Balancing costs and benefits at different stages of medical innovation: a systematic review of Multi-criteria decision analysis (MCDA)

Background The diffusion of health technologies from translational research to reimbursement depends on several factors included the results of health economic analysis. Recent research identified several flaws in health economic concepts. Additionally, the heterogeneous viewpoints of participating stakeholders are rarely systematically addressed in current decision-making. Multi-criteria Decision Analysis (MCDA) provides an opportunity to tackle these issues. The objective of this study was to review applications of MCDA methods in decisions addressing the trade-off between costs and benefits. Methods Using basic steps of the PRISMA guidelines, a systematic review of the healthcare literature was performed to identify original research articles from January 1990 to April 2014. Medline, PubMed, Springer Link and specific journals were searched. Using predefined categories, bibliographic records were systematically extracted regarding the type of policy applications, MCDA methodology, criteria used and their definitions. Results 22 studies were included in the analysis. 15 studies (68 %) used direct MCDA approaches and seven studies (32 %) used preference elicitation approaches. Four studies (19 %) focused on technologies in the early innovation process. The majority (18 studies - 81 %) examined reimbursement decisions. Decision criteria used in studies were obtained from the literature research and context-specific studies, expert opinions, and group discussions. The number of criteria ranged between three up to 15. The most frequently used criteria were health outcomes (73 %), disease impact (59 %), and implementation of the intervention (40 %). Economic criteria included cost-effectiveness criteria (14 studies, 64 %), and total costs/budget impact of an intervention (eight studies, 36 %). The process of including economic aspects is very different among studies. Some studies directly compare costs with other criteria while some include economic consideration in a second step. Conclusions In early innovation processes, MCDA can provide information about stakeholder preferences as well as evidence needs in further development. However, only a minority of these studies include economic features due to the limited evidence. The most important economic criterion cost-effectiveness should not be included from a technical perspective as it is already a composite of costs and benefit. There is a significant lack of consensus in methodology employed by the various studies which highlights the need for guidance on application of MCDA at specific phases of an innovation

Changes in Cognitive Function over 3 Years after First-Ever Stroke and Predictors of Cognitive Impairment and Long-Term Cognitive Stability: The Erlangen Stroke Project

Background and Purpose: Cognitive impairment (CI) is frequent after stroke, but data from population-based stroke cohorts on the natural course of CI are limited. The purpose of this study was to determine changes in cognitive status over 3 years after stroke. Methods: Data were collected from the Erlangen Stroke Project, an ongoing population-based stroke registry. The Mini-Mental State Examination (MMSE) for assessing global cognitive function was used; CI was defined as an MMSE score ! 24. Results: From February 1998 to January 2006, 630 patients with first-ever stroke were included. Prevalence rates of CI at 3 months, 1 and 3 years were 15, 13, and 12%. In multivariable analysis, stroke severity, i.e. Barthel index (p ! 0.001), age (OR = 1.03; 95% CI = 1.00–1.05) and diabetes mellitus (OR = 2.03; 95% CI = 1.13–3.67) were associated with CI at 3 months. Recovery rate from CI at 3 months after stroke was found to be 31% over the following 3 years. Intact cognitive function rate was 71% over 3 years and inversely associated with age (OR = 0.96; 95% CI = 0.96–0.94) and stroke severity (p ! 0.001). Conclusion: CI is frequent among stroke survivors and associated with age, stroke severity, and diabetes mellitus, but recovery occurs in approximately one third of the patients over the course of 3 years. Factors affecting intact cognitive function over time are increasing age and stroke severity

Tailored e-Health services for the dementia care setting: a pilot study of ‘eHealthMonitor’

Background The European eHealthMonitor project (eHM) developed a user-sensitive and interactive web portal for dementia care: the eHM Dementia Portal (eHM-DP). It aims to provide targeted and personalized support for informal caregivers of people with dementia in a home-based care setting. The objective of the pilot study was to obtain feedback on the eHM-DP from two user perspectives (caregivers and medical professionals), focusing on caregiver empowerment, decision aid, and the perceived benefits of the eHM-DP. Methods The study on the eHM-DP was conducted from March 2014 to June 2014. The methodological approach followed a user-participatory design with a total number of 42 participants. The study included caregivers of people with dementia and medical professionals (MPs) from the metropolitan region of Erlangen-Nürnberg (Bavaria, Germany). Study participants were interviewed face-to-face with semi-structured, written interviews. Results Caregivers indicated a high degree of perceived support by the eHM-DP and of provided decision aid. In total, 89 % of caregivers and 54 % of MPs would use the eHM-DP if access were provided. The primary benefits participants perceived were the acquisition of individualized information, computerized interaction between caregivers and MPs, empowerment in health-related decisions and comprehensive insights into the progress of the disease. Major recommendations for improving the eHM-DP encompassed: an active search functionality based on predefined terms, the implementation of a chatroom for caregivers, an upload function and alerts for MPs, as well as the overall design. Conclusions Our study is the first to have provided new insights and results on an interactive and needs-oriented web portal, endeavouring towards empowerment and assistance in decision making for caregivers as well as MPs within the realm of caring for patients with dementia. The acceptance and willingness to use the eHM-DP emphasizes the potential of eHealth services for community-based dementia care settings

A systematic review of Health Technology Assessment tools in sub-Saharan Africa: methodological issues and implications

Background Health technology assessment (HTA) is mostly used in the context of high- and middle-income countries. Many “resource-poor” settings, which have the greatest need for critical assessment of health technology, have a limited basis for making evidence-based choices. This can lead to inappropriate use of technologies, a problem that could be addressed by HTA that enables the efficient use of resources, which is especially crucial in such settings. There is a lack of clarity about which HTA tools should be used in these settings. This research aims to provide an overview of proposed HTA tools for “resource-poor” settings with a specific focus on sub-Saharan Africa (SSA). Methodology A systematic review was conducted using basic steps from the PRISMA guidelines. Studies that described HTA tools applicable for “resource-limited” settings were identified and critically appraised. Only papers published between 2003 and 2013 were included. The identified tools were assessed according to a checklist with methodological criteria. Results Six appropriate tools that are applicable in the SSA setting and cover methodological robustness and ease of use were included in the review. Several tools fulfil these criteria, such as the KNOW ESSENTIALS tool, Mini-HTA tool, and Multi-Criteria Decision Analysis but their application in the SSA context remains limited. The WHO CHOICE method is a standardized decision making tool for choosing interventions but is limited to their cost-effectiveness. Most evaluation of health technology in SSA focuses on priority setting. There is a lack of HTA tools that can be used for the systematic assessment of technology in the SSA context. Conclusions An appropriate HTA tool for “resource-constrained” settings, and especially SSA, should address all important criteria of decision making. By combining the two most promising tools, KNOW ESSENTIALS and Multi-Criteria Decision Analysis, appropriate analysis of evidence with a robust and flexible methodology could be applied for the SSA setting

Stroke risk associated with balloon based catheter ablation for atrial fibrillation: Rationale and design of the MACPAF Study

<p>Abstract</p> <p>Background</p> <p>Catheter ablation of the pulmonary veins has become accepted as a standard therapeutic approach for symptomatic paroxysmal atrial fibrillation (AF). However, there is some evidence for an ablation associated (silent) stroke risk, lowering the hope to limit the stroke risk by restoration of rhythm over rate control in AF. The purpose of the prospective randomized single-center study "Mesh Ablator versus Cryoballoon Pulmonary Vein Ablation of Symptomatic Paroxysmal Atrial Fibrillation" (MACPAF) is to compare the efficacy and safety of two balloon based pulmonary vein ablation systems in patients with symptomatic paroxysmal AF.</p> <p>Methods/Design</p> <p>Patients are randomized 1:1 for the Arctic Front^®or the HD Mesh Ablator^®catheter for left atrial catheter ablation (LACA). The predefined endpoints will be assessed by brain magnetic resonance imaging (MRI), neuro(psycho)logical tests and a subcutaneously implanted reveal recorder for AF detection. According to statistics 108 patients will be enrolled.</p> <p>Discussion</p> <p>Findings from the MACPAF trial will help to balance the benefits and risks of LACA for symptomatic paroxysmal AF. Using serial brain MRIs might help to identify patients at risk for LACA-associated cerebral thromboembolism. Potential limitations of the study are the single-center design, the existence of a variety of LACA-catheters, the missing placebo-group and the impossibility to assess the primary endpoint in a blinded fashion.</p> <p>Trial registration</p> <p>clinicaltrials.gov NCT01061931</p

Temporal trends in hospitalisation for stroke recurrence following incident hospitalisation for stroke in Scotland

&lt;p&gt;Background: There are few studies that have investigated temporal trends in risk of recurrent stroke. The aim of this study was to examine temporal trends in hospitalisation for stroke recurrence following incident hospitalisation for stroke in Scotland during 1986 to 2001.&lt;/p&gt; &lt;p&gt;Methods: Unadjusted survival analysis of time to first event, hospitalisation for recurrent stroke or death, was undertaken using the cumulative incidence method which takes into account competing risks. Regression on cumulative incidence functions was used to model the temporal trends of first recurrent stroke with adjustment for age, sex, socioeconomic status and comorbidity. Complete five year follow-up was obtained for all patients. Restricted cubic splines were used to determine the best fitting relationship between the survival events and study year.&lt;/p&gt; &lt;p&gt;Results: There were 128,511 incident hospitalisations for stroke in Scotland between 1986 and 2001, 57,351 (45%) in men. A total of 13,835 (10.8%) patients had a recurrent hospitalisation for stroke within five years of their incident hospitalisation. Another 74,220 (57.8%) patients died within five years of their incident hospitalisation without first having a recurrent hospitalisation for stroke. Comparing incident stroke hospitalisations in 2001 with 1986, the adjusted risk of recurrent stroke hospitalisation decreased by 27%, HR = 0.73 95% CI (0.67 to 0.78), and the adjusted risk of death being the first event decreased by 28%, HR = 0.72 (0.70 to 0.75).&lt;/p&gt; &lt;p&gt;Conclusions: Over the 15-year period approximately 1 in 10 patients with an incident hospitalisation for stroke in Scotland went on to have a hospitalisation for recurrent stroke within five years. Approximately 6 in 10 patients died within five years without first having a recurrent stroke hospitalisation. Using hospitalisation and death data from an entire country over a 20-year period we have been able to demonstrate not only an improvement in survival following an incident stroke, but also a reduction in the risk of a recurrent event.&lt;/p&gt

Exploring the perspectives and preferences for HTA across German healthcare stakeholders using a multi-criteria assessment of a pulmonary heart sensor as a case study

Background Health technology assessment and healthcare decision-making are based on multiple criteria and evidence, and heterogeneous opinions of participating stakeholders. Multi-criteria decision analysis (MCDA) offers a potential framework to systematize this process and take different perspectives into account. The objectives of this study were to explore perspectives and preferences across German stakeholders when appraising healthcare interventions, using multi-criteria assessment of a heart pulmonary sensor as a case study. Methods An online survey of 100 German healthcare stakeholders was conducted using a comprehensive MCDA framework (EVIDEM V2.2). Participants were asked to provide i) relative weights for each criterion of the framework; ii) performance scores for a health pulmonary sensor, based on available data synthesized for each criterion; and iii) qualitative feedback on the consideration of contextual criteria. Normalized weights and scores were combined using a linear model to calculate a value estimate across different stakeholders. Differences across types of stakeholders were explored. Results The survey was completed by 54 participants. The most important criteria were efficacy, patient reported outcomes, disease severity, safety, and quality of evidence (relative weight >0.075 each). Compared to all participants, policymakers gave more weight to budget impact and quality of evidence. The quantitative appraisal of a pulmonary heart sensor revealed differences in scoring performance of this intervention at the criteria level between stakeholder groups. The highest value estimate of the sensor reached 0.68 (on a scale of 0 to 1, 1 representing maximum value) for industry representatives and the lowest value of 0.40 was reported for policymakers, compared to 0.48 for all participants. Participants indicated that most qualitative criteria should be considered and their impact on the quantitative appraisal was captured transparently. Conclusions The study identified important variations in perspectives across German stakeholders when appraising a healthcare intervention and revealed that MCDA can demonstrate the value of a specified technology for all participating stakeholders. Better understanding of these differences at the criteria level, in particular between policymakers and industry representatives, is important to focus innovation aligned with patient health and healthcare system values and constraints