Cost-of-illness studies in rare diseases: a scoping review.
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ABSTRACT: Objective
The aim of this scoping review was to overview the cost-of-illness studies conducted in rare diseases.Methods
We searched papers published in English in PubMed from January 2007 to December 2018. We selected cost-of-illness studies on rare diseases defined as those with prevalence lower than 5 per 10,000 cases. Studies were selected by one researcher and verified by a second researcher. Methodological characteristics were extracted to develop a narrative synthesis.Results
We included 63 cost-of-illness studies on 42 rare diseases conducted in 25 countries, and 9 systematic reviews. Most studies (94%) adopted a prevalence-based estimation, where the predominant design was cross-sectional with a bottom-up approach. Only four studies adopted an incidence-based estimation. Most studies used questionnaires to patients or caregivers to collect resource utilisation data (67%) although an important number of studies used databases or registries as a source of data (48%). Costs of lost productivity, non-medical costs and informal care costs were included in 68%, 60% and 43% of studies, respectively.Conclusion
This review found a paucity of cost-of-illness studies in rare diseases. However, the analysis shows that the cost-of-illness studies of rare diseases are feasible, although the main issue is the lack of primary and/or aggregated data that often prevents a reliable estimation of the economic burden.
SUBMITTER: Garcia-Perez L
PROVIDER: S-EPMC8045199 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BACKGROUND:Rare Diseases (RDs), which are defined as diseases affecting no more than 5 out of 10,000 people, are often severe, chronic and life-threatening. A main problem is the delay in diagnosing RDs. Clinical decision support systems (CDSSs) for RDs are software systems to support clinicians in the diagnosis of patients with RDs. Due to their clinical importance, we conducted a scoping review to determine which CDSSs are available to support the diagnosis of RDs patients, whether the CDSSs are available to be used by clinicians and which functionalities and data are used to provide decision support. METHODS:We searched PubMed for CDSSs in RDs published between December 16, 2008 and December 16, 2018. Only English articles, original peer reviewed journals and conference papers describing a clinical prototype or a routine use of CDSSs were included. For data charting, we used the data items "Objective and background of the publication/project", "System or project name", "Functionality", "Type of clinical data", "Rare Diseases covered", "Development status", "System availability", "Data entry and integration", "Last software update" and "Clinical usage". RESULTS:The search identified 636 articles. After title and abstracting screening, as well as assessing the eligibility criteria for full-text screening, 22 articles describing 19 different CDSSs were identified. Three types of CDSSs were classified: "Analysis or comparison of genetic and phenotypic data," "machine learning" and "information retrieval". Twelve of nineteen CDSSs use phenotypic and genetic data, followed by clinical data, literature databases and patient questionnaires. Fourteen of nineteen CDSSs are fully developed systems and therefore publicly available. Data can be entered or uploaded manually in six CDSSs, whereas for four CDSSs no information for data integration was available. Only seven CDSSs allow further ways of data integration. thirteen CDSS do not provide information about clinical usage. CONCLUSIONS:Different CDSS for various purposes are available, yet clinicians have to determine which is best for their patient. To allow a more precise usage, future research has to focus on CDSSs RDs data integration, clinical usage and updating clinical knowledge. It remains interesting which of the CDSSs will be used and maintained in the future.
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Project description:BACKGROUND:Heart failure is a major and growing medical and economic problem worldwide as 1-2% of the healthcare budget are spent for heart failure. The prevalence of heart failure has increased over the past decades and it is expected that there will be further raise due to the higher proportion of elderly in the western societies. In this context cost-of-illness studies can significantly contribute to a better understanding of the drivers and problems which lead to the increasing costs in heart failure. The aim of this study was to perform a systematic review of published cost-of-illness studies related to heart failure to highlight the increasing cost impact of heart failure. METHODS:A systematic review was conducted from 2004 to 2016 to identify cost-of-illness studies related to heart failure, searching PubMed (Medline), Cochrane, Science Direct (Embase), Scopus and CRD York Database. RESULTS:Of the total of 16 studies identified, 11 studies reported prevalence-based estimates, 2 studies focused on incidence-based data and 3 articles presented both types of cost data. A large variation concerning cost components and estimates can be noted. Only three studies estimated indirect costs. Most of the included studies have shown that the costs for hospital admission are the most expensive cost element. Estimates for annual prevalence-based costs for heart failure patients range from $868 for South Korea to $25,532 for Germany. The lifetime costs for heart failure patients have been estimated to $126.819 per patient. CONCLUSIONS:Our review highlights the considerable and growing economic burden of heart failure on the health care systems. The cost-of-illness studies included in this review show large variations in methodology used and the cost results vary consequently. High quality data from cost-of-illness studies with a robust methodology applied can inform policy makers about the major cost drivers of heart failure and can be used as the basis of further economic evaluations.
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Project description:AIM:To estimate the economic burden of common health problems associated with pregnancy and childbirth, such as incontinence, mental health problems, or gestational diabetes, excluding acute complications of labour or birth, or severe acute adverse maternal outcomes. METHODS:Searches for relevant studies were carried out to November 2019 in Medline, Embase, CINAHL, PsycINFO and EconLit databases. After initial screening, all results were reviewed for inclusion by two authors. An adapted version of a previously developed checklist for cost-of-illness studies was used for quality appraisal. All costs were converted to 2018 Euro using national consumer price indices and purchasing power parity conversion factors. RESULTS:Thirty-eight relevant studies were identified, some of which reported incremental costs for more than one health problem (16 gestational diabetes, 13 overweight/obesity, 8 mental health, 4 hypertensive disorders, 2 nausea and vomiting, 2 epilepsy, 1 intimate partner violence). A high level of heterogeneity was observed in both the methods used, and the incremental cost estimates obtained for each morbidity. Average incremental costs tended to be higher in studies that modelled a hypothetical cohort of women using data from a range of sources (compared to analyses of primary data), and in studies set in the United States. No studies that examined the economic burden of some common pregnancy-related morbidities, such as incontinence, pelvic girdle pain, or sexual health problems, were identified. CONCLUSION:Our findings indicate that maternal morbidity is associated with significant costs to health systems and society, but large gaps remain in the evidence base for the economic burden of some common health problems associated with pregnancy and childbirth. More research is needed to examine the economic burden of a range of common maternal health problems, and future research should adopt consistent methodological approaches to ensure comparability of results.
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Project description:PurposeTo explore the extent to which musculoskeletal (MSK) complications have been reported following critical illness, identifying evidence gaps and providing recommendations for future research.Materials and methodsWe searched five databases from January 1st 2000 to March 31st 2021. We included published original research reporting MSK complications in patients discharged from hospital following an admission to an intensive care unit (ICU). Two reviewers independently screened English language articles for eligibility. Data extracted included the MSK area of investigation and MSK outcome measures. The overall quality of study was evaluated against standardised reporting guidelines.Results4512 titles were screened, and 32 met the inclusion criteria. Only one study included was interventional, with the majority being prospective cohort studies (n = 22). MSK complications identified included: muscle weakness or atrophy, chronic pain, neuromuscular dysfunction, peripheral joint impairment and fracture risk. The quality of the overall reporting in the studies was deemed adequate.ConclusionsWe identified a heterogenous body of literature reporting a high prevalence of a variety of MSK complications extending beyond muscle weakness, therefore future investigation should include evaluations of more than one MSK area. Further investigation of MSK complications could inform the development of future post critical illness rehabilitation programs.
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Project description:BackgroundThe term ?reproductive, maternal, newborn, and child health (RMNCH)? describes an integrated continuum of health states which is central to Millennium Development Goals 4 and 5. While the burden of mortality and morbidity associated with RMNCH is well known, knowledge is still limited about the economic burden of RMNCH. Concrete evidence of cost of illness (COI) of RMNCH may help policy makers in supporting investment in RMNCH.MethodsA systematic literature search of COI studies was performed in electronic databases. The time frame for the analysis was January 1990 ? April 2011. The databases checked were Medline (Pubmed), Embase and ECONbase, EconLit, the Cumulative Index to Nursing and Allied Health (CINAHL), the National Bureau of Economic Research, the Latin American and Caribbean Literature on Health Sciences Database (LILACS), and Popline. Furthermore, we searched working papers and reference lists of selected articles.ResultsAll the studies investigated address particular complications and issues of RMNCH, e.g., preterm birth, non-exclusive breastfeeding, and sexually transmitted diseases (STDs), but not RMNCH as an entire continuum. Most of the studies were conducted in high income countries, with limited data on low and middle income countries. The burden of disease is very high even for single complications. For example, the disease burden related to non-exclusive breastfeeding was given as 14.39 billion international dollars (ID) (2012, purchasing power parity) per year in the USA. Methodological differences in study design, costing approach, perspective of analysis, and time frame make it difficult to compare different studies.ConclusionThe continuum of RMNCH covers a large portion of the lifespan from birth through the reproductive age. From a methodological perspective, an ideal COI study would clearly describe the perspective of analysis and, hence, the cost items (direct or indirect), cost collection procedure, discounting, quality of data, time frame of analysis, related comorbidities, and robust sensitivity analysis for all the assumptions. Further research is needed to measure the economic impact of RMNCH, including identification of the most cost-effective policy and interventions for prevention, reduction, and elimination of the complications of RMNCH.
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