Exon-Skipping Therapy for DMD Linked to High Care Costs, Health Care Resource Utilization

Higher health care costs and utilization rates were seen among patients living with Duchenne muscular dystrophy (DMD) who were treated with exon-skipping therapy compared with patients who received branded or generic glucocorticoid treatment or no treatment, according to the results of a comprehensive comparative analysis presented at AMCP Nexus 2023.

Investigators evaluated health care costs and health care resource utilization (HCRU) among 4 groups of patients, stratified by treatment and before/after treatment periods: those receiving exon-skipping therapy (n = 43); those receiving glucocorticoids, branded (n = 172) or generic (n = 149); and a control group not on DMD therapy (n = 68). Data for July 1, 2018, through January 31, 2022 came from the Merative MarketScan Commercial claims database, and data for July 1, 2018, through December 31, 2020, came from Medicaid claims. They noted a lack of research on the economic implications and HCRU rates of glucocorticoid and exon skipping therapy as their reason for initiating this investigation. The index date was either the day a patient began DMD treatment or the diagnosis date if not on treatment.

Patient age ranged from 13 to 19 years, 81.08% of patients reported a White ethnicity (n = 180/222) and 9.46 (n = 21/222) a Black ethnicity, and most had insurance through Medicaid (71.06%).

In the pre-index period, mean (SD) total all-cause office visits were highest in the exon-skipping group at 9.27 (10.41), followed by 6.9 (6.4) in the generic glucocorticoid group, 4.4 (6.4) in the no-treatment group, and 0.3 (8.8) in the branded glucocorticoid group. Corresponding all-cause outpatients visits were 108.1 (134.7), 96.7 (150.9), 102.2 (134.4), and 78.2 (106.6). All-cause emergency department (ED) also were highest in the exon-skipping therapy group and lowest in the no-treatment group, at 0.8 (2.1) vs 0.2 (0.9), respectively. However, the average all-cause length of stay (LOS) was longest in the generic glucocorticoid group, at 4.2 (27.3) and lowest in the branded group, at 0.2 (1.9).

In the postindex period, the following results were seen:

• All-cause office visits: highest in the exon-skipping cohort (9.27 [10.41]) and lowest in the no-treatment group (5.41 [4.26])
• All-cause outpatient visits: highest in the exon-skipping cohort (139.72 [142.30]) and lowest in the branded-glucocorticoid group (7.14 [5.55])
• All-cause ED visits: highest in the generic glucocorticoid group (0.93 [1.70]) and lowest in the exon-skipping group (0.47 [0.77])
• All-cause LOS: highest in the generic glucocorticoid group (1.47 [5.35]) and lowest in the branded glucocorticoid group (0.64 [3.17])

Annual pharmacy spend was exponentially higher in the exon-skipping therapy group in both the pre- and postindex periods, at $10,843 and $648,869, respectively, compared with the following, which the investigators said equated to a 60-fold increase:

• Branded glucocorticoid group: $2540 and $66,218, a 26-fold increase
• Generic glucocorticoid group: $1016 and $2809, a 2-fold increase
• No treatment: $616 and $688

Health Cost Representation Concept | Image Credit: A stockphoto - stock.adobe.com

Higher, too, were annualized medical expenditures among the exon-skipping therapy group in the pre- and postindex periods, respectively:

• Exon-skipping therapy group: $115,934 and $1,010,571, an 8-fold increase
• Branded glucocorticoid group: $22,309 and $91,319, a 4-fold increase
• Generic glucocorticoid group: $30,035 and $35,020, a 1-fold increase
• No treatment: $36,080 and $48,369, a 1-fold increase

Overall, mean annual outpatient costs grew 3-fold in the exon-skipping therapy group, which the study authors attribute to potential use of the treatment in an outpatient setting, whereas those for the remaining patient groups had “only minimal increases,” they noted.

“The study found that the exon-skipping therapy group incurred higher costs and HCRU compared with both the branded and generic glucocorticoid therapy subgroups,” the authors concluded, “as well the subgroup without any DMD treatments, in both the pre- and postindex periods.”

Reference

Scavini M, Posner N, Dorling P, Talaga A, Kane AS, Alvir J. The economic impact of treatment initiation in Duchenne muscular dystrophy: a retrospective US claims analysis. Presented at: AMCP Nexus 2023; AMCP Nexus 2023; October 16-October 19, 2023; Orlando, Florida.