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

Abstract

Objectives:

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.

Keywords::

• Budget-impact
• Cushing’s disease
• Orphan disease

Transparency

Declaration of funding

This research was sponsored by Novartis Pharmaceuticals Corporation.

Declaration of financial/other relationships

H.L.T. and D.N. are employees of Analysis Group and have disclosed receiving consulting fees from Novartis Pharmaceuticals. M.P.N. and W.H.L. have disclosed that they are employees of Novartis and are stock option share-holders of Novartis Pharmaceuticals. The authors have disclosed that they have received no additional support or contributions from others in the preparation of this manuscript. The authors also declare that they have no competing interests. CMRO Peer Reviewers on this manuscript have received honorarium for their review work, but have no other relevant financial relationships to disclose.

Notes

*Signifor is a registered trademark of Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.