Budget impact of pasireotide for the treatment of Cushing’s disease, a rare endocrine disorder associated with considerable comorbidities

Figures & data

Figure 1. Patient flow for Cushing’s disease treatments (Scenario 1). TSS, transsphenoidal surgery.

Table 1. Treatment-eligible patient population estimates (Scenario 1).

	Reference	Value
Covered population (third party reimbursement perspective)		1,000,000
Prevalence (patients per million in general population, n)	Etxabe and Vazquez8	39
Total number of CD patients		39
Portion of patients eligible for 1st line TSS	Assumption	75%*
Portion of patients with recurrent CD after surgery	Alwani et al.27	50%
Number of patients with recurrent CD		15
Portion of patients ineligible for 1st line TSS	Assumption	25%*
Number of CD patients ineligible for TSS		10
Total number of CD patients eligible for pasireotide		25^†

*Portion of patients eligible for 1st line TSS may vary across different populations.

^†Value presented has been rounded for demonstration purposes. Unrounded value: 24.38.

CD, Cushing’s disease; TSS, transsphenoidal surgery.

Table 2. Total cost estimates associated with Cushing’s disease treatments (Scenario 1).

Treatment	Annual cost per patient*	Source
Medical procedures
Repeat TSS	$60,310	Calculated using micro-costing methods from Patel et al.34
BLA	$72,525	Calculated using micro-costing methods from Patel et al.34
Radiotherapy	$59,258	Calculated using micro-costing methods from Patel et al.34
Radiosurgery	$45,156	Calculated using micro-costing methods from Patel et al.34
Drug therapies
Pasireotide	$144,280	Based on a monthly cost of $14,383.56 for 60 ampules or 30 days, for an annual cost of $175,000 per year (365 days), regardless of starting dose from Medi-Span Price Rx, March 15, 201349, and incorporates a response rate of 66.3% after 2 months of full treatment.
Mifepristone	$207,562	Fleseriu et al.38; WAC (300 mg tabs) from First Databank50
Ketoconazole	$25,475	Biller et al.2; WAC (200 mg tabs) from Redbook39
Cabergoline	$32,179	Pivonello et al.37; WAC (0.5 mg tabs) from Redbook39
Mitotane	$40,893	Biller et al.2; 2013 WAC (500 mg tabs) from Drug Redbook39

*Includes treatment, complication, adverse event, comorbidity, and monitoring costs. See Supplementary Tables 7–9 for detailed cost data.

BLA, bilateral adrenalectomy; TSS, transsphenoidal surgery; WAC, weighted average cost.

Figure 2. Cushing’s disease budget impact results on a per-member per-month basis (Scenario 1).

Table 3. Cushing’s disease budget impact summary (Scenario 1).

	Total budget impact ($USD)			PMPM (cents)
	2013	2014	2015	2013	2014	2015
Without pasireotide on the market ($)	1,988,980	2,142,497	2,296,015	16.57	17.85	19.13
With pasireotide on the market ($)	2,126,485	2,362,389	2,527,969	17.72	19.69	21.07
Difference ($)	137,505	219,892	231,954	1.15	1.84	1.94

PMPM, per member per month.

Figure 3. Cushing’s disease budget impact model sensitivity analysis (Scenario 1). Input ranges for each parameter are as follows: pasireotide response rate/clinical benefit (60–73%), pasireotide treated share (8–10% in 2013, 14–18% in 2014, 17–21% in 2015), percentage of patients who fail first line TSS (45–55%), patients eligible for first line TSS (68–83%), and pasireotide percentage of controlled disease (50–61%). PMPM, per member per month; TSS, transsphenoidal surgery.

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