It Takes 2 to Tango. Setting Out the Conditions in Which Performance-Based Risk-Sharing Arrangements Work for Both Parties.

Objectives: Faster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements, to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes. One important reason for risk sharing is to resolve differences of opinion between innovators and payers about a technology's underlying value.

The value of treatment for infertility: A systematic literature review of willingness-to-pay thresholds and approaches for determining the cost effectiveness of fertility therapies.

Willingness to pay (WTP) for an infertility treatment is the maximum amount of money a patient is willing to pay per treatment, or to achieve a live birth or pregnancy. Such thresholds are important to determine the cost effectiveness of a treatment. A systematic review was conducted to identify and explore the studies that attempt to ascertain WTP for infertility and compare them with the cost-effectiveness studies that claimed to use WTP thresholds.

A Guide to Selecting Flexible Survival Models to Inform Economic Evaluations of Cancer Immunotherapies.

Objectives: Parametric models are routinely used to estimate the benefit of cancer drugs beyond trial follow-up. The advent of immune checkpoint inhibitors has challenged this paradigm, and emerging evidence suggests that more flexible survival models, which can better capture the shapes of complex hazard functions, might be needed for these interventions. Nevertheless, there is a need for an algorithm to help analysts decide whether flexible models are required and, if so, which should be chosen for testing.

Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?

Conventional cost-effectiveness analysis-i.e., assessing pharmaceuticals through a cost per quality-adjusted life year (QALY) framework-originated from a societal commitment to maximize population health given limited resources.

An Innovative Approach to Modelling the Optimal Treatment Sequence for Patients with Relapsing-Remitting Multiple Sclerosis: Implementation, Validation, and Impact of the Decision-Making Approach.

Introduction: An innovative computational model was developed to address challenges regarding the evaluation of treatment sequences in patients with relapsing-remitting multiple sclerosis (RRMS) through the concept of a 'virtual' physician who observes and assesses patients over time. We describe the implementation and validation of the model, then apply this framework as a case study to determine the impact of different decision-making approaches on the optimal sequence of disease-modifying therapies (DMTs) and associated outcomes.

Methods: A patient-level discrete event simulation (DES) was used to model heterogeneity in disease trajectories and outcomes.

Cost-effectiveness analysis of a single-inhaler triple therapy for COPD in the UK.

UK management costs for COPD, estimated at £1.9 billion/year, are rising. In the FULFIL (Lung Function and Quality of Life Assessment in Chronic Obstructive Pulmonary Disease with Closed Triple Therapy) study, single-inhaler triple therapy with fluticasone furoate/umeclidinium/vilanterol (100/62.

What Did Time Tell Us? A Comparison and Retrospective Validation of Different Survival Extrapolation Methods for Immuno-Oncologic Therapy in Advanced or Metastatic Renal Cell Carcinoma.

Background: The immuno-oncologic (IO) mechanism of action may lead to an overall survival (OS) hazard that changes over time, producing shapes that standard parametric extrapolation methods may struggle to reflect. Furthermore, selection of the most appropriate extrapolation method for health technology assessment is often based on trial data with limited follow-up.

Objective: To examine this problem, we fitted a range of extrapolation methods to patient-level survival data from CheckMate 025 (NCT01668784, CM-025), a phase III trial comparing nivolumab with everolimus for previously treated advanced renal cell carcinoma (aRCC), to assess their predictive accuracy over time.

Value of Information Analytical Methods: Report 2 of the ISPOR Value of Information Analysis Emerging Good Practices Task Force.

The allocation of healthcare resources among competing priorities requires an assessment of the expected costs and health effects of investing resources in the activities and of the opportunity cost of the expenditure. To date, much effort has been devoted to assessing the expected costs and health effects, but there remains an important need to also reflect the consequences of uncertainty in resource allocation decisions and the value of further research to reduce uncertainty. Decision making with uncertainty may turn out to be suboptimal, resulting in health loss.

Value of Information Analysis for Research Decisions-An Introduction: Report 1 of the ISPOR Value of Information Analysis Emerging Good Practices Task Force.

Healthcare resource allocation decisions made under conditions of uncertainty may turn out to be suboptimal. In a resource constrained system in which there is a fixed budget, these suboptimal decisions will result in health loss. Consequently, there may be value in reducing uncertainty, through the collection of new evidence, to make better resource allocation decisions.

Uncertainty and Cures: Discontinuation, Irreversibility, and Outcomes-Based Payments: What Is Different About a One-Off Treatment?

Payers are concerned that one-off "cures" bring great uncertainty with the consequential risk of incorrect adoption decisions, and significant budget impact from large one-off payments. Innovators worry about bias against "cures" in favor of repeat treatment, which is not in patients' interests. We find that even in the absence of a difference in uncertainty of outcomes, adverse pay-offs differ.

Cost-effectiveness of TYRX absorbable antibacterial envelope for prevention of cardiovascular implantable electronic device infection.

Aims: Infection is a major complication of cardiovascular implantable electronic device (CIED) therapy that usually requires device extraction and is associated with increased morbidity and mortality. The TYRX Antibacterial Envelope is a polypropylene mesh that stabilizes the CIED and elutes minocycline and rifampin to reduce the risk of post-operative infection.

Methods: A decision tree was developed to assess the cost-effectiveness of TYRX vs standard of care (SOC) following implantation of four CIED device types.

Adjuvant HPV vaccination for anal cancer prevention in HIV-positive men who have sex with men: The time is now.

Importance: Outcomes of treating high-grade squamous intraepithelial lesions (HSIL), a precursor to anal cancer, remain uncertain. Emerging evidence shows that post HSIL treatment adjuvant quadrivalent human papillomavirus (qHPV) vaccination improves the effectiveness of treatment. However, no recommendations exist regarding the use of qHPV vaccine as an adjuvant form of therapy.

Emerging Use of Early Health Technology Assessment in Medical Product Development: A Scoping Review of the Literature.

Early health technology assessment is increasingly being used to support health economic evidence development during early stages of clinical research. Such early models can be used to inform research and development about the design and management of new medical technologies to mitigate the risks, perceived by industry and the public sector, associated with market access and reimbursement. Over the past 25 years it has been suggested that health economic evaluation in the early stages may benefit the development and diffusion of medical products.

The CARE Plus study - a whole-system intervention to improve quality of life of primary care patients with multimorbidity in areas of high socioeconomic deprivation: exploratory cluster randomised controlled trial and cost-utility analysis.

Background: Multimorbidity is common in deprived communities and reduces quality of life. Our aim was to evaluate a whole-system primary care-based complex intervention, called CARE Plus, to improve quality of life in multimorbid patients living in areas of very high deprivation.

Methods: We used a phase 2 exploratory cluster randomised controlled trial with eight general practices in Glasgow in very deprived areas that involved multimorbid patients aged 30-65 years.

Cost-Effectiveness of HBV and HCV Screening Strategies--A Systematic Review of Existing Modelling Techniques.

Introduction: Studies evaluating the cost-effectiveness of screening for Hepatitis B Virus (HBV) and Hepatitis C Virus (HCV) are generally heterogeneous in terms of risk groups, settings, screening intervention, outcomes and the economic modelling framework. It is therefore difficult to compare cost-effectiveness results between studies. This systematic review aims to summarise and critically assess existing economic models for HBV and HCV in order to identify the main methodological differences in modelling approaches.

Practice nurse health checks for adults with intellectual disabilities: a cluster-design, randomised controlled trial.

Background: Adults with intellectual disabilities have substantial health inequalities and poor access to health care. We assessed whether practice nurse-delivered health checks could improve the health of adults with intellectual disabilities compared with standard care.

Methods: In this cluster-design, single-blind, randomised controlled trial, we included general practices in Scotland, UK.

The management of Otitis Media with Effusion in children with cleft palate (mOMEnt): a feasibility study and economic evaluation.

Background: Cleft lip and palate are among the most common congenital malformations, with an incidence of around 1 in 700. Cleft palate (CP) results in impaired Eustachian tube function, and 90% of children with CP have otitis media with effusion (OME) histories. There are several approaches to management, including watchful waiting, the provision of hearing aids (HAs) and the insertion of ventilation tubes (VTs).

Operationalising the capability approach as an outcome measure in public health: The development of the OCAP-18.

There is growing interest in operationalising the capability approach to measure quality of life. This paper reports the results of a research project undertaken in 2007 that sought to reduce and refine a longer survey in order to provide a summary measure of wellbeing and capability in the realm of public health. The reduction and refinement of the questionnaire took place across a number of stages, using both qualitative (five focus group discussions and 17 in-depth interviews) and quantitative (secondary data analysis, N = 1048 and primary data collection using postal surveys and interviews, N = 45) approaches.

Use of value of information in UK health technology assessments.

Objectives: The aim of this study was to identify and critically appraise the use of Value of Information (VOI) analyses undertaken as part of health technology assessment (HTA) reports in England and Wales.

Methods: A systematic review of National Institute for Health Research (NIHR) funded HTA reports published between 2004 and 2013 identified the use of VOI methods and key analytical details in terms of: (i) types of VOI methodology used; (ii) parameters and key assumptions; and (iii) conclusions drawn in terms of the need for further research.

Results: A total of 512 HTA reports were published during the relevant timeframe.

A model-based cost-effectiveness analysis of a grommets-led care pathway for children with cleft palate affected by otitis media with effusion.

There is a paucity of evidence to guide the management of otitis media with effusion (OME), which is a common problem causing significant hearing impairment in children with cleft palate. The insertion of grommets is currently being used to correct hearing impairment and prevent complications of unmanaged OME, but there is ongoing discussion about whether the benefits of grommets outweigh the costs and risks. A decision-tree model was developed to assess the surgical insertion of grommets with two non-surgical alternatives (hearing-aids and do-nothing strategies) in cleft palate children with persistent bilateral OME.

Communicating the parameter uncertainty in the IQWiG efficiency frontier to decision-makers.

The Institute for Quality and Efficiency in Health Care (IQWiG) developed-in a consultation process with an international expert panel-the efficiency frontier (EF) approach to satisfy a range of legal requirements for economic evaluation in Germany's statutory health insurance system. The EF approach is distinctly different from other health economic approaches. Here, we evaluate established tools for assessing and communicating parameter uncertainty in terms of their applicability to the EF approach.

A gender-sensitised weight loss and healthy living programme for overweight and obese men delivered by Scottish Premier League football clubs (FFIT): a pragmatic randomised controlled trial.

Background: The prevalence of male obesity is increasing but few men take part in weight loss programmes. We assessed the effect of a weight loss and healthy living programme on weight loss in football (soccer) fans.

Methods: We did a two-group, pragmatic, randomised controlled trial of 747 male football fans aged 35-65 years with a body-mass index (BMI) of 28 kg/m(2) or higher from 13 Scottish professional football clubs.

Double trouble: the impact of multimorbidity and deprivation on preference-weighted health related quality of life a cross sectional analysis of the Scottish Health Survey.

Objective: To investigate the association between multimorbidity and Preference_Weighted Health Related Quality of Life (PW_HRQoL), a score that combines physical and mental functioning, and how this varies by socioeconomic deprivation and age.

Design: The Scottish Health Survey (SHeS) is a cross-sectional representative survey of the general population which included the SF-12, a survey of HRQoL, for individuals 20 years and over.

Methods: For 7,054 participants we generated PW_HRQoL scores by running SF-12 responses through the SF-6D algorithm.