The unexploited potential of data systems tracking medicines utilization: an opportunity to improve access to oncology combination therapies.

Background: While progress has been made in oncology treatments, including the introduction of combination therapies, barriers affect patient access. There are approaches that could improve access including combination-specific pricing that allow the price to reflect whether a product is used in monotherapy or in combination. The feasibility of this solution requires data on the utilization of combination therapies.

Barriers to adoption of biosimilar insulins in Europe: A mixed-methods study.

Background: Despite the introduction of biosimilar insulins to improve affordability and enhance patient access, their adoption remains limited in Europe.

Objectives: This study aims to (i) provide an up-to-date overview of EMA-approved insulin products and their reimbursement status across selected European countries, and (ii) explore the k,ey factors influencing physicians' prescribing decisions regarding biosimilar and originator insulin.

Methods: A mixed-method design was applied, comprising (i) a quantitative analysis of EMA-approved insulin products and their reimbursement status in nine European countries, and (ii) semi-structured interviews with physicians (N = 20).

Advanced therapy medicinal products are coming of age: A pipeline analysis of the clinical trial landscape.

Advanced therapy medicinal products (ATMPs) are a unique class of biological medicines that are based on cells, genes, or tissues. Searching four databases, this analysis identified 2008 trials that investigated ATMPs and characterised them according to various characteristics, such as the sponsor type and phase of clinical development. This characterisation revealed that the market for ATMPs is maturing in terms of the number of products in development, the diseases being targeted, and the technologies being pursued.

Value attribution for oncology combination regimens: going beyond frameworks to balance innovation, access, and affordability.

Combination therapies are a mainstay in cancer treatment, but reimbursement access can be limited, owing to complexities around value assessment and pricing, and budget impact. Traditional frameworks for value assessment lack specific methodologies for evaluating combinations. A key challenge is value attribution between components.

Evaluating the Cost-Effectiveness of Air Pollution Mitigation Strategies: A Systematic Review.

Air pollution is the world's greatest environmental health risk. Pollutants that pose large health concerns are particulate matter (PM and PM), ozone (O), carbon monoxide (CO), nitrogen dioxide (NO), and sulphur dioxide (SO). These compounds (especially PM and PM) contribute to the onset of diseases, such as respiratory diseases (e.

Cost-Effectiveness of Strategies Addressing Environmental Noise: A Systematic Literature Review.

Environmental noise, a significant public health concern, is associated with adverse health effects, including cardiovascular diseases, cognitive impairments, and psychological distress. Noise reduction strategies are essential for mitigating these effects. Despite evidence of their health benefits, limited information exists on the cost-effectiveness of such strategies to guide resource allocation.

Evolving global regulatory landscape for approval of biosimilars: current challenges and opportunities for convergence.

Introduction: Biosimilars promote price competition, improving affordability and access to biologics without compromising on quality, efficacy, and safety. Biosimilar approvals initially followed a cautious approach, with regulatory requirements developed independently across jurisdictions, complicating global development and increasing costs. Advancements in analytical sciences and two decades of accumulated experience with biosimilar approvals offer an opportunity to reevaluate regulatory requirements.

Compulsory Licensing as an Instrument to Tackle High Medicine Prices: A Realist Review of Industrial and Health Consequences.

Although compulsory licensing of medicines is traditionally discussed in the context of low- and middle-income countries tackling high prices, it has recently sparked debate in several high-income countries. This study aims to examine the industrial and health consequences of compulsory licensing when applied by a high-income country. Our literature review found that the impact of compulsory licensing is challenging to predict as it can have multiple (opposing) consequences in terms of economic activity, patient outcomes and public health.

What role for real-world evidence in market access of biosimilars?

Experience with the use of biosimilars in real-life practice provides an excellent opportunity to collect real-world evidence aimed at addressing residual uncertainties about biosimilars. Hence, this aims to explore the role of real-world evidence on biosimilars by showcasing how real-world evidence studies have contributed to addressing key questions affecting biosimilar market access. We find that the comparable efficacy and safety of a biosimilar and the reference product is corroborated by real-world evidence.

Caregivers and multidisciplinary team members' perspectives on shared decision making in Duchenne muscular dystrophy: A qualitative study.

Background: As new therapies for Duchenne muscular dystrophy (DMD) are entering the market, shared decision making (SDM) will become increasingly important. Therefore, this study aimed to understand (1) Belgian stakeholders' knowledge and perceptions of SDM in DMD treatment decision making, (2) the current state of SDM implementation in DMD in Belgium, examining the role of all involved parties, and (3) the barriers and facilitators for SDM in DMD in the Belgian context.

Methods: In this qualitative study, semi-structured interviews with the multidisciplinary team (MDT) of individuals with DMD (n = 18) and caregivers thereof (n = 11) were conducted in Belgium.

Drawing on collective action theory to foster sustainable biosimilar markets - insights from co-creation workshops with UK and Belgian stakeholders.

Background: Research has signaled the need for reformed biosimilar policy frameworks that adopt a behavioral approach, are informed by consensus-generating strategies and thus better align with the requirements of local healthcare communities.

Research Design And Methods: Through a series of co-creation workshops, the current study explores the feasibility of applying learnings from Collective Action Theory to formulate evidence-based multistakeholder-supported policy recommendations.

Results: Insights from the conducted workshops indicate that future policy frameworks would benefit from: 1) a working system of incentives and rewards aligned with stakeholder needs; 2) evaluating the cost-benefit balance for stakeholders prior to policy implementation; 3) involving multistakeholder panels in policy co-design; 4) adopting a long-term vision; 5) fostering coordination at the interface between levels of governance; 6) defining shared goals and efficient systems to monitor policy compliance; and 7) using policy outcome data to adapt current policy frameworks based on evolving needs.

Clinical and Socioeconomic Burden of Retinal Diseases: Can Biosimilars Add Value? A Narrative Review.

Retinal diseases, such as neovascular age-related macular degeneration, diabetic retinopathy, and retinal vein occlusion, pose a significant global burden on individuals, families, and healthcare systems. Intravitreal anti-vascular endothelial growth factor (anti-VEGF) therapy has become the standard treatment for retinal diseases, improving clinical outcomes, while delaying disease progression. Although effective, biologics are associated with high costs, which can lead to underutilisation and, consequently, suboptimal patient treatment outcomes, further contributing to healthcare costs.

Real-world cost-effectiveness of nirmatrelvir-ritonavir as treatment for SARS-CoV-2 infection in the Belgian setting with omicron variant.

Background: Nirmatrelvir-ritonavir is an oral treatment for SARS-CoV-2 infection in patients who are at high risk of developing severe COVID-19 disease. This antiviral has proven to significantly reduce the risk of hospitalization and death compared to no anti-SARS-CoV-2 treatment in this target population. This paper aims to assess the cost-effectiveness of nirmatrelvir-ritonavir in Belgium using real-world evidence.

Towards implementing new payment models for the reimbursement of high-cost, curative therapies in Europe: insights from semi-structured interviews.

Background: New ways of reimbursement for high-cost, one-shot curative therapies such as advanced therapy medicinal products (ATMPs) are a growing area of interest to stakeholders in market access such as industry representatives, legislative and accounting experts, physicians, hospital managers, hospital pharmacists, patient representatives, policymakers, and sickness funds. Due to the complex nature of ATMPs, new payment models and reimbursement modalities are proposed yet not widely applied across Europe.

Objectives: This study aimed to elicit opinions on and insights into the governance aspect of implementing outcome-based spread payments (OBSP) in Belgium for the reimbursement of innovative therapies.

Mitigating the Nocebo Effect in Biosimilar Use and Switching: A Systematic Review.

Background: In the context of biosimilar use and switching, a patient's negative perception of a biosimilar might trigger a nocebo effect, where negative expectations unrelated to its pharmacologic action may lead to worsened symptoms, resulting in less perceived therapeutic benefit or worsened adverse events. Considering the crucial role biosimilars have in reducing health care costs and expanding access to safe biologics globally, it is essential to develop and implement effective strategies to mitigate the possible occurrence of such nocebo effects.

Objective: The aim of this systematic literature review was two-fold: (i) to review strategies that have been applied and/or tested for minimising the nocebo effect in clinical practice, within and outside the context of biosimilar switching, and (ii) to propose recommendations on useful mitigation strategies to minimise the occurrence of the nocebo effect in the context of biosimilar switching.

Expert Consensus Recommendations on a Biosimilars Value Framework for the Gulf Cooperation Council Countries.

Objective: This paper aims to develop a biosimilar value framework with local stakeholders in Gulf Cooperation Council (GCC) countries.

Methods: A convenience sample of ten key opinion leaders from the United Arab Emirates, Kingdom of Saudi Arabia, Kuwait, Oman and Qatar participated in an expert panel meeting in November 2022 that examined factors positively influencing biosimilar adoption in these countries. The discussion was structured around a conceptual biosimilar value framework and an overview of biosimilar policies as derived from a targeted review of the peer-reviewed and grey literature.

The broader benefits of vaccines: methodologies for inclusion in economic evaluation.

Background: As the societal value of vaccines is increasingly recognized, there is a need to examine methodological approaches that could be used to integrate these various benefits in the economic evaluation of a vaccine.

Research Design And Methods: A literature review and two expert panel meetings explored methodologies to value herd immunity, health spillover effects (beyond herd immunity), impact on antimicrobial resistance, productivity, and equity implications of vaccines.

Results: The consideration of broader benefits of vaccines in economic evaluation is complicated and necessitates technical expertise.

Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease.

Background And Objective: Treatment of sickle cell disease (SCD) has traditionally focused on symptomatic and preventative care. Recent advances in novel therapeutic developments, likely to be orphan-designated, are anticipated to carry a substantial price tag. This study assesses the potential budget impact of adopting disease-modifying treatments, crizanlizumab and voxelotor, and pioneering CRISPR gene-edited therapy, CTX001, in the Belgian healthcare system.

Mapping Health Technology Assessment Agency Approaches for Biosimilar Value Assessment: An ISPOR Special Interest Group Report.

Objectives: A systematic literature review undertaken by the ISPOR Biosimilar Special Interest Group highlighted that limited guidance exists on how to assess biosimilars value and on appropriate economic evaluation techniques. This study described current health technology assessment (HTA) agency approaches for biosimilar value assessment.

Methods: Semi-structured interviews (n = 16) were carried out with HTA experts in Africa, America, Asia, Australia, and Europe to investigate current HTA practices for biosimilars.

Implementing the EU HTA regulation: Insights from semi-structured interviews on patient expectations, Belgian and European institutional perspectives, and industry outlooks.

The goal of the Health Technology Assessment (HTA) Regulation 2021/2282 is to establish a more harmonized HTA framework, fostering member states cooperation and enabling equal patient access to innovative health technologies in Europe. This research aimed to assess the impact of the regulation on national HTAs, the strategic implications for health technology developers, and its influence on price and reimbursement negotiations. A scoping literature review encompassing peer-reviewed literature as well as grey literature was conducted.

An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators.

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation's (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group.

Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process.

Can Endangered Biosimilar Markets be Rescued? The Need to Bridge Competing Interests for Long-Term Gain.

Market signals such as: (1) the limited number of biosimilars in the development pipeline, (2) the focus of biosimilar development on high-profit therapeutic areas only, and (3) the increase in the number of biosimilar discontinuations and withdrawals, are indicative of sustainability threats facing biosimilar markets in Europe. Two prominent factors that undermine sustainability are: competing interests between the various stakeholders and a preferential focus on short-term gains, disregarding future sustainability threats, hence the need for effective policies that create sustainable competition in biologic markets. Thus far, measures implemented to foster biosimilar adoption have not been necessarily complied with and have had mixed success.