Gene Therapy Equity in Sickle Cell Disease: Distributional Cost-Effectiveness Analysis (DCEA) of Gene Therapy Vs. Standard-of-Care in Patients with Sickle Cell Disease in the United States

Goshua, George; Calhoun, Cecelia; Cheng, Vivien; James, Lyndon; Luviano, Andrea; Krishnamurti, Lakshmanan; Pandya, Ankur

doi:10.1182/blood-2022-157460

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“…Novel therapies such as transplant and gene therapy have the potential to lower expenditures, especially if pursued by those who are super-utilizers with high rates of vaso-occlusive pain episodes, because data suggest these can reduce vaso-occlusive pain episodes that require short-term health care use. 29 , 34 , 35 However, there are few disease-modifying therapies that have been shown to significantly lower the comorbidity burden of SCD. 36 Additional research is needed to examine whether these novel therapies are also able to improve or reverse costly SCD comorbidities.…”

Section: Discussionmentioning

confidence: 99%

Comparing super-utilizers and lower-utilizers among commercial- and Medicare-insured adults with sickle cell disease

MacEwan,

Chiang,

O’Brien

et al. 2023

Blood Advances

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Sickle cell disease (SCD) is a rare but costly condition in the US. Super-utilizers have been defined as a subset of the population with high healthcare encounters or expenditures. While super-utilizers have been described in other disease states, little is known about super-utilizers among adults with SCD. This study aimed to characterize differences in expenditures, overall healthcare encounters, and pain episode encounters between super-utilizers (top 10% expenditures) and lower-utilizers with SCD (high: top 10-24.9%, moderate: 25-49.9%, and low: bottom 50% expenditures). A retrospective longitudinal cohort of adults with SCD were identified using validated algorithms in MarketScan and Medicare claims databases from 2016-2020. Encounters and expenditures were analyzed from inpatient, outpatient, and emergency department settings. Differences in encounters and expenditures between lower- and super-utilizers were compared with logistic regression. Among super-utilizers, differences in encounters and expenditures were compared according to incidence of pain episode encounters. The study population included 5,666 commercial-insured and 8,600 Medicare-insured patients. Adjusted total annual healthcare expenditure was 43.46 times higher for super-utilizers as compared to low-utilizers among commercial-insured and 13.37 times higher in Medicare-insured patients. Among super-utilizers, there were patients with few pain episode encounters who had higher outpatient expenditures compared to patients with a high number of pain episode encounters. Our findings demonstrate the contribution of expensive outpatient care among SCD super-utilizers, where analyses of high expenditure have largely focused on acute care. Future studies are needed to better understand super-utilizers in the SCD population to inform the effective use of preventative interventions and/or curative therapies.

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