Lessons learned from the Canadian Fabry Disease Initiative for future risk-sharing and managed access agreements for pharmaceutical and advanced therapies in Canada.

Risk sharing agreements (RSAs) and managed access agreements have emerged as tools to overcome evidentiary uncertainty and contain costs of pharmaceuticals; however, Canada has relatively little experience with these health policy instruments. This article describes one of the few examples of national RSAs. Enzyme replacement therapies (ERT) were introduced in Canada to treat Fabry disease in the early 2000s through an RSA. Based on qualitative interviews with key participating actors, this article explains how this RSA ensured continuity of treatment for patients already on ERT, and collected robust real-world evidence to secure treatment for future Fabry patients. We show the importance of partnerships, collaborations, and active patient communities in establishing RSAs, as well as the critical role of robust registries for the collection, storage, and use of that real-world data. In doing so, this paper points to reasons that explain the relative dearth of RSAs in Canada, which can be resource (both human and finance) intensive and are difficult to broker in a federalist health system. Through these findings, policy lessons are developed concerning the need for technological and governance platforms on how RSA in Canada can be more effectively supported going forward in a broader move towards "social pharmaceutical innovation".

Implementing outcomes-based risk-sharing agreements: an integrative review of applications in blood cancer in the UK and beyond.

INTRODUCTION: Outcomes-based risk-sharing agreements (OBRSA) have been increasingly used worldwide to manage uncertainty in value assessments. This review aimed to summarize motivations, barriers, and facilitators to implementing OBRSAs with a specific focus on therapies for hematological cancer. AREAS COVERED: An integrative review was conducted based on a scoping of existing reviews on the topic and reports published by UK NICE. Findings from 16 articles and 10 reports were summarized and categorized into three themes: applications in blood cancer drugs, motivations for adoption, and barriers and facilitators to implementation. EXPERT OPINION: There was a dissociation between the theoretical basis for opting for OBRSAs, and reasons stated or inferred from practice. The administrative burden was considered a notable barrier to implementation, which affects not only payers and manufacturers but also healthcare providers. Effective stakeholder engagement and building mutual trust among key groups were identified as factors enabling successful implementation. The review raises essential considerations in implementing OBRSAs and implications for their future role, particularly for blood cancer drugs where uncertainty is rife. Carefully designed and managed schemes may remain an option for health systems to manage risks involved when funding high-cost treatments.

Opportunities and Threats of the Legally Facilitated Performance-Based Managed Entry Agreements in Slovakia: The Early-Adoption Perspective.

Slovakia has adopted an amendment to Act No. 363/2011, regulating, among other things, drug reimbursement and is undergoing a significant change in the availability of innovative treatments for patients. High expectations are associated with arrangements related to performance-based managed entry agreements. Opinions and positions towards this change appear to be inconsistent, and for the further application of the law in practice and when setting up the main implementation processes, it is necessary to understand the positions and opinions of the individual actors who are involved in the PB-MEA process. The interviews were conducted in the period from 20 May to 15 August 2022 around the same time as the finalisation of the amendment to Act No. 363/2011 and its adoption. A roughly one-hour open interview was conducted on a sample of 12 stakeholders in the following groups: representatives of the Ministry of Health, health-care providers, pharmaceutical companies and others, including a health insurance company. The main objective was to qualitatively describe the perception of this topic by key stakeholders in Slovakia. The responses were analysed using MAXQDATA 2022 software to obtain codes associated with key expressions. We identified three main strong top categories of expressions that strongly dominated the pro-management interviews with stakeholders: legislation, opportunities and threats. Ambiguity and insufficient coverage of the new law, improved availability of medicinal products and threats associated with data, IT systems and potentially unfavourable new reimbursement schemes were identified as key topics of each of the said top categories, respectively. Among individual sets of respondents, there is frequent consensus on both opportunities and threats in the area of implementing process changes in PB-MEA. For the successful implementation of the law in practice, some basic threats need to be removed, among which in particular is insufficient data infrastructure.

Globally optimal trial design and risk sharing arrangements are key to avoiding opportunity costs of delay and enabling equitable, feasible and effective global vaccine research and implementation in current or future pandemics.

Global vaccination in the face of pandemics such as COVID-19 and new variants is a race against time. Avoiding the opportunity costs of delay and the associated health, social, and downstream economic impacts is a challenge and an imperative. Failures to address the global challenges posed by COVID-19 have become increasingly evident as waves of vaccine-evading mutations have emerged, facilitated by unequal vaccination coverage and diminishing immunity against new variants worldwide. To address these challenges, societal decision-makers (governments) and industry manufacturer interests must be better aligned for rapid, globally optimal trial design, ideally with research coverage, implementation, and accessibility of effective vaccines across joint research, implementation, and distribution cycles to address pandemic evolution in real time. Value of information (VoI) methods for optimal global trial design and risk-sharing arrangements align the research, distribution, and implementation interests and efforts globally to meet head-on the imperative of avoiding opportunity costs of delay and enabling consistent global solutions with maximizing local and global net benefits. They uniquely enable feasible early adoption of the most promising strategies in real time while the best globally translatable evidence is collected and interests are aligned for global distribution and implementation. Furthermore, these methods are generally shown to be imperative for feasible, fast, and optimal solutions across joint research, reimbursement, and regulatory processes for current and future pandemics and other global existential threats. Establishing pathways for globally optimal trial designs, risk-sharing agreements, and efficient translation to practice is urgent on many fronts.

Country versus pharmaceutical company interests for hepatitis C treatment.

Hepatitis C virus (HCV) is one of the leading causes of liver disease and is responsible for massive health and economic burden worldwide. The disease is asymptomatic in its early stages, but it can progress over time to fatal end-stage liver disease. Thus, the majority of individuals infected with HCV are unaware of their chronic condition. Recent treatment options for HCV can completely cure the infection but are costly. We developed a game model between a pharmaceutical company (PC) and a country striving to maximize its citizens' utility. First, the PC determines the price of HCV treatment; then, the country responds with corresponding screening and treatment strategies. We employed an analytical framework to calculate the utility of the players for each selected strategy. Calibrated to detailed HCV data from Israel, we found that the PC will gain higher revenue by offering a quantity discount rather than using standard fixed pricing per treatment, by indirectly forcing the country to conduct more screening than it desired. By contrast, risk-sharing agreements, in which the country pays only for successful treatments are beneficial for the country. Our findings underscore that policy makers worldwide should prudently consider recent offers by PCs to increase screening either directly, via covering HCV screening, or indirectly, by providing discounts following a predetermined volume of sales. More broadly, our approach is applicable in other healthcare settings where screening is essential to determine treatment strategies.

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. A number of binary logit models captured the probability (Odds ratio (OR)) of a previous coverage rejection being reversed to positive after resubmission with vs. without a MEA. Gamma generalised linear models were used to understand if there is any association between time to final funding decision and the presence of MEA, among other decision-making variables, and if so, the strength and direction of this association (Beta coefficient (B)). RESULTS: Of the 59 previously rejected medicine-indication pairs studied, 88.2% (n = 45) received a favourable decision after resubmission with MEA vs. 11.8% (n = 6) without. Average time from original submission to final funding decision was 404 (± 254) and 452 (± 364) days for submissions without vs. with MEA respectively. Resubmissions with a MEA had a higher likelihood of receiving a favourable funding decision compared to those without MEA (43.36 < OR < 202, p < 0.05), although approval specifically with an outcomes-based agreement was associated with an increase in the time to final funding decision (B = 0.89, p < 0.01). A statistically significant decrease in time to final funding decision was observed for resubmissions in Australia and Scotland compared to England and Sweden, and for resubmissions with a clinically relevant instead of a surrogate endpoint. CONCLUSIONS: MEAs can improve availability of medicines by increasing the likelihood of reimbursement for medicines that would have otherwise remained rejected from reimbursement due to their evidentiary uncertainties. Nevertheless, approval with a MEA can increase the time to final funding decision, while the true, added value for patients and healthcare systems of the interventions approved with MEAs in comparison to other available interventions remains unknown.

Pfizer and Palbociclib in China: Analyzing an Oncology Pay-for-Performance Plan.

OBJECTIVES: China is poised to become the world's second-largest oncology drug market. Its ability to continue broadening health coverage is in question. Institutional innovations such as performance-based risk-sharing agreements (PBRSAs) have been developed to promote access to novel therapeutics beyond that provided by public health insurance and central procurement systems. We examine in depth the financial implications of a PBRSA developed in China for the breast cancer drug palbociclib. METHODS: We generated a 2-state Markov model from PBRSA information made publicly available. Model inputs included breast cancer outcomes data from the published literature. The primary analysis estimates the percentage reduction in overall drug expenditures due to the PBRSA. Sensitivity analyses explored the financial impact of varied computed tomography scan utilization, rebate rate, and rebate duration. RESULTS: Estimated palbociclib expenditures for the PBRSA cohort totaled $36 278 000. Based on the publicly available information for the PBRSA, an effective discount of 1.3% was estimated. The effective discount was insensitive to changes in computed tomography scan utilization. CONCLUSIONS: The palbociclib PBRSA likely had negligible impact on patient access to therapy and limited downstream financial impact to patients and payers. The short duration of the rebate window, small rebate, and disease indolence contributed to the low expected rebate percentage.

The current state of patient access to new drugs in South Korea under the positive list system: evaluation of the changes since the new review pathways.

Objective: This study aims to provide an up-to-date analysis of the current state of patient access to new drugs in South Korea, focusing on the effect of new review pathways for reimbursement. Methods: We analyzed patients' access to new drugs, listing rate and lead time until listing from marketing authorization. New pathways were defined as 'price negotiation waiver,' 'risk-sharing agreements,' and 'pharmacoeconomic evaluation exemption.' Results: The listing rate for drugs increased after the introduction of the new pathways (93.7% vs. 77.9%, p < 0.001). Before the new pathways, the median lead time for listing was 21.0 months (95% CI: 16.9-25.0), while afterward it was shortened to 10.9 months (95% CI: 10.2-11.7) (p < 0.001). Conclusion: Although it has strengthened national health insurance coverage by positively impacting the rate and lead time, the lead time for the oncology and orphan drugs is substantially longer as compared to other drugs. Expanding the eligibility criteria to include non-life-threatening but rare or intractable diseases, and resolving the system's operational issues are still necessary.

Inspiration from International Experience on Risk Sharing Agreements of Medical Insurance Access for CAR-T Products / 中国药房

OBJECTIVE：To learn from the experience of foreign listed chimeric antigen receptor T lymphocyte （CAR-T） products in signing risk sharing agreements in medical insurance access ，so as to provide references for relevant decisions of medical insurance departments in China. METHODS ：Taking 9 risk sharing agreements of CAR-T products marketed in the United Kingdom，France，Italy and Germany as samples ，the international experience of medical insurance payment of CAR-T products were analyzed from six dimensions ，such as agreement types ，monitoring indicators ，data collection metho ds，agreement periods ， payment conditions and payment methods. Some suggestions were put forward for the medical insurance access of these products in China. RESULTS & CONCLUSIONS ：Four sample countries generally signed risk sharing agreements of medical insurance access （financial agreement and performance-based agreement ）with pharmaceutical enterprises ；the indicators such as progressive disease and progression-free survival were collected by using data collection system or clinical research data ，so as to monitor the efficacy and safety of CAR-T products. The agreement periods and payment conditions were determined according to different agreement types；“medical insurance advance payment ”or“pharmaceutical enterprise advance payment ”combined with “staged payments ” were adopted for risk control. Solving the risk of medical insurance funds caused by “efficacy uncertainty ”is the core issue of CAR-T product access. The induction of risk sharing agreements may be the way to solve this problem ，and the scientific design of the various elements of risk sharing agreements is a prerequisite to ensure that the agreement is operational.

Compartilhamento de risco na saúde suplementar brasileira / Risk sharing in Brazilian supplementary health

Objetivos: Este trabalho discute os recentes impactos do avanço tecnológico no mercado da saúde e a crescente importância do aumento de custos em toda a sua cadeia e investiga uma alternativa para aumentar o acesso a essas novas tecnologias por um maior número de pessoas na saúde suplementar. Nesse contexto, o objetivo geral proposto para este trabalho foi evidenciar as visões de gestores de saúde com papel de destaque no Brasil a respeito do uso de modelos de compartilhamento de risco (risk sharing) em diferentes geografias e em diferentes financiadores no sistema. Pretendeu-se aprofundar o tema com o cumprimento dos seguintes objetivos específicos, também sob a perspectiva dos participantes da pesquisa: verificar a utilização desses modelos de pagamento e, consequentemente, determinar as características e adaptações necessárias a eles; identificar os desafios para a consolidação do risk sharing na saúde suplementar; verificar o impacto do risk sharing nos custos com saúde das operadoras a partir da percepção dos entrevistados. Métodos: Trata-se de estudo qualitativo, com entrevistas em profundidade com os principais gestores e formadores de opinião em saúde de diferentes instituições no Brasil, totalizando 25 entrevistas. A análise de conteúdo foi escolhida como método de interpretação e construção das categorias para a análise. Resultados: Os resultados foram agrupados nas seguintes categorias: as sugestões e adaptações sugeridas para o melhor funcionamento na realidade brasileira; as críticas e desafios aos modelos propostos. Os resultados sugerem que o compartilhamento de risco pode ser uma alternativa capaz de promover uma nova forma de relacionamento entre os pagadores e a indústria fabricante, substituindo o tradicional modelo de remuneração, que limita a relação linear com todos os setores, buscando sempre o objetivo de atender o paciente com novas alternativas de acesso. Conclusões: Em meio à complexidade do sistema de saúde brasileiro, é importante que os players avaliem novas alternativas de remuneração e incorporação tecnológica. Os desafios são inúmeros, desde a efetivação do perfil dos pacientes elegíveis até a aplicabilidade do risk sharing que ocorre a partir do interesse de ambas as partes em trazer novas tecnologias ao sistema, sem impactos orçamentários significativos, desde que seja viável clínica e economicamente, gerando valor em saúde, na efetividade e nos resultados de desfecho em taxa de sobrevida real

Objectives: This paper discusses the recent impacts of technological advancement on the health market, the growing importance of increasing costs throughout its chain, and investigates an alternative to increasing the access of these new technologies to a greater number of people within supplementary health. In this context, the general objective proposed for this work was to highlight the views of health managers with a prominent role in Brazil regarding the use of risk-sharing models in different regions and in different payers in the system. It was intended to deepen the theme with the fulfillment of the following specific objectives, also from the perspective of the research participants: verify the use of these payment models and consequently determine the characteristics and necessary adaptations for them; identify the challenges for the consolidation of risk-sharing in Supplementary Health; to verify the impact of risk-sharing on health costs of operators based on interviewees' perceptions. Methods: This is a qualitative study with in-depth interviews with main health managers and opinion makers from different institutions in Brazil, totaling 25 interviews. Content analysis was chosen as a method of interpretation and construction of categories for analysis. Results: The results were grouped into the following categories: the suggestions and adaptations suggested for the best operation in the Brazilian reality; criticisms and challenges to the proposed models. The results suggest that risk-sharing may be an alternative capable of promoting a new form of relationship between payers and the manufacturing industry, replacing the traditional remuneration model, which limits the linear relationship with all sectors, always seeking the objective of assisting the patient with new access alternatives. Conclusions: Amid the complexity of the Brazilian health system, it is important for players to evaluate new alternatives for remuneration and technological incorporation. The challenges are innumerable from the realization of the profile of eligible patients and the applicability of risk-sharing that occurs from the interest of both parties in bringing new technologies to the system, without significant budgetary impacts, as long as it is clinically and economically viable, generating health value, effectiveness, and outcomes in survival rates in real world evidences

The impact of managed entry agreements on pharmaceutical prices.

Managed entry agreements (MEAs) have been used for several years, with the aim of curbing the growth of pharmaceutical expenditure and enhancing patient access to innovation. Yet, much remains to be understood about their economic implications. This paper studies the impact of MEAs on list prices, that is, prices before the deduction of any discount. Using a theoretical model, we show that, under most price setting regimes, the introduction of an MEA leads to a higher list price. This is confirmed by our empirical analysis of a sample of 156 medicines in six countries, providing a conservative estimate of the increase in price due to the MEA of 5.9%. A relevant policy implication is that payers may overestimate the financial gains that can be achieved through this  tool.

Implementing Health Technology Assessments in Latin America: Looking at the Past, Mirroring the Future. A Perspective from the ISPOR Health Technology Assessment Roundtable in Latin America.

OBJECTIVES: In the last two decades, several countries in Latin America (LA) have shown an interest in developing health technology assessments (HTAs), but the process has not been uniform and has often been challenged by the health systems characteristics and the political or economic idiosyncrasies of these countries. METHODS: This article summarizes the discussions held by the participants at the 40th ISPOR HTA Council Roundtable for LA. An additional literature review was carried out to support some of the concepts included. RESULTS: This article includes a brief description of the implementation of HTA over the last 30 years and then a conceptual analysis using examples of the broader use of HTA to support procurement decisions and risk-sharing agreements, which might play a future role in healthcare priority-setting in LA. CONCLUSIONS: Formerly, HTA processes and methods played important although mostly isolated roles (with drug licensing or reimbursement being examples of this). Nowadays, with more and more innovative technologies and the establishment of value frameworks to support the priority setting in healthcare, HTA features a promising panorama for the health systems sustainability.

Acuerdos de Riesgo Compartido: Lecciones Para su Diseño e Implementación a la Luz de la Experiencia Internacional.

BACKGROUND: Despite the growing interest in risk-sharing agreements as appropriate payment mechanisms for high-cost treatments, few practical resources facilitate their adoption. OBJECTIVE: To identify and propose lessons for designing and implementing these models based on a review of the international experience, and to offer a concise model based on these lessons. METHODS: The steps of the Joanna Briggs Institute were adopted, which included identifying the concept and its relevant variants in scientific and gray literature. RESULTS: Forty-one references were examined in depth. The design of these payment mechanisms should be a process carried out by competent actors (payer, producer, specialists, patients, and a neutral entity); the design must be supported by a sound regulatory and contractual framework that structures its components and clarifies the functions of each actor. Finally, there are critical activities for each actor in each phase of the agreement's progress. CONCLUSIONS: The participation of all actors and the clarification of critical elements and tasks are fundamental for the optimal development of the experiences.

The emerging landscape of reimbursement of regenerative medicine products in the UK: publications, policies and politics.

AIM: This paper aims to map the trends and analyze key institutional dynamics that constitute the policies for reimbursement of regenerative medicine (RM), especially in the UK. MATERIALS & METHODS: Two quantitative publications studies using Google Scholar and a qualitative study based on a larger study of 43 semi-structured interviews. RESULTS: Reimbursement has been a growing topic of publications specific to RM and independent from orphan drugs. Risk-sharing schemes receive attention among others for dealing with RM reimbursement. Trade organizations have been especially involved on RM reimbursement issues and have proposed solutions. CONCLUSION: The policy and institutional landscape of reimbursement studies in RM is a highly variegated and conflictual one and in its infancy.

National reimbursement listing determinants of new cancer drugs: a retrospective analysis of 58 cancer treatment appraisals in 2007-2016 in South Korea.

BACKGROUND: Since the positive-list system was introduced, concerns have been raised over restricting access to new cancer drugs in Korea. Policy changes in the decision-making process, such as risk-sharing agreement and the waiver of pharmacoeconomic data submission, were implemented to improve access to oncology medicines, and other factors are also involved in the reimbursement for cancer drugs. The aim of this study is to investigate the reimbursement listing determinants of new cancer drugs in Korea. METHODS: All cancer treatment appraisals of Health Insurance Review and Assessment during 2007-2016 were analyzed based on 13 independent variables (comparative effectiveness, cost-effectiveness, drug-price comparison, oncology-specific policy, and innovation such as new mode of action). Univariate and multivariate logistic analyses were conducted. RESULTS: Of 58 analyzed submissions, 40% were listed in the national reimbursement formulary. In univariate analysis, four variables were related to listing: comparative effectiveness, drug-price comparison, new mode of action, and risk-sharing agreement. In multivariate logistic analysis, three variables significantly increased the likelihood of listing: clinical improvement, below alternative's price, and risk-sharing arrangement. Cancer drug's listing increased from 17% to 47% after risk-sharing agreement implementation. CONCLUSION: Clinical improvement, cost-effectiveness, and RSA application are critical to successful national reimbursement listing.

The implementing experience of pharmaceutical risk-sharing agreements in Australia, New Zealand, Taiwan area and its implication for mainland China / 中国卫生政策研究

With the rapid development of healthcare technologies, the improvement of patient health expecta-tions, and the increasing of the government or insurer's financial budget pressure, risk-sharing agreements has be-come the focus of the governments or insurer concerned. This article systematically analyzed Australia, New Zealand, Taiwan risk-sharing agreements from five aspects, including the operation main, scope, classification, application processes and the implementation effects. According to the results of the analysis, we suggests that China should im-prove risk-sharing agreements theoretical basis, diversify risk-sharing agreements models, establish risk-sharing a-greements standardization process and so on.

The implementing experience of patient access schemes in UK and its implication for China / 中国卫生政策研究

The Risk-sharing agreements have achieved remarkable success in improving patients'access to drugs, lowering the uncertainty of the drugs cost-effectiveness, financial risk control and other aspects of medical in-surance fund , so they have attracted widespread attention by the concerned governments and insurers .This paper sys-tematically reviewed the patient access schemes in UK from several aspects , including the origin of the program , clas-sification , application processes and the implementation effects as well .The results of the research indicated that Chi-na has basically met the conditions for implementation of the risk-sharing agreements .In order to gradually promote the risk-sharing agreements implementation , this paper suggests that China should clarify the main root of risk-sharing agreements implementation , establish risk-sharing agreements standardization process and strengthen the application of health technology assessment in health resources allocation to improve the Chinese medicines bargaining system more scientifically and efficiently .

Futurescapes: expectations in Europe for relative effectiveness evidence for drugs in 2020.

AIM: Explore key factors influencing future expectations for the production of evidence of relative effectiveness (RE) for drugs in Europe in 2020; construct three plausible future scenarios for RE evidence generation. MATERIALS & METHODS: Semi-structured key informant interviews and three rounds of modified Delphi to gather expert perspectives and develop future scenarios. RESULTS & CONCLUSION: Most influential factors were degree of regulator use of postmarketing authorization (postlaunch) efficacy studies and adaptive licensing; degree of pan-European health technology assessment body coordination in reviewing prelaunch evidence and demanding postlaunch studies; the nature of regulator - health technology assessment body interaction. The most likely scenario entailed some change with postlaunch regulatory studies driving the likely nature of RE evidence generated.

Dealing with uncertainty and high prices of new medicines: a comparative analysis of the use of managed entry agreements in Belgium, England, the Netherlands and Sweden.

Managed entry agreements are a set of instruments used to reduce the impact of uncertainty and high prices when introducing new medicines. This study develops a conceptual framework for these agreements and tests it by exploring variations in their implementation in Belgium, England, the Netherlands and Sweden and over time as well as their governance structures. Using publicly available data from HTA agencies and survey data from the European Medicines Information Network, a database of agreements implemented between 2003 and 2012 was developed. A review of governance structures was also undertaken. In December 2012 there were 133 active MEAs for different medicine-indications across the four countries. These corresponded to 110 unique medicine-indications. Over time there has been a steady growth in the number of agreements implemented, with the highest number in the Netherlands in 2012. The number of new agreements introduced each year followed a different pattern. In Belgium and England it increased over time, while it decreased in the Netherlands and fluctuated in Sweden. Only 18 (16%) of the unique medicine-indication pairs identified were part of an agreement in two or more countries. England uses mainly discounts and free doses to influence prices. The Netherlands and Sweden have focused more on addressing uncertainties through coverage with evidence development and, in Sweden, on monitoring use and compliance with restrictions through registries. Belgium uses a combination of the above. Despite similar reasons being cited for managed entry agreements implementation, only in a minority of cases have countries implemented an agreement for the same medicine-indication; when they do, a different agreement type is often implemented. Differences in governance across countries partly explain such variations. However, more research is needed to understand whether e.g. risk-perception and/or notion of what constitutes a high price differ between these countries.

International variability in the reimbursement of cancer drugs by publically funded drug programs.

PURPOSE: Evaluate inter-country variability in the reimbursement of publically funded cancer drugs, and identify factors such as cost containment measures that may contribute to variability. METHODS: As of February 28, 2010, licensed indications for 10 cancer drugs (bevacizumab, bortezomib, cetuximab, erlotinib, imatinib, pemetrexed, rituximab, sorafenib, sunitinib, and trastuzumab) were obtained from the drug registries of 6 licensing authorities corresponding to 13 countries or regions: Australia, Canada (Ontario), England, Finland, France, Italy, Germany, Japan, New Zealand, the Netherlands, Scotland, Sweden, and the United States (Medicare Parts B and D). Number of licensed indications reimbursed by public payers and the use of cost containment measures were obtained by survey of health authorities involved in reimbursement and through public documents. RESULTS: The 48 identified licensed indications varied between agencies (range: 36-44 indications). Finland, France, Germany, Sweden, and the United States reimbursed the highest percentage of indications (range: 90%-100%). Canada (54%), Australia (46%), Scotland (40%), England (38%), and New Zealand (25%) reimbursed the least. All 5 countries with the lowest rate of reimbursement incorporated a cost-effectiveness analysis into reimbursement decisions and rejected submissions for reimbursement mainly because of lack of cost effectiveness; in New Zealand, lack of cost effectiveness was the second leading cause of rejection after excessive cost. In 9 countries, risk-sharing agreements were used to contain costs. Indications initially not recommended for reimbursement (9 in Australia, 5 in Canada, and 3 in England, New Zealand, and Scotland) were subsequently approved with risk-sharing agreements or special pricing arrangements. CONCLUSIONS: Reimbursement of publically funded cancer drugs varies globally. The cause is multifactorial.