Public participation in decision-making on the coverage of new antivirals for hepatitis C.

Purpose - New hepatitis C medicines such as sofosbuvir underline the need to balance considerations of innovation, clinical evidence, budget impact and equity in health priority-setting. The purpose of this paper is to examine the role of public participation in addressing these considerations. Design/methodology/approach - The paper employs a comparative case study approach. It explores the experience of four countries - Brazil, England, South Korea and the USA - in making coverage decisions about the antiviral sofosbuvir and involving the public and patients in these decision-making processes. Findings - Issues emerging from public participation ac tivities include the role of the universal right to health in Brazil, the balance between innovation and budget impact in England, the effect of unethical medical practices on public perception in South Korea and the legitimacy of priority-setting processes in the USA. Providing policymakers are receptive to these issues, public participation activities may be re-conceptualized as processes that illuminate policy problems relevant to a particular context, thereby promoting an agenda-setting role for the public. Originality/value - The paper offers an empirical analysis of public involvement in the case of sofosbuvir, where the relevant considerations that bear on priority-setting decisions have been particularly stark. The perspectives that emerge suggest that public participation contributes to raising attention to issues that need to be addressed by policymakers. Public participation activities can thus contribute to setting policy agendas, even if that is not their explicit purpose. However, the actualization of this contribution is contingent on the receptiveness of policymakers.This is the author accepted manuscript. It is currently under an indefinite embargo pending publication by Emerald

Patterns of public participation: opportunity structures and mobilization from a cross-national perspective

Purpose: The paper summarizes data from twelve countries, chosen to exhibit wide variation, on the role and place of public participation in the setting of priorities. It seeks to exhibit cross-national patterns in respect of public participation, linking those differences to institutional features of the countries concerned. Design/methodology/approach: The approach is an example of case-orientated qualitative assessment of participation practices. It derives its data from the presentation of country case studies by experts on each system. The country cases are located within the historical development of democracy in each country. Findings: Patterns of participation are widely variable. Participation that is effective through routinized institutional processes appears to be inversely related to contestatory participation that uses political mobilization to challenge the legitimacy of the priority setting process. No system has resolved the conceptual ambiguities that are implicit in the idea of public participation. Originality/value: The paper draws on a unique collection of country case studies in participatory practice in prioritization, supplementing existing published sources. In showing that contestatory participation plays an important role in a sub-set of these countries it makes an important contribution to the field because it broadens the debate about public participation in priority setting beyond the use of minipublics and the observation of public representatives on decision-making bodies

Overview of managed entry agreements : an integrative review towards policy making in Brazil

Resuminho (60 palavras): In order to provide legal and scientific embasement for policy making in Brazil, the aim of this study was to provide a panorama of Managed Entry Agreements around the world. A systematic review was conducted and the information about the agreements were summarized. It was included 25 studies, which described 446 agreements performed in 29 countries. Introduction (100 palavras): Managed Entry Agreements (MEA) are a reality in many countries. They are used as a tool to reduce the impact of uncertainty and the high cost of new drugs by providing access to new technologies under pre-established conditions. In Brazil, the Ministry of Health approved a high-cost technology under performance evaluation, being the first experience in the country. The aim of this study was to conduct a review to identify MEA performed worldwide to provide embasement to inform public health policy making in Brazil, as well as critical considerations surrounding the implementation of performance based agreements. Methods (75 palavras): A review of MEA for health technologies was conducted, using the question ‘What are the health technology managed entry agreements that have being performed around the world?’. The searches were conducted in april 2019, through PUBMED, EMBASE, LILACS and Cochrane Library databases, as well as manual search and gray literature. The selection of studies was performed by two independent reviewers and, in cases of disagreement, solved by a third reviewer. Results (75 palavras): A total of 25 studies were included, describing 446 agreements in 29 countries, being Australia (122), Italy (96), the United States (48) and Scotland (42) more frequent. Financial risk-sharing agreements were the most prevalent (43%). About 95% of the agreements involved medicines - more than half antineoplastic agents. The outcomes assessed and the impact of the agreements were not addressed in most studies, which may be due to the confidentiality character of them. Conclusions (100 palavras): We are likely to see a growth in MEA in the future with the continual launch of new high priced and complex treatments, coupled with increasing demands on available resources. They are an important tool to improve access to innovative and high cost medicines to achieve universal health coverage, although there are critical issues to consider. Besides the embedded confidentiality of most of the agreements, learning from already stablished knowledge, experiences and practices across countries can be a crucial strategy to guide Brazil’s initial experiences in this area

Integrative review of managed entry agreements : chances and limitations

Introduction: Managed Entry Agreements (MEAs) consist of a set of instruments to reduce the uncertainty and the budget impact of new high priced medicines; however, there are concerns. There is a need to critically appraise MEAs with their planned introduction in Brazil. Accordingly, the objective is to identify and appraise key attributes and concerns with MEAs among payers and their advisers, with the findings providing critical considerations for Brazil and other high- and middle-income countries. Methods: An integrative review approach was adopted. This involved a review of MEAs across countries. The review question was ‘What are the health technology MEAs that have been applied around the world?’ This review was supplemented with studies not retrieved in the search known to the senior level co-authors including key South American markets. Afterall, involved senior level decision makers and advisers providing guidance on potential advantages and disadvantages of MEAs and ways forward. Results: 25 studies were included in the review. Most MEAs included medicines (96.8%), focused on financial arrangements (43%), and included mostly antineoplastic medicines. Most countries kept key information confidential including discounts or had not published such data. Few details were found in the literature regarding South America. Our findings and inputs resulted in both advantages including reimbursement and disadvantages including concerns with data collection for outcome-based schemes. Conclusion: We are likely to see a growth in MEAs with the continual launch of new high priced and often complex treatments, coupled with increasing demands on resources. Whilst outcome based MEAs could be an important tool to improve access to new innovative medicines there are critical issues to address. Comparing knowledge, experiences and practices across countries is crucial to guide high- and middle-income countries when designing their future MEAs

Time to Review Authorisation and Funding for New Cancer Medicines in Europe? Inferences from the Case of Olaratumab

The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety and added value when new medicines are authorised, and subsequently funded based on initial preliminary data only, have important implications. In 2016 olaratumab received accelerated conditional approval from both the European Medicines Agency and the US Food and Drug Administration for the treatment of soft-tissue sarcoma, based on the claims of a substantial reduction in the risk of death with an 11.8-month improvement in median overall survival in a phase II trial in combination with doxorubicin vs. doxorubicin alone. The failure to confirm these benefits in the post-authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable clinical and economic costs, so that patients may have received suboptimal treatment and any money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential pathways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair manner, with minimal patient exposure as required to achieve robust evidence. Additionally, it may be time to review early access systems, and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post-marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments