Cost-Effectiveness of a Digital Leakage Notification System (Heylo™) for People with Ileostomies or Colostomies in the United Kingdom.

Background: People with stomas report a substantial negative impact of stomal effluent leaking outside the ostomy baseplate and subsequent complications in their professional and social lives, causing immense worry. However, many patients are not able to recognize leakages in a timely manner. We conducted a cost-effectiveness study to evaluate the impact of a digital leakage notification system (DLNS) to reduce leakages outside the baseplate (LOB) and worry about leakage for people with intestinal stomas from a UK National Health Service and Personal Social Services perspective.

The economic burden of stomas in the UK: a retrospective observational study of health records and hospital encounters.

Background: Stomas divert waste from the small intestine (ileostomy), large intestine (colostomy) or ureters (urostomy), and complications are common.

Aims: This study evaluated healthcare resource utilisation (HCRU) and costs of stomas from a UK perspective.

Methods: This was a retrospective observational study of adults with new stomas (New Stoma Group) or new/existing stomas and >6 months of follow-up (Established Stoma Group) using health records linked with hospital encounters (January 2009-December 2018).

Complications and Healthcare Costs Associated With the First Year Following Colostomy and Ileostomy Formation: A Retrospective Study.

Purpose: The purpose of this study was to evaluate clinical and economic outcomes during the first year following ostomy formation.

Design: Single-center retrospective audit.

Subjects And Setting: The sample comprised 200 patients who underwent surgery leading to ileostomy or colostomy at a large English National Health Service (NHS) Trust.

Cost-effectiveness analysis of HIV pre-exposure prophylaxis in Japan.

Background: While global efforts have been made to prevent transmission of HIV, the epidemic persists. Men who have sex with men (MSM) are at high risk of infection. Despite evidence of its cost-effectiveness in other jurisdictions, pre-exposure prophylaxis (PrEP) for MSM is neither approved nor reimbursed in Japan.

Examining the impact of haemophilia treatment on health-related quality of life.

Introduction: Haemophilia has substantial SD effects on health-related quality of life (HRQoL), particularly for people with severe haemophilia. How certain aspects of haemophilia influence HRQoL is not well understood.

Aim: To develop predictive models of variables influencing HRQoL in people with severe haemophilia A or B.

Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: Evaluating the impact of treatment crossover on outcomes.

Introduction/aims: Trials incorporating placebo-to-active treatment crossover are encouraged in fatal conditions like amyotrophic lateral sclerosis (ALS) but may underestimate active treatment survival benefit. Here, we apply methods for modeling survival without crossover, including the rank-preserving structural failure time model (RPSFTM), to data from the CENTAUR trial of sodium phenylbutyrate and taurursodiol (PB and TURSO) in ALS incorporating both randomized placebo-controlled and open-label extension (OLE) phases.

Methods: Intent-to-treat (ITT) and RPSFTM survival analyses were performed with final data at a July 2020 cutoff date.

Examining patient and professional perspectives in the UK for gene therapy in haemophilia.

Introduction: With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions.

Objective: To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs).

Methods: Semi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance.

Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment.

Introduction: Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.

Aim: We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes.

Methods: We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21.

Budget Impact Analysis of Gemtuzumab Ozogamicin for the Treatment of CD33-Positive Acute Myeloid Leukemia.

Background: Gemtuzumab ozogamicin (GO) was approved in 2017 in the US for the treatment of adults with newly diagnosed CD33-positive (CD33+) acute myeloid leukemia (AML), and adults and pediatric patients with CD33+ relapsed/refractory (R/R) AML.

Objective: The aim of this study was to estimate the budgetary impact of introducing GO to a 1-million-member US health plan over a 5-year period.

Methods: We developed models to estimate the impact of introducing GO in combination with conventional induction chemotherapy or as monotherapy for newly diagnosed AML, and as monotherapy for R/R AML.

Systematic review, network meta-analysis and economic evaluation of biological therapy for the management of active psoriatic arthritis.

Background: An updated economic evaluation was conducted to compare the cost-effectiveness of the four tumour necrosis factor (TNF)-α inhibitors adalimumab, etanercept, golimumab and infliximab in active, progressive psoriatic arthritis (PsA) where response to standard treatment has been inadequate.

Methods: A systematic review was conducted to identify relevant, recently published studies and the new trial data were synthesised, via a Bayesian network meta-analysis (NMA), to estimate the relative efficacy of the TNF-α inhibitors in terms of Psoriatic Arthritis Response Criteria (PsARC) response, Health Assessment Questionnaire (HAQ) scores and Psoriasis Area and Severity Index (PASI). A previously developed economic model was updated with the new meta-analysis results and current cost data.