The introduction of direct-acting antivirals (DAAs) represents a potential clinical cure for hepatitis C virus (HCV) infection. Identification of costs associated with different stages of untreated disease through cost-of-illness (COI) evaluation helps inform policy decisions and cost-effectiveness analyses (CEAs). This study’s objective was to review published real-world costs for patients with HCV to estimate the COI across different stages of disease progression. A literature search of EMBASE...
The Orphan Drug Act extends exclusivity of branded drugs by 7 years for each rare disease approval. By extending market exclusivity, manufacturers can forestall generic competition. We determined the prevalence of drugs with multiple orphan approvals, the duration for which manufacturers are able to maintain exclusivity using this mechanism, and the budget impact of these additional exclusivity periods on US spending on orphan drugs. We analyzed a retrospective cohort of US orphan drug approvals...
BACKGROUND Paroxysmal nocturnal hemoglobinuria, characterized by intravascular hemolysis and venous thrombosis, can be managed with eculizumab, an inhibitor of the complement system; however, patients may periodically experience breakthrough hemolysis. Ravulizumab is a newly approved treatment for paroxysmal nocturnal hemoglobinuria that may reduce breakthrough hemolysis risk, thus improving health-related quality of life and reducing treatment costs. OBJECTIVE The objective of this study was to...
GW Research Ltd. provided two separate, but similar, submissions to the National Institute for Health and Care Excellence (NICE) on the clinical and cost-effectiveness of cannabidiol (CBD) 10 mg/kg/day, trade name Epidyolex®, for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS). This paper highlights important methodological issues related to the company submissions, the Evidence Review Group (ERG) reports, and the subsequent development...
#2W. Ken Redekop(EUR: Erasmus University Rotterdam)H-Index: 12
Last. Carl V. Asche(UIC: University of Illinois at Chicago)H-Index: 25
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The development of evidence to demonstrate 'value for money' is regarded as an important step in facilitating the search for the optimal allocation of limited resources and has become an essential component in healthcare decision making. Real-world evidence collected from de-identified individuals throughout the continuum of healthcare represents the most valuable source in technology evaluation. However, in the European Union, the value assessment based on real-world data has become challenging...
The current US drug innovation financing framework rests on the notion that a defined period of marketing exclusivity combined with the expectation of reimbursement for clinically valuable, cost-effective therapies, followed by vigorous price competition from generic drugs and biosimilars ensures a sufficient return on investment (ROI) to incent private sector risk-based investment and research and development activities while providing access for new treatments to patients. While periodically, ...
INTRODUCTION: Cannabinoid oils are being increasingly used to treat Dravet syndrome, yet the long-term costs and outcomes of this approach are unknown. Thus, we examined the cost effectiveness of cannabinoid oil as an adjunctive treatment (added to clobazam and valproate), compared with adjunctive stiripentol or with clobazam and valproate alone, for the treatment of Dravet syndrome in children. METHODS: We performed a probabilistic cost-utility analysis from the perspective of the Canadian publ...
BACKGROUND: Economic modeling is widely used in estimating cost-effectiveness in type 2 diabetes mellitus. Because type 2 diabetes is complex and patients are heterogenous, the cohort modeling approach may generate biased estimates of costeffectiveness. The IHE Diabetes Cohort Model (IHE-DCM) was constructed using the cohort approach as an alternative for stakeholders with limited resources, some of whom have voiced reasonable concerns about a lack of transparency with type 2 diabetes micro-simu...