Background: There are potential conflicts between authorities and companies to fund new premium priced drugs especially where there are safety and/or budget concerns. Dabigatran, a new oral anticoagulant for the prevention of stroke in patients with non-valvular atrial fibrillation (AF), exemplifies this issue. Whilst new effective treatments are needed, there are issues in the elderly with dabigatran due to variable drug concentrations, no known antidote and dependence on renal elimination. Published studies have shown dabigatran to be cost-effective but there are budget concerns given the prevalence of AF. There are also issues with potentially re-designing anticoagulant services. This has resulted in activities across countries to better manage its use.Objective: To (i) review authority activities in over 30 countries and regions, (ii) use the findings to develop new models to better manage the entry of new drugs, and (iii) review the implications for all major stakeholder groups.Methodology: Descriptive review and appraisal of activities regarding dabigatran and the development of guidance for groups through an iterative process.Results: There has been a plethora of activities among authorities to manage the prescribing of dabigatran including extensive pre-launch activities, risk sharing arrangements, prescribing restrictions, and monitoring of prescribing post-launch. Reimbursement has been denied in some countries due to concerns with its budget impact and/or excessive bleeding. Development of a new model and future guidance is proposed to better manage the entry of new drugs, centering on three pillars of pre-, peri-, and post-launch activities.Conclusion: Models for introducing new drugs are essential to optimize their prescribing especially where there are concerns. Without such models, new drugs may be withdrawn prematurely and/or struggle for funding.

Background and objectivesThe expanding diabetes epidemic worldwide could have potentially devastating effects on the development of healthcare systems and economies in emerging countries, both in terms of direct health care costs and loss of working time and disability. This study aims to review evidence on the burden, expenditure, complications, treatment, and outcomes of diabetes in Indonesia and its implications on the current health system developments.MethodsWe conducted a comprehensive literature review together with a review of unpublished data from the Ministry of Health and a public health insurer (Askes). Studies presenting evidence on prevalence, incidence, mortality, costs, complications and cost of complications, treatment, and outcomes were included in the analysis.ResultsA limited number of international, national and local studies on the burden and cost of diabetes in Indonesia were identified. National survey data suggests that in 2007 the prevalence of diabetes was 5.7%, of which more than 70% of cases were undiagnosed. This estimate hides large intracountry variation. There was very limited data available on direct costs and no data on indirect costs. The most commonly-identified complication was diabetic neuropathy.DiscussionThere were a number of limitations in the data retrieved including the paucity of data representative at the national level, lack of a clear reference date, lack of data from primary care, and lack of data from certain regions of the country.ConclusionsIf left unaddressed, the growing prevalence of diabetes in the country will pose a tremendous challenge to the Indonesian healthcare system, particularly in view of the Government’s 2010 mandate to achieve universal health coverage by 2014. Essential steps to address this issue would include: placing diabetes and non-communicable diseases high on the Government agenda and creating a national plan; identifying disparities and priority areas for Indonesia; developing a framework for coordinated actions between all relevant stakeholders.

Introduction: There is continued unmet medical need for new medicines across countries especially for cancer, immunological diseases, and orphan diseases. However, there are growing challenges with funding new medicines at ever increasing prices along with funding increased medicine volumes with the growth in both infectious diseases and non-communicable diseases across countries. This has resulted in the development of new models to better manage the entry of new medicines, new financial models being postulated to finance new medicines as well as strategies to improve prescribing efficiency. However, more needs to be done. Consequently, the primary aim of this paper is to consider potential ways to optimize the use of new medicines balancing rising costs with increasing budgetary pressures to stimulate debate especially from a payer perspective.Methods: A narrative review of pharmaceutical policies and implications, as well as possible developments, based on key publications and initiatives known to the co-authors principally from a health authority perspective.Results: A number of initiatives and approaches have been identified including new models to better manage the entry of new medicines based on three pillars (pre-, peri-, and post-launch activities). Within this, we see the growing role of horizon scanning activities starting up to 36 months before launch, managed entry agreements and post launch follow-up. It is also likely there will be greater scrutiny over the effectiveness and value of new cancer medicines given ever increasing prices. This could include establishing minimum effectiveness targets for premium pricing along with re-evaluating prices as more medicines for cancer lose their patent. There will also be a greater involvement of patients especially with orphan diseases. New initiatives could include a greater role of multicriteria decision analysis, as well as looking at the potential for de-linking research and development from commercial activities to enhance affordability.Conclusion: There are a number of ongoing activities across countries to try and fund new valued medicines whilst attaining or maintaining universal healthcare. Such activities will grow with increasing resource pressures and continued unmet need.

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Background Managed entry agreements (MEAs) are a set of instruments to facilitate access to new medicines. This study surveyed the implementation of MEAs in Central and Eastern Europe (CEE) where limited comparative information is currently available. Method We conducted a survey on the implementation of MEAs in CEE between January and March 2017.Results Sixteen countries participated in this study. Across five countries with available data on the number of different MEA instruments implemented, the most common MEAs implemented were confidential discounts (n = 495, 73%), followed by paybacks (n = 92, 14%), price-volume agreements (n = 37, 5%), free doses (n = 25, 4%), bundle and other agreements (n = 19, 3%), and payment by result (n = 10, [1%). Across seven countries with data on MEAs by therapeutic group, the highest number of brand names associated with one or more MEA instruments belonged to the Anatomical Therapeutic Chemical (ATC)-L group, antineoplastic and immunomodulating agents (n = 201, 31%). The second most frequent therapeutic group for MEA implementation was ATC-A, alimentary tract and metabolism (n = 87, 13%), followed by medicines for neurological conditions (n = 83, 13%). Conclusions Experience in implementing MEAs varied substantially across the region and there is considerable Alessandra Ferrario was a Research Officer at the LSE Health at the time this research was conducted. She is now a postdoctoral Research

BackgroundDiabetes and its complications are a major cause of morbidity and mortality in India, and the prevalence of type 2 diabetes is on the rise. This calls for an assessment of the economic burden of the disease.ObjectiveTo conduct a critical review of the literature on cost of illness studies of diabetes and its complications in India.MethodsA comprehensive literature review addressing the study objective was conducted. An extraction table and a scoring system to assess the quality of the studies reviewed were developed.ResultsA total of nineteen articles from different regions of India met the study inclusion criteria. The third party payer perspective was the most common study design (17 articles) while fewer articles (n =2) reported on costs from a health system or societal perspective. All the articles included direct costs and only a few (n =4) provided estimates for indirect costs based on income loss for patients and carers. Drug costs proved to be a significant cost component in several studies (n =12). While middle and high-income groups had higher expenditure in absolute terms, costs constituted a higher proportion of income for the poor. The economic burden was highest among urban groups. The overall quality of the studies is low due to a number of methodological weaknesses. The most frequent epidemiological approach employed was the prevalence-based one (n =18) while costs were mainly estimated using a bottom up approach (n =15).ConclusionThe body of literature on the costs of diabetes and its complications in India provides a fragmented picture that has mostly concentrated on the direct costs borne by individuals rather than the healthcare system. There is a need to develop a robust methodology to perform methodologically rigorous and transparent cost of illness studies to inform policy decisions.

Key points for Decision Makers: European countries apply different pharmaceutical pricing and reimbursement policies. These policies are frequently assessed against their financial consequences and their ability to contain costs but less so in terms of access to medicines. Policies should be accompanied by regular evaluations, facilitated by the use of the appropriate methodology and access to the relevant data. There appears to be a need for additional changes beyond traditional pharmaceutical pricing and reimbursement policies. Collaborative approaches (e.g. between countries or between regulatory authorities, pricing and reimbursement agencies) and more transparency in terms of real medicine prices, R+D costs and medicines in the pipeline are considered as possible pathways for the future. AbstractThe paper discusses pharmaceutical pricing and reimbursement policies in European countries with regard to their ability to ensure affordable access to medicines. A frequently applied pricing policy is external price referencing. While it provides some benchmark for policy-makers and has shown to be able to generate savings, it may also contribute to delay in product launch in countries where medicine prices are low. Value-based pricing has been proposed as a policy that promotes access while rewarding useful innovation, however implementing it has proven quite challenging. For high-priced medicines, managed-entry agreements are increasingly used. These agreements allow policy-makers to manage uncertainty and obtain lower prices. They can also facilitate earlier market access in case of limited evidence about added therapeutic value of the medicine. However, these agreements raise transparency concerns due to the confidentiality clause. Tendering as used in the 2 hospital and offpatent outpatient sectors has proven to reduce medicine prices but it requires a robust framework and appropriate design with clear strategic goals in order to prevent shortages. These pricing and reimbursement policies are supplemented by the widespread use of Health Technology Assessment to inform decision-making, and by strategies to improve the uptake of generics, and also biosimilars. While European countries have been implementing a set of policy options, there is a lack of thorough impact assessments of several pricing and reimbursement policies on affordable access. Increased cooperation between authorities, experience sharing, and improving transparency on price information, including the disclosure of confidential discounts, are opportunities to address current challenges.3

BackgroundWith an estimated 74% of all deaths attributable to non-communicable diseases (NCDs) in 2010, NCDs have become a major health priority in Brazil. The objective of the study was to conduct a comprehensive literature review on diabetes in Brazil; specifically: the epidemiology of type 2 diabetes, the availability of national and regional sources of data (particularly in terms of direct and indirect costs) and health policies for the management of diabetes and its complications.MethodsA literature search was conducted using PubMed to identify articles containing information on diabetes in Brazil. Official documents from the Brazilian government and the World Health Organization, as well as other grey literature and official government websites were also reviewed.ResultsFrom 2006 to 2010, an approximate 20% increase in the prevalence of self-reported diabetes was observed. In 2010, it was estimated that 6.3% of Brazilians aged 18 years or over had diabetes. Diabetes was estimated to be responsible for 278,778 years of potential life lost for every 100,000 people. In 2013, it is estimated that about 7% of patients with diabetes has had one or more of the following complications: diabetic foot ulcers, amputation, kidney disease, and fundus changes. The estimated annual direct cost of diabetes was USD $3.952 billion in 2000; the estimated annual indirect cost was USD $18.6 billion. The two main sources of data on diabetes are the information systems of the Ministry of Health and surveys. In the last few years, the Brazilian Ministry of Health has invested considerably in improving surveillance systems for NCDs as well as implementing specific programmes to improve diagnosis and access to treatment.ConclusionsBrazil has the capacity to address and respond to NCDs due to the leadership of the Ministry of Health in NCD prevention activities, including an integrated programme currently in place for diabetes. Strengthening the surveillance of NCDs is a national priority along with recognising the urgent need to invest in improving the coverage and quality of mortality data. It is also essential to conduct regular surveys of risk factors on a national scale in order to design effective preventive strategies.