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

Abstract

Vol. 17, No. 4, 2014, 288-295 Cushing's disease (CD) is a rare condition with a prevalence of roughly 39 cases per million in the general population. Healthcare costs are substantial for CD patients with either untreated or inadequately controlled disease. This study assesses the 3-year budget impact of pasireotide on a US managed care health plan following pasireotide (Signifor*) availability. Methods:Two scenarios were evaluated to understand the differences in costs associated with the introduction of pasireotide. The first scenario evaluates the budget impact of pasireotide from the perspective of an entire health plan (total budget impact) and the second from the perspective of the pharmacy budget (pharmacy budget impact). Both scenarios evaluate the annual incremental budget impact with and without pasireotide. Scenario 1 includes costs for medical procedures, drug therapies, monitoring, surgical complications, comorbidities for patients with controlled or uncontrolled CD, and adverse events. Procedures include transsphenoidal surgery, bilateral adrenalectomy, radiotherapy and radiosurgery. Drugs include pasireotide (indicated for CD), mifepristone (indicated to control hyperglycemia secondary to hypercortisolism in patients with Cushing's syndrome) as well as several off-label treatments (ketoconazole, cabergoline, mitotane). Scenario 2 considers costs solely from the perspective of a health plan pharmacy. Costs are in $2013. Results:The estimated total budget impact is $0.0115 per-member per-month (PMPM) in the first year following FDA approval, $0.0184 in the second year, and $0.0194 in the third year. Introduction of pasireotide is expected to increase the pharmacy budget by $0.0257 PMPM in the first year, $0.0363 in the second year, and $0.0360 in the third year. Limitations:Model inputs rely on the small body of literature available for Cushing's disease. Conclusions:Cushing's disease is severe disease with debilitating comorbidities and substantial healthcare costs when untreated or inadequately controlled. The inclusion of pasireotide in a health plan formulary appears to have only a small impact on the total health plan or pharmacy budget.

A Methodological Review of US Budget-Impact Models for New Drugs

“…Ten of the studies included acquisition only [30, 32-35, 37, 38, 40, 42, 43]; two studies included acquisition, administration, and monitoring [31,44]; one study included acquisition, adverse events, and monitoring [36]; two studies included acquisition and administration [39,46]; and two studies included acquisition and monitoring [41,45]. Of those studies that did not include administration costs, nine were studies of oral drugs [30, 32-35, 38, 40, 42, 45] and three were studies with the same administration method [37,41,43], but one study [36] compared a subcutaneously administered drug with oral drugs and should have included administration costs. Only one study included adverse events [36], and five studies included monitoring [31,36,41,44,45].…”

“…In these studies, the drug for which the budget impact was estimated took treatment share from all drugs (six studies) [33,35,36,42,45,46] or one drug (four studies) [31,38,40,43]. In one study, the drug was added to current therapy [39], and the treatment shares before and after introduction of a new drug were taken from a database analysis in two studies [30,32].…”

“…In one study, the drug was added to current therapy [39], and the treatment shares before and after introduction of a new drug were taken from a database analysis in two studies [30,32]. Two of the studies that took treatment shares from all drugs assumed equi-proportionate contributions of the current drugs to the treatment share for the new drug [36,42]. In addition, the base case in the Berenson et al [43] study compared treatment with the drug of interest for all patients to treatment with one of two other drugs for all patients.…”

“…The fifth decision-analytic model [43] Although these studies focused on chronic conditions, the time horizon assumed was 1 year in seven of the 12 cost-calculator models [31,32,34,37,38,40,41] and only 6 months in one of the cost-calculator models using data from a database analysis [30], implicitly making the assumption that the budget impact would be the same for subsequent periods. In the other four cost-calculator models [33,35,36,39] and the five decision-analytic models [42][43][44][45][46], time horizons varied from 2 to 7 years.…”

“…Of the 17 analyses in which the new drug would not change the mortality or disease progression rates within the model time horizon, 12 used a simple cost-calculator model structure [30][31][32][33][34][35][36][37][38][39][40][41] and five used formal decision-analytic model structures [42][43][44][45][46]. The cost-calculator approach can be appropriate for chronic conditions when the mortality and/or disease progression rates remain constant, and this approach generally has the advantage of being simpler and more transparent for the user.…”