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IntroductionHealth technology assessment (HTA) is an area that remains less implemented in low- and lower middle-income countries. The aim of the study is to understand the perceptions of stakeholders in Uganda towards HTA and its role in decision making, in order to inform its potential implementation in the country.MethodsThe study takes a cross-sectional mixed methods approach, utilising an adapted version of an International Decision Support Initiative questionnaire with both semi-structured and open-ended questions. We interviewed thirty key informants from different stakeholder institutions in Uganda that have decision making roles in the health sector.ResultsAll participants perceived HTA as an important tool for decision making. Allocative efficiency was regarded as the most important use of HTA receiving the highest average score (8.8 out of 10), followed by quality of healthcare (7.8/10), transparency (7.6/10), budget control (7.5/10) and equity (6.5/10). There was concern that some of the uses of HTA may not be achieved in reality if there was political interference during the HTA process. The technology areas that interviewees highlighted as needing HTA type evaluations urgently were identified as medicines (60.0% of the participants), diagnostics (53.3%), vaccines (40.0%), and public health programs (26.7%). The study participants identified development partners as the most likely potential users of HTA (66.7% of participants), followed by Ministry of Health (43.3%).ConclusionInterviewed stakeholders in Uganda viewed the role of HTA positively, suggesting that there exists a promising environment for the establishment and operationalisation of HTA as a tool for decision making within the health sector. However, sustainable development and application of HTA in Uganda will require adequate capacity both to undertake HTAs and to support their use and uptake.

I.AbstractA.ObjectiveClinical Decision Support (CDS) systems (CDSSs) that integrate clinical guidelines need to reflect real-world co-morbidity. In patient-specific clinical contexts, transparent recommendations that allow for contraindications and other conflicts arising from co-morbidity are a requirement. We aimed to develop and evaluate a non-proprietary, standards-based approach to the deployment of computable guidelines with explainable argumentation, integrated with a commercial Electronic Health Record (EHR) system in a middle-income country.B.Materials and MethodsWe used an ontological framework, the Transition-based Medical Recommendation (TMR) model, to represent, and reason about, guideline concepts, and chose the 2017 International Global Initiative for Chronic Obstructive Lung Disease (GOLD) guideline and a Serbian hospital as the deployment and evaluation site, respectively. To mitigate potential guideline conflicts, we used a TMR-based implementation of the Assumptions-Based Argumentation framework extended with preferences and Goals (ABA+G). Remote EHR integration of computable guidelines was via a microservice architecture based on HL7 FHIR and CDS Hooks. A prototype integration was developed to manage COPD with comorbid cardiovascular or chronic kidney diseases, and a mixed-methods evaluation was conducted with 20 simulated cases and five pulmonologists.C.ResultsPulmonologists agreed 97% of the time with the GOLD-based COPD symptom severity assessment assigned to each patient by the CDSS, and 98% of the time with one of the proposed COPD care plans. Comments were favourable on the principles of explainable argumentation; inclusion of additional co-morbidities were suggested in the future along with customisation of the level of explanation with expertise.D.ConclusionAn ontological model provided a flexible means of providing argumentation and explainable artificial intelligence for a long-term condition. Extension to other guidelines and multiple co-morbidities is needed to test the approach further.E.FundingThe project was funded by the British government through the Engineering and Physical Sciences Research Council (EPSRC) – Global Challenges Research Fund.1

Background: Better data on medicines use can inform better decisions, better health systems, and better patient outcomes. More African studies of medicines use are emerging, especially those using routinely-collected data. Such work is even more pressing given the growing burden of non-communicable diseases (NCD) - where medicines are often the mainstay of treatment - and additional challenges in financing sustainable health systems for universal health coverage and the ongoing COVID-19 pandemic. Objective: To describe how medicines use studies can strengthen health systems (focus on Ghana). Methods: We identified sources of data: national health insurance systems (public and private);district level health information systems (DHIMS2);health service and hospital data;together with commercial local, regional, and global data. Data users need to be mindful of appropriate data governance and privacy of patient's records. Results and Conclusions: 1. Rational use of medicines: adherence with standard treatment guidelines, treatment pathways, potential cost savings, pharmacovigilance 2. Health Technology Assessment: key inputs for cost-effectiveness and budget impact analysis of medicines for inclusion within a benefits package. 3. Patterns of disease: explore prevalence and incidence of disease (especially NCDs) plus comorbidities using medicines use as a proxy for diseases, particularly in the absence of other high-quality epidemiological data. 4. Health Policy + Monitoring and Evaluation: developing essential medicines lists and treatment guidelines;key indicators in M&E of health systems. 5. Building research capacity: promoting researchers in pharmacoepidemiology using identified and accessible data sources. Initial steps in Africa can develop with continued training and support.

BACKGROUND Pediatric endocrinology is a specialty that is struggling worldwide to maintain adequately trained professionals. Pediatric endocrine care in Central America and Caribbean countries is often performed by pediatricians or adult endocrinologists due to the limited number of pediatric endocrinologists. These health care providers are seldom members of endocrine societies and frequently lack formal training in the field. OBJECTIVE In this study, we describe the scope of a virtual conference in pediatric endocrinology and diabetes targeted to low- and middle-income countries to provide equal opportunities for access to medical education for health care professionals. METHODS The virtual conference was sponsored by the Pediatric Endocrine Society (North America), Asociación Costarricense de Endocrinología (previously, Asociación Nacional Pro Estudio de la Diabetes, Endocrinología y Metabolismo), and Asociacion Centroamericana y del Caribe de Endocrinologia Pediátrica. The conference was free to participants and comprised 23 sessions that were either synchronous with ability for real-time interactive sessions or asynchronous sessions, where content was available online to access at their convenience. Topics included idiopathic short stature, polycystic ovarian syndrome, diabetes mellitus, telemedicine, Turner syndrome, congenital adrenal hyperplasia, obesity, central precocious puberty, and subclinical hypothyroidism. The participants were asked to evaluate the conference after its completion with a questionnaire. RESULTS A total of 8 speakers from Spain, Canada, Costa Rica, and the United States delivered the virtual event to 668 health care professionals from Guatemala, Venezuela, Dominican Republic, Costa Rica, Ecuador, Peru, Uruguay, Mexico, Honduras, Argentina, the United States, Bolivia, Chile, Panama, El Salvador, Nicaragua, Paraguay, Belize, Spain, and Colombia. Name, profession, and country were fully disclosed by 410 (61.4%) of the 668 health care professionals. The profession or level of training of participants were as follows: pediatric endocrinologists (n=129, 19.3%), pediatricians (n=116, 17.4%), general practitioners (n=77, 11.5%), adult endocrinologists (n=34, 5.1%), medical students (n=23, 3.4%), residents in various specialties (n=14, 2.1%), and others (n=17, 2.6%). A total of 23 sessions were offered, most of which were bilingual (Spanish and English). Feedback from the evaluation questionnaire indicated that the content of the conference was very relevant to the participants’ professional practice. Additionally, the participants reported that they were very satisfied with the organization, the web-based platform, and the sessions of the conference. CONCLUSIONS Lack of accessibility to the latest and cutting-edge medical education in pediatric endocrinology and diabetes for medical professionals from low- and middle-income countries can be overcome with a virtual conference. Online availability, low cost, and easy-to-use technology were well received from the participants, who were overall very satisfied by the quality and the relevance of the sessions to their professional practice.

The race to find an effective treatment for coronavirus disease 2019 (COVID-19) is still on, with only two treatment options currently authorized for emergency use and/or recommended for patients hospitalized with severe respiratory symptoms: low-dose dexamethasone and remdesivir. The USA decision to stockpile the latter has resulted in widespread condemnation and in similar action being taken by some other countries. In this commentary we discuss whether stockpiling remdesivir is justified in light of the currently available evidence.

BackgroundThe COVID-19 vaccine supply shortage in 2021 constrained rollout efforts in Africa while populations experienced waves of epidemics. As supply picks up, a key question becomes if vaccination remains an impactful and cost-effective strategy given changes in the timing of implementation.MethodsWe assessed the impact of timing using an epidemiological and economic model. We fitted our mathematical epidemiological model to reported COVID-19 deaths in 27 African countries to estimate the existing immunity (resulting from infection) before substantial vaccine rollout. We then projected health outcomes for different programme start dates (2021-01-01 to 2021-12-01, n = 12) and roll-out rates (slow, medium, fast; 275, 826, and 2066 doses/ million population-day, respectively) for viral vector and mRNA vaccines. Rollout rates used were derived from observed uptake trajectories. We collected data on vaccine delivery costs by country income group. Lastly, we calculated incremental cost-effectiveness ratios and relative affordability.FindingsVaccination programmes with early start dates incur the most health benefits and are most cost-effective. While incurring the most health benefits, fast vaccine roll-outs are not always the most cost-effective. At a willingness-to-pay threshold of 0.5xGDP per capita, vaccine programmes starting in August 2021 using mRNA and viral vector vaccines were cost-effective in 6-10 and 17-18 of 27 countries, respectively.InterpretationAfrican countries with large proportions of their populations unvaccinated by late 2021 may find vaccination programmes less cost-effective than they could have been earlier in 2021. Lower vaccine purchasing costs and/or the emergence of new variants may improve cost-effectiveness.FundingBill and Melinda Gates Foundation, World Health Organization, National Institute of Health Research (UK), Health Data Research (UK)

Objectives Evidence-informed priority setting, in particular cost-effectiveness analysis (CEA), can help target resources better to achieve universal health coverage. Central to the application of CEA is the use of a cost-effectiveness threshold. We add to the literature by looking at what thresholds have been used in published CEA and the proportion of interventions found to be cost-effective, by type of threshold. Methods We identified CEA studies in low- and middle-income countries from the Global Health Cost-Effectiveness Analysis Registry that were published between January 1, 2015, and January 6, 2020. We extracted data on the country of focus, type of interventions under consideration, funder, threshold used, and recommendations. Results A total of 230 studies with a total 713 interventions were included in this review; 1 to 3× gross domestic product (GDP) per capita was the most common type of threshold used in judging cost-effectiveness (84.3%). Approximately a third of studies (34.2%) using 1 to 3× GDP per capita applied a threshold at 3× GDP per capita. We have found that no study used locally developed thresholds. We found that 79.3% of interventions received a recommendation as “cost-effective” and that 85.9% of studies had at least 1 intervention that was considered cost-effective. The use of 1 to 3× GDP per capita led to a higher proportion of study interventions being judged as cost-effective compared with other types of thresholds. Conclusions Despite the wide concerns about the use of 1 to 3× GDP per capita, this threshold is still widely used in the literature. Using this threshold leads to more interventions being recommended as “cost-effective.” This study further explore alternatives to the 1 to 3× GDP as a decision rule.

Public payers around the world are increasingly using cost-effectiveness thresholds (CETs) to assess the value-for-money of an intervention and make coverage decisions. However, there is still much confusion about the meaning and uses of the CET, how it should be calculated, and what constitutes an adequate evidence base for its formulation. One widely referenced and used threshold in the last decade has been the 1-3 GDP per capita, which is often attributed to the Commission on Macroeconomics and WHO guidelines on Choosing Interventions that are Cost Effective (WHO-CHOICE). For many reasons, however, this threshold has been widely criticised; which has led experts across the world, including the WHO, to discourage its use. This has left a vacuum for policy-makers and technical staff at a time when countries are wanting to move towards Universal Health Coverage. This article seeks to address this gap by offering five practical options for decision-makers in low- and middle-income countries that can be used instead of the 1-3 GDP rule, to combine existing evidence with fair decision-rules or develop locally relevant CETs. It builds on existing literature as well as an engagement with a group of experts and decision-makers working in low, middle and high income countries.

La diabetes mellitus neonatal es un raro desorden metabólico usualmente desarrollado en las primeras 6 semanas de vida, secundario a un grupo de mutaciones y defectos del desarrollo pancreático que puede desembocar en una catástrofe clínica si no se identifica tempranamente; se divide en una variante transitoria y una permanente, siendo la primera la más frecuente, con cerca de un 60% de los casos. El manejo inicial de ambas variantes es la insulinoterapia intensiva, que en la variante transitoria puede suspenderse usualmente en los primeros meses de vida. El síndrome de disregulación inmunológica, poliendocrinopatía y enteropatía ligada a X (IPEX por sus siglas en inglés), es una causa extremadamente rara de la variante permanente, casi siempre mortal, caracterizada por un inmunocompromiso severo, enteropatía, diabetes y dermatitis. En el estudio se describen 4 casos de diabetes mellitus neonatal diagnosticados en el Hospital Nacional de Niños de San José, Costa Rica: 2 correspondientes a una diabetes mellitus neonatal transitoria, 2 a una diabetes mellitus neonatal permanente y 1 de ellos correspondiente a un síndrome de IPEX. Neonatal diabetes mellitus s a rare metabolic disease that usually develops in the first 6 weeks of life. It is caused by a group of mutations and birth defects in the organogenesis of the pancreas that could be catastrophic if not identified early. It is divided in a transient and a permanent variant, the first one is more frequent as it is related to 60% of cases. The initial treatment in both variants is intensive insulin therapy, but in the transient variant it may be suspended in the first months of life. IPEX syndrome is an extremely rare cause of permanent neonatal diabetes, usually mortal, characterized by severe immunological compromise, enteropathy, diabetes and dermatitis. In this study we describe four cases of NDM diagnosed at the National Children’s Hospital in San José, Costa Rica; 2 cases correspond to the transitory variant and 2 to the permanent variant, one of the latter is an IPEX syndrome case.

Summary box The COVID-19 pandemic underscores that infectious disease is a global challenge. For many infectious diseases, vaccines are the best tools available for control and elimination. Vaccines helped eliminate smallpox and reduce annual measles deaths from 2.6 million before widespread vaccination to 140 000 in 2018.1 Vaccines avert two to three million deaths annually, and they are often touted as one of the most cost-effective health interventions.1 Since infectious diseases do not recognise borders, vaccine deployment requires global cooperation to achieve the best outcomes. Presently, many countries have halted vaccination programmes and campaigns, including for measles and polio, where vaccination has had transformative impact on the burden of disease. Gavi estimates that at least 13.5 million people are missing vaccinations, and that will rise as the pandemic continues.2 Hence, the burden of vaccine-preventable diseases will increase as a consequence of COVID-19: this is especially true in low-income and middle-income countries (LMICs), which already suffer from a greater infectious disease burden than high-income countries and where the pandemic could, as elsewhere, overwhelm health systems that have a lower capacity. Sustaining routine vaccination programmes in Africa, for example, is estimated to prevent 140 deaths for every …

Background: Health technology assessment (HTA) is an effective tool for supporting priority setting (PS) in health. Stakeholder groups need to understand HTA appropriate to their role and to interpret and critique the evidence produced. We aimed to rapidly assess current health system priorities and policy areas of demand for HTA in Sub-Saharan Africa, and identify key gaps in data and skills to inform targeted capacity building. Methods: We revised an existing survey, delivered it to 357 participants, then analysed responses and explored key themes. Results: There were 51 respondents (14%, 30 full completions) across 14 countries. HTA was considered an important and valuable PS tool with a key role in the design of health benefits packages, clinical guideline development, and service improvement. Medicines were identified as a technology type that would especially benefit from the application of HTA. Using HTA to address safety issues (e.g. low-quality medicines) and value for money concerns was particularly highlighted. The perceived availability and accessibility of suitable local data to support HTA varied widely but was mostly considered inadequate and limited. Respondents also noted a need for training support in research methodology and data gathering. Conclusions: While important in raising awareness of HTA as a tool for PS, this study had a low response rate, and that respondents were self-selected. A more refined survey will be developed to support engagement strategies and capacity building.

Background Evidence-based decision-making for prioritising health is assisted by health technology assessment (HTA) to integrate data on effectiveness, costs and equity to support transparent decisions. Ghana is moving towards universal health coverage, facilitated mainly by the National Health Insurance Scheme (NHIS) established in 2003. The Government of Ghana is committed to institutionalising HTA for priority-setting. We aimed to identify and describe the sources of accessible data to support HTA in Ghana. Methods We identified and described data sources encompassing six main domains using an existing framework. The domains were epidemiology, clinical efficacy, costs, health service use and consumption, quality of life, and equity. We used existing knowledge, views of stakeholders, and searches of the literature and internet. Results The data sources for each of the six domains vary in extent and quality. Ghana has several large data sources to support HTA (e.g. Demographic Health Surveys) that have rigorous quality assurance processes. Few accessible data sources were available for costs and resource utilisation. The NHIS is a potentially rich source of data on resource use and costs but there are some limits on access. There are some data on equity but data on quality of life are limited. Conclusions A small number of quality data sources are available in Ghana but there are some gaps with respect to HTA based on greater use of local and contextualised information. Although more data are becoming available for monitoring, challenges remain in terms of their usefulness for HTA, and some information may not be available in disaggregated form to enable specific analyses. We support recent initiatives for the routine collection of comprehensive and reliable data that is easily accessible for HTA users. A commitment to HTA will require concerted efforts to leverage existing data sources, for example, from the NHIS, and develop and maintain new data (e.g. local health utility estimates). It will be critical that an overarching strategic and mandatory approach to the collection and use of health information is developed for Ghana in parallel to, and informed by, the development of HTA approaches to support resource allocation decisions. The key to HTA is to use the best available data while being open about its limitations and the impact on uncertainty.

Objectives Universal healthcare coverage in low- and middle-income countries requires challenging resource allocation decisions. Health technology assessment is one important tool to support such decision making. The International Decision Support Initiative worked with the Ghanaian Ministry of Health to strengthen health technology assessment capacity building, identifying hypertension as a priority topic area for a relevant case study. Methods Based on guidance from a national technical working group of researchers and policy makers, an economic evaluation and budget impact analysis were undertaken for the main antihypertensive medicines used for uncomplicated, essential hypertension. The analysis aimed to address specific policy questions relevant to the National Health Insurance Scheme. Results The evaluation found that first-line management of essential hypertension with diuretics has an incremental cost per disability-adjusted life-year avoided of GH¢ 276 ($179 in 2017, 4% of gross national income per capita) compared with no treatment. Calcium channel blockers were more effective than diuretics but at a higher incremental cost: GH¢ 11 061 per disability-adjusted life-year avoided ($7189 in 2017; 160% of gross national income per capita). Diuretics provide better health outcomes at a lower cost than angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, or beta-blockers. Budget impact analysis highlighted the potential for cost saving through enhanced price negotiation and increased use of better-value drugs. We also illustrate how savings could be reinvested to improve population health. Conclusions Economic evaluation enabled decision makers to assess hypertension medicines in a Ghanaian context and estimate the impact of using such evidence to change policy. This study contributes to addressing challenges associated with the drive for universal healthcare coverage in the context of constrained budgets., Highlights • This article provides details of a technical modeling work collaboratively built in Ghana with a high-level policy orientation. • Recent developments in Ghana further highlight the constraints imposed on current resources available for health. • This is the first national health technology assessment study to be commissioned and conducted under Ghanaian government supervision. There are no other studies that offer the same level of contextualized information to support the National Health Insurance Authority and the Ghanaian Ministry of Health in their policy-making efforts toward improving value for money in hypertension management. • A range of cost-saving and health-improving policy scenarios were identified that could have a major impact on reducing the burden of hypertension and contribute to the financial sustainability of the National Health Insurance Scheme.

Ghana is one of the few African countries to enact legislation and earmark significant funding to establish universal health coverage (UHC) through the National Health Insurance Scheme, although donor funds have declined recently. Given a disproportionate level of spending on medicines, health technology assessment (HTA) can support resource allocation decisions in the face of highly constrained budgets, as commonly found in low-resource settings. The Ghanaian Ministry of Health, supported by the International Decision Support Initiative (iDSI), initiated a HTA study in 2016 to examine the cost-effectiveness of antihypertensive medicines. We aimed to summarize key insights from this work that highlights success factors beyond producing purely technical outputs. These include the need for capacity building, academic collaboration, and ongoing partnerships with a broad range of experts and stakeholders. By building on this HTA study, and with ongoing interactions with iDSI, HTAi, WHO, and others, Ghana will be well positioned to institutionalize HTA in resource allocation decisions and support progress toward UHC.

Objectives Transferability of economic evaluations to low- and middle-income countries through adaptation of models is important; however, several methodological and practical challenges remain. Given its significant costs and the quality-of-life burden to patients, adjuvant treatment of early breast cancer was identified as a priority intervention by the South African National Department of Health. This study assessed the cost-effectiveness of docetaxel and paclitaxel-containing chemotherapy regimens (taxanes) compared with standard (non-taxane) treatments. Methods A cost-utility analysis was undertaken based on a UK 6-health-state Markov model adapted for South Africa using the Mullins checklist. The analysis assumed a 35-year time horizon. The model was populated with clinical effectiveness data (hazard ratios, recurrence rates, and adverse events) using direct comparisons from clinical trials. Resource use patterns and unit costs for estimating cost parameters (drugs, diagnostics, consumables, personnel) were obtained from South Africa. Uncertainty was assessed using probabilistic and deterministic sensitivity analyses. Results The incremental cost per patient for the docetaxel regimen compared with standard treatment was R6774. The incremental quality-adjusted life years (QALYs) were 0.24, generating an incremental cost-effectiveness ratio of R28430 per QALY. The cost of the paclitaxel regimen compared with standard treatment was estimated as −R578 and −R1512, producing an additional 0.03 and 0.025 QALYs, based on 2 trials. Paclitaxel, therefore, appears to be a dominant intervention. The base case results were robust to all sensitivity analyses. Conclusions Based on the adapted model, docetaxel and paclitaxel are predicted to be cost-effective as adjuvant treatment for early breast cancer in South Africa.

Background: Mathematical models have been used throughout the COVID-19 pandemic to inform policymaking decisions. The COVID-19 Multi-Model Comparison Collaboration (CMCC) was established to provide country governments, particularly low- and middle-income countries (LMICs), and other model users with an overview of the aims, capabilities and limits of the main multi-country COVID-19 models to optimise their usefulness in the COVID-19 response. Methods: Seven models were identified that satisfied the inclusion criteria for the model comparison and had creators that were willing to participate in this analysis. A questionnaire, extraction tables and interview structure were developed to be used for each model, these tools had the aim of capturing the model characteristics deemed of greatest importance based on discussions with the Policy Group. The questionnaires were first completed by the CMCC Technical group using publicly available information, before further clarification and verification was obtained during interviews with the model developers. The fitness-for-purpose flow chart for assessing the appropriateness for use of different COVID-19 models was developed jointly by the CMCC Technical Group and Policy Group. Results: A flow chart of key questions to assess the fitness-for-purpose of commonly used COVID-19 epidemiological models was developed, with focus placed on their use in LMICs. Furthermore, each model was summarised with a description of the main characteristics, as well as the level of engagement and expertise required to use or adapt these models to LMIC settings. Conclusions: This work formalises a process for engagement with models, which is often done on an ad-hoc basis, with recommendations for both policymakers and model developers and should improve modelling use in policy decision making.

Summary box In the last two decades, performance-based financing (PBF) has gained worldwide prominence. As of September 2016, the Health Results Innovation Trust Fund (HRITF) at the World Bank supported 29 low-income and middle-income countries in the introduction, implementation and evaluation of 35 PBF programmes, with expenditure near $2.5 billion. Although PBF is perceived as a tool to achieve the Sustainable Development Goals, several global health experts have pointed to its mixed evidence base.1 In recent years, PBF has become one of the most divisive topics in the global health community, as illustrated by the lively discussions following the publication of Paul et al ’s piece.2 Policy-makers need to assess whether PBF is an appropriate policy choice for their countries, given the substantial budget constraints they face and that are likely to worsen with transition from aid. In practice, whether the investments required to implement PBF are ‘worthwhile’ is a question that can be answered by a cost-effectiveness analysis (CEA). However, a recent review found no studies making clear connections between the costs and effects of PBF.3 Our search yielded three CEAs …

Background: Resource allocation in health is universally challenging, but especially so in resource-constrained contexts in the Global South. Pursuing a strategy of evidence-based decision-making and using tools such as Health Technology Assessment (HTA), can help address issues relating to both affordability and equity when allocating resources. Three BRICS and Global South countries, China, India and South Africa have committed to strengthening HTA capacity and developing their domestic HTA systems, with the goal of getting evidence translated into policy. Through assessing and comparing the HTA journey of each country it may be possible to identify common problems and shareable insights. Objectives: This collaborative paper aimed to share knowledge on strengthening HTA systems to enable enhanced evidence-based decision-making in the Global South by: Identifying common barriers and enablers in three BRICS countries in the Global South; and Exploring how South-South collaboration can strengthen HTA capacity and utilisation for better healthcare decision-making. Methods: A descriptive and explorative comparative analysis was conducted comprising a Within-Case analysis to produce a narrative of the HTA journey in each country and an Across-Case analysis to explore both knowledge that could be shared and any potential knowledge gaps. Results: Analyses revealed that China, India and South Africa share many barriers to strengthening and developing HTA systems such as: (1) Minimal HTA expertise; (2) Weak health data infrastructure; (3) Rising healthcare costs; (4) Fragmented healthcare systems; and (5) Significant growth in non-communicable diseases. Stakeholder engagement and institutionalisation of HTA were identified as two conducive factors for strengthening HTA systems. Conclusion: China, India and South Africa have all committed to establishing robust HTA systems to inform evidence-based priority setting and have experienced similar challenges. Engagement among countries of the Global South can provide a supportive platform to share knowledge that is more applicable and pragmatic.

Background In attempting to constrain healthcare expenditure growth, health technology assessment (HTA) can enable policy-makers to look beyond budget impact and facilitate more rational decision-making. However lack of technical capacity and poor governance can limit use in some countries. Undertaking de facto HTA by adapting decisions taken in countries with established processes is a method that may be applied while building domestic HTA capacity. We explored the potential for applying this approach in Romania. Methods As part of a review of the basic health benefits available to insured Romanians we examined the listing process and content of the Romanian drug reimbursement formulary. We assessed value for money indirectly by drawing on appraisals by UK's NICE, and for products considered cost effective in the UK, adjusting prices by the ratio of Romanian per capita GDP to UK per capita GDP. Findings We found more than 30 of the top 50 medicines on the Romanian formulary unlikely to be cost-effective, suggesting that existing external reference pricing mechanisms may not be delivering good value for money. Conclusions While not taking into account local costs or treatment patterns, absent local considerations of value for money, this method offers a guide for both drug selection and pricing. Until robust local HTA processes are established this approach could support further analysis of existing prices and pricing mechanisms. Applied more generally, it is arguably preferable to external reference pricing, product delisting or arbitrary price cuts, and may support the future development of more rigorous, evidence-based decision-making.

Objectives Grading of Recommendations Assessment, Development and Evaluation (GRADE) is a system for rating the confidence in estimates of effect and grading guideline recommendations. It promotes evaluation of the quality of the evidence for each outcome and an assessment of balance between desirable and undesirable outcomes leading to a judgment about the strength of the recommendation. In 2007, the National Institute for Health and Clinical Excellence began introducing GRADE across its clinical guideline program to enable separation of judgments about the evidence quality from judgments about the strength of the recommendation. Study Design and Setting We describe the process of implementing GRADE across guidelines. Results Use of GRADE has been positively received by both technical staff and guideline development group members. Conclusion A shift in thinking about confidence in the evidence was required leading to a more structured and transparent approach to decision making. Practical problems were also encountered; these have largely been resolved, but some areas require further work, including the application of imprecision and presenting results from analyses considering more than two alternative interventions. The use of GRADE for nonrandomized and diagnostic accuracy studies needs to be refined.

Objectives: The development of a new economic model for every single country that an intervention is used may be seen an inefficient. As such, model adaptation has often been proposed as a potential solution which could save scarce resources for decision makers in low and middle-income countries (LMICs). Using adjuvant treatment of early breast cancer as a case study, this study assessed the appropriateness of adapting an economic evaluation model originally developed in the UK for use in South Africa. The study aimed to determine the likely challenges that can arise from the routine adaptation of models to similar settings around the world. Methods: Eight model adaptation methods were identified from a systematic review and a citation search performed using Web of Science and Scopus databases. The eight methods were then appraised against criteria developed by the study team to select the most appropriate approach for LMICs. The Mullins checklist was formally applied in a case study assessing the cost-effectiveness of docetaxel and paclitaxel-containing chemotherapy regimens compared with standard treatments for adjuvant early breast cancer in South Africa. South African resource use patterns and unit cost data were obtained for estimating cost parameters, whilst targeted literature reviews were used to update the clinical effectiveness and utility data in the model. Results: Several methods are available to guide the process of adapting economic evaluation models for countries other than those in which they were conducted. The Mullins checklist seems the most appropriate approach for LMICs. We recommend that a consensus should be developed by a well-conducted Delphi panel. Conclusions: Based on model adaptation using resource use data from South Africa, the Mullins checklist appears to be an appropriate existing method to guide the transferability of economic evaluations done elsewhere to LMICs. However, research on refining the guidance further is urgently needed.

Copyright @ Queen’s Printer and Controller of HMSO 2013. This work was produced by Lord et al. under the terms of a commissioning contract issued by the Secretary of State for Health. This issue may be freely reproduced for the purposes of private research and study and extracts (or indeed, the full report) may be included in professional journals provided that suitable acknowledgement is made and the reproduction is not associated with any form of advertising. Applications for commercial reproduction should be addressed to: NIHR Journals Library, National Institute for Health Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK. Background - National Institute for Health and Care Excellence (NICE) clinical guidelines (CGs) make recommendations across large, complex care pathways for broad groups of patients. They rely on cost-effectiveness evidence from the literature and from new analyses for selected high-priority topics. An alternative approach would be to build a model of the full care pathway and to use this as a platform to evaluate the cost-effectiveness of multiple topics across the guideline recommendations. Objectives - In this project we aimed to test the feasibility of building full guideline models for NICE guidelines and to assess if, and how, such models can be used as a basis for cost-effectiveness analysis (CEA). Data sources - A ‘best evidence’ approach was used to inform the model parameters. Data were drawn from the guideline documentation, advice from clinical experts and rapid literature reviews on selected topics. Where possible we relied on good-quality, recent UK systematic reviews and meta-analyses. Review methods - Two published NICE guidelines were used as case studies: prostate cancer and atrial fibrillation (AF). Discrete event simulation (DES) was used to model the recommended care pathways and to estimate consequent costs and outcomes. For each guideline, researchers not involved in model development collated a shortlist of topics suggested for updating. The modelling teams then attempted to evaluate options related to these topics. Cost-effectiveness results were compared with opinions about the importance of the topics elicited in a survey of stakeholders. Results - The modelling teams developed simulations of the guideline pathways and disease processes. Development took longer and required more analytical time than anticipated. Estimates of cost-effectiveness were produced for six of the nine prostate cancer topics considered, and for five of eight AF topics. The other topics were not evaluated owing to lack of data or time constraints. The modelled results suggested ‘economic priorities’ for an update that differed from priorities expressed in the stakeholder survey. Limitations - We did not conduct systematic reviews to inform the model parameters, and so the results might not reflect all current evidence. Data limitations and time constraints restricted the number of analyses that we could conduct. We were also unable to obtain feedback from guideline stakeholders about the usefulness of the models within project time scales. Conclusions - Discrete event simulation can be used to model full guideline pathways for CEA, although this requires a substantial investment of clinical and analytic time and expertise. For some topics lack of data may limit the potential for modelling. There are also uncertainties over the accessibility and adaptability of full guideline models. However, full guideline modelling offers the potential to strengthen and extend the analytical basis of NICE’s CGs. Further work is needed to extend the analysis of our case study models to estimate population-level budget and health impacts. The practical usefulness of our models to guideline developers and users should also be investigated, as should the feasibility and usefulness of whole guideline modelling alongside development of a new CG. Medical Research Council and the National Institute for Health Research.

OBJECTIVES: To assess how long the UK's National Institute for Health and Clinical Excellence's (NICE) Technology Appraisal Programme has taken to produce guidance and to determine independent predictors of time to guidance. DESIGN: Retrospective time to event (survival) analysis. SETTING: Technology Appraisal guidance produced by NICE. DATASOURCE: All appraisals referred to NICE by February 2010 were included, except those referred prior to 2001 and a number that were suspended. OUTCOME MEASURE: Duration from the start of an appraisal (when the scope document was released) until publication of guidance. RESULTS: Single Technology Appraisals (STAs) were published significantly faster than Multiple Technology Appraisals (MTAs) with median durations of 48.0 (IQR; 44.3-75.4) and 74.0 (IQR; 60.9-114.0) weeks, respectively (p

Objectives In this article, we describe how to include considerations about resource utilization when making recommendations according to the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach. Study Design and Settings We focus on challenges with rating the confidence in effect estimates (quality of evidence) and incorporating resource use into evidence profiles and Summary of Findings (SoF) tables. Results GRADE recommends that important differences in resource use between alternative management strategies should be included along with other important outcomes in the evidence profile and SoF table. Key steps in considering resources in making recommendations with GRADE are the identification of items of resource use that may differ between alternative management strategies and that are potentially important to decision makers, finding evidence for the differences in resource use, making judgments regarding confidence in effect estimates using the same criteria used for health outcomes, and valuing the resource use in terms of costs for the specific setting for which recommendations are being made. Conclusions With our framework, decision makers will have access to concise summaries of recommendations, including ratings of the quality of economic evidence, and better understand the implications for clinical decision making.

Management of type 2 diabetes is complex and aims to prevent and reduce the impact of complications by encouraging a healthy lifestyle, controlling blood pressure and blood lipids, and lowering blood glucose concentrations. In recent years new drugs have become available for blood glucose control, including the long acting insulin analogues (insulin detemir and insulin glargine); glucagon-like peptide-1 (GLP-1) mimetics (exenatide); and dipeptidylpeptidase-4 (DPP-4) inhibitors (sitagliptin and vildagliptin). In addition, safety concerns have surfaced recently about the use of the thiazolidinediones (pioglitazone and rosiglitazone). This article summarises the most recent recommendations from the National Institute for Health and Clinical Excellence (NICE) on the use of newer agents for control of blood glucose in type 2 diabetes1 and updates the relevant section of the NICE clinical guideline on the management of type 2 diabetes.2 NICE recommendations are based on systematic reviews of best available evidence. When minimal evidence is available, recommendations are based on the Guideline Development Group’s experience and opinion of what constitutes good practice. Evidence levels for the recommendations are given in italic in square brackets. ### DPP-4 inhibitors and thiazolidinediones DPP-4 inhibitors stabilise concentrations of endogenous glucagon-like peptide and increase insulin secretion. [ Based on evidence ranging from low to moderate quality from randomised controlled trials; a meta-analysis; current NICE guidance 2 ; and a health economic analysis ]