Cost-Effectiveness of Acthar Gel versus Standard of Care for the Treatment of Advanced Symptomatic Sarcoidosis

Figures & data

Figure 1 Schematic of the probabilistic cohort-level state-transition model.

Note: Response was assessed based on sarcoidosis treatment score.

Table 1 Clinical Parameters Among Patients with Symptomatic Pulmonary Sarcoidosis

Parameter	Proportion of Patients
Clinical response	No Response	Partial Response	Complete Response
Acthar Gel
3 months	44.4%	37.0%	18.5%
6 months	40.7%	29.6%	29.6%
Standard of care
3 months	60.7%	21.4%	17.9%
6 months	53.6%	28.6%	17.9%
Lung function (Forced vital capacity percent predicted)^a	No Response	Partial Response	Complete Response
60.0–69.9	33.3%	20.0%	16.7%
50.0–59.9	16.7%	20.0%	0.0%
Less than 50	11.1%	0.0%	0.0%
Pain^b	No Response	Partial Response	Complete Response
Mild	27.8%	66.7%	75.0%
Moderate	50.0%	13.3%	16.7%
Severe	22.2%	0.0%	0.0%
Depression^c	No Response	Partial Response	Complete Response
Mild	33.3%	26.7%	33.3%
Moderate	33.3%	6.7%	16.7%
Severe	5.6%	6.7%	0.0%
3-month probability^d	Acthar Gel	Standard of care
Partial response to no response	0.200	0.333
Partial response to partial response	0.400	0.500
Partial response to complete response	0.400	0.167
Complete response to no response	0.000	0.200
Complete response to partial response	0.600	0.400
Complete response to complete response	0.400	0.400

Notes: ^aPatients with forced vital capacity percent predicted ≥70 not included. ^bPatients with “no pain” not included. ^cPatients with “no depression” not included. ^dThis only considers the movement of patients between response states and from a response state to a no-response state. The probability of transition from a no-response state to a response state is based on clinical response probability; patients moving from no-response to a response state in any model cycle are assumed to have the same probability of clinical response as that of the first cycle. Source: Data was derived from the PULSAR trial.

Table 2 Healthcare Resource Use and Cost Inputs

Parameter	Value			Reference
Treatment costs
Cost of Acthar Gel	$42,703			Red Book 2023^Citation22
Acthar Gel use (12 months)	9.27 packs			Data on file^a
Other medications
Medication utilization	No Response	Partial Response
Acthar Gel
Oral corticosteroids	25.5%	12.9%		Chopra 2019^Citation16
Biologics	10.3%	4.3%		Chopra 2019^Citation16
Antimalarial	12.6%	6.0%		Chopra 2019^Citation16
Immunosuppressants	11.9%	6.6%		Chopra 2019^Citation16
Standard of care
Oral corticosteroids	95.5%	95.5%		PULSAR trial^Citation23
Biologics	24.0%	24.0%		Chopra 2019^Citation16
Antimalarial	22.7%	22.7%		PULSAR trial^Citation23
Immunosuppressants	63.6%	63.6%		PULSAR trial^Citation23
Annual cost of medications
Oral corticosteroids	$2326			El Jammal 2020;^Citation24 Drugs.com^Citation25
Biologics	$63,694			El Jammal 2020;^Citation24 Drugs.com^Citation25
Antimalarial	$404			El Jammal 2020;^Citation24 Drugs.com^Citation25
Immunosuppressants	$2262			El Jammal 2020;^Citation24 Drugs.com^Citation25
Sarcoidosis-related annual healthcare costs
Inpatient	$28,634			Baughman 2016^Citation5
Physician office	$719			Baughman 2016^Citation5
Laboratory	$182			Baughman 2016^Citation5
Outpatient	$2049			Baughman 2016^Citation5
Emergency department	$1027			Baughman 2016^Citation5
Home Health	$3153			Baughman 2016^Citation5
Durable medical equipment	$1839			Baughman 2016^Citation5
Other	$3397			Baughman 2016^Citation5
Respiratory insufficiency
Lung transplant costs (per transplant)	$496,061			Peel 2021^Citation26
Extrapulmonary organ involvement annual costs
Skin	$9309			Tripathi 2019^Citation27
Cardiac	$12,238			Foy 2018^Citation28
Eyes	$1599			Rein 2006^Citation29
Bones/Joints	$20,845			Williams 2020^Citation30
Liver	$2858			Nguyen 2019^Citation31
Pain-related annual costs
Mild	$13,158			Lalonde 2014^Citation32
Moderate	$18,779			Lalonde 2014^Citation32
Severe	$17,034			Lalonde 2014^Citation32
Sarcoidosis-related opioid abuse	$52,643			White 2005^Citation33
Depression-related annual costs
Mild	$7810			Chow 2019^Citation34
Moderate	$9811			Chow 2019^Citation34
Severe	$13,691			Chow 2019^Citation34
Corticosteroid toxicity
Corticosteroid dosage group	No Response	Partial Response	Complete Response
Intermittent use	5.6%	20.0%	25.0%	PULSAR trial^Citation23
Low dose	38.9%	46.7%	50.0%	PULSAR trial^Citation23
Moderate dose	38.9%	33.3%	25.0%	PULSAR trial^Citation23
High dose	16.7%	0.0%	0.0%	PULSAR trial^Citation23
Corticosteroid-related toxicity annual costs
Intermittent use	$40,134			Rice 2018^Citation10
Low dose	$39,161			Rice 2018^Citation10
Moderate dose	$43,581			Rice 2018^Citation10
High dose	$83,995			Rice 2018^Citation10

Note: ^aUsing dispensing data from specialty pharmacies, from the last 12 months as of March 29, 2019.

Table 3 Health Utilities and Disutilities

Parameter	Value	Reference
EQ-5D utility
No response	0.215	PULSAR trial^Citation23
Partial response	0.612	PULSAR trial^Citation23
Complete response	0.680	PULSAR trial^Citation23
Disutilities
Lung transplant	−0.510	Anyanwu 2001^Citation48
Extrapulmonary organ involvement
Skin	−0.057	Chen 2004^Citation49
Cardiac	−0.076	Di Tanna 2021^Citation50
Eyes	−0.220	Brown 2009^Citation51
Bone or joint	−0.030	Törmälehto 2018^Citation52
Liver	−0.130	Dan 2008^Citation53
Chronic medication use
Chronic oral corticosteroid use	−0.023	ICER 2018^Citation54
Substance use disorder	−0.132	ICER 2018^Citation55
Pain
Mild	−0.170	Wetherington 2014^Citation56
Moderate	−0.400	Wetherington 2014^Citation56
Severe	−0.430	Wetherington 2014^Citation56
Depression
Mild	−0.130	Kolovos 2017^Citation57
Moderate	−0.180	Kolovos 2017^Citation57
Severe	−0.310	Kolovos 2017^Citation63

Abbreviation: EQ-5D, EuroQol five-dimension.

Table 4 Base Case Results for Incremental Cost-Effectiveness Among Patients with Symptomatic Sarcoidosis (2023 USD)

Acthar Gel Versus SoC	Incremental Costs	Incremental QALYs	ICER (Incremental Cost per QALY)
Payer perspective
2 years	$65,915	0.489	$134,796
3 years	$29,306	0.748	$39,179
Societal perspective
2 years	$57,517	0.489	$117,622
3 years	$16,431	0.748	$21,967

Notes: Base case analysis considered ICER over 2 years from the payer perspective. Results are presented on a per-person basis.

Abbreviations: ICER, incremental cost-effectiveness ratio; QALY, quality-adjusted life years; SoC, standard of care; USD, United States Dollars.

Table 5 Base Case per Patient-Year Costs Among Patients with Symptomatic Sarcoidosis Over 2 Years (2023 USD)

Cost Component	Acthar Gel	Standard of Care
Treatment	$254,882	$0
Direct Medical
Sarcoidosis-related	$29,121	$34,668
Lung transplant	$102,627	$192,300
Concomitant medications	$5729	$26,142
Corticosteroid-related toxicity	$10,018	$64,273
Extrapulmonary organ involvement	$4398	$8241
Pain	$53,643	$67,789
Depression	$8630	$9720
Total direct medical	$214,166	$403,133
Indirect costs
Disability	$1453	$1775
Disability-related work training	$3766	$5300
Substance use disorder	$13,654	$19,472
Caregiving	$1776	$2500
Total indirect	$20,649	$29,047

Abbreviation: USD, United States Dollars.

Figure 2 Tornado chart: DSA comparing Acthar Gel and standard of care. DSA was performed for analysis comparing a time horizon of 2 years and a payer perspective.

Abbreviations: DSA, deterministic sensitivity analysis; QALY, quality-adjusted life-year.

Figure 3 Cost-effectiveness plane comparing Acthar Gel and standard of care.

Note: Base case is presented [Time horizon of 2 years and a payer perspective].

Abbreviations: QALY, quality-adjusted life-year; USD, United States dollars.

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