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Background Public health care payer organizations face increasing pressures to make transparent and sustainable coverage decisions about ever more expensive prescription drugs, suggesting a need for public engagement in coverage decisions. However, little is known about countries’ approaches to integrating public preferences in existing funding decisions. The aim of this study was to describe how Belgium and New Zealand used deliberative processes to engage the public and to identify lessons learned from these countries’ approaches. Methods To describe two countries’ deliberative processes, we first reviewed key country policy documents and then conducted semi-structured interviews with five leaders of the processes from Belgium and New Zealand. We assessed each country’s rationales for and approaches to engaging the public in pharmaceutical coverage decisions and identified lessons learned. We used qualitative content analysis of the interviews to describe key themes and subthemes. Results In both countries, the national public payer organization initiated and led the process of integrating public preferences into national coverage decision making. Reimbursement criteria considered outdated and changing societal expectations prompted the change. Both countries chose a deliberative process of public engagement with a multi-year commitment of many stakeholders to develop new reimbursement processes. Both countries’ new reimbursement processes put a stronger emphasis on quality of life, the separation of individual versus societal perspectives, and the importance of final reimbursement decisions being taken in context rather than based largely on cost-effectiveness thresholds. Conclusions To face the growing financial pressure of sustainable funding of medicines, Belgium’s and New Zealand’s public payers have developed processes to engage the public in defining the reimbursement system’s priorities. Although these countries differ in context and geographic location, they came up with overlapping lessons learnt which include the need for 1) political commitment to initiate change, 2) broad involvement of all stakeholders, and 3) commitment of all to engage in a long-term process. To evaluate these changes, further research is required to understand how coverage decisions in systems with and without public engagement differ.

Background Reimbursing orphan medicinal products (OMPs) presents both opportunities and challenges for national healthcare payers and health technology assessment (HTA) bodies because of their potential high benefits, large clinical uncertainties and high prices. To support a more structured application of (outcome-based) managed entry agreements (MEAs) intended to mitigate these OMP-related reimbursement challenges, a matrix was developed to facilitate reimbursement negotiations and, ultimately, patient access. Methods A systematic literature review was performed, searching PubMed, Embase, and grey literature from 1 January 2000 until 1 January 2024 to globally identify reimbursement challenges (clinical-, cost-effectiveness uncertainties, or financial risks) described in relation to MEAs for OMPs. The data retrieved were used to develop a matrix that structures the drivers of managed access agreements to reduce financial risk and reimbursement challenges specific to OMPs. Results A total of 77 studies were included in the review, identifying 23 different types of MEAs for OMPs. The results indicated that more commonly known MEAs were designed to mitigate different reimbursement challenges, and more innovative MEAs and combinations thereof have been frequently described in literature. The selected case study of Myozyme ® illustrated how the matrix can present stakeholders with additional mitigation strategies for the relevant reimbursement challenges. Conclusion To address reimbursement challenges for OMPs along their life cycle, it is valuable to consider both established and innovative, e.g., outcome-based MEAs. Combining reimbursement and payment models has the potential to address multifaceted reimbursement challenges. The developed matrix fills a gap in providing a structure for drivers of MEAs tailored to OMPs, enhancing decision-making processes and ultimate patient access to OMPs targeting high unmet medical needs.

To identify pharmaceutical policy changes during the economic recession in eight European countries and to determine whether policy measures resulted in lower sales of, and less expenditure on, pharmaceuticals. Information on pharmaceutical policy changes between 2008 and 2011 in eight European countries was obtained from publications and pharmaceutical policy databases. Data on the volume and value of the quarterly sales of products between 2006 and 2011 in the 10 highest-selling therapeutic classes in each country were obtained from a pharmaceutical market research database. We compared these indicators in economically stable countries; Austria, Estonia and Finland, to those in economically less stable countries, Greece, Ireland, Portugal, Slovakia and Spain. Economically stable countries implemented two to seven policy changes each, whereas less stable countries implemented 10 to 22 each. Of the 88 policy changes identified, 33 occurred in 2010 and 40 in 2011. They involved changing out-of-pocket payments for patients in 16 cases, price mark-up schemes in 13 and price cuts in 11. Sales volumes increased moderately in all countries except Greece and Portugal, which experienced slight declines after 2009. Sales values decreased in both groups of countries, but fell more in less stable countries. Less economically stable countries implemented more pharmaceutical policy changes during the recession than economically stable countries. Unexpectedly, pharmaceutical sales volumes increased in almost all countries, whereas sales values declined, especially in less stable countries.

Abstract Nearly 2 billion people globally have no access to essential medicines. This means essential medicines are unavailable, unaffordable, inaccessible, unacceptable or of low quality for more than a quarter of the population worldwide. This supplement demonstrates the implications of poor medicine access and highlights recent innovations to improve access to essential medicines by presenting new research findings from low- and middle-income countries (LMICs). These studies answer key questions such as: Can performance-based financing improve availability of essential medicines? How affordable are cardiovascular treatments for children? Which countries’ legal frameworks promote universal access to medicines? How appropriately are people using medicines? Do poor-quality medicines impact equity? Answers to these questions are important as essential medicines are vital to the Sustainable Development Goals and are central to the goal of achieving Universal Health Coverage. Access to affordable, quality-assured essential medicines is crucial to reducing the financial burden of care, preventing greater pain and suffering, shortening the duration of illness, and averting needless disabilities and deaths worldwide. This supplement was organized by the Medicines in Health Systems Thematic Working Group of Health Systems Global, a membership organization dedicated to promoting health systems research and knowledge translation. The five studies in the supplement further our understanding by showcasing recent successes and challenges of improving access to quality-assured medicines through health systems in LMICs.

This paper reviews the main pricing policies in Latin American countries, discussing their shortcomings. It also gives an overview of the most common pricing and reimbursement policies in Europe and describes in detail three well-established approaches — international price referencing, value-based pricing, including setting up of health technology assessment, and generic and biosimilar policies — building on country examples.

Objective To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions. Methods A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population. To demonstrate the potential of the framework, three case studies were used: onasemnogene abeparvovec (Zolgensma ® ), brexucabtagene autoleucel (Tecartus ® ) and etranacogene dezaparvovec (Hemgenix ® ). A hypothetical case study was used to illustrate the output of the calculation tool. Results Part 1 of the framework presents steps for matching a suitable reimbursement and payment model with the disease and treatment characteristics. The reimbursement and payment models are further specified in Part 2. Part 3 guides end users through the setup of a calculation tool with which the financial impact can be calculated of two payment models: a price discount model and an outcome-based spread payment model with a discount. Part 4 concerns the output of the calculation tool, showing how different payment models lead to different financial consequences under three assumptions of longer term effectiveness. Conclusions The presented framework provides decision makers with insight into the financial consequences of their chosen payment model under different assumptions. This can aid reimbursement negotiations by clarifying the optimal choice given a therapy’s characteristics.

Managing expenditures on pharmaceuticals is important for health systems to sustain universal access to necessary medicines. We sought to estimate the size and sources of differences in expenditures on primary care medications among high-income countries with universal health care systems. We compared data on the 2015 volume and cost per day of primary care prescription drug therapies purchased in 10 high-income countries with various systems of universal health care coverage (7 from Europe, in addition to Australia, Canada and New Zealand). We measured total per capita expenditure on 6 categories of primary care prescription drugs: hypertension treatments, pain medications, lipid-lowering medicines, noninsulin diabetes treatments, gastrointestinal preparations and antidepressants. We quantified the contributions of 5 drivers of the observed differences in per capita expenditures. Across countries, the average annual per capita expenditure on the primary care medicines studied varied by more than 600%: from $23 in New Zealand to $171 in Switzerland. The volume of therapies purchased varied by 41%: from 198 days per capita in Norway to 279 days per capita in Germany. Most of the differences in average expenditures per capita were driven by a combination of differences in the average mix of drugs selected within therapeutic categories and differences in the prices paid for medicines prescribed. Significant international differences in average expenditures on primary care medications are driven primarily by factors that contribute to the average daily cost of therapy, rather than differences in the volume of therapy used. Average expenditures were lower among single-payer financing systems that appeared to promote lower prices and the selection of lower-cost treatment options.

IntroductionFindable, structured, and understandable data from health technology assessment (HTA) reports is the core of HTA policy research. Available databases with this information, such as the International Network of Agencies for Health Technology Assessment (INAHTA) database, may be incomplete and their common manual data collection is time-consuming. Automated data extraction may offer a solution by creating a standardized, real-time-updating, comprehensive, open-access HTA database.MethodsIn this research, we explore the possibilities of automated data extraction in the context of creating a standardized and comprehensive HTA policy research database. Data points were extracted from publicly available guidance reports of the National Institute for Health and Care Excellence (NICE) using different text extraction techniques such as natural language processing (NLP) and generative pre-trained transformers (GPTs). Future efforts are aiming to expand the database to other HTA bodies and link it to the European Medicines Regulatory Database (EMRD) that is also being developed.ResultsPreliminary results of our research show that it is possible to use existing text extraction techniques to extract relevant information from publicly available HTA recommendations. Scaling the system to include more HTA bodies and data points is challenging as extraction based on document structure is complicated by heterogeneity in document structure within HTA bodies and between HTA bodies. Future results will focus on finding the best data extraction approach for each data point and on validating the system.ConclusionsUsing automated data extraction to extract data from HTA reports can be a viable option for creating a comprehensive database that can be used to enhance comparative HTA policy research. Challenges remain in scaling the system to include more HTA bodies and data points. Results regarding best-performing extraction techniques and data validation of the system are expected soon.