The prognostic value of peak oxygen uptake in obstructive hypertrophic cardiomyopathy: a literature review to inform economic model development.

Aims: Patients with obstructive hypertrophic cardiomyopathy (oHCM) experience significant clinical burden which is associated with a high economic burden. Peak oxygen uptake (pVO2), measured by cardiopulmonary exercise testing, is used to quantify functional capacity, and has been studied as a primary endpoint in recent clinical trials. This study aimed to gather evidence to consolidate the prognostic value of pVO2 in oHCM and to assess whether it is feasible to predict health outcomes in an economic model based on changes in pVO2.

Cost-Effectiveness Analysis of a Prescription Digital Therapeutic in Type 2 Diabetes.

Introduction: BT-001 (AspyreRx™) prescription digital therapy, a form of personalized cognitive behavioral therapy, has demonstrated clinically meaningful and durable hemoglobin A1c reductions in patients with type 2 diabetes (T2D). The current study examined the cost-effectiveness of BT-001 plus standard of care (SoC) versus SoC alone in T2D over a lifetime horizon from a healthcare payer perspective.

Methods: We modeled the T2D pathway using an individual patient-level simulation; clinical data were sourced from the intention-to-treat subset of the BT-001 randomized clinical trial (RCT).

R and Shiny for Cost-Effectiveness Analyses: Why and When? A Hypothetical Case Study.

Introduction: Health economics models are typically built in Microsoft Excel owing to its wide familiarity, accessibility and perceived transparency. However, given the increasingly rapid and analytically complex decision-making needs of both the pharmaceutical industry and the field of health economics and outcomes research (HEOR), the demands of cost-effectiveness analyses may be better met by the programming language R.

Objective: This case study provides an explicit comparison between Excel and R for contemporary cost-effectiveness analysis.

A Picture is Worth a Thousand Words: The Role of Survey Training Materials in Stated-Preference Studies.

Background: Online survey-based methods are increasingly used to elicit preferences for healthcare. This digitization creates an opportunity for interactive survey elements, potentially improving respondents' understanding and/or engagement.

Objective: Our objective was to understand whether, and how, training materials in a survey influenced stated preferences.

A Micro-Costing Study of Screening for Lynch Syndrome-Associated Pathogenic Variants in an Unselected Endometrial Cancer Population: Cheap as NGS Chips?

Lynch syndrome is the most common inherited cause of endometrial cancer. Identifying individuals affected by Lynch syndrome enables risk-reducing interventions including colorectal surveillance, and cascade testing of relatives. We conducted a micro-costing study of screening all women with endometrial cancer for Lynch syndrome using one of four diagnostic strategies combining tumor microsatellite instability testing (MSI), immunohistochemistry (IHC), and/or methylation testing, and germline next generation sequencing (NGS).

Generating EQ-5D-3L Utility Scores from the Dermatology Life Quality Index: A Mapping Study in Patients with Psoriasis.

Objectives: To develop an algorithm to predict the three-level EuroQol five-dimensional questionnaire (EQ-5D-3L) utility scores from the Dermatology Life Quality Index (DLQI) in psoriasis.

Methods: This mapping study used data from the British Association of Dermatologists Biologic Interventions Register-a pharmacovigilance register comprising patients with moderate to severe psoriasis on systemic therapies. Conceptual overlap between the EQ-5D-3L and DLQI was assessed using Spearman rank correlation coefficients and exploratory factor analysis.

Toward health technology assessment of whole-genome sequencing diagnostic tests: challenges and solutions.

Whole-genome sequencing (WGS) is being applied within research settings across Europe to develop genomic WGS-based diagnostic tests. The focus of this perspective paper is to describe if, and how, current approaches of health technology assessment could be applied to WGS-based diagnostic tests. This perspective draws on the collective view from a trans-European multidisciplinary consortium of methodologists, clinicians and scientists.

Exploring the feasibility of delivering standardized genomic care using ophthalmology as an example.

Purpose: Broadening access to genomic testing and counseling will be necessary to realize the benefits of personalized health care. This study aimed to assess the feasibility of delivering a standardized genomic care model for inherited retinal dystrophy (IRD) and of using selected measures to quantify its impact on patients.

Methods: A pre-/post- prospective cohort study recruited 98 patients affected by IRD to receive standardized multidisciplinary care.