Patient Preferences to Assess Value IN Gene Therapies: Protocol Development for the PAVING Study in Hemophilia.

Gene therapies are innovative therapies that are increasingly being developed. However, health technology assessment (HTA) and payer decision making on these therapies is impeded by uncertainties, especially regarding long-term outcomes. Through measuring patient preferences regarding gene therapies, the importance of unique elements that go beyond health gain can be quantified and inform value assessments.

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies.

The challenging market access of high-cost one-time curative therapies has inspired the development of alternative reimbursement structures, such as outcome-based spread payments, to mitigate their unaffordability and answer remaining uncertainties. This study aimed to provide a broad overview of barriers and possible opportunities for the practical implementation of outcome-based spread payments for the reimbursement of one-shot therapies in European healthcare systems. A systematic literature review was performed investigating published literature and publicly available documents to identify barriers and implementation opportunities for both spreading payments and for implementing outcome-based agreements.

Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions.

This review can inform gene therapy developers on challenges that can be encountered when seeking market access. Moreover, it provides an overview of trends among challenges and potential solutions.

A Systematic Review of the Value Assessment Frameworks Used within Health Technology Assessment of Omics Technologies and Their Actual Adoption from HTA Agencies.

Background: Omics technologies, enabling the measurements of genes (genomics), mRNA (transcriptomics), proteins (proteomics) and metabolites (metabolomics), are valuable tools for personalized decision-making. We aimed to identify the existing value assessment frameworks used by health technology assessment (HTA) doers for the evaluation of omics technologies through a systematic review.

Methods: PubMed, Scopus, Embase and Web of Science databases were searched to retrieve potential eligible articles published until 31 May 2020 in English.

Evaluation of precision medicine assessment reports of the Belgian healthcare payer to inform reimbursement decisions.

Introduction: Precision medicines rely on companion diagnostics to identify patient subgroups eligible for receiving the pharmaceutical product. Until recently, the Belgian public health payer, RIZIV-INAMI, assessed precision medicines and companion diagnostics separately for reimbursement decisions. As both components are considered co-dependent technologies, their assessment should be conducted jointly from a health technology assessment (HTA) perspective.

The effect of traffic-light labels and time pressure on estimating kilocalories and carbon footprint of food.

Food consumption decisions require consumers to evaluate the characteristics of products. However, the literature has given limited attention to how consumers determine the impact of food on health (e.g.

Variability in hospital treatment costs: a time-driven activity-based costing approach for early-stage invasive breast cancer patients.

Objectives: Using a standardised diagnostic and generic treatment path for breast cancer, and the molecular subtype perspective, we aim to measure the impact of several patient and disease characteristics on the overall treatment cost for patients. Additionally, we aim to generate insights into the drivers of cost variability within one medical domain.

Design, Setting And Participants: We conducted a retrospective study at a breast clinic in Belgium.

Shedding Light on Reimbursement Policies of Companion Diagnostics in European Countries.

Objectives: Ensuring access to precision medicine has been an issue because in some European countries, desynchronized reimbursement decision-making occurs between the medicine and the companion diagnostic (CDx). This has resulted in cases in which precision medicine is reimbursed but not the CDx. In overcoming this issue, an alignment of the decision-making process for reimbursement between the 2 entities should be considered.

Variability in Hospital Costs of Adult Spinal Deformity Care.

Study Design: Retrospective, single-center analysis.

Objective: To calculate the total clinical hospital cost of the Adult Spinal Deformity (ASD) care trajectory, to explain cost variability by patient and surgery characteristics, and to identify areas of process improvement opportunities.

Summary Of Background Data: ASD is associated with a high financial and clinical burden on society.

Patient-Level Effectiveness Prediction Modeling for Glioblastoma Using Classification Trees.

Objectives: Little research has been done in pharmacoepidemiology on the use of machine learning for exploring medicinal treatment effectiveness in oncology. Therefore, the aim of this study was to explore the added value of machine learning methods to investigate individual treatment responses for glioblastoma patients treated with temozolomide.

Methods: Based on a retrospective observational registry covering 3090 patients with glioblastoma treated with temozolomide, we proposed the use of a two-step iterative exploratory learning process consisting of an initialization phase and a machine learning phase.

The unhealthy = tasty belief is associated with BMI through reduced consumption of vegetables: A cross-national and mediational analysis.

Obesity is one of the greatest public health challenges of modern times and its prevalence is increasing worldwide. With food so abundant in developed countries, many people face a conflict between desires for short-term taste and the goal of long-term health, multiple times a day. Recent research suggests that consumers often resolve these conflicts based on their lay beliefs about the healthiness and tastiness of food.

Nearest Neighbour Propensity Score Matching and Bootstrapping for Estimating Binary Patient Response in Oncology: A Monte Carlo Simulation.

Nearest Neighbour (NN) propensity score (PS) matching methods are commonly used in pharmacoepidemiology to estimate treatment response using observational data. Unfortunately, there is limited evidence on the optimal approach for accurately estimating binary treatment response and, more so, to estimate its variance. Bootstrapping, although commonly used to accurately estimate variance, is rarely used together with PS matching.

When Holding in Prevents From Reaching Out: Emotion Suppression and Social Support-Seeking in Multicultural Groups.

Members of multicultural groups benefit from developing diverse social support networks. Engaging openly with people who have a different worldview (i.e.

Real-World Evidence Gathering in Oncology: The Need for a Biomedical Big Data Insight-Providing Federated Network.

Moving toward new adaptive pathways for the development and access to innovative medicines implies that real-world data (RWD) collected throughout the medicinal product life cycle is becoming increasingly important. Big data analytics on RWD can obtain new and powerful insights into medicines' effectiveness. However, the healthcare ecosystem still faces many sector-specific challenges that hamper the use of big data analytics delivering real world evidence (RWE).

Comparative and combined effectiveness of innovative therapies in cancer: a literature review.

To achieve therapeutic innovation in oncology, already expensive novel medicines are often concomitantly combined to potentially enhance effectiveness. While this aggravates the pricing problem, comparing effectiveness of novel yet expensive (concomitant) treatments is much needed for healthcare decision-making to deliver effective but affordable treatments. This study reviewed published clinical trials and real-world studies of targeted and immune therapies.

Cross-national investigation of the drivers of obesity: Re-assessment of past findings and avenues for the future.

In this paper we question whether prior cross-national differences in food attitudes still exist and if so, to what extent. Due to societal evolutions such as sedentarism and globalization, international variations in food attitudes may not be as pronounced as currently believed. A cross-sectional web-based survey was carried out in the United States, the United Kingdom, France and Belgium.

To squeeze or not to squeeze: How squeeze tubes affect consumers' serving sizes.

Squeeze tubes increasingly complement traditional packaging. But, would squeeze tubes - besides offering ease of use - also affect consumers' serving sizes? And if so, in what way? To answer these questions, we contrast the motor fluency hypothesis (i.e.

Due time driven surgery scheduling.

In many hospitals there are patients who receive surgery later than what is medically indicated. In one of Europe's largest hospitals, the University Hospital Leuven, this is the case for approximately every third patient. Serving patients late cannot always be avoided as a highly utilized OR department will sometimes suffer capacity shortage, occasionally leading to unavoidable delays in patient care.

A perspective on the economic valorization of gene manipulated biotechnology: Past and future.

Three distinct fields of gene manipulated biotechnology have so far been economically exploited: medical biotechnology, plant biotechnology and industrial biotechnology. This article analyzes the economic evolution and its drivers in the three fields over the past decades, highlighting strong divergences. Product and market characteristics, affecting firms' financing options, are shown to be important enablers or inhibitors.

Reversed item bias: an integrative model.

In the recent methodological literature, various models have been proposed to account for the phenomenon that reversed items (defined as items for which respondents' scores have to be recoded in order to make the direction of keying consistent across all items) tend to lead to problematic responses. In this article we propose an integrative conceptualization of three important sources of reversed item method bias (acquiescence, careless responding, and confirmation bias) and specify a multisample confirmatory factor analysis model with 2 method factors to empirically test the hypothesized mechanisms, using explicit measures of acquiescence and carelessness and experimentally manipulated versions of a questionnaire that varies 3 item arrangements and the keying direction of the first item measuring the focal construct. We explain the mechanisms, review prior attempts to model reversed item bias, present our new model, and apply it to responses to a 4-item self-esteem scale (N = 306) and the 6-item Revised Life Orientation Test (N = 595).