Cancer remains one of the leading causes of morbidity and mortality worldwide. It is predicted that by 2020, the number of new cases of cancer in the world will increase to more than 15 million, with deaths increasing to 12 million. Much of the burden of cancer incidence, morbidity, and mortality will occur in the developing world. This forms part of a larger epidemiological transition in which the burden of chronic, non-communicable disease-once limited to industrialized nations-is now increasing in less developed countries. In addition to the accumulating risks associated with diet, tobacco, alcohol, lack of exercise, and industrial exposures, the developing world is already burdened by cancers some of which are attributable to infectious diseases. These disparities in cancer risk combined with poor access to epidemiological data, research, treatment, and cancer control and prevention combine to result in significantly poorer survival rates in developing countries for a range of specific malignancies. This paper summarizes the recent trends in the epidemiology and survival of cancers in the developing and developed world, and explores potential causes and policy responses to the disproportionate and growing cancer burden in less developed countries. Such responses may include raising awareness as well as education and training to foster better informed decision-making, together with improved cancer surveillance, early detection and emphasis on prevention. Improved health care financing and international initiatives and/or partnerships could also provide additional impetus in targeting resources where needed urgently.

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

BackgroundAlthough health technology assessment (HTA) systems base their decision making process either on economic evaluations or comparative clinical benefit assessment, a central aim of recent approaches to value measurement, including value based assessment and pricing, points towards the incorporation of supplementary evidence and criteria that capture additional dimensions of value.ObjectiveTo study the practices, processes and policies of value-assessment for new medicines across eight European countries and the role of HTA beyond economic evaluation and clinical benefit assessment.MethodsA systematic (peer review and grey) literature review was conducted using an analytical framework examining: (1) ‘Responsibilities and structure of HTA agencies’; (2) ‘Evidence and evaluation criteria considered in HTAs’; (3) ‘Methods and techniques applied in HTAs’; and (4) ‘Outcomes and implementation of HTAs’. Study countries were France, Germany, England, Sweden, Italy, Netherlands, Poland and Spain. Evidence from the literature was validated and updated through two rounds of feedback involving primary data collection from national experts.ResultsAll countries assess similar types of evidence; however, the specific criteria/endpoints used, their level of provision and requirement, and the way they are incorporated (e.g. explicitly vs. implicitly) varies across countries, with their relative importance remaining generally unknown. Incorporation of additional ‘social value judgements’ (beyond clinical benefit assessment) and economic evaluation could help explain heterogeneity in coverage recommendations and decision-making.ConclusionMore comprehensive and systematic assessment procedures characterised by increased transparency, in terms of selection of evaluation criteria, their importance and intensity of use, could lead to more rational evidence-based decision-making, possibly improving efficiency in resource allocation, while also raising public confidence and fairness.Electronic supplementary materialThe online version of this article (doi:10.1007/s10198-017-0871-0) contains supplementary material, which is available to authorized users.

A c c e p t e d M a n u s c r i p t 2 Highlights  We systematically reviewed the socioeconomic cost literature for 10 rare diseases. Direct and indirect costs incurred by patients, carers and society were searched. 77 studies were included with an unequal distribution of studies over disease types. Level of existing evidence is highest for diseases with available drug treatments. Indirect costs are in most cases of similar or higher magnitude than direct costs. found to range between 1-2 per 100,000 population (PWS, a sub-type of Histiocytosis, and EB) up to 42 per 100,000 population (Scleroderma). Overall, cost evidence on rare diseases appears to be very scarce (a total of 77 studies were identified across all diseases), with CF (n=29) and Haemophilia (n=22) being relatively well studied, compared to the other conditions, where very limited cost of illness information was available. In terms of data availability, total lifetime cost figures were found only across four diseases, and total annual costs (including indirect costs) across five diseases.Overall, data availability was found to correlate with the existence of a pharmaceutical treatment and indirect costs tended to account for a significant proportion of total costs.Although methodological variations prevent any detailed comparison between conditions, most of the rare diseases examined are associated with significant economic burden, both direct and indirect.

Chronic kidney disease (CKD) is a prevalent condition in many countries, and it is estimated that over $1 trillion is spent globally on end-stage renal disease (ESRD) care. There is a clear clinical and economic rationale for designing timely and appropriate health system responses to limit progression from CKD to ESRD. This article reviews the gaps in our knowledge about which early CKD interventions are appropriate, the optimal time to intervene, and what model of care to adopt. The available diagnostic tests exhibit key limitations. Clinical care may improve if early-stage (1–3) CKD with risk for progression towards ESRD is differentiated from early CKD that is unlikely to advance. It is possible that CKD should be re-conceptualized as a part of primary care. Additional research is needed to better understand the risk factors for CKD progression. Systems modelling can be used to evaluate the impact of different care models on CKD outcomes and costs. The US Indian Health Service experience has demonstrated that an integrated, system-wide approach, even in an underfunded system, can produce significant benefits.

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Escalating drug prices have catalysed the generation of numerous "value frameworks" with the aim of informing payers, clinicians and patients on the assessment and appraisal process of new medicines for the purpose of coverage and treatment selection decisions. Although this is an important step towards a more inclusive Value Based Assessment (VBA) approach, aspects of these frameworks are based on weak methodologies and could potentially result in misleading recommendations or decisions. In this paper, a Multiple Criteria Decision Analysis (MCDA) methodological process, based on Multi Attribute Value Theory (MAVT), is adopted for building a multi-criteria evaluation model. A five-stage model-building process is followed, using a top-down "value-focused thinking" approach, involving literature reviews and expert consultations. A generic value tree is structured capturing decision-makers' concerns for assessing the value of new medicines in the context of Health Technology Assessment (HTA) and in alignment with decision theory. The resulting value tree (Advance Value Tree) consists of three levels of criteria (top level criteria clusters, mid-level criteria, bottom level sub-criteria or attributes) relating to five key domains that can be explicitly measured and assessed: (a) burden of disease, (b) therapeutic impact, (c) safety profile (d) innovation level and (e) socioeconomic impact. A number of MAVT modelling techniques are introduced for operationalising (i.e. estimating) the model, for scoring the alternative treatment options, assigning relative weights of importance to the criteria, and combining scores and weights. Overall, the combination of these MCDA modelling techniques for the elicitation and construction of value preferences across the generic value tree provides a new value framework (Advance Value Framework) enabling the comprehensive measurement of value in a structured and transparent way. Given its flexibility to meet diverse requirements and become readily adaptable across different settings, the Advance Value Framework could be offered as a decision-support tool for evaluators and payers to aid coverage and reimbursement of new medicines.

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