ISPOR 2025
Tuesday, May 13 - Friday, May 16
Participation in a Patient Support Program is Associated With Improved Access to Efgartigimod Treatment for Generalized Myasthenia Gravis in Patients Facing Social Determinants of Health (SDOH) Challenges
Author(s)
Tom Hughes, BSc, PhD1, Dominic Robert Nunag, B.S.2, Avery Mohan, B.A.2, Matthew Davis, M.A.2, Glenn Phillips, Ph.D.3.
1Director, argenx, Boston, MA, USA, 2Medicus Economics, Boston, MA, USA, 3argenx, Boston, MA, USA.
1Director, argenx, Boston, MA, USA, 2Medicus Economics, Boston, MA, USA, 3argenx, Boston, MA, USA.
Presentation Documents
OBJECTIVES: A manufacturer-provided patient support program (PSP) for efgartigimod (EFG) assists patients initiating treatment for generalized myasthenia gravis, but evidence is limited on its effectiveness. This study quantifies the PSP’s impact on treatment access, especially within patients facing SDOH challenges
METHODS: Specialty pharmacy and PSP data from December 2021-May 2024 were linked to identify PSP participation within 30 days of an initial EFG script (index). Baseline characteristics were assessed using patients’ index script and ≥3 months of follow-up observation was required. SDOH subgroups with access challenges historically included patients in the Midwest, with Medicaid coverage, and with non-specialist prescribers. Access outcomes, including initial script approval, successful dispensing, and time to dispense were compared between PSP and non-PSP cohorts. Multivariate regressions controlling for baseline characteristics estimated risk-adjusted outcomes, odds ratios, and rate ratios.
RESULTS: Among 3,199 patients meeting the inclusion criteria, 64% participated in the PSP (n = 2,061 PSP; n = 1,138 non-PSP) and baseline characteristics were similar across cohorts. PSP participants had 21% higher adjusted odds of initial script approval (53.5% vs. 48.9%; p=0.01), 47% higher odds of receiving a dispense (61.6% vs. 52.5%; p<0.01), and 30% lower odds of never receiving approval (29.5% vs. 37.3%; p<0.01) compared to non-PSP patients. Additionally, PSP patients were 56% more likely to receive a dispense within 3 months (53.6% vs. 42.8%; p<0.01), had a 29% faster time to coverage decision (31.2 vs. 44.1 days; p<0.01), and a 27% faster time to dispense (36.5 vs. 50.0 days; p<0.01). The relative benefits of PSP participation on coverage approval were greatest within three subgroups facing SDOH challenges: 1.5x, 1.9x, and 3.0x higher for patients in the Midwest, with Medicaid coverage, and with non-specialist prescribers, respectively.
CONCLUSIONS: PSP participation may help patients initiate EFG sooner and more successfully, especially patients facing SDOH challenges.
METHODS: Specialty pharmacy and PSP data from December 2021-May 2024 were linked to identify PSP participation within 30 days of an initial EFG script (index). Baseline characteristics were assessed using patients’ index script and ≥3 months of follow-up observation was required. SDOH subgroups with access challenges historically included patients in the Midwest, with Medicaid coverage, and with non-specialist prescribers. Access outcomes, including initial script approval, successful dispensing, and time to dispense were compared between PSP and non-PSP cohorts. Multivariate regressions controlling for baseline characteristics estimated risk-adjusted outcomes, odds ratios, and rate ratios.
RESULTS: Among 3,199 patients meeting the inclusion criteria, 64% participated in the PSP (n = 2,061 PSP; n = 1,138 non-PSP) and baseline characteristics were similar across cohorts. PSP participants had 21% higher adjusted odds of initial script approval (53.5% vs. 48.9%; p=0.01), 47% higher odds of receiving a dispense (61.6% vs. 52.5%; p<0.01), and 30% lower odds of never receiving approval (29.5% vs. 37.3%; p<0.01) compared to non-PSP patients. Additionally, PSP patients were 56% more likely to receive a dispense within 3 months (53.6% vs. 42.8%; p<0.01), had a 29% faster time to coverage decision (31.2 vs. 44.1 days; p<0.01), and a 27% faster time to dispense (36.5 vs. 50.0 days; p<0.01). The relative benefits of PSP participation on coverage approval were greatest within three subgroups facing SDOH challenges: 1.5x, 1.9x, and 3.0x higher for patients in the Midwest, with Medicaid coverage, and with non-specialist prescribers, respectively.
CONCLUSIONS: PSP participation may help patients initiate EFG sooner and more successfully, especially patients facing SDOH challenges.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR95
Topic
Health Policy & Regulatory
Topic Subcategory
Health Disparities & Equity
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases