J. Mark. Access Health Policy, Volume 3, Issue 1 (January 2015) – 24 articles

Objectives: To take inventory of the current state of affairs of Market Access Launch Excellence in the life sciences industry. To identify key gaps and challenges for Market Access (MA) and discuss how they can be addressed. To generate a baseline for benchmarking MA launch excellence. Methodology: An online survey was conducted with pharmaceutical executives primarily working in MA, marketing, or general management. The survey aimed to evaluate MA excellence prerequisites across the product life cycle (rated by importance and level of implementation) and to describe MA activity models in the respective companies. Composite scores were calculated from respondents’ ratings and answers. Results: Implementation levels of MA excellence prerequisites generally lagged behind their perceived importance. Item importance and the respective level of implementation correlated well, which can be interpreted as proof of the validity of the questionnaire. The following areas were shown to be particularly underimplemented: 1) early integration of MA and health economic considerations in research and development decision making, 2) developing true partnerships with payers, including the development of services ‘beyond the pill’, and 3) consideration of human resource and talent management. The concept of importance-adjusted implementation levels as a hybrid parameter was introduced and shown to be a viable tool for benchmarking purposes. More than 70% of respondents indicated that their companies will invest broadly in MA in terms of capital and headcount within the next 3 years. Conclusions: MA (launch) excellence needs to be further developed in order to close implementation gaps across the entire product life cycle. As MA is a comparatively young pharmaceutical discipline in a complex and dynamic environment, this effort will require strategic focus and dedication. The Market Access Launch Excellence Inventory benchmarking tool may help guide decision makers to prioritize their endeavors. Full article

The slow reaction of French authorities to the so-called Mediator^® saga in 2009 in France led to investigations that questioned the way conflicts of interest are reported. France implemented the Loi Bertrand (‘Bertrand Law’) in May 2013, known as the ‘French Sunshine Act’, with the aim of specifying the scope of disclosure obligations. This policy research reviewed the Loi Bertrand and reported case law from the French Council of State (COS) related to conflicts of interest in French Health technology assessment (HTA) opinion. The Loi Bertrand requires the publication of most of the agreements concluded between health-care professionals and companies and covers a vast range of health products. Commercial sales agreements of goods and services concluded between manufacturers and health-care professionals are a strong exception to this disclosure obligation. Six cases examined by the COS were analyzed, most of them related to the publication of guidelines or the removal of products from the list of reimbursed drugs and devices. These cases have been reviewed, as well as the impact of the ruling on reimbursement decisions. Four cases led to suspension or invalidation of decisions based on the Haute Autorité de Santé (HAS) recommendations due to conflicts of interest. In the two other cases, the HAS provided post hoc declarations of interest when required by the COS, and the COS considered the conflicts of interest as irrelevant for the decision. It appears that the COS based its decisions on two main criteria: the acknowledgement of negative conflicts of interest (a link with competitors) and the absence of declarations of conflicts of interest, which have to be presented when required by legal authorities irrespective of when they were completed (even posterior to the HAS opinion). However, the number of cases that have been decided against the HAS remains very limited with respect to the volume of assessments performed yearly. The strengthening of the regulation on declarations of interest might lead to more transparency but also to more cases decided by the COS. A new press investigation (in March 2015) related to alleged cases of conflict of interests led policy makers to amend the Bertrand Law in April 2015 and require the disclosure of amounts paid to health-care professionals by the industry. Full article

Introduction and objective: Cell therapies can be classified into three main categories of products: advanced therapy medicinal products (ATMPs), ATMPs prepared on a non-routine basis (hospital exemptions), and minimally manipulated cells. Despite the benefits that cell therapies can bring to patients, they are subject to complex pathways to reach the market in France. The objective of this study was to identify and describe routes to market access for cell therapies in France and how these vary by regulatory status. Methodology: The research was structured following five main steps: (1) identification of the French regulatory framework for cell therapies; (2) identification of the health products categorised as cell therapies in France; (3) mapping of the market access pathways per category of cell therapy; (4) validation of findings by interviewing experts; and (5) development of a roadmap summarising market access pathways for cell therapies in France. The secondary research methodology included a comprehensive literature review conducted on websites of French public health institutions, complemented by a research for peer-reviewed articles, abstracts, and grey literature. Results: Different market access pathways are possible depending on the cell therapy category. For ATMPs, market access pathways depend on the licensing status of the therapy. Licensed ATMPs followed the same market access pathways as ‘conventional’ pharmaceuticals, whereas not-yet-licensed ATMPs can be funded via a specific financial allowance under the framework of a Temporary Authorisation for Use procedure or various research programmes. For new ATMPs that are associated with a separate medical device (not considered as ‘combined ATMPs’) or associated with a new medical procedure, additional pathways will apply for the medical device and/or medical procedure to be reimbursed in the ambulatory settings or at hospital. The most likely funding option for ATMPs prepared on a non-routine basis is outside the diagnosis-related group (DRG) system through Missions of General Interest and Support to Contracting (MIGAC). For minimally manipulated cells, four different funding processes are applicable, depending on the type of activity: (1) inclusion in a DRG; (2) inclusion in the list of products and services qualifying for reimbursement (LPPR) (as a medical device); (3) an annual lump sum provided by regional health agencies; and (4) a financial allowance under Missions of General Interest (MIG). Conclusion: Cell therapy is a diverse and promising category of medical interventions. Its heterogeneity and complexity mean that several funding options and market access pathways apply. The main challenges facing cell therapies relate to (1) the identification of the most appropriate path to reimbursement, and (2) price setting, whereas high manufacturing costs of these therapies will dictate a high price that could only be achieved by a product that leads to important additional patient benefits compared to available treatment options. More specific funding options could emerge as the number of cell therapies increases and the authorities face the need to structure and stabilise funding. It will be vital for manufacturers to have a clear understanding of the various temporary funding opportunities early in a product's lifecycle for the adoption of a stepwise approach to secure permanent funding. Furthermore, due to the very limited Health Technology Assessment (HTA) bodies experience for cell therapies, manufacturers should enter into dialogues with HTA agencies at an early stage to optimise market access conditions. Full article

Background: The use of pharmacokinetics is associated with cost savings in anti-tumor necrosis factor (anti-TNF) therapy, but the long-term cost savings in a large cohort of Crohn's disease (CD) patients are unknown. Aim: The goal of this study was to compare the cost of anti-TNF therapy in two cohorts of CD patients losing response to infliximab, one using a test-based strategy and one an empirical dose escalation. Methods: We used a selected mathematical model to describe the trajectories of CD patients based on a discrete event system. This design allowed us to track over a given period a double cohort of patients who moved randomly and asynchronously from one state to another, while keeping all the information on their entire trajectory. Both cohorts were modeled using state diagram parameters where transition probabilities from one state to another are derived from literature data. Costs were estimated based on the French health care system. Results: Cost savings among the 10,000 CD patients using a test-based strategy were €131,300,293 at 5 years. At 5 years the mean cost saving was €13,130 per patient. The direct cost of the test had no impact on the results until the cost per test reached €2,000. Conclusions: A test-based strategy leads to major cost savings related to anti-TNF therapy in CD. Full article

Objective: The aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the key drivers for securing adoption at a commercially viable reimbursed price. Methodology: The research was structured following three main steps: 1) Identifying the market access pathways relevant to ATMPs through secondary research; 2) Validating the secondary research findings and addressing any data gaps in primary research, by qualitative interviews with national, regional, and local-level payers and their clinical and economic advisors; 3) Collating of primary and secondary findings to compare results across countries. Results: The incremental clinical benefit forms the basis for all P&R&MA processes. Budget impact is a key consideration, regardless of geography. Cost-effectiveness analyses are increasingly applied; however, only the United Kingdom has a defined threshold that links the cost per quality-adjusted life year (QALY) specifically and methodologically to the reimbursed price. Funding mechanisms to enable adoption of new and more expensive therapies exist in all countries, albeit to varying extents. Willingness to pay is typically higher in smaller patient populations, especially in populations with high disease burden. Outcomes modelling and risk-sharing agreements (RSAs) provide strategies to address the data gap and uncertainties often associated with trials in niche populations. Conclusions: The high cost of ATMPs, coupled with the uncertainty at launch around their long-term claims, present challenges for their adoption at a commercially viable reimbursed price. Targeting populations of high disease burden and unmet needs may be advantageous, as the potential for improvement in clinical benefit is greater, as well as the potential for capitalising on healthcare cost offsets. Also, targeting small populations can also help reduce both payers’ budget impact concerns and the risk of reimbursement restrictions being imposed. Full article

Objective: To assess the variation of relative efficacy and tolerability of an antidepressant versus others based on both pre-marketing (registration studies) and post-marketing studies versus pre-marketing studies only in patients with major depressive disorder. Methods: The relative efficacy and tolerability of antidepressants was assessed by mixed treatment comparisons (MTCs) using data acquired over two time periods: before registration of the reference drug escitalopram (1989–2002) and up to 5 years later (1989–2007). Ranking probability outputs were presented for efficacy, using change from baseline to 8 weeks on Montgomery–Åsberg Depression Rating Scale total score, and tolerability, using withdrawals due to adverse events. Results: The relative efficacy and tolerability of some selected antidepressants, including escitalopram, varied considerably over the two time periods. The improved relative efficacy and tolerability of escitalopram over time, compared with citalopram, was demonstrated by greater separation of ranking probability curves for efficacy and tolerability. In 2002, escitalopram ranked low with 13.9% and 5.1% probability of being in the top four antidepressants’ relative efficacy and tolerability, respectively. In 2007, ranking probabilities for relative efficacy and tolerability of escitalopram increased to 52.5% and 82.1%, respectively. Conclusions: Time of marketing authorization may not be the most appropriate time to evaluate the relative efficacy and tolerability of a new antidepressant based on MTC approach due to the asymmetry of information between new and older compounds. However, the first evaluation of relative effect of a new drug for health technology assessment recommendations is commonly done at this time. Re-evaluation of a drug several years after its launch is likely to provide a more accurate indication of its relative efficacy and tolerability. Full article

Background: Little information is available about the long-term management of ischemic stroke (IS) in West Scotland. In this study we aim to describe the management of IS at onset, admission, and during follow-up among patients who survived an IS event. Methods: General practitioners (GPs) (n = 20) were randomly selected to recruit IS patients and extract data about patient characteristics, hospitalizations, discharge, and ambulatory care from GP databases, hospital letters, and direct contact with patients and their relatives. Descriptive analyses were conducted. Results: One hundred and one patients were included, with a mean age of 65.6 ± 13.4. About half of the patients contacted their GPs at the time of onset (45.4%). Cardiovascular history was prevalent in 29.7% of cases, and 14% of all cases were recurrences. Of the patients, 89 (88%) were hospitalized with mean length of stay (LOS) 11.8 days. Treatment was administered on average within 12.9 hours of admission and 23.6% of the admitted patients received thrombolytic treatment. During the 1-year follow-up period, 33.6% of patients were rehospitalized and the mean LOS was 15.1 ± 29.5 days. Further, patients on average sought nursing care (10.9%), physical therapy (45.5%), occupational therapy (27.7%), speech therapy (12.9%), and professional caregivers (12%). Conclusion: The health-care resource utilization of IS patients is a major driver of economic burden. Full article

Background: Ischemic stroke (IS) poses physical, emotional, and economic burdens on both patients and the healthcare system in Germany. However, the management of IS is not well described, especially after hospital discharge. In this study, we aim to describe the management of IS at onset, admission, and during follow-up. Methods: German general practitioners (GPs) (n = 40) extracted data on patient characteristics, hospitalizations, discharge, and ambulatory care from both GPs patient databases and hospital letters. Descriptive analyses were conducted. Results: The sample included 185 patients with a mean age of 70 years [standard deviation (SD) = 11.7]. Most patients (63%) contacted the Emergency Medical Services, while 36% contacted their GPs. The majority of patients were hospitalized within 1 h from onset, and the length of stay was on average 14 days. Half of the patients (50%) were admitted to the stroke unit, and 16% of patients received thrombolysis treatment with 2 h (SD = 2.6) of time to treatment. Of the admitted patients, 32% were discharged to their homes, while the remaining patients were discharged to nursing homes (16.2%) and rehabilitation centers (47.6%). During the 12 months follow-up, 22% of patients were re-hospitalized and patients visited their GP (11.7 times), psychologist or psychiatrist (9.5 times), and neurologist (2.2 times). Death rate after stroke event was 13%. Conclusion: The rate of patients who received thrombolysis is lower than the optimal rate in Germany. More research is needed to determine the factors that could predict the utilization of thrombolysis treatment. Full article

Background: Appropriate use of inhaled therapies for asthma and chronic obstructive pulmonary disease (COPD) is critical to ensuring good patient outcomes, efficient use of healthcare resources and limiting the effects of high-morbidity. The appropriate choice of inhaler and active therapy, incorporating patient preferences, can help improve treatment adherence and long-term outcomes. Despite this, many current inhalers are non-intuitive to use, and require extensive training. Methods: In this review, an expert panel considers the evidence for the use of inhaler devices in management of COPD and asthma. The panel also evaluates the value of innovation in inhaler technologies, which optimise the use of existing molecules from a clinical, economic and societal perspective. Conclusions: The panel conclusion is that there remains a substantial unmet need in inhaler technology and that innovation in inhaler devices can provide real-world health benefits to patients. Furthermore, we recommend that these innovations should be supported by healthcare systems through appropriate pricing and reimbursement mechanisms. Full article

Background and objectives: External reference pricing (ERP) is a price regulation tool widely used by policy makers in the European Union (EU) Member States (MS) to contain drug cost, although in theory, it may contribute to modulate prices up and down. The objective of this article was to summarise and discuss the main findings of part of a large project conducted for the European Commission (‘External reference pricing of medicinal products: simulation-based considerations for cross-country coordination’; see www.ec.europa. eu/health/healthcare/docs/erp_reimbursement_medicinal_products_en.pdf) that aimed to provide an over- view of ERP systems, both on processes and potential issues in 31 European countries (28 EU MS, Iceland, Norway, and Switzerland). Methods: A systematic structured literature review was conducted to identify and characterise the use of ERP in the selected countries, to describe its impact on the prices of pharmaceuticals, and to discuss the possible cross-country coordination issues in EU MS. This research was complemented with a consultation of competent authorities’ and international organisations’ representatives to address the main issues or uncertainties identified through the literature review. Results: All selected countries applied ERP, except the United Kingdom and Sweden. Twenty-three countries used ERP as the main systematic criterion for pricing. In the majority of European countries, ERP was based on legislated pricing rules with different levels of accuracy. ERP was applied either for all marketed drugs or for specific categories of medicines; it was mainly used for publicly reimbursed medicines. The number of reference countries included in the basket varied from 1 to 31. There was a great variation in the calculation methods used to compute the price; 15 countries used the average price, 7 countries used the lowest price, and 7 countries used other calculation methods. Reported limitations of ERP application included the lack of reliable sources of price information, price heterogeneity, exchange rate volatility, and hidden discounts. Spill-over effect and downward price convergence have often been mentioned as ERP’s consequences leading to pricing strategies from pharmaceutical companies. Conclusion: While ERP is widely used in Europe, processes and availability of price information vary from one country to another, thus limiting ERP implementation. Furthermore, ERP spill-over effect is a major concern of pharmaceutical firms leading to implementation of the so-called ‘launch sequence strategies’. Full article

Background: Treatment with long-acting injectable (LAI) antipsychotic medication is an important element of relapse prevention in schizophrenia. Recently, the intramuscular once-monthly formulation of aripiprazole received marketing approval in Europe and the United States for schizophrenia. Objective: This study aimed to compare aripiprazole once-monthly with other LAI antipsychotics in terms of efficacy, tolerability, and safety. Data sources: A systematic literature review was conducted to identify relevant double-blind randomized clinical trials of LAIs conducted in the maintenance treatment of schizophrenia. MEDLINE, MEDLINE In-Process, Embase, the Cochrane Library, PsycINFO, conference proceedings, clinical trial registries, and the reference lists of key review articles were searched. The literature search covered studies dating from January 2002 to May 2013. Study selection: Studies were required to have ≥24 weeks of follow-up. Patients had to be stable at randomization. Studies were not eligible for inclusion if efficacy of acute and maintenance phase treatment was not reported separately. Six trials were identified (0.5% of initially identified studies), allowing comparisons of aripiprazole once-monthly, risperidone LAI, paliperidone palmitate, olanzapine pamoate, haloperidol depot, and placebo. Data extraction: Data extracted included study details, study duration, the total number of patients in each treatment arm, efficacy, tolerability, and safety outcomes. The efficacy outcome contained the number of patients that experienced a relapse, tolerability outcomes included the number of patients that discontinued treatment due to treatment-related adverse events (AEs), and that discontinued treatment due to reasons other than AEs (e.g., loss to follow-up). Safety outcomes included the incidence of clinically relevant weight gain and extrapyramidal symptoms. Data synthesis: Data were analyzed by applying a mixed treatment comparison competing risks model (efficacy) and using binary models (safety). There was no statistically significant difference between any study outcome, including the risk of relapse, the risk of discontinuations, and safety outcomes. Conclusions: Aripiprazole once-monthly is similarly efficacious to other LAIs with relatively low rates of discontinuation due to AEs and due to reasons other than AEs than other LAIs. Full article

In the context of current economic difficulties across Europe, accurate budgeting and resource allocation have become increasingly important. Vaccination programmes can respond to the needs of governments to budget with confidence. It may be more reliable and accurate to forecast budget and resource allocation for a vaccination programme than for unpredictable seasonal disease peaks of infections such as rotavirus gastroenteritis, influenza, and pneumonia. In addition, prevention through vaccination involves low levels of investment relative to the substantial benefits that may be obtained. In France, total lifelong vaccination costs, per fully compliant individual, ranged from €865 to €3,313, covering 12 to 16 diseases, which is comparable to, or lower than, costs of other preventive measures. In addition, effectively implemented vaccination programmes have the potential to generate substantial savings both in the short and in the long term. For example, vaccination programmes for rotavirus, meningitis C, human papillomavirus, influenza, and pneumonia have all been shown to significantly reduce the disease burden, and thus the associated costs, in the first years following vaccination implementation. These programmes demonstrate the potential for health authorities to obtain early, and often substantial, return on investment. Full article

The health of a population is important from a public health and economic perspective as healthy individuals contribute to economic growth. Vaccination has the potential to contribute substantially to improving population health and thereby economic growth. Childhood vaccination programmes in Europe can offer protection against 15 important infectious diseases, thus preventing child fatalities and any serious temporary and permanent sequelae that can occur. Healthy children are more able to participate in education, thus preparing them to become healthy and productive adults. Vaccination programmes can also prevent infectious diseases in adolescents, thus allowing them to continue their development towards a healthy adulthood. Protecting adults against infectious diseases ensures that they can fully contribute to productivity and economic development by avoiding sick leave and lower productivity. Vaccination in older adults will contribute to the promotion of healthy ageing, enabling them to assist their familiy with, for instance, childcare, and also help them avoid functional decline and the related impacts on health and welfare expenditure. Effective vaccination programmes for all ages in Europe will thus contribute to the European Union's 2020 health and economic strategies. Indeed, beyond their impact on healthcare resources and productivity, reductions in mortality and morbidity also contribute to increased consumption and gross domestic product. Therefore, assessment of the value of vaccines and vaccination needs to consider not just the direct impact on health and healthcare but also the wider impact on economic growth, which requires a macroeconomic analysis of vaccination programmes. Full article

The use of vaccines to prevent diseases in children, adults, and the elderly results in fewer medical visits, diagnostic tests, treatments, and hospitalisations, which leads to substantial savings in healthcare costs each year in Europe and elsewhere. Vaccines also contribute to reducing resource utilisation by preventing nosocomial infections, such as rotavirus gastroenteritis, which can increase hospital stays by 4–12 days. Vaccination also has an important role in the prevention of cancers with, for example, human papillomavirus or hepatitis B vaccines. Since the financial impact of cancer is high for patients, healthcare systems, and society as a whole, any cases prevented will reduce this impact. Newer vaccines, such as the herpes zoster vaccine, can provide an answer to unmet medical needs by preventing and reducing the severity of shingles and associated post-herpetic neuralgia, which are difficult conditions to treat. Thus, in the context of increasing pressure on healthcare budgets, vaccination can contribute to the sustainability of healthcare systems through reduced and more efficient use of healthcare resources. Full article

Vaccination has made an important contribution to the decreased incidence of numerous infectious diseases and associated mortality. In 2013, it was estimated that 103 million cases of childhood diseases in the United States had been prevented by the use of vaccines since 1924. These health effects translate into positive economic results, as vaccination can provide significant savings by avoiding the direct and indirect costs associated with treating the disease and possible long-term disability. A recent US study estimated that every dollar spent on childhood vaccination could save US$3 from a payer perspective and US$10 from a societal perspective. The first vaccines set a high standard from a public health ‘return on investment’ perspective, because they are highly cost-saving. Today, however, where only a few healthcare interventions are considered to be cost-saving, the challenge that decision-makers typically face is to identify such healthcare interventions that are deemed cost-effective, that is, provide extra benefit at a reasonable extra cost. Some of the newer vaccines provide a solution to some of today's important health issues, such as cervical cancers with human papillomavirus vaccines, or debilitating diseases with herpes zoster vaccines. These recent, more expensive vaccines have been shown to be cost-effective in several economic analyses. Overall, vaccination can still be regarded as one of the most cost-effective healthcare interventions. Full article

The poor perception of the benefits of vaccines, and their subsequent underuse, can result in substantial economic, societal, and political burden. Adequate support and communication from health authorities and governments is essential to promote the benefits of vaccination and reduce the risk of infectious diseases outbreaks. Cost-containment policies in the vaccine procurement processes could also be a threat to the long-term sustainability of the vaccine industry and manufacturing sites in Europe. Biologicals, such as vaccines, are highly technical and complex products to manufacture and only a few industries are engaged in this activity. Developing incentives to encourage vaccine manufacturers and identifying means of taking into consideration the specificities of vaccines in economic evaluations could allow the full value of vaccination to be appreciated. In conclusion, governments, international agencies, and other stakeholders have an important role to play to help society regain confidence in vaccination and ensure that the benefits of vaccination programmes are fully recognised and valued. Full article

Previous economic evaluations of new vaccines largely focussed on a narrow set of benefit categories, including primarily health gains and disease-related medical cost-savings, which probably resulted in underestimates of the true value of these vaccines. Other economic benefits of vaccines could be considered to assess the full economic value of vaccination, such as, for example, impact of the human papillomavirus vaccine on women's fertility through the decrease in precancerous lesions and, therefore, in the number of diagnostic and treatment interventions, which can be associated with an increased risk of subsequent pregnancy complications. Vaccines’ impact on resource allocation at hospital level or on antimicrobial resistance, such as pneumococcal conjugate vaccines that have substantially reduced infections due to antimicrobial non-susceptible strains, thereby rendering the residual disease easier to treat, are other examples of intangible benefits of vaccination. These benefits are generally not considered in economic evaluations because they may not be immediately visible and are difficult to quantify. However, they should be taken into consideration in health technology assessments to enable those responsible for healthcare policies to make well-informed decisions on vaccination. Full article

The full economic and societal value of vaccination is complex to assess. Although direct protection is the immediate goal of vaccination programmes, it is rare that 100% uptake is attained. An important facet of vaccines value comes from the indirect (or herd) protection they provide. The evolving dynamics of our society, including the increase in the proportion of older individuals enhances the value of indirect protection in reducing disease transmission within the family setting and the society as a whole. For example, grandparents are increasingly involved in childcare, putting them at risk of disease transmission if they or the children are not vaccinated. Preventing disease in children can also reduce absenteeism for parents who otherwise would take days off work to care for their sick children, leading to a substantial societal burden. Preventing disease in working adults reduces absenteeism and presenteeism, enhancing productivity and contributing in turn to economic growth. Quality of life is essential at all ages. It is fundamental in children for their life chances, educational achievements, and healthy wellbeing. Additionally, preventing common diseases in adults and the elderly also contributes to their quality of life and helps to assure healthy ageing for growing ageing populations. These wider economic and societal values, although difficult to measure, should be taken into consideration in assessments of the economic value and cost-effectiveness of vaccination programmes. Full article

Objective: We describe and compare the availability and accessibility of administrative healthcare databases (AHDB) in several Asia-Pacific countries: Australia, Japan, South Korea, Taiwan, Singapore, China, Thailand, and Malaysia. Methods: The study included hospital records, reimbursement databases, prescription databases, and data linkages. Databases were first identified through PubMed, Google Scholar, and the ISPOR database register. Database custodians were contacted. Six criteria were used to assess the databases and provided the basis for a tool to categorise databases into seven levels ranging from least accessible (Level 1) to most accessible (Level 7). We also categorised overall data accessibility for each country as high, medium, or low based on accessibility of databases as well as the number of academic articles published using the databases. Results: Fifty-four administrative databases were identified. Only a limited number of databases allowed access to raw data and were at Level 7 [Medical Data Vision EBM Provider, Japan Medical Data Centre (JMDC) Claims database and Nihon-Chouzai Pharmacy Claims database in Japan, and Medicare, Pharmaceutical Benefits Scheme (PBS), Centre for Health Record Linkage (CHeReL), HealthLinQ, Victorian Data Linkages (VDL), SA-NT DataLink in Australia]. At Levels 3–6 were several databases from Japan [Hamamatsu Medical University Database, Medi-Trend, Nihon University School of Medicine Clinical Data Warehouse (NUSM)], Australia [Western Australia Data Linkage (WADL)], Taiwan [National Health Insurance Research Database (NHIRD)], South Korea [Health Insurance Review and Assessment Service (HIRA)], and Malaysia [United Nations University (UNU)-Casemix]. Countries were categorised as having a high level of data accessibility (Australia, Taiwan, and Japan), medium level of accessibility (South Korea), or a low level of accessibility (Thailand, China, Malaysia, and Singapore). In some countries, data may be available but accessibility was restricted based on requirements by data custodians. Conclusions: Compared with previous research, this study describes the landscape of databases in the selected countries with more granularity using an assessment tool developed for this purpose. A high number of databases were identified but most had restricted access, preventing their potential use to support research. We hope that this study helps to improve the understanding of the AHDB landscape, increase data sharing and database research in Asia-Pacific countries. Full article

The Social Security Funding Law for 2012 introduced the Economic and Public Health Assessment Committee (Commission Evaluation Economique et de Santé Publique, or CEESP) in the Social Security Code as a specialised committee affiliated with the Haute Autorité de Santé in charge of providing recommendations and health economic opinions. This article provides an in-depth description of the CEESP's structure and working methods, and analyses the impact of health economic assessment on market access of drugs in France. It also points out the areas of uncertainty and the conflicting rules following the introduction of the health economic assessment in France. The authors also provide their personal opinion on the likely future of health economic assessment of drugs in France, including the possible merge of the CEESP and the Transparency Committee, the implementation of a French threshold, and the extension of health economic assessment to a larger number of products. Full article

Background: Health technology assessment (HTA) has been reinforced in France, notably with the introduction of economic evaluation in the pricing process for the most innovative and expensive treatments. Similarly to the National Institute for Clinical Excellence (NICE) in England, the National Authority for Health (HAS), which is responsible for economic evaluation of new health technologies in France, has published recommendations on the methods of economic evaluation. Since economic assessment represents a major element of HTA in England, exploring the differences between these methodological guidelines might help to comprehend both the shape and the role economic assessment is intended to have in the French health care system. Methods: Methodological guidelines for economic evaluation in France and England have been compared topic-by-topic in order to bring out key differences in the recommended methods for economic evaluation. Results: The analysis of both guidelines has revealed multiple similarities between France and England, although a number of differences were also noted regarding the elected methodology of analysis, the comparison of studies’ outcomes with cost-effectiveness thresholds, the study population to consider, the quality of life valuation methods, the perspective on costs, the types of resources considered and their valuation, the discount rates to apply in order to reflect the present value of interventions, etc. To account for these differences, modifications will be required in order to adapt economic models from one country to the other. Conclusion: Changes in HTA assessment methods occur in response to different challenges determined by the different philosophical and cultural considerations surrounding health and welfare as well as the political considerations regarding the role of public policies and the importance of their evaluation. Full article