Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease

**Background:** “On-demand” treatments approved in the United States (US) for “OFF” episodes in Parkinson’s disease (PD) include apomorphine hydrochloride injection (SC-APO), apomorphine sublingual film (APL), and levodopa inhalation powder (CVT-301). APL received US approval in 2020, and its cost-e...

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Autores principales: Andrew Thach, Noam Kirson, Miriam L. Zichlin, Ibrahima Dieye, Eric Pappert, G. Rhys Williams
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Publicado: Columbia Data Analytics, LLC 2021
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spelling oai:doaj.org-article:a5f68444c1634208b50b603887e8c7cb2021-11-17T13:59:04ZCost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease10.36469/jheor.2021.294882327-2236https://doaj.org/article/a5f68444c1634208b50b603887e8c7cb2021-11-01T00:00:00Zhttps://jheor.scholasticahq.com/article/29488-cost-effectiveness-of-apomorphine-sublingual-film-as-an-on-demand-treatment-for-off-episodes-in-patients-with-parkinson-s-disease.pdfhttps://doaj.org/toc/2327-2236**Background:** “On-demand” treatments approved in the United States (US) for “OFF” episodes in Parkinson’s disease (PD) include apomorphine hydrochloride injection (SC-APO), apomorphine sublingual film (APL), and levodopa inhalation powder (CVT-301). APL received US approval in 2020, and its cost-effectiveness has not been compared with SC-APO and CVT-301. **Objective:** To develop a cost-effectiveness analysis model comparing APL versus SC-APO and CVT-301 for treatment of patients with PD experiencing “OFF” episodes from a US payer perspective. **Methods:** The model estimated total costs and effectiveness for each comparator arm, informed from the treatments’ pivotal studies or literature, over a 10-year horizon. Total and incremental patient costs (in 2020 US dollars), total time spent without “OFF” episode symptoms, and quality-adjusted life years (QALY) gained were summarized and compared. Incremental cost-effectiveness ratios for APL versus SC-APO and CVT-301 were estimated and expressed as incremental patient costs per patient QALY gained and incremental cost per “OFF” hour avoided. Scenario analyses varying inputs and including caregiver costs were also conducted. **Results:** In the base case, APL had the lowest total “on-demand” treatment costs (\$42,095) compared with SC-APO (\$276,320; difference: –\$234,225) and CVT-301 (\$69,577; difference: –\$27,482) over the 10-year horizon. APL was also associated with the highest utility, with incremental QALYs of 0.019 versus SC-APO and 0.235 versus CVT-301. APL was dominant over CVT-301 in terms of incremental cost per “OFF” hour, and dominant over both CVT-301 and SC-APO in terms of incremental cost per QALY gained. In all scenario analyses, APL was dominant against both SC-APO and CVT-301, confirming the robustness of the base-case results. **Discussion:** APL was dominant compared with both comparator arms, being less costly and more effective on average than SC-APO and CVT-301 in terms of QALYs. For SC-APO, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and adverse event–related disutilities. For CVT-301, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and substantially higher efficacy. **Conclusions:** From a US payer perspective, APL represents a cost-effective option compared with SC-APO and CVT-301 for treatment of “OFF” episodes in patients with PD.Andrew ThachNoam KirsonMiriam L. ZichlinIbrahima DieyeEric PappertG. Rhys WilliamsColumbia Data Analytics, LLCarticleComputer applications to medicine. Medical informaticsR858-859.7ENJournal of Health Economics and Outcomes Research (2021)
institution DOAJ
collection DOAJ
language EN
topic Computer applications to medicine. Medical informatics
R858-859.7
spellingShingle Computer applications to medicine. Medical informatics
R858-859.7
Andrew Thach
Noam Kirson
Miriam L. Zichlin
Ibrahima Dieye
Eric Pappert
G. Rhys Williams
Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
description **Background:** “On-demand” treatments approved in the United States (US) for “OFF” episodes in Parkinson’s disease (PD) include apomorphine hydrochloride injection (SC-APO), apomorphine sublingual film (APL), and levodopa inhalation powder (CVT-301). APL received US approval in 2020, and its cost-effectiveness has not been compared with SC-APO and CVT-301. **Objective:** To develop a cost-effectiveness analysis model comparing APL versus SC-APO and CVT-301 for treatment of patients with PD experiencing “OFF” episodes from a US payer perspective. **Methods:** The model estimated total costs and effectiveness for each comparator arm, informed from the treatments’ pivotal studies or literature, over a 10-year horizon. Total and incremental patient costs (in 2020 US dollars), total time spent without “OFF” episode symptoms, and quality-adjusted life years (QALY) gained were summarized and compared. Incremental cost-effectiveness ratios for APL versus SC-APO and CVT-301 were estimated and expressed as incremental patient costs per patient QALY gained and incremental cost per “OFF” hour avoided. Scenario analyses varying inputs and including caregiver costs were also conducted. **Results:** In the base case, APL had the lowest total “on-demand” treatment costs (\$42,095) compared with SC-APO (\$276,320; difference: –\$234,225) and CVT-301 (\$69,577; difference: –\$27,482) over the 10-year horizon. APL was also associated with the highest utility, with incremental QALYs of 0.019 versus SC-APO and 0.235 versus CVT-301. APL was dominant over CVT-301 in terms of incremental cost per “OFF” hour, and dominant over both CVT-301 and SC-APO in terms of incremental cost per QALY gained. In all scenario analyses, APL was dominant against both SC-APO and CVT-301, confirming the robustness of the base-case results. **Discussion:** APL was dominant compared with both comparator arms, being less costly and more effective on average than SC-APO and CVT-301 in terms of QALYs. For SC-APO, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and adverse event–related disutilities. For CVT-301, cost-effectiveness of APL was driven by lower “on-demand” treatment costs and substantially higher efficacy. **Conclusions:** From a US payer perspective, APL represents a cost-effective option compared with SC-APO and CVT-301 for treatment of “OFF” episodes in patients with PD.
format article
author Andrew Thach
Noam Kirson
Miriam L. Zichlin
Ibrahima Dieye
Eric Pappert
G. Rhys Williams
author_facet Andrew Thach
Noam Kirson
Miriam L. Zichlin
Ibrahima Dieye
Eric Pappert
G. Rhys Williams
author_sort Andrew Thach
title Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
title_short Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
title_full Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
title_fullStr Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
title_full_unstemmed Cost-Effectiveness of Apomorphine Sublingual Film as an “On-Demand” Treatment for “OFF” Episodes in Patients with Parkinson's Disease
title_sort cost-effectiveness of apomorphine sublingual film as an “on-demand” treatment for “off” episodes in patients with parkinson's disease
publisher Columbia Data Analytics, LLC
publishDate 2021
url https://doaj.org/article/a5f68444c1634208b50b603887e8c7cb
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