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1.  Funding Decisions for Newborn Screening: A Comparative Review of 22 Decision Processes in Europe 
Decision-makers need to make choices to improve public health. Population-based newborn screening (NBS) is considered as one strategy to prevent adverse health outcomes and address rare disease patients’ needs. The aim of this study was to describe key characteristics of decisions for funding new NBS programmes in Europe. We analysed past decisions using a conceptual framework. It incorporates indicators that capture the steps of decision processes by health care payers. Based on an internet survey, we compared 22 decisions for which answers among two respondents were validated for each observation. The frequencies of indicators were calculated to elicit key characteristics. All decisions resulted in positive, mostly unrestricted funding. Stakeholder participation was diverse focusing on information provision or voting. Often, decisions were not fully transparent. Assessment of NBS technologies concentrated on expert opinion, literature review and rough cost estimates. Most important appraisal criteria were effectiveness (i.e., health gain from testing for the children being screened), disease severity and availability of treatments. Some common and diverging key characteristics were identified. Although no evidence of explicit healthcare rationing was found, processes may be improved in respect of transparency and scientific rigour of assessment.
PMCID: PMC4053875  PMID: 24852389
coverage; reimbursement; decision-making; internet survey; European Union; tandem mass spectronomy
2.  Screening of the hearing of newborns - Update 
Permanent congenital bilateral hearing loss (CHL) of moderate or greater degree (≥40 dB HL) is a rare disease, with a prevalence of about 1 to 3 per 1000 births. However, it is one of the most frequent congenital diseases. Reliance on physician observation and parental recognition has not been successful in the past in detecting significant hearing loss in the first year of life. With this strategy significant hearing losses have been detected in the second year of life. With two objective technologies based on physiologic response to sound, otoacoustic emissions (OAE) and auditory brainstem response (ABR) hearing screening in the first days of life is made possible.
The objective of this health technology assessment report is to update the evaluation on clinical effectiveness and cost-effectiveness of newborn hearing screening programs. Universal newborn hearing screening (UHNS) (i), selective screening of high risk newborns (ii), and the absence of a systematic screening program are compared for age at identification and age at hearing aid fitting of children with hearing loss. Secondly the potential benefits of early intervention are analysed. Costs and cost-effectiveness of newborn hearing screening programs are determined. This report is intended to make a contribution to the decision making whether and under which conditions a newborn hearing screening program should be reimbursed by the statutory sickness funds in Germany.
This health technology assessment report updates a former health technology assessment (Kunze et al. 2004 [1]). A systematic review of the literature was conducted, based on a documented search and selection of the literature using predefined inclusion and exclusion criteria and a documented extraction and appraisal of the included studies. To assess the cost-effectiveness of the different screening strategies in Germany the decision analytic Markov state model which had been developed in our former health technology assessment report was updated.
Universal newborn hearing screening programs are able to substantially reduce the age at identification and the age at intervention of children with CHL to six months of age in the German health care setting. High coverage rates, low fail rates and - if tracking systems are implemented – high follow-up-rates to diagnostic evaluation for test positives were achieved. New publications on potential benefits of early intervention could not be retrieved. For a final assessment of cost-effectiveness of newborn hearing screening evidence based long-term data are lacking. Decision analytic models with lifelong time horizon assuming that early detection results in improved language abilities and lower educational costs and higher life time productivity showed a potential of UNHS for long term cost savings compared to selective screening and no screening. For the short-term cost-effectiveness with a time horizon up to diagnostic evaluation more evidence based data are available. The average costs per case diagnosed range from 16,000 EURO to 33,600 EURO in Germany and hence are comparable to the cost of other implemented newborn screening programs. Empirical data for cost of selective screening in the German health care setting are lacking. Our decision analytic model shows that selective screening is more cost-effective but detects only 50% of all cases of congenital hearing loss.
There is good evidence that UNHS-Programs with appropriate quality management can reduce the age at start of intervention below six months. Up to now there is no indication of considerable negative consequences of screening for children with false positive test results and their parents. However, it is more difficult to prove the efficacy of early intervention to improve long-term outcomes. Randomized clinical trials of the efficacy of early intervention for children with CHL hearing losses are inappropriate because of ethical reasons. Prospective cohort studies with long-term outcomes of rare diseases are costly, take a long time and simultaneously substantial benefits of early intervention for language development seem likely.
A UNHS-Program should be implemented in Germany and be reimbursed by the statutory sickness funds. To achieve high coverage and because of better conditions for obtaining low false positive rates UNHS should be performed in hospital after birth. For outpatient deliveries additionally screening measures in an outpatient setting must be provided.
PMCID: PMC3011344  PMID: 21289971
3.  Developing a decision aid to guide public sector health policy decisions: A study protocol 
Decision aids have been developed in a number of health disciplines to support evidence-informed decision making, including patient decision aids and clinical practice guidelines. However, policy contexts differ from clinical contexts in terms of complexity and uncertainty, requiring different approaches for identifying, interpreting, and applying many different types of evidence to support decisions. With few studies in the literature offering decision guidance specifically to health policymakers, the present study aims to facilitate the structured and systematic incorporation of research evidence and, where there is currently very little guidance, values and other non-research-based evidence, into the policy making process. The resulting decision aid is intended to help public sector health policy decision makers who are tasked with making evidence-informed decisions on behalf of populations. The intent is not to develop a decision aid that will yield uniform recommendations across jurisdictions, but rather to facilitate more transparent policy decisions that reflect a balanced consideration of all relevant factors.
The study comprises three phases: a modified meta-narrative review, the use of focus groups, and the application of a Delphi method. The modified meta-narrative review will inform the initial development of the decision aid by identifying as many policy decision factors as possible and other features of methodological guidance deemed to be desirable in the literatures of all relevant disciplines. The first of two focus groups will then seek to marry these findings with focus group members' own experience and expertise in public sector population-based health policy making and screening decisions. The second focus group will examine issues surrounding the application of the decision aid and act as a sounding board for initial feedback and refinement of the draft decision aid. Finally, the Delphi method will be used to further inform and refine the decision aid with a larger audience of potential end-users.
The product of this research will be a working version of a decision aid to support policy makers in population-based health policy decisions. The decision aid will address the need for more structured and systematic ways of incorporating various evidentiary sources where applicable.
PMCID: PMC3108337  PMID: 21569255
4.  Provincial prenatal record revision: a multiple case study of evidence-based decision-making at the population-policy level 
There is a significant gap in the knowledge translation literature related to how research evidence actually contributes to health care decision-making. Decisions around what care to provide at the population (rather than individual) level are particularly complex, involving considerations such as feasibility, cost, and population needs in addition to scientific evidence. One example of decision-making at this "population-policy" level involves what screening questions and intervention guides to include on standardized provincial prenatal records. As mandatory medical reporting forms, prenatal records are potentially powerful vehicles for promoting population-wide evidence-based care. However, the extent to which Canadian prenatal records reflect best-practice recommendations for the assessment of well-known risk factors such as maternal smoking and alcohol consumption varies markedly across Canadian provinces and territories. The goal of this study is to better understand the interaction of contextual factors and research evidence on decision-making at the population-policy level, by examining the processes by which provincial prenatal records are reviewed and revised.
Guided by Dobrow et al.'s (2004) conceptual model for context-based evidence-based decision-making, this study will use a multiple case study design with embedded units of analysis to examine contextual factors influencing the prenatal record revision process in different Canadian provinces and territories. Data will be collected using multiple methods to construct detailed case descriptions for each province/territory. Using qualitative data analysis techniques, decision-making processes involving prenatal record content specifically related to maternal smoking and alcohol use will be compared both within and across each case, to identify key contextual factors influencing the uptake and application of research evidence by prenatal record review committees. All study participants will be required to give written informed consent prior to participating in data collection.
This study will advance knowledge in the field of evidence-based decision-making by illustrating the complex interaction of contextual factors and evidence on health policy decision-making by provincial-level committees. By increasing the transparency of decision-making within provincial prenatal record committees, this study will help inform more effective strategies for enhancing the integration of best-practice evidence into prenatal records.
PMCID: PMC2642799  PMID: 19099585
5.  The Cost and Impact of Scaling Up Pre-exposure Prophylaxis for HIV Prevention: A Systematic Review of Cost-Effectiveness Modelling Studies 
PLoS Medicine  2013;10(3):e1001401.
Gabriela Gomez and colleagues systematically review cost-effectiveness modeling studies of pre-exposure prophylaxis (PrEP) for preventing HIV transmission and identify the main considerations to address when considering the introduction of PrEP to HIV prevention programs.
Cost-effectiveness studies inform resource allocation, strategy, and policy development. However, due to their complexity, dependence on assumptions made, and inherent uncertainty, synthesising, and generalising the results can be difficult. We assess cost-effectiveness models evaluating expected health gains and costs of HIV pre-exposure prophylaxis (PrEP) interventions.
Methods and Findings
We conducted a systematic review comparing epidemiological and economic assumptions of cost-effectiveness studies using various modelling approaches. The following databases were searched (until January 2013): PubMed/Medline, ISI Web of Knowledge, Centre for Reviews and Dissemination databases, EconLIT, and region-specific databases. We included modelling studies reporting both cost and expected impact of a PrEP roll-out. We explored five issues: prioritisation strategies, adherence, behaviour change, toxicity, and resistance. Of 961 studies retrieved, 13 were included. Studies modelled populations (heterosexual couples, men who have sex with men, people who inject drugs) in generalised and concentrated epidemics from Southern Africa (including South Africa), Ukraine, USA, and Peru. PrEP was found to have the potential to be a cost-effective addition to HIV prevention programmes in specific settings. The extent of the impact of PrEP depended upon assumptions made concerning cost, epidemic context, programme coverage, prioritisation strategies, and individual-level adherence. Delivery of PrEP to key populations at highest risk of HIV exposure appears the most cost-effective strategy. Limitations of this review include the partial geographical coverage, our inability to perform a meta-analysis, and the paucity of information available exploring trade-offs between early treatment and PrEP.
Our review identifies the main considerations to address in assessing cost-effectiveness analyses of a PrEP intervention—cost, epidemic context, individual adherence level, PrEP programme coverage, and prioritisation strategy. Cost-effectiveness studies indicating where resources can be applied for greatest impact are essential to guide resource allocation decisions; however, the results of such analyses must be considered within the context of the underlying assumptions made.
Please see later in the article for the Editors' Summary
Editors' Summary
Every year approximately 2.5 million people are infected with HIV, the virus that causes AIDS. Behavioral strategies like condom use and reduction of sexual partners have been the hallmarks of HIV prevention efforts. However, biological prevention measures have also recently been shown to be effective. These include male circumcision, treatment as prevention (treating HIV-infected people with antiretroviral drugs to reduce transmission), and pre-exposure prophylaxis (PrEP), where people not infected with HIV take antiretroviral drugs to reduce the probability of transmission. Strategies such as PrEP may be viable prevention measure for couples in long-term relationships where one partner is HIV-positive and the other is HIV-negative (HIV serodiscordant couples) or groups at higher risk of HIV infection, such as men who have sex with men, and injection drug users.
Why Was This Study Done?
The findings from recent clinical trials that demonstrate PrEP can reduce HIV transmission have led to important policy discussions and in the US, Southern Africa, and the UK new clinical guidelines have been developed on the use of PrEP for the prevention of HIV infection. For those countries that are considering whether to introduce PrEP into HIV prevention programs, national policy and decision makers need to determine potential costs and health outcomes. Cost-effectiveness models—mathematical models that simulate cost and health effects of different interventions—can help inform such decisions. However, the cost-effectiveness estimates that could provide guidance for PrEP programs are dependent on, and limited by, the assumptions included in the models, which can make their findings difficult to generalize. A systematic comparison of published cost-effectiveness models of HIV PrEP interventions would be useful for policy makers who are considering introducing PrEP intervention programs.
What Did the Researchers Do and Find?
The researchers performed a systematic review to identify published cost-effectiveness models that evaluated the health gains and costs of HIV PrEP interventions. Systematic reviews attempt to identify, appraise, and synthesize all the empirical evidence that meets pre-specified eligibility criteria to answer a given research question by using explicit methods aimed at minimizing bias. By searching databases the authors identified 13 published studies that evaluated the impact of PrEP in different populations (heterosexual couples, men who have sex with men, and injection drug users) in different geographic settings, which included Southern Africa, Ukraine, US, and Peru.
The authors identified seven studies that assessed the introduction of PrEP into generalized HIV epidemics in Southern Africa. These studies suggest that PrEP may be a cost effective intervention to prevent heterosexual transmission. However, the authors note that funding PrEP while other cost-effective HIV prevention methods are underfunded in this setting may have high opportunity costs. The authors identified five studies where PrEP was introduced for concentrated epidemics among men who have sex with men (four studies in the US and one in Peru). These studies suggest that PrEP may have a substantial impact on the HIV epidemic but may not be affordable at current drug prices. The authors also identified a single study that modeled the introduction of PrEP for people who inject drugs in the Ukraine, which found PrEP not to be cost effective.
In all settings the price of antiretroviral drugs was found to be a limiting factor in terms of affordability of PrEP programs. Behavioral changes and adherence to PrEP were estimated to have potentially significant impacts on program effectiveness but the emergence of drug resistance or PrEP-related toxicity did not significantly affect cost-effectiveness estimates. Several PrEP prioritization strategies were explored in included studies and delivering PrEP to populations at highest risk of HIV exposure was shown to improve cost-effectiveness estimates. However, the extra costs of identifying and engaging with high-risk populations were not taken into consideration. The authors note that the geographic coverage of identified studies was limited and that the findings are very dependent on the setting which limits generalizability.
What Do these Findings Mean?
These findings suggest that PrEP could be a cost-effective tool to reduce new HIV infections in some settings. However, the cost-effectiveness of PrEP is dependent upon cost, the epidemic context, program coverage and prioritization strategies, participants' adherence to the drug regimen, and PrEP efficacy estimates. These findings will aid decision makers quantify and compare the reductions in HIV incidence that could be achieved by implementing a PrEP program.
Additional Information
Please access these Web sites via the online version of this summary at
The US National Institute of Allergy and Infectious Diseases has information on HIV/AIDS
aidsmap provides basic information about HIV/AIDS, summaries of recent research findings on HIV care and treatment, and has a section on PrEP
Information is available from Avert, an international AIDS charity, on many aspects of HIV/AIDS, including HIV prevention
AVAC Global Advocacy for HIV Prevention provides information on HIV prevention, including PrEP
The US Centers for Disease Control and Prevention also has information on PrEP
The World Health Organization has a page on its WHO-CHOICE criteria for cost-effectiveness
PMCID: PMC3595225  PMID: 23554579
6.  Patchy ‘coherence’: using normalization process theory to evaluate a multi-faceted shared decision making implementation program (MAGIC) 
Implementing shared decision making into routine practice is proving difficult, despite considerable interest from policy-makers, and is far more complex than merely making decision support interventions available to patients. Few have reported successful implementation beyond research studies. MAking Good Decisions In Collaboration (MAGIC) is a multi-faceted implementation program, commissioned by The Health Foundation (UK), to examine how best to put shared decision making into routine practice. In this paper, we investigate healthcare professionals’ perspectives on implementing shared decision making during the MAGIC program, to examine the work required to implement shared decision making and to inform future efforts.
The MAGIC program approached implementation of shared decision making by initiating a range of interventions including: providing workshops; facilitating development of brief decision support tools (Option Grids); initiating a patient activation campaign (‘Ask 3 Questions’); gathering feedback using Decision Quality Measures; providing clinical leads meetings, learning events, and feedback sessions; and obtaining executive board level support. At 9 and 15 months (May and November 2011), two rounds of semi-structured interviews were conducted with healthcare professionals in three secondary care teams to explore views on the impact of these interventions. Interview data were coded by two reviewers using a framework derived from the Normalization Process Theory.
A total of 54 interviews were completed with 31 healthcare professionals. Partial implementation of shared decision making could be explained using the four components of the Normalization Process Theory: ‘coherence,’ ‘cognitive participation,’ ‘collective action,’ and ‘reflexive monitoring.’ Shared decision making was integrated into routine practice when clinical teams shared coherent views of role and purpose (‘coherence’). Shared decision making was facilitated when teams engaged in developing and delivering interventions (‘cognitive participation’), and when those interventions fit with existing skill sets and organizational priorities (‘collective action’) resulting in demonstrable improvements to practice (‘reflexive monitoring’). The implementation process uncovered diverse and conflicting attitudes toward shared decision making; ‘coherence’ was often missing.
The study showed that implementation of shared decision making is more complex than the delivery of patient decision support interventions to patients, a portrayal that often goes unquestioned. Normalizing shared decision making requires intensive work to ensure teams have a shared understanding of the purpose of involving patients in decisions, and undergo the attitudinal shifts that many health professionals feel are required when comprehension goes beyond initial interpretations. Divergent views on the value of engaging patients in decisions remain a significant barrier to implementation.
PMCID: PMC3848565  PMID: 24006959
Shared decision making; Implementation; Patient-centered care; Normalization Process Theory
7.  Why integrated care? Conclusions from an international expert survey 
Exploring the indicators most crucial for actors in health and social care services and identifying those which lead up to the initiation and implementation of integrated care. By analysing the reasons why integrated care is chosen and initiated, the underlying mechanisms and decision-making processes of integrated care are highlighted.
Even though integrated care projects and programmes are implemented in very different settings and health systems one can find the same main actors everywhere. They share similar goals and principles which are universal and not unique to one specific country or system. Hence, it is likely that the type of actor, i.e. health insurance, is more influential on the decision-making process than the health system and surrounding setting. In other words, it was stipulated that a health insurer in the Netherlands will have similar priorities for integrated care as a health insurer in Singapore and hence will choose similar integrated care approaches.
On the other hand, system administrators can strongly incentivise or discourage innovation and cooperation within the health and social care dominions, i.e. by passing laws or (re)organising the financing system. It is suggested that integrated care is implemented more widely in countries where stakeholders receive targeted incentives and fragmentation within the system is less pronounced.
The hypotheses were tested using an international expert questionnaire, contacting integrated care managers and decision-makers in Europe, North America and Australasia. The results were quantitatively analyzed using SPSS.
Results and conclusions
Integrated care is stipulated to offer solutions to the demographic changes, the concurring increase of chronic disease and the pressures on restricted resources experienced in the modern health systems of today. The survey conducted suggested a more diversified picture regarding the expectations and priorities set into the concept by health care decision-makers. While all of these challenges are perceived by them, their undisputed priority is on the introduction and enhancement of management structures on all levels. They do not value financial restrictions as severely as may have been anticipated and they revealed that neither active patient participation nor the introduction of outcome measurement are high priorities when introducing integrated care. The findings also suggest that the same stakeholders follow similar priorities disregarding the different health systems they act in.
In conclusion, two levels of priority setting have been identified as highly important for integrated care initiation: on the policy level, prioritisation of integrated care along with specific promotion measures influence decision-making; on the organisational level, the need for clear structures and better management tools both in the organisation itself and in the management of the targeted patient population, reflect the highest priorities for the decision-making process.
The response rate was 18% with the majority of responses coming from Germany, The Netherlands, UK and the USA.
Integrated care has come a long way from the first projects implemented by health insurance organisations to a colourful array of projects and permanent programmes, spanning from local to national level and from very targeted to very broad inclusion criteria, initiated, owned and financed by a mix of all stakeholders and agents in the system. Hence, integrated care developed into a ‘Jack-of-all-trades’ approach, stimulating change and confusion at the same time. Still, in most countries, it has not entered mainstream health care organisation and management and continues to struggle in proving its value. Asking why integrated care is initiated and implemented in the first place helps understand the priorities and objectives of decision-makers in health care, and hence can lead to a more targeted development and application of integrated care models.
PMCID: PMC3184810
decision-making; priority setting in integrated care; stakeholder-specific differences
8.  Falls prevention for the elderly 
An ageing population, a growing prevalence of chronic diseases and limited financial resources for health care underpin the importance of prevention of disabling health disorders and care dependency in the elderly. A wide variety of measures is generally available for the prevention of falls and fall-related injuries. The spectrum ranges from diagnostic procedures for identifying individuals at risk of falling to complex interventions for the removal or reduction of identified risk factors. However, the clinical and economic effectiveness of the majority of recommended strategies for fall prevention is unclear. Against this background, the literature analyses in this HTA report aim to support decision-making for effective and efficient fall prevention.
Research questions
The pivotal research question addresses the effectiveness of single interventions and complex programmes for the prevention of falls and fall-related injuries. The target population are the elderly (> 60 years), living in their own housing or in long term care facilities. Further research questions refer to the cost-effectiveness of fall prevention measures, and their ethical, social and legal implications.
Systematic literature searches were performed in 31 databases covering the publication period from January 2003 to January 2010. While the effectiveness of interventions is solely assessed on the basis of randomised controlled trials (RCT), the assessment of the effectiveness of diagnostic procedures also considers prospective accuracy studies. In order to clarify social, ethical and legal aspects all studies deemed relevant with regard to content were taken into consideration, irrespective of their study design. Study selection and critical appraisal were conducted by two independent assessors. Due to clinical heterogeneity of the studies no meta-analyses were performed.
Out of 12,000 references retrieved by literature searches, 184 meet the inclusion criteria. However, to a variable degree the validity of their results must be rated as compromised due to different biasing factors. In summary, it appears that the performance of tests or the application of parameters to identify individuals at risk of falling yields little or no clinically relevant information. Positive effects of exercise interventions may be expected in relatively young and healthy seniors, while studies indicate opposite effects in the fragile elderly. For this specific vulnerable population the modification of the housing environment shows protective effects. A low number of studies, low quality of studies or inconsistent results lead to the conclusion that the effectiveness of the following interventions has to be rated unclear yet: correction of vision disorders, modification of psychotropic medication, vitamin D supplementation, nutritional supplements, psychological interventions, education of nursing personnel, multiple and multifactorial programs as well as the application of hip protectors.
For the context of the German health care system the economic evaluations of fall prevention retrieved by the literature searches yield very few useful results. Cost-effectiveness calculations of fall prevention are mostly based on weak effectiveness data as well as on epidemiological and cost data from foreign health care systems.
Ethical analysis demonstrates ambivalent views of the target population concerning fall risk and the necessity of fall prevention. The willingness to take up preventive measures depends on a variety of personal factors, the quality of information, guidance and decision-making, the prevention program itself and social support.
The analysis of papers regarding legal issues shows three main challenges: the uncertainty of which standard of care has to be expected with regard to fall prevention, the necessity to consider the specific conditions of every single case when measures for fall prevention are applied, and the difficulty to balance the rights to autonomous decision making and physical integrity.
Discussion and conclusions
The assessment of clinical effectiveness of interventions for fall prevention is complicated by inherent methodological problems (esp. absence of blinding) and meaningful clinical heterogeneity of available studies. Therefore meta-analyses are not appropriate, and single study results are difficult to interpret. Both problems also impair the informative value of economic analyses. With this background it has to be stated that current recommendations regarding fall prevention in the elderly are not fully supported by scientific evidence. In particular, for the generation of new recommendations the dependency of probable effects on specific characteristics of the target populations or care settings should be taken into consideration. This also applies to the variable factors influencing the willingness of the target population to take up and pursue preventive measures.
In the planning of future studies equal weight should be placed on methodological rigour (freedom from biases) and transferability of results into routine care. Economic analyses require input of German data, either in form of a “piggy back study“ or in form of a modelling study that reflects the structures of the German health care system and is based on German epidemiological and cost data.
PMCID: PMC3334922  PMID: 22536299
accidental falls; accidents, home/*; activities of daily living; aged/*; aged/*psychology; adjustment of the living environment; cataract surgery; correction of the visual acuity; customisation of the living environment; diagnosis; dietary supplements; dose-response relationship, drug; EBM; economic evaluation; elderly; environment design; evidence-based medicine; exercise program; exercise/physiology; eye test; eyesight; eyesight test; fall; fall prevention; fall prophylaxis; fall risk; fall risk factors; falling consequences; falling danger; fall-related injuries; fracture; freedom/*; freedom-depriving measures; geriatric nursing home; health technology assessment; hip fracture; hip fractures; hip protectors; homes for the aged; HTA; humans; interventions; medical adjustment; meta-analysis as topic; motor activity; motor activity/drug effects; motor skills; motor function; multi-factorial programs; multimodal programs; nursing homes; peer review; power of movement; prevention; primary prevention; private domesticity; prophylaxis; randomized controlled trial; randomized controlled trials as topic; RCT; review literature as topic; risk assessment; risk factors; risk reduction behavior; seniors; sight; stabilized; systematic review; technology assessment, biomedical; training program; visual acuity; Vitamin D/administration & dosage
9.  Acceptance of shared decision making with reference to an electronic library of decision aids (arriba-lib) and its association to decision making in patients: an evaluation study 
Decision aids based on the philosophy of shared decision making are designed to help patients make informed choices among diagnostic or treatment options by delivering evidence-based information on options and outcomes. A patient decision aid can be regarded as a complex intervention because it consists of several presumably relevant components. Decision aids have rarely been field tested to assess patients' and physicians' attitudes towards them. It is also unclear what effect decision aids have on the adherence to chosen options.
The electronic library of decision aids (arriba-lib) to be used within the clinical encounter has a modular structure and contains evidence-based decision aids for the following topics: cardiovascular prevention, atrial fibrillation, coronary heart disease, oral antidiabetics, conventional and intensified insulin therapy, and unipolar depression. We conducted an evaluation study in which 29 primary care physicians included 192 patients. After the consultation, patients filled in questionnaires and were interviewed via telephone two months later. We used generalised estimation equations to measure associations within patient variables and traditional crosstab analyses.
Patients were highly satisfied with arriba-lib and the process of shared decision making. Two-thirds of patients reached in the telephone interview wanted to be counselled again with arriba-lib. There was a high congruence between preferred and perceived decision making. Of those patients reached in the telephone interview, 80.7% said that they implemented the decision, independent of gender and education. Elderly patients were more likely to say that they implemented the decision.
Shared decision making with our multi-modular electronic library of decision aids (arriba-lib) was accepted by a high number of patients. It has positive associations to general aspects of decision making in patients. It can be used for patient groups with a wide range of individual characteristics.
PMCID: PMC3143082  PMID: 21736724
10.  Parental Decision-Making and Acceptance of Newborn Bloodspot Screening: An Exploratory Study 
PLoS ONE  2013;8(11):e79441.
Newborn bloodspot screening is an internationally established public health measure. Despite this, there is a paucity of information relating to the decision-making process that parents go through when accepting newborn screening. This is important as screening panels are expanding; potentially leading to an increasing amount of complex information. This study sought to understand the factors that influence parental decisions and roles they play in the decision-making process.
Patients and Methods
Qualitative thematic evaluation of semi structured interviews with parents whose children had recently undergone newborn screening in the Merseyside and Cheshire region of England, UK.
Eighteen interviews with first time parents (n = 12) and those with previous children (n = 6). Seven factors were identified as being either explicitly or implicitly related to parental decision-making: Experience, Attitudes to medicine, Information-seeking behaviour, Perceived knowledge, Attitudes to screening, and Perceived choice, all of which ultimately impact on Perceived decisional quality.
These results indicate that while content is important, other contextual factors such as personal experience, perceived choice, and general attitudes toward medicine, are also highly influential. In particular, relationships with key healthcare professionals are central to information collection, attitudes toward screening, and the level of deliberation that is invested in decisions to accept newborn bloodspot screening.
PMCID: PMC3827133  PMID: 24265771
11.  Field testing of a multicriteria decision analysis (MCDA) framework for coverage of a screening test for cervical cancer in South Africa 
Systematic and transparent approaches to priority setting are needed, particularly in low-resource settings, to produce decisions that are sound and acceptable to stakeholders. The EVIDEM framework brings together Health Technology Assessment (HTA) and multi-criteria decision analysis (MCDA) by proposing a comprehensive set of decision criteria together with standardized processes to support decisionmaking. The objective of the study was to field test the framework for decisionmaking on a screening test by a private health plan in South Africa.
Liquid-based cytology (LBC) for cervical cancer screening was selected by the health plan for this field test. An HTA report structured by decision criterion (14 criteria organized in the MCDA matrix and 4 contextual criteria) was produced based on a literature review and input from the health plan. During workshop sessions, committee members 1) weighted each MCDA decision criterion to express their individual perspectives, and 2) to appraise LBC, assigned scores to each MCDA criterion on the basis of the by-criterion HTA report.
Committee members then considered the potential impacts of four contextual criteria on the use of LBC in the context of their health plan. Feedback on the framework and process was collected through discussion and from a questionnaire.
For 9 of the MCDA matrix decision criteria, 89% or more of committee members thought they should always be considered in decisionmaking. Greatest weights were given to the criteria "Budget impact", "Cost-effectiveness" and "Completeness and consistency of reporting evidence". When appraising LBC for cervical cancer screening, the committee assigned the highest scores to "Relevance and validity of evidence" and "Disease severity". Combination of weights and scores yielded a mean MCDA value estimate of 46% (SD 7%) of the potential maximum value. Overall, the committee felt the framework brought greater clarity to the decisionmaking process and was easily adaptable to different types of health interventions.
The EVIDEM framework was easily adapted to evaluating a screening technology in South Africa, thereby broadening its applicability in healthcare decision making.
PMCID: PMC3330006  PMID: 22376143
12.  An evidence-based shared decision making programme on the prevention of myocardial infarction in type 2 diabetes: protocol of a randomised-controlled trial 
BMC Family Practice  2013;14:155.
Lack of patient involvement in decision making has been suggested as one reason for limited treatment success. Concepts such as shared decision making may contribute to high quality healthcare by supporting patients to make informed decisions together with their physicians.
A multi-component shared decision making programme on the prevention of heart attack in type 2 diabetes has been developed. It aims at improving the quality of decision-making by providing evidence-based patient information, enhancing patients’ knowledge, and supporting them to actively participate in decision-making. In this study the efficacy of the programme is evaluated in the setting of a diabetes clinic.
A single blinded randomised-controlled trial is conducted to compare the shared decision making programme with a control-intervention. The intervention consists of an evidence-based patient decision aid on the prevention of myocardial infarction and a corresponding counselling module provided by diabetes educators. Similar in duration and structure, the control-intervention targets nutrition, sports, and stress coping. A total of 154 patients between 40 and 69 years of age with type 2 diabetes and no previous diagnosis of ischaemic heart disease or stroke are enrolled and allocated either to the intervention or the control-intervention. Primary outcome measure is the patients’ knowledge on benefits and harms of heart attack prevention captured by a standardised knowledge test. Key secondary outcome measure is the achievement of treatment goals prioritised by the individual patient. Treatment goals refer to statin taking, HbA1c-, blood pressure levels and smoking status. Outcomes are assessed directly after the counselling and at 6 months follow-up. Analyses will be carried out on intention-to-treat basis. Concurrent qualitative methods are used to explore intervention fidelity and to gain insight into implementation processes.
Interventions to facilitate evidence-based shared decision making represent an innovative approach in diabetes care. The results of this study will provide information on the efficacy of such a concept in the setting of a diabetes clinic in Germany.
Trial registration
PMCID: PMC4016600  PMID: 24138325
Diabetes mellitus; Type 2; Myocardial infarction; Primary prevention; Patient education; Patient participation; Decision making; Evidence-based medicine
13.  HIV Treatment as Prevention: Systematic Comparison of Mathematical Models of the Potential Impact of Antiretroviral Therapy on HIV Incidence in South Africa 
PLoS Medicine  2012;9(7):e1001245.
Many mathematical models have investigated the impact of expanding access to antiretroviral therapy (ART) on new HIV infections. Comparing results and conclusions across models is challenging because models have addressed slightly different questions and have reported different outcome metrics. This study compares the predictions of several mathematical models simulating the same ART intervention programmes to determine the extent to which models agree about the epidemiological impact of expanded ART.
Methods and Findings
Twelve independent mathematical models evaluated a set of standardised ART intervention scenarios in South Africa and reported a common set of outputs. Intervention scenarios systematically varied the CD4 count threshold for treatment eligibility, access to treatment, and programme retention. For a scenario in which 80% of HIV-infected individuals start treatment on average 1 y after their CD4 count drops below 350 cells/µl and 85% remain on treatment after 3 y, the models projected that HIV incidence would be 35% to 54% lower 8 y after the introduction of ART, compared to a counterfactual scenario in which there is no ART. More variation existed in the estimated long-term (38 y) reductions in incidence. The impact of optimistic interventions including immediate ART initiation varied widely across models, maintaining substantial uncertainty about the theoretical prospect for elimination of HIV from the population using ART alone over the next four decades. The number of person-years of ART per infection averted over 8 y ranged between 5.8 and 18.7. Considering the actual scale-up of ART in South Africa, seven models estimated that current HIV incidence is 17% to 32% lower than it would have been in the absence of ART. Differences between model assumptions about CD4 decline and HIV transmissibility over the course of infection explained only a modest amount of the variation in model results.
Mathematical models evaluating the impact of ART vary substantially in structure, complexity, and parameter choices, but all suggest that ART, at high levels of access and with high adherence, has the potential to substantially reduce new HIV infections. There was broad agreement regarding the short-term epidemiologic impact of ambitious treatment scale-up, but more variation in longer term projections and in the efficiency with which treatment can reduce new infections. Differences between model predictions could not be explained by differences in model structure or parameterization that were hypothesized to affect intervention impact.
Please see later in the article for the Editors' Summary
Editors' Summary
Following the first reported case of AIDS in 1981, the number of people infected with HIV, the virus that causes AIDS, increased rapidly. In recent years, the number of people becoming newly infected has declined slightly, but the virus continues to spread at unacceptably high levels. In 2010 alone, 2.7 million people became HIV-positive. HIV, which is usually transmitted through unprotected sex, destroys CD4 lymphocytes and other immune system cells, leaving infected individuals susceptible to other infections. Early in the AIDS epidemic, half of HIV-infected people died within eleven years of infection. Then, in 1996, antiretroviral therapy (ART) became available, and, for people living in affluent countries, HIV/AIDS gradually became considered a chronic condition. But because ART was expensive, for people living in developing countries HIV/AIDS remained a fatal condition. Roll-out of ART in developing countries first started in the early 2000s. In 2006, the international community set a target of achieving universal ART coverage by 2010. Although this target has still not been reached, by the end of 2010, 6.6 million of the estimated 15 million people in need of ART in developing countries were receiving ART.
Why Was This Study Done?
Several studies suggest that ART, in addition to reducing illness and death among HIV-positive people, reduces HIV transmission. Consequently, there is interest in expanding the provision of ART as a strategy for reducing the spread of HIV (“HIV treatment as prevention"), particularly in sub-Saharan Africa, where one in 20 adults is HIV-positive. It is important to understand exactly how ART might contribute to averting HIV transmission. Several mathematical models that simulate HIV infection and disease progression have been developed to investigate the impact of expanding access to ART on the incidence of HIV (the number of new infections occurring in a population over a year). But, although all these models predict that increased ART coverage will have epidemiologic (population) benefits, they vary widely in their estimates of the magnitude of these benefits. In this study, the researchers systematically compare the predictions of 12 mathematical models of the HIV epidemic in South Africa, simulating the same ART intervention programs to determine the extent to which different models agree about the impact of expanded ART.
What Did the Researchers Do and Find?
The researchers invited groups who had previously developed mathematical models of the epidemiological impact of expanded access to ART in South Africa to participate in a systematic comparison exercise in which their models were used to simulate ART scale-up scenarios in which the CD4 count threshold for treatment eligibility, access to treatment, and retention on treatment were systematically varied. To exclude variation resulting from different model assumptions about the past and current ART program, it was assumed that ART is introduced into the population in the year 2012, with no treatment provision prior to this, and interventions were evaluated in comparison to an artificial counterfactual scenario in which no treatment is provided. A standard scenario based on the World Health Organization's recommended threshold for initiation of ART, although unrepresentative of current provision in South Africa, was used to compare the models. In this scenario, 80% of HIV-infected individuals received treatment, they started treatment on average a year after their CD4 count dropped below 350 cells per microliter of blood, and 85% remained on treatment after three years. The models predicted that, with a start point of 2012, the HIV incidence would be 35%–54% lower in 2020 and 32%–74% lower in 2050 compared to a counterfactual scenario where there was no ART. Estimates of the number of person-years of ART needed per infection averted (the efficiency with which ART reduced new infections) ranged from 6.3–18.7 and from 4.5–20.2 over the periods 2012–2020 and 2012–2050, respectively. Finally, estimates of the impact of ambitious interventions (for example, immediate treatment of all HIV-positive individuals) varied widely across the models.
What Do These Findings Mean?
Although the mathematical models used in this study had different characteristics, all 12 predict that ART, at high levels of access and adherence, has the potential to reduce new HIV infections. However, although the models broadly agree about the short-term epidemiologic impact of treatment scale-up, their longer-term projections (including whether ART alone can eliminate HIV infection) and their estimates of the efficiency with which ART can reduce new infections vary widely. Importantly, it is possible that all these predictions will be wrong—all the models may have excluded some aspect of HIV transmission that will be found in the future to be crucial. Finally, these findings do not aim to indicate which specific ART interventions should be used to reduce the incidence of HIV. Rather, by comparing the models that are being used to investigate the feasibility of “HIV treatment as prevention," these findings should help modelers and policy-makers think critically about how the assumptions underlying these models affect the models' predictions.
Additional Information
Please access these websites via the online version of this summary at
This study is part of the July 2012 PLoS Medicine Collection, Investigating the Impact of Treatment on New HIV Infections
Information is available from the US National Institute of Allergy and Infectious Diseases on HIV infection and AIDS
NAM/aidsmap provides basic information about HIV/AIDS and summaries of recent research findings on HIV care and treatment
Information is available from Avert, an international AIDS charity on many aspects of HIV/AIDS, including information on HIV/AIDS treatment and care, on HIV treatment as prevention, and on HIV/AIDS in South Africa (in English and Spanish)
The World Health Organization provides information about universal access to AIDS treatment (in English, French, and Spanish); its 2010 ART guidelines can be downloaded
The HIV Modelling Consortium aims to improve scientific support for decision-making by coordinating mathematical modeling of the HIV epidemic
Patient stories about living with HIV/AIDS are available through Avert; the charity website Healthtalkonline also provides personal stories about living with HIV, including stories about taking anti-HIV drugs and the challenges of anti-HIV drugs
PMCID: PMC3393664  PMID: 22802730
14.  Interpersonal Communication from the Patient Perspective: Comparison of Primary Healthcare Evaluation Instruments 
Healthcare Policy  2011;7(Spec Issue):108-123.
The operational definition of interpersonal communication is “the ability of the provider to elicit and understand patient concerns, to explain healthcare issues and to engage in shared decision-making if desired.”
To examine how well interpersonal communication is captured in validated instruments that evaluate primary healthcare from the patient's perspective.
645 adults with at least one healthcare contact in the previous 12 months responded to instruments that evaluate primary healthcare. Eight subscales measure interpersonal communication: the Primary Care Assessment Survey (PCAS, two subscales); the Components of Primary Care Index (CPCI, one subscale); the first version of the EUROPEP (EUROPEP-I); and the Interpersonal Processes of Care Survey, version II (IPC-II, four subscales). Scores were normalized for descriptive comparison. Exploratory and confirmatory (structural equation) factor analysis examined fit to operational definition, and item response theory analysis examined item performance.
Items not pertaining to interpersonal communication were removed from the EUROPEP-I. Most subscales are skewed positively. Normalized mean scores are similar across subscales except for IPC-II Patient-Centred Decision-Making and IPC-II Hurried Communication. All subscales load reasonably well on a single factor, presumed to be interpersonal communication. The best model has three underlying factors corresponding to eliciting (eigenvalue = 26.56), explaining (eigenvalue = 2.45) and decision-making (eigenvalue = 1.34). Both the PCAS Communication and the EUROPEP-I Clinical Behaviour subscales capture all three dimensions. Individual subscales within IPC-II measure each sub-dimension.
The operational definition is well reflected in the available measures, although shared decision-making is poorly represented. These subscales can be used with confidence in the Canadian context to measure this crucial aspect of patient-centred care.
PMCID: PMC3399440  PMID: 23205039
15.  Cost effectiveness of pediatric pneumococcal conjugate vaccines: a comparative assessment of decision-making tools 
BMC Medicine  2011;9:53.
Several decision support tools have been developed to aid policymaking regarding the adoption of pneumococcal conjugate vaccine (PCV) into national pediatric immunization programs. The lack of critical appraisal of these tools makes it difficult for decision makers to understand and choose between them. With the aim to guide policymakers on their optimal use, we compared publicly available decision-making tools in relation to their methods, influential parameters and results.
The World Health Organization (WHO) requested access to several publicly available cost-effectiveness (CE) tools for PCV from both public and private provenance. All tools were critically assessed according to the WHO's guide for economic evaluations of immunization programs. Key attributes and characteristics were compared and a series of sensitivity analyses was performed to determine the main drivers of the results. The results were compared based on a standardized set of input parameters and assumptions.
Three cost-effectiveness modeling tools were provided, including two cohort-based (Pan-American Health Organization (PAHO) ProVac Initiative TriVac, and PneumoADIP) and one population-based model (GlaxoSmithKline's SUPREMES). They all compared the introduction of PCV into national pediatric immunization program with no PCV use. The models were different in terms of model attributes, structure, and data requirement, but captured a similar range of diseases. Herd effects were estimated using different approaches in each model. The main driving parameters were vaccine efficacy against pneumococcal pneumonia, vaccine price, vaccine coverage, serotype coverage and disease burden. With a standardized set of input parameters developed for cohort modeling, TriVac and PneumoADIP produced similar incremental costs and health outcomes, and incremental cost-effectiveness ratios.
Vaccine cost (dose price and number of doses), vaccine efficacy and epidemiology of critical endpoint (for example, incidence of pneumonia, distribution of serotypes causing pneumonia) were influential parameters in the models we compared. Understanding the differences and similarities of such CE tools through regular comparisons could render decision-making processes in different countries more efficient, as well as providing guiding information for further clinical and epidemiological research. A tool comparison exercise using standardized data sets can help model developers to be more transparent about their model structure and assumptions and provide analysts and decision makers with a more in-depth view behind the disease dynamics. Adherence to the WHO guide of economic evaluations of immunization programs may also facilitate this process.
Please see related article:
PMCID: PMC3117724  PMID: 21569402
16.  Prioritizing Congenital Syphilis Control in South China: A Decision Analytic Model to Inform Policy Implementation 
PLoS Medicine  2013;10(1):e1001375.
Nicholas Tan and colleagues use a decision analytic model to quantify the impact of the ten-year national syphilis control plan in China and conclude that earlier and more extensive screening are also necessary for reaching policy goals.
Syphilis is a major public health problem in many regions of China, with increases in congenital syphilis (CS) cases causing concern. The Chinese Ministry of Health recently announced a comprehensive 10-y national syphilis control plan focusing on averting CS. The decision analytic model presented here quantifies the impact of the planned strategies to determine whether they are likely to meet the goals laid out in the control plan.
Methods and Findings
Our model incorporated data on age-stratified fertility, female adult syphilis cases, and empirical syphilis transmission rates to estimate the number of CS cases associated with prenatal syphilis infection on a yearly basis. Guangdong Province was the focus of this analysis because of the availability of high-quality demographic and public health data. Each model outcome was simulated 1,000 times to incorporate uncertainty in model inputs. The model was validated using data from a CS intervention program among 477,656 women in China. Sensitivity analyses were performed to identify which variables are likely to be most influential in achieving Chinese and international policy goals. Increasing prenatal screening coverage was the single most effective strategy for reducing CS cases. An incremental increase in prenatal screening from the base case of 57% coverage to 95% coverage was associated with 106 (95% CI: 101, 111) CS cases averted per 100,000 live births (58% decrease). The policy strategies laid out in the national plan led to an outcome that fell short of the target, while a four-pronged comprehensive syphilis control strategy consisting of increased prenatal screening coverage, increased treatment completion, earlier prenatal screening, and improved syphilis test characteristics was associated with 157 (95% CI: 154, 160) CS cases averted per 100,000 live births (85% decrease).
The Chinese national plan provides a strong foundation for syphilis control, but more comprehensive measures that include earlier and more extensive screening are necessary for reaching policy goals.
Please see later in the article for the Editors' Summary
Editors' Summary
Every year, 1.5 million pregnant women are infected with syphilis, a bacterial infection that is usually transmitted during sexual contact but that can also pass from a mother to her unborn child. In many of these women, the disease is detected through routine antenatal testing and is successfully treated with penicillin. But among those women who are not treated, about half experience adverse outcomes—the death of their baby during early or late pregnancy (fetal death and stillbirth, respectively) or soon after birth (neonatal death), or the birth of an infected baby. Babies born with syphilis (congenital syphilis) often fail to thrive and can develop problems such as blindness, deafness, and seizures if not treated. In 2008, syphilis in pregnancy contributed to 305,000 fetal deaths, stillbirths, and neonatal deaths, and 215,000 babies were affected by other adverse consequences of congenital syphilis. Yet congenital syphilis is simple and cheap to eliminate—a few injections of penicillin can clear the infection in a pregnant woman, and screening all pregnant women for syphilis is feasible even in low-resource settings.
Why Was This Study Done?
Congenital syphilis has recently reemerged in China. In the 1990s, there were very few cases of congenital syphilis in China, but by 2009, the reported incidence of congenital syphilis had risen to 139 cases per 100,000 live births. In 2010 the Chinese Ministry of Health announced a ten-year National Syphilis Prevention and Control Plan (NSCP) that, in line with World Health Organization (WHO) recommendations, aims to reduce the incidence of congenital syphilis to fewer than 30 cases per 100,000 live births by 2015 and to fewer than 15 cases per 100,000 live births by 2020. China's strategy for achieving these targets includes increasing prenatal syphilis screening coverage to 80% in urban areas and 60% in rural areas, increasing treatment rates among infected women to 90% in urban areas and 70% in rural areas, and increasing syphilis awareness among adults. But will this strategy be sufficient? Here, the researchers develop a mathematical model to quantify the likely impact of the NSCP's strategy on the incidence of congenital syphilis in southern China.
What Did the Researchers Do and Find?
The researchers developed a decision analytic model in which women move through a sequence of health states from uninfected, through infection and pregnancy, and to the development of congenital syphilis, and fed data collected in Guangdong Province between 2005 and 2008 on women's fertility, female syphilis cases, and syphilis transmission rates into the model. The researchers checked that their model provided estimates of the incidence of congenital syphilis that matched the reported incidence for Guangdong Province (internal validation) and the reported incidence in a congenital syphilis intervention program in Shenzhen, Guangdong (external validation). They then used their model to identify which parts of the NSCP strategy are likely to have the greatest effect on the incidence of congenital syphilis. Increasing prenatal screening coverage was the single most effective strategy for the reduction of congenital syphilis, but neither this strategy alone nor implementation of the whole NPSC strategy achieved the plan's target outcomes. By contrast, a four-pronged approach (95% coverage of prenatal screening, 75% of screening during the first two-thirds of pregnancy, 95% treatment completion, and having an accurate screening test) reduced the estimated incidence of congenital syphilis cases to 27 per 100,000 live births.
What Do These Findings Mean?
These findings suggest that although the NSCP is a strong foundation for control of congenital syphilis in China, more comprehensive measures will be needed to reach the plan's policy goals. In particular, the findings suggest that earlier and more extensive screening will be needed to reduce the incidence of congenital syphilis to below 30 cases per 100,000 live births, WHO's benchmark for congenital syphilis control. The accuracy of these findings is limited by the assumptions included in the model and by the data fed into it. Moreover, because the data included in the model came from Guangdong Province, these findings may not apply elsewhere in China or in other countries. Nevertheless, this study illustrates the importance of using multi-pronged approaches to address the complex problem of congenital syphilis control and identifies some strategies that are likely to improve the control of this important public health problem.
Additional Information
Please access these websites via the online version of this summary at
The World Health Organization provides information on sexually transmitted infections, including details of its strategy for the global elimination of congenital syphilis, the investment case for the elimination of mother-to-child transmission of syphilis, and regional updates on progress towards elimination (some information is available in several languages)
The US Centers for Disease Control and Prevention has a fact sheet on syphilis
The UK National Health Service Choices website also has a page on syphilis
MedlinePlus provides information on congenital syphilis and links to additional syphilis resources (in English and Spanish)
Haiti: Congenital Syphilis on the Way Out is a YouTube video describing the introduction of rapid diagnostic tests for syphilis in remote parts of Haiti
PMCID: PMC3551934  PMID: 23349624
17.  Vaccination of children with a live-attenuated, intranasal influenza vaccine – analysis and evaluation through a Health Technology Assessment 
Background: Influenza is a worldwide prevalent infectious disease of the respiratory tract annually causing high morbidity and mortality in Germany. Influenza is preventable by vaccination and this vaccination is so far recommended by the The German Standing Committee on Vaccination (STIKO) as a standard vaccination for people from the age of 60 onwards. Up to date a parenterally administered trivalent inactivated vaccine (TIV) has been in use almost exclusively. Since 2011 however a live-attenuated vaccine (LAIV) has been approved additionally. Consecutively, since 2013 the STIKO recommends LAIV (besides TIV) for children from 2 to 17 years of age, within the scope of vaccination by specified indications. LAIV should be preferred administered in children from 2 to 6 of age. The objective of this Health Technology Assessment (HTA) is to address various research issues regarding the vaccination of children with LAIV. The analysis was performed from a medical, epidemiological and health economic perspective, as well as from an ethical, social and legal point of view.
Method: An extensive systematic database research was performed to obtain relevant information. In addition a supplementary research by hand was done. Identified literature was screened in two passes by two independent reviewers using predefined inclusion and exclusion criteria. Included literature was evaluated in full-text using acknowledged standards. Studies were graded with the highest level of evidence (1++), if they met the criteria of European Medicines Agency (EMA)-Guidance: Points to consider on applications with 1. meta-analyses; 2. one pivotal study.
Results: For the medical section, the age of the study participants ranges from 6 months to 17 years. Regarding study efficacy, in children aged 6 months to ≤7 years, LAIV is superior to placebo as well as to a vac-cination with TIV (Relative Risk Reduction – RRR – of laboratory confirmed influenza infection approx. 80% and 50%, respectively). In children aged >7 to 17 years (= 18th year of their lives), LAIV is superior to a vaccination with TIV (RRR 32%). For this age group, no studies that compared LAIV with placebo were identified. It can be concluded that there is high evidence for superior efficacy of LAIV (compared to placebo or TIV) among children aged 6 months to ≤7 years. For children from >7 to 17 years, there is moderate evidence for superiority of LAIV for children with asthma, while direct evidence for children from the general population is lacking for this age group. Due to the efficacy of LAIV in children aged 6 months to ≤7 years (high evidence) and the efficacy of LAIV in children with asthma aged >7 to 17 years (moderate evidence), LAIV is also very likely to be efficacious among children in the general population aged >7 to 17 years (indirect evidence). In the included studies with children aged 2 to 17 years, LAIV was safe and well-tolerated; while in younger children LAIV may increase the risk of obstruction of the airways (e.g. wheezing).
In the majority of the evaluated epidemiological studies, LAIV proved to be effective in the prevention of influenza among children aged 2–17 years under everyday conditions (effectiveness). The trend appears to indicate that LAIV is more effective than TIV, although this can only be based on limited evidence for methodological reasons (observational studies). In addition to a direct protective effect for vaccinated children themselves, indirect protective ("herd protection") effects were reported among non-vaccinated elderly population groups, even at relatively low vaccination coverage of children. With regard to safety, LAIV generally can be considered equivalent to TIV. This also applies to the use among children with mild chronically obstructive conditions, from whom LAIV therefore does not have to be withheld. In all included epidemiological studies, there was some risk of bias identified, e.g. due to residual confounding or other methodology-related sources of error.
In the evaluated studies, both the vaccination of children with previous illnesses and the routine vaccination of (healthy) children frequently involve cost savings. This is especially the case if one includes indirect costs from a societal perspective. From a payer perspective, a routine vaccination of children is often regarded as a highly cost-effective intervention. However, not all of the studies arrive at consistent results. In isolated cases, relatively high levels of cost-effectiveness are reported that make it difficult to perform a conclusive assessment from an economic perspective. Based on the included studies, it is not possible to make a clear statement about the budget impact of using LAIV. None of the evaluated studies provides results for the context of the German healthcare setting.
The efficacy of the vaccine, physicians' recommendations, and a potential reduction in influenza symptoms appear to play a role in the vaccination decision taken by parents/custodians on behalf of their children. Major barriers to the utilization of influenza vaccination services are a low level of perception and an underestimation of the disease risk, reservations concerning the safety and efficacy of the vaccine, and potential side effects of the vaccine. For some of the parents surveyed, the question as to whether the vaccine is administered as an injection or nasal spray might also be important.
Conclusion: In children aged 2 to 17 years, the use of LAIV can lead to a reduction of the number of influenza cases and the associated burden of disease. In addition, indirect preventive effects may be expected, especially among elderly age groups. Currently there are no data available for the German healthcare setting. Long-term direct and indirect effectiveness and safety should be supported by surveillance programs with a broader use of LAIV.
Since there is no general model available for the German healthcare setting, statements concerning the cost-effectiveness can be made only with precaution. Beside this there is a need to conduct health eco-nomic studies to show the impact of influenza vaccination for children in Germany. Such studies should be based on a dynamic transmission model. Only these models are able to include the indirect protective effects of vaccination correctly.
With regard to ethical, social and legal aspects, physicians should discuss with parents the motivations for vaccinating their children and upcoming barriers in order to achieve broader vaccination coverage.
The present HTA provides an extensive basis for further scientific approaches and pending decisions relating to health policy.
PMCID: PMC4219018  PMID: 25371764
Health Technology Assessment; HTA; LAIV; live attenuated vaccine; TIV; trivalent inactivated vaccine
18.  Multi-dimensional Problems in Health Settings: A Review of Approaches to Decision Making 
There appears to be a growing number of prioritization exercises, for example of diseases, in health related settings (1). The decision process around these exercises involves comparing competing alternatives, i.e. diseases, and irreducible objectives. In addition to the multi-dimensional nature of the problem, the lack of reliable data, group dynamics associated to the involvement of experts, and the multiplicity of stakeholders, among other contextual factors, add complexity to the decision process. Here we review trends in such prioritization exercises and applications in different settings and for different events of interest, for example the management of emerging risks. Based on our findings, we discuss a conceptual framework based on multi-attribute utility theory presented to the World Organization for Animal Health (OIE) for the modification of its qualitative assessment of veterinary services performance into a quantifiable decision support system.
We searched PubMed for articles containing the key words ‘multi-criteria’, ‘multi-attribute’, ‘multi-objective’, ‘prioritization’, ‘decision making’ and their variations (e.g. without hyphenation) for the period 1990 to 2011 for human and veterinary medicine. We focused on prioritization methodologies and their sound application.
A large number of prioritization efforts in health settings aim to produce a rank order of diseases to help allocation of scarce surveillance and disease control budgets. A number of applications target the prioritization of competing health interventions against specific diseases. Fewer target different events, for example emerging threats. Common mistakes found in multi-attribute prioritization approaches reported in the social sciences (2) appear also in public and animal health settings. In particular, the application of linear additive models to non-preferentially independent evaluation criteria, the poor design of attributes to assess the decision alternatives, the failure to define suitable criteria scales, and mistakes in defining trade-off weights were prevalent. In addition, most decision support tools tend to be overly complex. This not only compromises their acceptability and long-term sustainability but also increases the likelihood of methodological mistakes in their design and regular application. For example, the failure to properly identify and separate ‘ends’ objectives, such as the improvement of a country’s health, from ‘means’ objectives, i.e. required resources, in the definition of the fundamental drivers in any decision process.
Our findings, and experience in the practical application of formal prioritization methodologies (3), informed our advice to the OIE for the quantification of its tools for the assessment of veterinary services performance. The current framework used by the OIE produces a purely qualitative output with ordinal scales. The suggested quantitative extension allows additional outputs not available in their current form, for example, the aggregation of assessment scores at any level within the framework to produce a country’s overall score. It also permits the assessment of marginal performance improvements for every criterion and the consideration of trade-offs among the different criteria. The final output of our extension is the identification of the best portfolio of actions that will maximize the overall capability of national veterinary services given available resources. Quantification of the existing tool will deliver obvious benefits such as enhanced accountability and transparency in the decision making process, and will allow the historical analysis of a country’s veterinary services performance. The approach suggested to the OIE is adaptable to similar decision problems, such as monitoring the implementation of the International Health Regulations in a given country.
PMCID: PMC3692762
Prioritisation; Multi-attribute utility theory; Decision support
19.  Development of a Transparent Interactive Decision Interrogator to Facilitate the Decision-Making Process in Health Care 
Value in Health  2011;14(5):768-776.
Decisions about the use of new technologies in health care are often based on complex economic models. Decision makers frequently make informal judgments about evidence, uncertainty, and the assumptions that underpin these models.
Transparent interactive decision interrogator (TIDI) facilitates more formal critique of decision models by decision makers such as members of appraisal committees of the National Institute for Health and Clinical Excellence in the UK. By allowing them to run advanced statistical models under different scenarios in real time, TIDI can make the decision process more efficient and transparent, while avoiding limitations on pre-prepared analysis.
TIDI, programmed in Visual Basic for applications within Excel, provides an interface for controlling all components of a decision model developed in the appropriate software (e.g., meta-analysis in WinBUGS and the decision model in R) by linking software packages using RExcel and R2WinBUGS. TIDI's graphical controls allow the user to modify assumptions and to run the decision model, and results are returned to an Excel spreadsheet. A tool displaying tornado plots helps to evaluate the influence of individual parameters on the model outcomes, and an interactive meta-analysis module allows the user to select any combination of available studies, explore the impact of bias adjustment, and view results using forest plots. We demonstrate TIDI using an example of a decision model in antenatal care.
Use of TIDI during the NICE appraisal of tumor necrosis factor-alpha inhibitors (in psoriatic arthritis) successfully demonstrated its ability to facilitate critiques of the decision models by decision makers.
PMCID: PMC3161376  PMID: 21839417
bias adjustment; decision model; interactive; meta-analysis; RExcel; software; TIDI
20.  Assessing Generalisability in Model-Based Economic Evaluation Studies: A Structured Review in Osteoporosis 
PharmacoEconomics  2006;24(12):1181-1197.
To support decision making many countries have now introduced some formal assessment process to evaluate whether health technologies represent good ‘value for money’. These often take the form of decision models which can be used to explore elements of importance to generalisability of study results across clinical settings and jurisdictions. The objectives of the present review were to assess: (i) whether the published studies clearly defined the decision-making audience for the model; (ii) the transparency of the reporting in terms of study question, structure and data inputs; (iii) the relevance of the data inputs used in the model to the stated decision-maker or jurisdiction; and (iv) how fully the robustness of the model's results to variation in data inputs between locations was assessed.
Articles reporting decision-analytic models in the area of osteoporosis were assessed to establish the extent to which the information provided enabled decision makers in different countries/jurisdictions to fully appreciate the variability of results according to location, and the relevance to their own.
Of the 18 articles included in the review, only three explicitly stated the decision-making audience. It was not possible to infer a decision-making audience in eight studies. Target population was well reported, as was resource and cost data, and clinical data used for estimates of relative risk reduction. However, baseline risk was rarely adapted to the relevant jurisdiction, and when no decision-maker was explicit it was difficult to assess whether the reported cost and resource use data was in fact relevant. A few studies used sensitivity analysis to explore elements of generalisability, such as compliance rates and baseline fracture risk rates, although such analyses were generally restricted to evaluating parameter uncertainty.
This review found that variability in cost-effectiveness across locations is addressed to a varying extent in modelling studies in the field of osteoporosis, limiting their use for decision-makers across different locations. Transparency of reporting is expected to increase as methodology develops, and decision-makers publish “reference case” type guidance.
PMCID: PMC2230686  PMID: 17129074
21.  Implementation science: a role for parallel dual processing models of reasoning? 
A better theoretical base for understanding professional behaviour change is needed to support evidence-based changes in medical practice. Traditionally strategies to encourage changes in clinical practices have been guided empirically, without explicit consideration of underlying theoretical rationales for such strategies. This paper considers a theoretical framework for reasoning from within psychology for identifying individual differences in cognitive processing between doctors that could moderate the decision to incorporate new evidence into their clinical decision-making.
Parallel dual processing models of reasoning posit two cognitive modes of information processing that are in constant operation as humans reason. One mode has been described as experiential, fast and heuristic; the other as rational, conscious and rule based. Within such models, the uptake of new research evidence can be represented by the latter mode; it is reflective, explicit and intentional. On the other hand, well practiced clinical judgments can be positioned in the experiential mode, being automatic, reflexive and swift. Research suggests that individual differences between people in both cognitive capacity (e.g., intelligence) and cognitive processing (e.g., thinking styles) influence how both reasoning modes interact. This being so, it is proposed that these same differences between doctors may moderate the uptake of new research evidence. Such dispositional characteristics have largely been ignored in research investigating effective strategies in implementing research evidence. Whilst medical decision-making occurs in a complex social environment with multiple influences and decision makers, it remains true that an individual doctor's judgment still retains a key position in terms of diagnostic and treatment decisions for individual patients. This paper argues therefore, that individual differences between doctors in terms of reasoning are important considerations in any discussion relating to changing clinical practice.
It is imperative that change strategies in healthcare consider relevant theoretical frameworks from other disciplines such as psychology. Generic dual processing models of reasoning are proposed as potentially useful in identifying factors within doctors that may moderate their individual uptake of evidence into clinical decision-making. Such factors can then inform strategies to change practice.
PMCID: PMC1523359  PMID: 16725023
22.  Development of a Support Tool for Complex Decision-Making in the Provision of Rural Maternity Care 
Healthcare Policy  2010;5(3):82-96.
Decisions in the organization of safe and effective rural maternity care are complex, difficult, value laden and fraught with uncertainty, and must often be based on imperfect information. Decision analysis offers tools for addressing these complexities in order to help decision-makers determine the best use of resources and to appreciate the downstream effects of their decisions.
To develop a maternity care decision-making tool for the British Columbia Northern Health Authority (NH) for use in low birth volume settings.
Based on interviews with community members, providers, recipients and decision-makers, and employing a formal decision analysis approach, we sought to clarify the influences affecting rural maternity care and develop a process to generate a set of value-focused objectives for use in designing and evaluating rural maternity care alternatives.
Four low-volume communities with variable resources (with and without on-site births, with or without caesarean section capability) were chosen.
Participants: Physicians (20), nurses (18), midwives and maternity support service providers (4), local business leaders, economic development officials and elected officials (12), First Nations (women [pregnant and non-pregnant], chiefs and band members) (40), social workers (3), pregnant women (2) and NH decision-makers/administrators (17).
We developed a Decision Support Manual to assist with assessing community needs and values, context for decision-making, capacity of the health authority or healthcare providers, identification of key objectives for decision-making, developing alternatives for care, and a process for making trade-offs and balancing multiple objectives. The manual was deemed an effective tool for the purpose by the client, NH.
Beyond assisting the decision-making process itself, the methodology provides a transparent communication tool to assist in making difficult decisions. While the manual was specifically intended to deal with rural maternity issues, the NH decision-makers feel the method can be easily adapted to assist decision-making in other contexts in medicine where there are conflicting objectives, values and opinions. Decisions on the location of new facilities or infrastructure, or enhancing or altering services such as surgical or palliative care, would be examples of complex decisions that might benefit from this methodology.
PMCID: PMC2831735  PMID: 21286270
23.  Do personal stories make patient decision aids more effective? A critical review of theory and evidence 
Patient decision aids support people to make informed decisions between healthcare options. Personal stories provide illustrative examples of others’ experiences and are seen as a useful way to communicate information about health and illness. Evidence indicates that providing information within personal stories affects the judgments and values people have, and the choices they make, differentially from facts presented in non-narrative prose. It is unclear if including narrative communications within patient decision aids enhances their effectiveness to support people to make informed decisions.
A survey of primary empirical research employing a systematic review method investigated the effect of patient decision aids with or without a personal story on people’s healthcare judgements and decisions. Searches were carried out between 2005-2012 of electronic databases (Medline, PsycINFO), and reference lists of identified articles, review articles, and key authors. A narrative analysis described and synthesised findings.
Of 734 citations identified, 11 were included describing 13 studies. All studies found participants’ judgments and/or decisions differed depending on whether or not their decision aid included a patient story. Knowledge was equally facilitated when the decision aids with and without stories had similar information content. Story-enhanced aids may help people recall information over time and/or their motivation to engage with health information. Personal stories affected both “system 1” (e.g., less counterfactual reasoning, more emotional reactions and perceptions) and “system 2” (e.g., more perceived deliberative decision making, more stable evaluations over time) decision-making strategies. Findings exploring associations with narrative communications, decision quality measures, and different levels of literacy and numeracy were mixed. The pattern of findings was similar for both experimental and real-world studies.
There is insufficient evidence that adding personal stories to decision aids increases their effectiveness to support people’s informed decision making. More rigorous research is required to elicit evidence about the type of personal story that a) encourages people to make more reasoned decisions, b) discourages people from making choices based on another’s values, and c) motivates people equally to engage with healthcare resources.
PMCID: PMC4044102  PMID: 24625283
24.  Rationality versus reality: the challenges of evidence-based decision making for health policy makers 
Current healthcare systems have extended the evidence-based medicine (EBM) approach to health policy and delivery decisions, such as access-to-care, healthcare funding and health program continuance, through attempts to integrate valid and reliable evidence into the decision making process. These policy decisions have major impacts on society and have high personal and financial costs associated with those decisions. Decision models such as these function under a shared assumption of rational choice and utility maximization in the decision-making process.
We contend that health policy decision makers are generally unable to attain the basic goals of evidence-based decision making (EBDM) and evidence-based policy making (EBPM) because humans make decisions with their naturally limited, faulty, and biased decision-making processes. A cognitive information processing framework is presented to support this argument, and subtle cognitive processing mechanisms are introduced to support the focal thesis: health policy makers' decisions are influenced by the subjective manner in which they individually process decision-relevant information rather than on the objective merits of the evidence alone. As such, subsequent health policy decisions do not necessarily achieve the goals of evidence-based policy making, such as maximizing health outcomes for society based on valid and reliable research evidence.
In this era of increasing adoption of evidence-based healthcare models, the rational choice, utility maximizing assumptions in EBDM and EBPM, must be critically evaluated to ensure effective and high-quality health policy decisions. The cognitive information processing framework presented here will aid health policy decision makers by identifying how their decisions might be subtly influenced by non-rational factors. In this paper, we identify some of the biases and potential intervention points and provide some initial suggestions about how the EBDM/EBPM process can be improved.
PMCID: PMC2885987  PMID: 20504357
25.  Prioritising health service innovation investments using public preferences: a discrete choice experiment 
Prioritising scarce resources for investment in innovation by publically funded health systems is unavoidable. Many healthcare systems wish to foster transparency and accountability in the decisions they make by incorporating the public in decision-making processes. This paper presents a unique conceptual approach exploring the public’s preferences for health service innovations by viewing healthcare innovations as ‘bundles’ of characteristics. This decompositional approach allows policy-makers to compare numerous competing health service innovations without repeatedly administering surveys for specific innovation choices.
A Discrete Choice Experiment (DCE) was used to elicit preferences. Individuals chose from presented innovation options that they believe the UK National Health Service (NHS) should invest the most in. Innovations differed according to: (i) target population; (ii) target age; (iii) implementation time; (iv) uncertainty associated with their likely effects; (v) potential health benefits; and, (vi) cost to a taxpayer. This approach fosters multidimensional decision-making, rather than imposing a single decision criterion (e.g., cost, target age) in prioritisation. Choice data was then analysed using scale-adjusted Latent Class models to investigate variability in preferences and scale and valuations amongst respondents.
Three latent classes with considerable heterogeneity in the preferences were present. Each latent class is composed of two consumer subgroups varying in the level of certainty in their choices. All groups preferred scientifically proven innovations, those with potential health benefits that cost less. There were, however, some important differences in their preferences for innovation investment choices: Class-1 (54%) prefers innovations benefitting adults and young people and does not prefer innovations targeting people with ‘drug addiction’ and ‘obesity’. Class- 2 (34%) prefers innovations targeting ‘cancer’ patients only and has negative preferences for innovations targeting elderly, and Class-3 (12%) prefers spending on elderly and cancer patients the most.
DCE can help policy-makers incorporate public preferences for health service innovation investment choices into decision making. The findings provide useful information on the public’s valuation and acceptability of potential health service innovations. Such information can be used to guide innovation prioritisation decisions by comparing competing innovation options. The approach in this paper makes, these often implicit and opaque decisions, more transparent and explicit.
PMCID: PMC4166469  PMID: 25167926

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