Challenges with cost-utility analyses of behavioural interventions among older adults at risk for dementia Estimating the Differential Costs of Criminal Activity for Juvenile Drug Court Participants: Challenges and Recommendations Erratum to: Estimating the Differential Costs of Criminal Activity for Juvenile Drug Court Participants: Challenges and Recommendations Falls prevention for the elderly Within-trial economic evaluation of diabetes-specific cognitive behaviour therapy in patients with type 2 diabetes and subthreshold depression Brief physician advice for problem drinking among older adults: an economic analysis of costs and benefits. Economic Evaluation of Occupational Safety and Health Interventions From the Employer Perspective Economic Evaluation of Bone Morphogenetic Protein Versus Autogenous Iliac Crest Bone Graft in Single-Level Anterior Lumbar Fusion: An Evidence-Based Modeling Approach (Cost)-effectiveness of case-management by district nurses among primary informal caregivers of older adults with dementia symptoms and the older adults who receive informal care: design of a randomized controlled trial [ISCRTN83135728] Measuring improved targeting of health interventions to the poor in the context of a community-randomised trial in rural India. Application of the development stages of a cluster randomized trial to a framework for evaluating complex health interventions A cost utility analysis of treatment options for gallstone disease: methodological issues and results. Systematic care for caregivers of people with dementia in the ambulatory mental health service: designing a multicentre, cluster, randomized, controlled trial (Cost)effectiveness of life review for Older Adults: Design of a randomized controlled trial Methodological Challenges in Online Trials The Monetary Value of Informal Care: Obtaining Pure Time Valuations Using a Discrete Choice Experiment From Outcomes Research to Disease Management: A Guide for the Perplexed Multifactorial day hospital intervention to reduce falls in high risk older people in primary care: a multi-centre randomised controlled trial [ISRCTN46584556] The effect of improved rural sanitation on diarrhoea and helminth infection: design of a cluster-randomized trial in Orissa, India Interventions for enhancing medication compliance/adherence with benefits in treatment outcomes The Cost-Effectiveness and Cost-Benefit of Screening and Brief Intervention for Unhealthy Alcohol Use in Medical Settings Promotion of self-management in vulnerable older people: a narrative literature review of outcomes of the Chronic Disease Self-Management Program (CDSMP) Informative value of Patient Reported Outcomes (PRO) in Health Technology Assessment (HTA)
Background Cognitive decline is one of the most prominent healthcare issues of the 21st century . Within the context of combating cognitive decline through behavioural interventions , physical activity is a promising approach . There is a dearth of health economic data in the area of behavioural interventions for dementia prevention . Yet , economic evaluations are essential for providing information to policy makers for re source allocation . It is essential we first address population and intervention-specific method ological challenges prior to building a larger evidence base . We use a cost-utility analysis conducted alongside the exercise for cognition and everyday living ( EXCEL ) study to illustrate method ological challenges specific to assessing the cost-effectiveness of behavioural interventions aim ed at older adults at risk of cognitive decline . Methods A cost-utility analysis conducted concurrently with a 6-month , three-arm r and omised controlled trial ( ie , the EXCEL study ) was used as an example to identify and discuss method ological challenges . Results Both the aerobic training and resistance training interventions were less costly than twice weekly balance and tone classes . In critically evaluating the economic evaluation of the EXCEL study we identified four category-specific challenges : ( 1 ) analysing costs ; ( 2 ) assessing quality -adjusted life-years ; ( 3 ) Incomplete data ; and ( 4 ) ‘ Intervention ’ activities of the control group . Conclusions Resistance training and aerobic training result ed in healthcare cost saving and were equally effective to balance and tone classes after only 6 months of intervention . To ensure this population is treated fairly in terms of cl aims on re sources , we first need to identify areas for method ological improvement Juvenile  drug court ( JDC ) programs have exp and ed rapidly over the past 20 years and are an increasingly popular option for rehabilitating juvenile offenders with substance use problems . Given the high cost of crime to society , an important economic question is whether and to what extent JDC programs reduce criminal activity among juvenile offenders . To address this question , the present study added an economic cost analysis to an ongoing r and omized trial of JDC conducted in Charleston , South Carolina . Four treatment conditions were included in the parent study : Family Court with usual community-based treatment ( FC , the comparison group ) , Drug Court with usual community-based treatment ( DC ) , DC with Multisystemic Therapy ( DC/MST ) , and DC/MST enhanced with Contingency Management ( DC/MST/CM ) . The economic study estimated the cost of criminal activity for nine specific crimes at baseline ( pretreatment ) and 4 and 12 months thereafter . A number of method ological challenges were encountered , suggesting that it may be more difficult to economically quantify frequency and type of criminal activity for adolescents than for adults . The present paper addresses method ological approaches and challenges , and proposes guidelines for future economic evaluations of adolescent substance abuse and crime prevention programs Juvenile  drug court ( JDC ) programs have exp and ed rapidly over the past 20 years and are an increasingly popular option for rehabilitating juvenile offenders with substance use problems . Given the high cost of crime to society , an important economic question is whether and to what extent JDC programs reduce criminal activity among juvenile offenders . To address this question , the present study added an economic cost analysis to an ongoing r and omized trial of JDC conducted in Charleston , South Carolina . Four treatment conditions were included in the parent study : Family Court with usual community-based treatment ( FC , the comparison group ) , Drug Court with usual community-based treatment ( DC ) , DC with Multisystemic Therapy ( DC/MST ) , and DC/MST enhanced with Contingency Management ( DC/MST/CM ) . The economic study estimated the cost of criminal activity for nine specific crimes at baseline ( pretreatment ) and 4 and 12 months thereafter . A number of method ological challenges were encountered , suggesting that it may be more difficult to economically quantify frequency and type of criminal activity for adolescents than for adults . The present paper addresses method ological approaches and challenges , and proposes guidelines for future economic evaluations of adolescent substance abuse and crime prevention programs Background An ageing population , a growing prevalence of chronic diseases and limited financial re sources 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 implication s. Methods Systematic literature search es were performed in 31 data bases covering the publication period from January 2003 to January 2010 . While the effectiveness of interventions is solely assessed on the basis of r and omised controlled trials ( RCT ) , the assessment of the effectiveness of diagnostic procedures also considers prospect i ve 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 . Results Out of 12,000 references retrieved by literature search es , 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 clinical ly 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 search es 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 st and ard 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 method ological 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 population s or care setting s 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 method ological 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 Background Despite the high prevalence of subthreshold depression in patients with type 2 diabetes , evidence on cost-effectiveness of different therapy options for these patients is currently lacking . Methods / Design Within-trial economic evaluation of the diabetes-specific cognitive behaviour therapy for subthreshold depression . Patients with diabetes and subthreshold depression are r and omly assigned to either 2 weeks of diabetes-specific cognitive behaviour group therapy  ( n = 104 ) or to st and ard diabetes education programme only  ( n = 104 ) . Patients are followed for 12 months . During this period data on total health sector costs , patient costs and societal productivity costs are collected in addition to clinical data . Health related quality of life ( the SF-36 and the EQ-5D ) is measured at baseline , immediately after the intervention , at 6 and at 12 months after the intervention . Quality adjusted life years ( QALYs ) , and cumulative costs will be estimated for each arm of the trial . Cost-effectiveness of the diabetes-specific cognitive behaviour group therapy will be analysed from the perspective of the German statutory health insurance and from the societal perspective . To this end , incremental cost-effectiveness ratio ( ICER ) in terms of cost per QALY gained will be calculated . Discussion Some method ological issues of the described economic evaluation are discussed . Trial registration The trial has been registered at the Clinical Trials Register ( NCT01009138 ) OBJECTIVE Problem alcohol use among elderly persons can have a variety of health-related consequences , complicating management of chronic illnesses and increasing health care utilization and costs . This study evaluates the economic cost and benefits of brief intervention for at-risk drinking older adults . METHOD A controlled clinical trial with 24-month follow-up tested effectiveness of brief physician advice in reducing alcohol use , health care utilization and other consequences among older ( age 65 or older ) adult problem drinkers . Of 6,073 patients screened for problem drinking in 24 community-based primary care practice s in Wisconsin , 158 patients met inclusion criteria and were r and omized into control ( n = 71 ) or intervention ( n = 87 ) groups . Intervention group patients received two 10- to 15-minute physician-delivered counseling sessions including professional advice , education and contracting using scripted workbooks . RESULTS The intervention group demonstrated significant reductions in alcohol use ( p = 0.001 ) and frequency of excessive drinking ( p = 0.03 ) compared with the control group over 24 months , but no significant differences emerged in economic outcomes , including hospital days , emergency department visits , office visits , medications , lab and x-ray procedures , injuries , legal events or mortality . CONCLUSIONS Although the clinical benefits of brief alcohol interventions with older adults are clear , the economic results in this age group are less certain . Older adult problem drinkers may require more intensive and costly interventions to achieve economic benefits similar to those seen in younger adult problem drinkers . Method ological issues , such as statistical power , outcome measures , outlier cases and follow-up periods , are identified for future evaluations Objectives : The aim of this systematic review was to evaluate the cost-effectiveness of  occupational safety and health interventions  from the employer perspective . Methods : A comprehensive literature search ( 2005 to 2016 ) in five electronic data bases was conducted . Pre-2005 studies were identified from the reference lists of previous studies and systematic review s , which have similar objective to those of this search . Results : A total of 19 r and omized controlled trials and quasi-experimental studies were included , targeting diverse health problems in a number of setting s. Few studies included organizational-level interventions . When viewed in relation to the method ological quality and the sufficiency of economic evidence , five of 11  cost-effective occupational safety and health ( OSH ) interventions appear to be promising . Conclusion : The present systematic review highlights the need for high- quality economic evidence to evaluate the cost-effectiveness of OSH interventions , especially at organizational-level , in all areas of worker health Study Design . Economic evaluation provides a framework to explicitly measure and compare the value of alternative medical interventions in terms of their clinical , health-related quality -of-life , and economic outcomes . Comput-erized economic models can help inform the design of future prospect i ve studies by identifying the cost-drivers , the most uncertain parameter estimates , and the parameters with the greatest impact on the results and inferences . Objective . An economic analysis of bone morphogenetic protein versus autogenous iliac crest bone graft for single-level anterior lumbar fusion poses several method ologic challenges . This article describes how such an economic evaluation may be framed and design ed , while enumerating challenges , offering some solutions , and suggesting an agenda for future research . Summary of Background Data . An evidence -based modeling approach can incorporate epidemiologic , clinical , and economic data from several sources including r and omized clinical trials , peer- review ed literature , and expert opinion . Sensitivity analyses can be conducted by varying key parameter estimates within a reasonable range to assess the impact on the results and inferences . Results . Preliminary results suggest that from a payer perspective , the upfront price of bone morphogenetic protein is likely to be entirely offset by reductions in the use of other medical re sources . That is , bone morphogenetic protein appears to be cost neutral . The cost offsets were attributable largely to prevention of pain and complications associated with autogenous iliac crest bone graft , as well as reduction of the costs associated with fusion failures . Conclusions . Future research should focus on quantifying the health-related quality -of-life impact of bone morphogenetic protein relative to autogenous iliac crest bone graft , as well as the impact on lost productivity Background Dementia is an incurable disease with devastating consequences for both patients and their relatives . The objective of this study is to describe the study protocol of a r and omized controlled trial with assignment to either usual care or case-management by district nurses , among informal caregivers of older adults with dementia symptoms who live at home and the older adults who receive informal care . Methods / design In this r and omized controlled trial , effectiveness as well as cost-effectiveness of case-management is evaluated . It concerns case-management in early-detected patients with dementia symptoms and their primary informal caregivers . Participants are followed up to twelve months after baseline assessment . The main outcome measure of the effect evaluation is the caregiver 's sense of competence to care for the older person with dementia symptoms . The economic evaluation is performed from a societal perspective . Discussion This is one of the first trials on case-management that includes an economic evaluation . In addition , it concerns a tailor-made intervention in early-detected patients with dementia symptoms and their caregivers . The results of this r and omized controlled trial will provide valuable information for health professionals and policy makers on effectiveness and cost-effectiveness of early tailor-made case-management for patients and their informal caregivers . Moreover , positive effects will challenge current health care systems to move to more pro-active approaches for this group In spite of growing interest in socioeconomic differentials in health outcomes and access to health services , little has been written about method ologies for assessing the impact of equity-enhancing policies or programs . This paper describes three method ological challenges involved in design ing a r and omised trial with an equity outcome , and how these were met in a trial of alternative strategies to improving the uptake of benefits of a health insurance scheme among its poorest members . The Vimo SEWA trial is nested within a community-based insurance scheme in rural India . While conducting this trial , three method ological problems were encountered : ( i ) measuring poverty ( or " wealth " , or " socioeconomic status " ) ( ii ) assessing beneficiaries against an appropriate reference st and ard population and ( iii ) settling on an appropriate equity measure as an outcome indicator . These problems are likely to arise in any policy or program assessment that has an equity outcome . In the Vimo SEWA trial , the socioeconomic status of beneficiaries ( cl aim ants ) is assessed relative to that of all scheme members living in same sub-district by applying a rapid assessment question naire -- which reduces to an integrated index of socioeconomic status -- to both a r and om sample of members in each sub-district , and to all cl aim ants . The results are used to estimate the full distribution of socioeconomic status of members in each sub-district , with each member given a rank score between 0 and 100 . Interpolation is used to estimate the rank scores of cl aim ants relative to the membership base . The primary outcome measure for the trial is the mean socioeconomic rank score of cl aim ants . In developing country setting s , using an index of socioeconomic status is simpler than assessing household income or the value of household consumption . It is also relatively straightforward to compare the socioeconomic status of health program beneficiaries with a relevant reference population , although two independent surveys are required . Expressing relative wealth on a scale from zero to 100 is conceptually appealing , and the mean value of this rank score provides an equity-specific outcome measure readily integrated into the usual analytic framework for cluster-r and omised trials Introduction Trials of complex health interventions often pose difficult method ologic challenges . The objective of this paper is to assess the extent to which the various development steps of a cluster r and omized trial to optimize antibiotic use in nursing homes are represented in a recently published framework for the design and evaluation of complex health interventions . In so doing , the utility of the framework for health services research ers is evaluated . Methods Using the five phases of the framework ( theoretical , identification of components of the intervention , definition of trial and intervention design , method ological issues for main trial , promoting effective implementation ) , corresponding stages in the development of the cluster r and omized trial using diagnostic and treatment algorithms to optimize the use of antibiotics in nursing homes are identified and described . Results Synthesis of evidence needed to construct the algorithms , survey and qualitative research used to define components of the algorithms , a pilot study to assess the feasibility of delivering the algorithms , method ological issues in the main trial including choice of design , allocation concealment , outcomes , sample size calculation , and analysis are adequately represented using the stages of the framework . Conclusions The framework is a useful re source for research ers planning a r and omized clinical trial of a complex intervention The techniques of cost utility analysis ( CUA ) were used to evaluate the treatment of gallstone disease by open and laparoscopic cholecystectomy and by extracorporeal shockwave lithotripsy ( ESWL ) .  The application of the techniques in this context raised three method ological questions which are not satisfactorily resolved in the literature . The first is whether an ex ante or ex post perspective is best adopted for the measurement of quality of life ( QoL ) . The second is the method for converting a short term deterioration in QoL followed by full health into QALYs and the reliability of the methods available . The third is the issue of indirect costs which , in the context of a temporary disease state , can not be easily avoided . The economic evaluation found laparoscopic cholecystectomy to be generally superior than the competitor technologies ( entailing lower costs and better outcomes ) . However , the results were sensitive to assumptions about the perspective for measuring benefits and the inclusion of indirect costs Background Care for people with dementia and their informal caregivers is a challenging aim in healthcare . There is an urgent need for cost-effective support programs that prevent informal caregivers of people with dementia from becoming overburdened , which might result in a delay or decrease of patient institutionalization . For this reason , we have developed the Systematic Care Program for Dementia ( SCPD ) . The SCPD consists of an assessment of caregiver 's sense of competence and suggestions on how to deal with competence deficiencies . The efficiency of the SCPD will be evaluated in our study . Methods and design In our ongoing , cluster , r and omized , single-blind , controlled trial , the participants in six mental health services in four regions of the Netherl and s have been r and omized per service . Professionals of the ambulatory mental health services ( psychologists and social psychiatric nurses ) have been r and omly allocated to either the intervention group or the control group . The study population consists of community-dwelling people with dementia and their informal caregivers ( patient-caregiver dyads ) coming into the health service . The dyads have been clustered to the professionals . The primary outcome measure is the patient 's admission to a nursing home or home for the elderly at 12 months of follow-up . This measure is the most important variable for estimating cost differences between the intervention group and the control group . The secondary outcome measure is the quality of the patient 's and caregiver 's lives . Discussion A novelty in the SCPD is the pro-active and systematic approach . The focus on the caregiver 's sense of competence is relevant to economical healthcare , since this sense of competence is an important determinant of delay of institutionalization of people with dementia . The SCPD might be able to facilitate this with a relatively small cost investment for caregivers ' support , which could result in a major decrease in costs in the management of dementia . Implementation on a national level will be started if the SCPD proves to be efficient . Trial Registration Background  Depression in older adults is a serious health problem with a poor prognosis . There is a need for indicated preventive psychological interventions for older adults , that show to be promising in preventing depressive disorders . Methods / design This manuscript describes the design of a study evaluating ' Looking for Meaning ' , a newly developed prevention course for older adults with depressive symptoms , based on life- review . Both clinical and economic effectiveness are evaluated in a pragmatic r and omized controlled trial . The control condition of this 12-session preventive intervention is a 20-minute video movie . The primary outcome is symptoms of depression at post-treatment and follow-up ( 6 months after post-treatment ) . Secondary outcomes are symptoms of anxiety , satisfaction with life , mastery , reminiscence styles , quality of life , and health care costs . An additional result of this study is the insight into the working elements of the course , provided by the qualitative study . The qualitative data , mainly based on 20 open-ended interviews with participants , are to be analyzed with an emphasis on newly emerging insight . Discussion This study will add to the existing scientific knowledge in several ways , especially by also including an economic evaluation and a qualitative study to gain insight into the working mechanisms of the course , both rather new in the field of life review . Positive results of this study will make an evidence -based intervention to improve public health among older people available . Trial registration Current Controlled Trials Ltd , IS RCT Health care and health care services are increasingly being delivered over the Internet . There is a strong argument that interventions delivered online should also be evaluated online to maximize the trial ’s external validity . Conducting a trial online can help reduce research costs and improve some aspects of internal validity . To date , there are relatively few trials of health interventions that have been conducted entirely online . In this paper we describe the major method ological issues that arise in trials ( recruitment , r and omization , fidelity of the intervention , retention , and data quality ) , consider how the online context affects these issues , and use our experience of one online trial evaluating an intervention to help hazardous drinkers drink less ( DownYourDrink ) to illustrate potential solutions . Further work is needed to develop online trial methodology Background Interventions in health care often not only have an effect on patients , but also on their informal caregivers . Caregiving can have a profound impact on the health and wellbeing of carers . Ignoring these spillovers in economic evaluations risks labelling interventions mistakenly as cost-effective , at the expense of informal caregivers . Objective This paper investigates willingness-to-accept ( WTA ) values for an hour of informal care , corrected for positive and negative impacts of informal care , to facilitate the inclusion of informal care hours on the cost side of economic evaluations without double-counting spillover effects . Methods A discrete choice experiment ( DCE ) was conducted among a representative sample of the adult population in the Netherl and s ( n = 552 ) in September 2011 . An experimental design minimizing the D-error was used to construct choice sets with two unlabelled alternatives with the attributes ‘ hours caregiving ’ , ‘ monetary compensation for caregiving ’ and seven impacts of caregiving . To operationalize the r and om utility model , we used a panel mixed multinomial logit ( MMNL ) parameter model . For calculation of WTA , we used both population -level parameters and individual-level parameters . Results The mean WTA for an additional hour of informal care , corrected for positive and negative impacts of informal care , was € 14.57 . The signs of the coefficients were all in the expected directions . Conclusions This study reports a preference-based monetary value for informal care , corrected for other impacts . This valuation facilitates the inclusion of informal care hours on the cost side in economic evaluations without double-counting any spillover effects included on the effects side The well-being of the soul can be obtained only after that of the body has been secured , said M aim onides in his classical treatise [ 1 ] . This early comment pre date s the current appreciation of the effect of physical health on quality of life . Outcomes research has exp and ed dramatically in recent years [ 2 - 5 ] through multidisciplinary efforts involving health services research ers , epidemiologists , economists , sociologists , statisticians , and ethicists . Driving this interest are concerns about rapidly increasing health care costs , growing consumer involvement in medical decision making , and incorporation of information systems into clinical medicine . The health care industry has also increased its focus on efficiency and productivity , with particular emphasis on outcomes . The latest concept in this field , disease management , refers to the use of an explicit systematic population -based approach to identify persons at risk , intervene with specific programs of care , and measure clinical and other outcomes . Disease management is not new ; for centuries , clinicians have been identifying and treating patients using information gleaned from their training , the medical literature , and personal experience . What is new about the current model of disease management is that it does much more than evaluate known practice s and suggest guidelines . It is a systematic information-driven approach in which clinical encounters are computerized , summarized , and shared ; opportunities for intervention are not always detected solely at the bedside , but sometimes from a central data -base ; practice guidelines may be implemented by computer screens in the practice setting ; and initiatives are measured and compared in terms of health outcomes . At the core of disease management are principles described as outcomes research , an evolving and somewhat embryonic field . Confusion has arisen over terminology and the application of principles to the management of patients . We identify core concepts and highlight some issues that become apparent as outcomes principles are applied to practice . Measurement of Outcomes An important aspect of disease management is the ability to measure and report health outcomes . The movement to collect this information has recently escalated , stimulated by federal initiatives to compare mortality among hospitals [ 6 , 7 ] , by comparative outcomes studies [ 8 ] , and by payers seeking to improve quality and reduce costs [ 9 ] . Donabedian [ 10 - 12 ] described quality health care as an optimal triad of structure , process , and outcomes . And erson and colleagues [ 13 ] extended this model by suggesting that relations among these factors should guide decision making . The outcome measures have evolved from simple dichotomous ones such as survival or occurrence of a clinical event to patient-oriented measures such as satisfaction , quality of life , and functional status . The term outcomes has been linked to different measures , ranging from physiologic values to quality of life assessment s. One approach to taxonomy is shown in Table 1 . The reporting of outcomes may need to differ , depending on the priorities of those examining the data . A clinician is likely to be primarily interested in clinical or humanistic outcomes , whereas a health plan administrator may be interested in economic ones . Patients are interested in humanistic outcomes and have trouble interpreting clinical outcomes . As clinicians ' financial risk increases , they are also developing greater interest in economic outcomes . Current disease management programs include mixtures of all measures . Table 1 . Types of Outcome Measures and Selected Examples Several points should be emphasized as these measures gain broader application . Selection of specific measures is currently a somewhat arbitrary process . For asthma , for example , several disease-specific measures of quality of life are available [ 14 - 17 ] . One way to distinguish among these measures is to examine the evidence behind the development of each measure : Has each one been vali date d ? This is now a st and ardized process of item generation , item reduction , reproducibility , responsiveness , and validation [ 18 , 19 ] ; however , multiple question naires can meet these criteria and still leave the clinician confused . Many outcomes measures were developed for use in population surveys or clinical trials and not for use in monitoring individual patients . The within-person variability can be great , but it is often offset with aggregate data ; thus , in the context of disease management , some measures must be used with care . Even when measures are aggregated , statistical power may be insufficient to detect a significant improvement in outcomes . For example , in a modestly sized health plan with 25 000 members , one might estimate that 150 persons have diabetes and use insulin . Even if all 150 participated in a program that reduced the number of diabetes-related complications by 50 % through improved diet , exercise , and appropriate use of insulin , the statistical power would be too low to document the program value compared with complication rates with a previous program . Thus , not all outcomes can be collected on all population s ; if outcomes are measured , statistical issues need sufficient attention . Finally , the interpretation of the change in outcomes is not yet clear for all measures . For example , what is the meaning of a 4-point change on a 100-point quality -of-life scale ? Is a $ 0.50 increase in the per-member per-month charge enough to warrant a disease management program ? Research ers attempting to define clinical significance are beginning to address these issues by anchoring degree of change to other events [ 20 - 24 ] . For example , Brook and colleagues [ 25 ] noted that a three-point difference on a st and ardized mental health scale was analogous to being laid off from a job . Considerable additional research into the meaning and application of outcomes is needed . Effectiveness Studies The distinction between effectiveness and efficacy is critical to disease management ; treatments with proven efficacy do not always perform as well under conditions of typical clinical practice ( effectiveness ) . In most efficacy studies , highly specialized practitioners treat selected patients on tight protocol s in a setting in which both patients and practitioners have economic incentives to comply . In effectiveness studies , on the other h and , treatments can be viewed as they would be in usual setting s ( Table 2 ) [ 26 - 28 ] . Table 2 . Effectiveness Studies Compared with Efficacy Studies * Most therapies require evaluation of efficacy before being implemented . This evaluation generally requires a priori hypotheses , r and omization ( to eliminate selection bias and confounding ) , homogeneous patients at high risk for the outcome , experienced investigators who follow a protocol , a comparative measure such as placebo ( if ethical ) , and intensive follow-up to ensure compliance . Under these circumstances , if a treatment proves to be better than placebo ( or a comparative measure ) , one can be reassured that the treatment can work . However , questions may remain about the ability of the treatment to work adequately in a broader range of patients and in usual practice setting s in which both patients and providers face natural barriers to care . These issues are of central importance in assessing relative cost-effectiveness in the community . To address these questions , most effectiveness studies have been done as observational studies , wherein observed groups are not r and omly assigned to therapy and neither patients nor providers generally know that they are being studied ( usually retrospective studies ) . This eliminates issues related to inclusion of special patients and providers and allows the treatment to be observed as it is typically used . Selection bias , which occurs when patients are not r and omly assigned to alternative treatments , becomes potentially problematic , and adjustment for case mix and severity of illness becomes important . In response to this problem , prospect i ve effectiveness trials are beginning to emerge [ 29 - 31 ] . As in typical clinical trials , these trials include the development of a protocol with a priori hypotheses , recruitment of participants with informed consent , r and omization to eliminate selection bias , and routine collection of data at prespecified time periods . Prospect i ve effectiveness trials differ from typical clinical trials in that they enroll heterogeneous participants , use providers more similar to those who treat the disease , impose few protocol -driven interventions , and incorporate outcome measures relevant to the disease and delivery system . Such trials attempt to provide unbiased information in relation to selection factors while still addressing outcomes and effectiveness . In disease management , clinical trials are not always possible . When a health plan has developed a new clinical program , members may not feel comfortable having an intervention withheld , and employers may be unwilling to conduct a trial before disseminating the program . However , if the program is implemented without a concurrent comparison group , what remains is a before- and -after comparison ; within a rapidly changing health care environment , the aging of the population and other factors can confound results . This issue is an especially important method ologic challenge of program evaluation . Health Economic Evaluation The types of studies that are considered to be economic evaluations are shown in Table 3 . Of these , cost-effectiveness and costutility analyses overlap with outcomes research because economic measures are frequently related to outcomes such as mortality , quality of life , or value assigned to years of life saved ( or quality -adjusted life-years ) . Table 3 . Health Economic Evaluations Suppose the intervention is design ed for middle-aged persons with a disease that leads to death and only requires treatment for 3 years . If the therapy costs $ 3/d , the total cost of Falls in older people are a major public health concern in terms of morbidity , mortality and cost . Previous studies suggest that multifactorial interventions can reduce falls , and many geriatric day hospitals are now offering falls intervention programmes . However , no studies have investigated whether these programmes , based in the day hospital are effective , nor whether they can be successfully applied to high-risk older people screened in primary care . The hypothesis is that a multidisciplinary falls assessment and intervention at Day hospitals can reduce the incidence of falls in older people identified within primary care as being at high risk of falling . This will be tested by a pragmatic parallel-group r and omised controlled trial in which the participants , identified as at high risk of falling , will be r and omised into either the intervention Day hospital arm or to a control ( current practice ) arm . Those participants preferring not to enter the full r and omised study will be offered the opportunity to complete brief diaries only at monthly intervals . This data will be used to vali date the screening question naire . Three day hospitals ( 2 Nottingham , 1 Derby ) will provide the interventions , and the University of Nottingham 's Departments of Primary Care , the Division of Rehabilitation and Ageing Unit , and the Trent Institute for Health Service Research will provide the method ological and statistical expertise . Four hundred subjects will be r and omised into the two arms . The primary outcome measure will be the rate of falls over one year . Secondary outcome measures will include the proportion of people experiencing at least one fall , the proportion of people experiencing recurrent falls ( > 1 ) , injuries , fear of falling , quality of life , institutionalisation rates , and use of health services . Cost-effectiveness analyses will be performed to inform health commissioners about re source allocation issues . The importance of this trial is that the results may be applicable to any UK day hospital setting .SitesGeneral practice s across Nottinghamshire and Derbyshire . Day hospitals : Derbyshire Royal Infirmary ( Southern Derbyshire Acute Hospitals NHS Trust)Sherwood Day Service ( Nottingham City Hospital Trust)Leengate Day Hospital ( Queen 's Medical Centre Nottingham University Hospital NHS Trust Background Infectious diseases associated with poor sanitation such as diarrhoea , intestinal worms , trachoma and lymphatic filariasis continue to cause a large disease burden in low income setting s and contribute substantially to child mortality and morbidity . Obtaining health impact data for rural sanitation campaigns poses a number of method ological challenges . Here we describe the design of a village-level cluster-r and omised trial in the state of Orissa , India to evaluate the impact of an ongoing rural sanitation campaign conducted under the umbrella of  India  ’s Total Sanitation Campaign (TSC).We r and omised 50 villages to the intervention and 50 villages to control . In the intervention villages the implementing non-governmental organisations conducted community mobilisation and latrine construction with subsidies given to poor families . Control villages receive no intervention . Outcome measures include ( 1 ) diarrhoea in children under 5 and in all ages , ( 2 ) soil-transmitted helminth infections , ( 3 ) anthropometric measures , ( 4 ) water quality , ( 5 ) number of insect vectors ( flies , mosquitoes ) , ( 6 ) exposure to faecal pathogens in the environment . In addition we are conducting process documentation ( latrine construction and use , intervention reach ) , cost and cost-effectiveness analyses , spatial analyses and qualitative research on gender and water use for sanitation . Results R and omisation result ed in an acceptable balance between trial arms . The sample size requirements appear to be met for the main study outcomes . Delays in intervention roll-out caused logistical problems especially for the planning of health outcome follow-up surveys . Latrine coverage at the end of the construction period ( 55 % ) remained below the target of 70 % , a result that may , however , be in line with many other TSC intervention areas in India . Conclusion We discuss a number of method ological problems encountered thus far in this study that may be typical for sanitation trials . Nevertheless , it is expected that the trial procedures will allow measuring the effectiveness of a typical rural sanitation campaign , with sufficient accuracy and validity Scientific background Poor compliance or adherence in  drug therapy  can cause increased morbidity , mortality and enormous costs in the healthcare system ( in Germany annually approximately 10 billion euros ) . Different methods are used for enhancing the compliance or adherence . Research questions The evaluation addresses the questions about existence , efficacy , cost-benefit relation as well as ethical-social and juridical implication s of strategies for enhancing compliance or adherence in drug therapy with concomitant improvements in treatment outcomes . Methods A systematic literature search was conducted in the medical , also health economic relevant , literature data bases in January 2007 , beginning from 2002 . Systematic review s on the basis of ( r and omised controlled trials ( RCT ) concerning interventions to enhance compliance or adherence with regard to treatment outcomes as well as systematic review s of health economic analyses were included in the evaluation . Additionally , it was also search ed for publications which primarily considered ethical-social and juridical aspects of these interventions for the German context . Results One systematic review with data for 57 RCT was included in the medical evaluation and one systematic review with data for six studies into the health economic evaluation . No publication primary concerning ethical-social or juridical implication s could be identified . A significant positive effect on the treatment outcome was reported for 22 evaluated interventions . For many interventions the results can be classified as reliable : counseling with providing an information leaflet and compliance diary chart followed by phone consultation for helicobacter pylori positive patients , repeated counseling for patients with acute asthma symptoms , telephone calls to establish the level of compliance and to make recommendations based on that for the therapy of cardiovascular diseases , calls of an automated telephone system with phone counseling in problem cases for diabetics , different family based interventions including repeated family counseling , education and " culturally modified family therapy " in patients with schizophrenia , repeated " compliance therapy " sessions for patients with acute psychosis . For other interventions the results should be viewed with more concern ( because of the poor method ical quality of the underlying studies ) . The effect size of the interventions can not be estimated from the available data . From the available data , no reliable results can be provided concerning the cost-benefit relation of these strategies . Discussion Many of the reported studies had a poor reporting and method ological quality . The reliability of the conclusions of the studies is restricted because of method ical shortcomings . Efficacy and cost estimates determined in the health economic studies are not transferable to the current situation in Germany . It has been discussed recently that the compliance or adherence enhancing interventions can restrict the autonomy and the privacy of the patients . Conclusions In drug therapy some compliance or adherence enhancing interventions with concomitant positive effect on the treatment outcome may be used . The cost-benefit relation of these interventions is to be estimated . Using these interventions the patient ’s autonomy and privacy are to be restricted as few as possible SUMMARY Economic evaluation can be a valuable tool for assessing the efficiency and value of health care programs . To examine the literature on the economic evaluation of  alcohol screening and brief intervention in medical setting s , relevant studies were identified in the MEDLINE data base ( 1966 through November 2006 ) and by h and - search ing the references of identified articles and relevant journals . The 15 identified studies used a range of economic evaluation methods , including cost analysis , cost-benefit , cost-effectiveness , and cost-utility . Nearly all of the studies supported the use of alcohol screening and brief intervention . The studies that prospect ively collected cost and effect data and /or conformed closely to method ological guidelines demonstrated a strong economic benefit of alcohol screening and brief intervention when compared to usual care . Overall , the review ed studies support alcohol SBI in medical setting s as a wise use of health care re sources and illustrate the usefulness of economic evaluation for assessing alcohol prevention and treatment programs With ageing , older people can become frail , and this has been shown to be associated with a decrease in well-being . Observational studies provide evidence of a positive effect of coping re sources on well-being . The question is : can coping re sources be improved in vulnerable older people ? The Chronic Disease Self-Management Program ( CDSMP ) is a target group-specific intervention which aims to promote the self-management of older people who are confronted with deteriorating health . The aim of this study was to review intervention studies focusing on the CDSMP and to draw conclusions on the benefits of the program . A systematic search was conducted in PubMed and PsychINFO to identify r and omized controlled trials ( RCTs ) focusing on the CDSMP .  Nine RCTs focusing on relatively young older adults , 75 % of whom with an average age between 49 and 65 years , were included . We found that the CDSMP was consistently beneficial for Health behaviour , especially with regard to the variables of exercise and self-care . For Health status , the majority of studies only showed improvement in the domain of health distress . Most of the studies that investigated Self-efficacy showed convincing improvement in self-efficacy , cognitive symptom management and mental stress management . In Health care utilization , there was no significant decrease . On the whole , the studies showed that CDSMP led to an increase in physical exercise , a decrease in health distress , an improvement in self-care , and it had a beneficial effect on self-efficacy Background “ Patient-Reported Outcome ” ( PRO ) is used as an umbrella term for different concepts for measuring subjectively perceived health status e. g. as treatment effects . Their common characteristic is , that the appraisal of the health status is reported by the patient himself . In order to describe the informative value of PRO in Health Technology Assessment ( HTA ) first an overview of concepts , classifications and methods of measurement is given . The overview is complemented by an empirical analysis of clinical trials and HTA-reports on rheumatoid arthritis and breast cancer in order to report on type , frequency and consequences of PRO used in these documents . Methods For both issues systematic review s of the literature have been performed . The search for method ological literature covers the publication period from 1990 to 2009 , the search for clinical trials of rheumatoid arthritis and breast cancer covers the period 2005 to 2009 . Both search es were performed in the medical data bases of the German Institute of Medical Documentation and Information ( DIMDI ) . The search for HTA-reports and method ological papers of HTA-agencies was performed in the CRD- Data bases ( CRD = Centre for Review s and Dissemination ) and by h and search ing the websites of INAHTA member agencies ( INAHTA = International Network of Agencies for Health Technology Assessment ) . For all issues specific inclusion and exclusion criteria were defined . The method ological quality of r and omized controlled trials ( RCT ) was assessed by a modified version of the Cochrane Risk of Bias Tool . For the method ological part information extraction from the literature is structured by the report ’s chapters , for the empirical part data extraction sheets were constructed . All information is summarized in a qualitative manner . Results Concerning the method ological issues the literature search retrieved 158 documents ( 87 documents related to definition or classification , 125 documents related to operationalisation of PRO ) . For the empirical analyses 225 RCT ( rheumatoid arthritis : 77 ; breast cancer : 148 ) and 40 HTA-reports and method papers were found . The analysis of the method ological literature confirms the role of PRO as an umbrella term for a variety of different concepts . The newest classification system facilitates the description of PRO measures by construct , target population and the method of measurement . Steps of operationalisation involve defining a conceptual framework , instrument development , exploration of measurement properties or , possibly , the modification of existing instruments . Seven out of 59 RCT analysing the effects of antibody therapy for rheumatoid arthritis define PRO as the primary endpoint , 38 trials utilize composite measures ( ACR , DAS ) and ten trials report clinical or radiological parameters as the primary endpoint . Six out of 123 chemotherapy trials for breast cancer define PRO as the primary endpoint , while 98 trials report clinical endpoints ( survival , tumour response , progression ) in their primary analyses . Discrepancies in the number of trials result from inaccurate specifications of endpoints in the publications . This distribution is reflected in the HTA-reports : while almost all reports on rheumatoid arthritis refer to PRO , this is only the case in about half of the reports on breast cancer . Conclusions As definition and classification of PRO are concerned , coherent concepts are found in the literature . Their operationalisation and implementation must be guided by scientific principles . The type and frequency of PRO used in clinical trials largely depend on the disease analysed . The HTA-community seems to pursue the utilization of PRO proactively – in case of missing data the need for further research is stated
Disregarding these method ological obstacles could implicitly lead to discrimination against the elderly in terms of health promotion and disease prevention and , hence , an age-based rationing of public health care
Background In the light of demographic developments health promotion interventions for older people are gaining importance .
In addition to method ological challenges arising from the economic evaluation of health promotion interventions in general , there are specific method ological problems for the particular target group of older people .
There are especially four main method ological challenges that are discussed in the literature .
They concern measurement and valuation of informal caregiving , accounting for productivity costs , effects of unrelated cost in added life years and the inclusion of ‘ beyond-health ’ benefits .
This paper focuses on the question whether and to what extent specific method ological requirements are actually met in applied health economic evaluations .