Use of the ESMO-Magnitude of Clinical Benefit Scale to guide HTA recommendations on coverage and reimbursement for cancer medicines: a retrospective analysis.

Background: Recommendations by countries' health technology assessment (HTA) agencies are used to decide which new therapies warrant the allocation of limited health-care resources to make them available through publicly funded health systems. This process is of public health importance for balancing the dual aims of optimising patient outcomes while ensuring financial sustainability. We evaluated which factors affect HTA outcomes and the time to positive HTA outcome, focussing on the role of clinical benefit evaluated with the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).

The Evolving Nature of Health Technology Assessment: A Critical Appraisal of NICE's New Methods Manual.

Objectives: The National Institute for Health and Care Excellence (NICE) recently completed a review of its methods for health technology assessment, involving a 2-stage public consultation. We appraise proposed methodological changes and analyze key decisions.

Methods: We categorize all changes proposed in the first consultation as "critical," "moderate" or "limited" updates, considering the importance of the topic and the degree of change or the level of reinforcement.

Funding Sources of Therapeutic and Vaccine Clinical Trials for COVID-19 vs Non-COVID-19 Indications, 2020-2021.

Importance: Effective COVID-19 vaccines and therapeutics reached the market within the first year of the pandemic. This rate of development and availability was an unprecedented achievement that required attention to numerous research and development, regulatory, and policy challenges. However, only limited evidence is currently available on the sources of funding for COVID-19 clinical trials.

Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies.

Recently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as cost-effectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions.

Early Health Technology Assessment during Nonalcoholic Steatohepatitis Drug Development: A Two-Round, Cross-Country, Multicriteria Decision Analysis.

The assessment of value along the clinical development of new biopharmaceutical compounds is a challenging task. Complex and uncertain evidence has to be analyzed, considering a multitude of value preferences from different stakeholders. To investigate the use of multicriteria decision analysis (MCDA) to support decision making during drug development while considering payer and health technology assessment (HTA) value concerns, by applying the Advance Value Framework in nonalcoholic steatohepatitis (NASH) and testing for the consistency of the results.

ICER Value Framework 2020 Update: Recommendations on the Aggregation of Benefits and Contextual Considerations.

The Institute for Clinical and Economic Review (ICER) in the United States recently published a 2020 update to its value assessment framework. We are commenting on the method by which the benefits of health interventions are integrated, relating to contextual considerations and other factors relevant to an intervention's value. We start by discussing the theoretical foundations of decision analysis and its extension to multiple criteria decision analysis (MCDA).

Advancing structured decision-making in drug regulation at the FDA and EMA.

The recent benefit-risk framework (BRF) developed by the Food and Drug Administration (FDA) is intended to improve the clarity and consistency in communicating the reasoning behind the FDA's decisions, acting as an important advancement in US drug regulation. In the PDUFA VI implementation plan, the FDA states that it will continue to explore more structured or quantitative decision analysis approaches; however, it restricts their use within the current BRF that is purely qualitative. By contrast, European regulators and researchers have been long exploring the use of quantitative decision analysis approaches for evaluating drug benefit-risk balance.

Quantifying Preferences in Drug Benefit-Risk Decisions.

Benefit-risk assessment is used in various phases along the drug lifecycle, such as marketing authorization and surveillance, health technology assessment (HTA), and clinical decisions, to understand whether, and for which patients, a drug has a favorable or more valuable profile with reference to one or more comparators. Such assessments are inherently preference-based as several clinical and nonclinical outcomes of varying importance might act as evaluation criteria, and decision makers must establish acceptable trade-offs between these outcomes. Different healthcare stakeholder perspectives, such as those from patients and healthcare professionals, are key for informing benefit-risk trade-offs.

An EU-wide approach to HTA: An irrelevant development or an opportunity not to be missed?

An EU-wide cooperation on HTA has been proposed recently by the European Commission, focusing on relative effectiveness assessment (REA) for pharmaceuticals and medical devices. This cooperation is operationalised through a proposal for a regulation. While a good step in the right direction, this HTA cooperation framework needs to be more explicit and pragmatic about clinical value definition, what constitutes quality of evidence, how real-world evidence is handled, whether the same assessment requirements will apply for medical devices as they do for pharmaceuticals, and how to safeguard consistency in REA interpretation.

Evaluating the Benefits of New Drugs in Health Technology Assessment Using Multiple Criteria Decision Analysis: A Case Study on Metastatic Prostate Cancer With the Dental and Pharmaceuticals Benefits Agency (TLV) in Sweden.

Multiple criteria decision analysis (MCDA) has been identified as a prospective methodology for assisting decision makers in evaluating the benefits of new medicines in health technology assessment (HTA); however, limited empirical evidence exists from real-world applications. To test in practice a recently developed MCDA methodological framework for HTA, the Advance Value Framework, in a proof-of-concept case study with decision makers. A multi-attribute value theory methodology was adopted applying the MACBETH questioning protocol through a facilitated decision-analysis modelling approach as part of a decision conference with four experts.

Multiple criteria decision analysis in the context of health technology assessment: a simulation exercise on metastatic colorectal cancer with multiple stakeholders in the English setting.

Background: Multiple criteria decision analysis (MCDA) has appeared as a methodology to address limitations of economic evaluation in health technology assessment (HTA), however there are limited empirical evidence from real world applications. The aim of this study is to test in practice a recently developed MCDA methodological framework known as Advance Value Framework (AVF) through a proof-of-concept case study engaging multiple stakeholders.

Methods: A multi-attribute value theory methodological process was adopted involving problem structuring, model building, model assessment and model appraisal phases.

Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework.

Escalating drug prices have catalysed the generation of numerous "value frameworks" with the aim of informing payers, clinicians and patients on the assessment and appraisal process of new medicines for the purpose of coverage and treatment selection decisions. Although this is an important step towards a more inclusive Value Based Assessment (VBA) approach, aspects of these frameworks are based on weak methodologies and could potentially result in misleading recommendations or decisions. In this paper, a Multiple Criteria Decision Analysis (MCDA) methodological process, based on Multi Attribute Value Theory (MAVT), is adopted for building a multi-criteria evaluation model.

Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries.

Background: Although health technology assessment (HTA) systems base their decision making process either on economic evaluations or comparative clinical benefit assessment, a central aim of recent approaches to value measurement, including value based assessment and pricing, points towards the incorporation of supplementary evidence and criteria that capture additional dimensions of value.

Objective: To study the practices, processes and policies of value-assessment for new medicines across eight European countries and the role of HTA beyond economic evaluation and clinical benefit assessment.

Methods: A systematic (peer review and grey) literature review was conducted using an analytical framework examining: (1) 'Responsibilities and structure of HTA agencies'; (2) 'Evidence and evaluation criteria considered in HTAs'; (3) 'Methods and techniques applied in HTAs'; and (4) 'Outcomes and implementation of HTAs'.

PARS PLANA VITRECTOMY FOR DIABETIC MACULAR EDEMA: A Systematic Review, Meta-Analysis, and Synthesis of Safety Literature.

Purpose: To assess the risk and benefit of pars plana vitrectomy for diabetic macular edema.

Methods: The authors conducted a systematic literature review using PubMed, EMBASE, Web of Science, and Cochrane Central Database of Controlled Trials until September 2014. The population was patients with diabetic macular edema, intervention vitrectomy, comparator macular laser or observation, and efficacy outcome visual acuity and central retinal thickness.

Socioeconomic costs and health-related quality of life in juvenile idiopathic arthritis: a cost-of-illness study in the United Kingdom.

Background: Juvenile idiopathic arthritis (JIA) refers to a number of rare chronic inflammatory diseases. Although JIA imposes a significant societal burden, limited data are available on the cost of JIA. The study's objective is to quantify the socioeconomic burden of JIA patients in the United Kingdom (UK), along with their health-related quality of life (HRQoL).

Social/economic costs and health-related quality of life in patients with epidermolysis bullosa in Europe.

Background: The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states.

Methods: We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers.

Value-Based Assessment of New Medical Technologies: Towards a Robust Methodological Framework for the Application of Multiple Criteria Decision Analysis in the Context of Health Technology Assessment.

In recent years, multiple criteria decision analysis (MCDA) has emerged as a likely alternative to address shortcomings in health technology assessment (HTA) by offering a more holistic perspective to value assessment and acting as an alternative priority setting tool. In this paper, we argue that MCDA needs to subscribe to robust methodological processes related to the selection of objectives, criteria and attributes in order to be meaningful in the context of healthcare decision making and fulfil its role in value-based assessment (VBA). We propose a methodological process, based on multi-attribute value theory (MAVT) methods comprising five distinct phases, outline the stages involved in each phase and discuss their relevance in the HTA process.