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).

An assessment of the implications of distribution remuneration and taxation policies on the final prices of prescription medicines: evidence from 35 countries.

This paper analyses the structure of and variability in taxation and prescription drug distribution policies and quantifies the impact of such policies on the cost of prescription drugs to health systems in 35 countries. Taxes on prescription drugs remain highly prevalent (83% of the sample) although 63% of the sample countries implement a lower than standard VAT rate. Three remuneration types of the wholesale and retail distribution chain have been identified.

The 30-Billion-Dollar Distribution Markups and Taxes of Pharmaceuticals in Latin American Countries: Impact, Options, and Trade-Offs.

Objectives: The World Health Organization provides 10 specific guidelines for managing the prices of pharmaceutical products. Many of those are widely known and used such as reference pricing, value-based pricing, price transparency, and tendering. Less attention and knowledge is concentrated in markup regulation across the pharmaceutical supply chain and distribution and in tax exemptions or reductions.

A Systematic Review of Value Criteria for Next-Generation Sequencing/Comprehensive Genomic Profiling to Inform Value Framework Development.

Objectives: To comprehensively identify and map an exhaustive list of value criteria for the assessment of next-generation sequencing/comprehensive genomic profiling (NGS/CGP), to be used as an aid in decision making.

Methods: We conducted a systematic review to identify existing value frameworks (VFs) applicable to any type of healthcare technology. VFs and criteria were mapped to a previously published Latin American (LA) VF to harmonize definitions and identify additional criteria and or subcriteria.

How can health technology assessment be improved to optimise access to medicines? Results from a Delphi study in Europe : Better access to medicines through HTA.

Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe.

Assessing the Value of Provider-Facing Digital Health Technologies Used in Chronic Disease Management: Toward a Value Framework Based on Multistakeholder Perceptions.

Objectives: Hardly any value frameworks exist that are focused on provider-facing digital health technologies (DHTs) for managing chronic disease with diverse stakeholder participation in their creation. Our study aimed to 1) understanding different stakeholder opinions on where value lies in provider-facing technologies and 2) create a comprehensive value assessment framework for DHT assessment.

Methods: Mixed-methods comprising both primary and secondary evidence were used.

Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries.

Context: Positive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes.

Objective: We analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes.

A Value Framework to Assess Patient-Facing Digital Health Technologies That Aim to Improve Chronic Disease Management: A Delphi Approach.

Objectives: Digital health technologies (DHTs) can optimise healthcare costs and improve quality and efficiency of care. However, the fast-paced rate of innovation and varying evidence standards can make it difficult for decision-makers to assess these technologies in an efficient and evidence-based manner. We sought to develop a comprehensive framework to assess the value of novel patient-facing DHTs used to manage chronic diseases by eliciting stakeholder value preferences.

The patient pathway in ATTR-CM in Greece and how to improve it: A multidisciplinary perspective.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed disease associated with high mortality rates and the patient journey is characterized by increased complexities. Accurate and timely diagnosis and prompt initiation of disease-modifying treatment constitute the contemporary unmet need in ATTR-CM. ATTR-CM diagnosis is characterized by considerable delays and high rates of misdiagnosis.

Road traffic mortality and economic uncertainty: Evidence from the United States.

Previous studies have shown that financial turbulence is associated with a short-term increase in road traffic collisions, largely due to drivers' emotional state, distraction, sleep deprivation and alcohol consumption. In this paper we advance this debate by studying the association between economic uncertainty and road traffic mortality in the United States. We used a State-level uncertainty index and State fatalities for the period 2008-2017 and found that a one standard deviation increase in economic uncertainty is associated with an additional 0.

Healthcare Payer Perspectives on the Assessment and Pricing of Oncology Multi-Indication Products: Evidence from Nine OECD Countries.

Background: New pharmaceuticals are increasingly being developed for use across multiple indications. Countries across Europe and North America have adopted a range of different approaches to capture differences in the value of individual indications.

Objective: The three aims of this study were (i) to review the price-setting practice over the past 5 years for multi-indication products across England, France, Italy, Spain, Belgium, Switzerland, Turkey, Canada and the USA; (ii) to assess the impact of current practices on launch strategy; and (iii) to identify issues in the implementation of indication-based pricing.

Do Reimbursement Recommendations by the Canadian Agency for Drugs and Technology in Health Translate Into Coverage Decisions for Orphan Drugs in the Canadian Province of Ontario?

Objectives: Unlike other high-income countries, Canada has no national policy for drugs treating rare diseases (orphan drugs). Nevertheless, in 2022, the Canadian government committed to creating a national strategy to make access to these drugs more consistent. Our aim was to study whether recommendations made by the Canadian Agency for Drugs and Technology in Health (CADTH) translated into coverage decisions for orphan drugs in Ontario, the largest Canadian province.

Launch sequencing of pharmaceuticals with multiple therapeutic indications: evidence from seven countries.

Background: New medicines are increasingly being identified as efficacious across multiple indications. The impact of current pricing and reimbursement policies on launch decisions across these indications remains unclear.

Objective: This paper, first, maps marketing authorisation and HTA coverage recommendation sequences of multi-indication medicines across Germany, France, England, Scotland, Canada, Australia, and the USA, and, second, evaluates the clinical characteristics, clinical development time and coverage recommendation time of multi-indication medicines, drawing comparisons between the first and subsequent indications of an approved molecule.

Value Insider Season 1 Episode 6: How Will Market Access and Value Demonstration Evolve? (Future Outlook) [Podcast].

How will market access and value demonstration evolve? In this episode of the Value Insider podcast, host Mike Chambers speaks with Ass. Prof. Panos Kanavos about the future of value assessments in healthcare.

How do HTA agencies perceive conditional approval of medicines? Evidence from England, Scotland, France and Canada.

There is a growing disconnect between regulatory agencies that are promoting expedited approval to medicines based on early phase clinical evidence and health technology assessment (HTA) agencies that require robust clinical evidence to inform coverage decisions. This paper provides an assessment of the evidence gap between regulatory and HTA agencies on medicines receiving conditional marketing authorisation (CMA) and examines how HTA agencies in France, England, Scotland, and Canada interpret and appraise evidence for these medicines. A mixed methods research design was used to identify the types and frequency of parameters raised in the context of HTA decision-making for all conditional approvals in Europe and Canada between 2010 and 2017.

Impact of Managed Entry Agreements on availability of and timely access to medicines: an ex-post evaluation of agreements implemented for oncology therapies in four countries.

Background: Despite the increased utilisation of Managed Entry Agreements (MEAs), empirical studies assessing their impact on achieving better access to medicines remains scarce. In this study we evaluated the role of MEAs on enhancing availability of and timely access to a sample of oncology medicines that had received at least one prior rejection from reimbursement.

Methods: Funding decisions and their respective timelines for all oncology medicines approved between 2009 and 2018 in Australia, England, Scotland and Sweden were studied.

The future of oncology policy.

To ensure the previous progress seen in cancer survival rates continues as we move through the 21st Century it is important to determine future effective policy related to oncology healthcare delivery and funding. Recent successes with, for example, the COVID vaccine response, the decision-making agility exhibited by governments and healthcare systems and the effective use of telehealth and real-world evidence highlight the progress that can be made with pooled efforts and innovative thinking. This shared approach is the basis for the European Beating Cancer Plan which outlines action points for governments and health systems for the period 2021-2025.

Challenges for health systems seeking to embrace virtual health care for population health.

A gradual move to proactive illness prevention requires a strategic shift towards population health management by health care systems. Such a shift becomes necessary to improve outcomes, reduce inequalities and manage costs better, as life expectancy increases and chronic illness becomes more prevalent. Health system digitisation and greater focus on virtual health care (VHC) can contribute to active population health management.

Value and Price of Multi-indication Cancer Drugs in the USA, Germany, France, England, Canada, Australia, and Scotland.

Purpose: Oncology drugs are often approved for multiple indications, for which their clinical benefit varies. Aligning a single price to this differing value remains a challenge. This study examines the clinical and economic value, price, and reimbursement of multi-indication cancer drugs across seven countries, representing different approaches to value assessment, pricing, and coverage decisions: the USA, Germany, France, England, Canada, Australia, and Scotland.

A Proposal for Value-Based Managed Entry Agreements in an Environment of Technological Change and Economic Challenge for Publicly Funded Healthcare Systems.

Managed entry agreements (MEA) represent one of the main topics of discussion between the European National Payers Authorities. Several initiatives on the subject have been organized over the past few years and the scientific literature is full of publications on the subject. There is currently little international sharing of information between payers, mainly as a result of the confidentiality issues.

Initial and supplementary indication approval of new targeted cancer drugs by the FDA, EMA, Health Canada, and TGA.

Background: Previous research focused on the clinical evidence supporting new cancer drugs' initial US Food and Drug Administration (FDA) approval. However, targeted drugs are increasingly approved for supplementary indications of unknown evidence and benefit.

Objectives: To examine the clinical trial evidence supporting new targeted cancer drugs' initial and supplementary indication approval in the US, EU, Canada, and Australia.

Similarities and Differences in Health Technology Assessment Systems and Implications for Coverage Decisions: Evidence from 32 Countries.

Health technology assessment (HTA) systems across countries vary in the way they are set up, according to their role and based on how funding decisions are reached. Our objective was to study the characteristics of these systems and their likely impact on the funding of technologies undergoing HTA. Based on a literature review, we created a conceptual framework that captures key operating features of HTA systems.

Assessing pricing and reimbursement policies for generic pharmaceuticals in the MENA region for improved efficiency, affordability and generic penetration.

Background: Healthcare systems in the Middle East & North Africa (MENA) region face challenges in healthcare service funding and delivery. Greater use of generics satisfies the objectives of macroeconomic efficiency and contributes to healthcare financial sustainability.

Objective: To examine and critically appraise the key features of supply- and demand-side policies for generic pharmaceuticals in eleven MENA countries.

Sin taxes and their effect on consumption, revenue generation and health improvement: a systematic literature review in Latin America.

Sin or public health taxes are excise taxes imposed on the consumption of potentially harmful goods for health [sugar-sweetened beverages (SSBs), tobacco, alcohol, among others], aiming to reduce consumption, raise additional revenue and/or improve population health. This paper assesses the extent to which sin taxes (a) can reduce consumption of potentially harmful goods, (b) raise revenue for national health systems and (c) contribute to population health in Latin America. A systematic literature review was conducted on peer-reviewed and grey literature; endpoints included: impact of raising sin taxes on consumption, ability to raise revenue for health and the possibility of population health improvements.