The estimated costs and benefits of a hypothetical supervised consumption site in Providence, Rhode Island.

Background: Overdose deaths have increased dramatically in the United States, including in Rhode Island. In July 2021, the Rhode Island government passed legislation supporting a two-year pilot program authorizing supervised consumption sites (SCSs) in response to this crisis. We estimated the costs and benefits of a hypothetical SCS in Providence, Rhode Island.

Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model.

Aims: Hemophilia B (HB) is a rare congenital disorder characterized by bleeding-related complications which are managed by prophylactic or post-bleeding event ("on-demand") replacement of clotting factor IX (FIX). The standard of care for severe HB is life-long prophylaxis with standard half-life (SHL) or extended half-life (EHL) products given every 2-3 or 7-14 days, respectively. FIX treatment costs in the US have been investigated, but the lifetime costs of HB treatment have not been well characterized, particularly related to the impact of joint health deterioration and associated health resource utilization.

Estimating the cost-effectiveness of an infant 13-valent pneumococcal conjugate vaccine national immunization program in China.

Background: The burden of pneumococcal disease in China is high, and a 13-valent pneumococcal conjugate vaccine (PCV13) recently received regulatory approval and is available to Chinese infants. PCV13 protects against the most prevalent serotypes causing invasive pneumococcal disease (IPD) in China, but will not provide full societal benefits until made broadly available through a national immunization program (NIP).

Objective: To estimate clinical and economic benefits of introducing PCV13 into a NIP in China using local cost estimates and accounting for variability in vaccine uptake and indirect (herd protection) effects.

Next-Generation Sequencing Panels for the Diagnosis of Colorectal Cancer and Polyposis Syndromes: A Cost-Effectiveness Analysis.

Purpose: To evaluate the cost effectiveness of next-generation sequencing (NGS) panels for the diagnosis of colorectal cancer and polyposis (CRCP) syndromes in patients referred to cancer genetics clinics.

Patients And Methods: We developed a decision model to evaluate NGS panel testing compared with current standard of care in patients referred to a cancer genetics clinic. We obtained data on the prevalence of genetic variants from a large academic laboratory and calculated the costs and health benefits of identifying relatives with a pathogenic variant, in life-years and quality-adjusted life-years (QALYs).

A model of cost-effectiveness of tissue plasminogen activator in patient subgroups 3 to 4.5 hours after onset of acute ischemic stroke.

Study Objective: The European Cooperative Acute Stroke Study III (ECASS III) showed that recombinant tissue plasminogen activator (rtPA) administered 3 to 4.5 hours after acute ischemic stroke led to improvement in patient disability versus placebo. We evaluate the long-term incremental cost-effectiveness of rtPA administered 3 to 4.

Meeting the governance challenges of next-generation biorepository research.

Advances in clinical translational research have led to an explosion of interest in infrastructure development and data sharing facilitated by biorepositories of specimens and linked health information. These efforts are qualitatively different from the single-center sample collections that preceded them and pose substantial new ethics and regulatory challenges for investigators and institutions. New research governance approaches, which can address current and anticipated challenges, promote high-quality research, and provide a robust basis for ongoing research participation, are urgently required.

A novel GLRA1 mutation associated with an atypical hyperekplexia phenotype.

Hyperekplexia (MIM #149400) is a rare neurological disorder characterized by an exaggerated startle response, infantile hypertonia and hyperreflexia without spasticity, a hesitant gait that usually improves by 3 years of age, and nocturnal myoclonus. Familial hyperekplexia is usually autosomal dominant resulting from mutations in the inhibitory glycine receptor subunit alpha 1 (GLRA1) gene on chromosome 5q. We identified a 3-generation family with progressively severe phenotypes of hyperekplexia.