Implications of Hematology Therapies Spotlighted Among EHA Research


With the number of treatment options in hematology and oncology rapidly expanding, so too are the strategies for determining and ensuring cost-effectiveness, according to session speakers at the European Hematology Association (EHA) 2023 Congress.

With the number of treatment options in hematology and oncology rapidly expanding, so too are the strategies for determining and ensuring cost-effectiveness. A session at the European Hematology Association 2023 Congress brought together presenters sharing their research centering on health economics in various disease states and countries.

First, Melina Sophie Kurte, MA, of the University Duisburg-Essen and VITIS Healthcare Group in Cologne, Germany, presented findings from a cost-effectiveness evaluation of third-line interventions for transplant-ineligible relapsed or refractory (R/R) diffuse large B-cell lymphoma from the German payer perspective. As the treatment landscape has changed remarkably in recent years, including the advent of chimeric antigen receptor (CAR) T-cell therapies, there is a need to compare these interventions in terms of their clinical benefit and cost.

Kurte and coauthors noted that CAR T costs were highest due to inpatient administration and drug costs. Their efficiency frontier model determined that gemcitabine/oxaliplatin, tafasitamab plus lenalidomide, and axicabtagene ciloleucel were cost-effective.

“The efficiency frontier can be seen as a guidance tool for our reimbursement decisions for new interventions,” Kurte concluded, acknowledging that these results may change with the emergence of new outcomes data, longer follow-up times, or decreases in treatment costs as patents expire.

Next, Monia Marchetti, MD, of the University of Eastern Piedmont in Alessandria, Italy, presented another angle of CAR T-cell therapy cost-effectiveness: that of brexucabtagene autoleucel for R/R acute lymphoblastic leukemia from the perspective of the Italian public health care system. Using outcome data from numerous trials, she and her colleagues extrapolated and constructed models to yield the incremental costs per quality-adjusted life-year (QALY).

These values were €46,415 ($49,912) vs inotuzumab and €76,384 ($82,139) vs chemoimmunotherapy, both below the €80,000 per QALY threshold indicating cost-effectiveness in Italy. Despite the need to incorporate longer-term follow-up data, Marchetti said, “the acquisition cost of brexucabtagene autoleucel is proportional to the expected QALY gain.”

“The real world is changing quite rapidly, so even these pharmacoeconomic models should be updated quite rapidly,” she suggested.

Moving away from CAR T treatments, next Jun Ho Jang, MD, PhD, of Samsung Medical Center in Seoul, Republic of Korea, took the podium to discuss the efficacy of intravenous (IV) iron treatment for patients with cancer who are experiencing anemia induced by their cancer or chemotherapy. This trial randomly assigned patients 2:1 to receive either IV iron or standard of care and measured their hemoglobin response.

The patients in the treatment arm were significantly more likely to achieve a hemoglobin response by 8 weeks (52.1% vs 32.9%; P = .0039), and their mean hemoglobin increase from baseline was higher as well (1.04 vs 0.42 g/dL; P = .0027).

Considering the significant quality of life and economic burdens stemming from anemia, Jang noted the clear evidence that IV iron monotherapy can achieve these outcomes “without treatment-related serious adverse events in cancer-induced anemia compared with standard of care.” He called for further studies to define the predictive serum hepcidin response threshold for IV iron.

Next up was Elisa Nevalainen, PhD, of IQVIA Nordics in Espoo, Finland, to present findings on persistence with oral therapy for chronic lymphocytic leukemia (CLL) in Swedish patients. She mentioned that real-world patients differ significantly from clinical trial participants in terms of their comorbidities, age, and other factors, so such an analysis was necessary to determine whether patients are continuing on therapy outside of trials.

The treatment with the highest median days of persistence was ibrutinib, followed by venetoclax, acalabrutinib, and idelalisib. At 36 months, the persistence rate was 50% for ibrutinib vs less than 10% for idelasib. Acalabrutinib had a short follow-up period due to its more recent approval, but its 9-month persistence rate was around 90%. The presence of some comorbidities decreased the likelihood of persistence.

“Only the drug you take can help you,” Nevalainen said in regard to the implications of these findings, and she suggested that persistence may be a possible proxy for progression-free survival. An audience member recommended repeating the analysis in 5 years, considering the rapidly evolving chronic lymphocytic leukemia landscape; persistence may also increase as clinicians adapt to the learning curve of how to manage new therapies.

Finally, Keith Pratz, MD, of the University of Pennsylvania in Philadelphia, delivered findings from a long-term follow-up of the VIALE-A trial that looked at the impact of venetoclax plus azacytidine on health-related quality of life (HRQOL) in acute myeloid leukemia (AML). The median follow-up was 43.2 months, and HRQOL data were collected from patient-reported outcomes scales.

Median time to deterioration on physical functioning and emotional functioning subscales was significantly longer with venetoclax plus azacytidine vs placebo and azacytidine. The former regimen consistently showed longer time to deterioration across most patient-reported outcomes in key subgroups vs the placebo regimen. Pratz also mentioned that clinical factors such as complete response and minimal residual disease negativity appear to be linked with the HRQOL outcomes.

“The overall conclusion is that venetoclax appears to have a positive impact on [HRQOL] for AML patients who are elderly or ineligible to receive intensive chemotherapy, leading to a slower deterioration of functioning and overall health status,” Pratz concluded.

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