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A Review of the Challenges of Using Biomedical Big Data for Economic Evaluations of Precision Medicine.

ABSTRACT: There is potential value in incorporating biomedical big data (BBD)-observational real-world patient-level genomic and clinical data in multiple sub-populations-into economic evaluations of precision medicine. However, health economists face practical and methodological challenges when using BBD in this context. We conducted a literature review to identify and summarise these challenges. Relevant articles were identified in MEDLINE, EMBASE, EconLit, University of York Centre for Reviews and Dissemination and Cochrane Library from 2000 to 2018. Articles were included if they studied issues relevant to the interconnectedness of biomedical big data, precision medicine, and health economic evaluation. Nineteen articles were included in the review. Challenges identified related to data management, data quality and data analysis. The availability of large volumes of data from multiple sources, the need to conduct data linkages within an environment of opaque data access and sharing procedures, and other data management challenges are primarily practical and may not be long-term obstacles if procedures for data sharing and access are improved. However, the existence of missing data across linked datasets, the need to accommodate dynamic data, and other data quality and analysis challenges may require an evolution in economic evaluation methods. Health economists face challenges when using BBD in economic evaluations of technologies that facilitate precision medicine. Potential solutions to some of these challenges do, however, exist. Going forward, health economists who present work that uses BBD should document challenges and the solutions they have applied to the challenges to support future researcher endeavours.

SUBMITTER: Fahr P

PROVIDER: S-EPMC6647451 | biostudies-literature | 2019 Aug

REPOSITORIES: biostudies-literature

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Project description:Background and objectivePrecision (stratified or personalised) medicine is underpinned by the premise that it is feasible to identify known heterogeneity using a specific test or algorithm in patient populations and to use this information to guide patient care to improve health and well-being. This study aimed to understand if, and how, previous economic evaluations of precision medicine had taken account of the impact of capacity constraints.MethodsA meta-review was conducted of published systematic reviews of economic evaluations of precision medicine (test-treat interventions) and individual studies included in these reviews. Due to the volume of studies identified, a sample of papers published from 2007 to 2015 was collated. A narrative analysis identified whether potential capacity constraints were discussed qualitatively in the studies and, if relevant, which quantitative methods were used to account for capacity constraints.ResultsA total of 45 systematic reviews of economic evaluations of precision medicine were identified, from which 222 studies focusing on test-treat interventions, published between 2007 and 2015, were extracted. Of these studies, 33 (15%) qualitatively discussed the potential impact of capacity constraints, including budget constraints; quality of tests and the testing process; ease of use of tests in clinical practice; and decision uncertainty. Quantitative methods (nine studies) to account for capacity constraints included static methods such as capturing inefficiencies in trials or models and sensitivity analysis around model parameters; and dynamic methods, which allow the impact of capacity constraints on cost effectiveness to change over time.ConclusionsUnderstanding the cost effectiveness of precision medicine is necessary, but not sufficient, evidence for its successful implementation. There are currently few examples of evaluations that have quantified the impact of capacity constraints, which suggests an area of focus for future research.

Project description:BackgroundExamples of precision medicine are complex interventions featuring both testing and treatment components. Because of this complexity, there are often barriers to the introduction of such interventions. Few economic evaluations attempt to determine the impact of these barriers on the cost-effectiveness of the intervention. This study presents a case study economic evaluation that illustrates how the value of implementation methods may be used to quantify the impact of capacity constraints in a decision-analytic model.MethodsA baseline decision-analytic model-based economic evaluation of ALK mutation testing was reproduced from a published technology appraisal. Three constraints (commissioning awareness, localization of testing, and pathology laboratory capacity) were identified using qualitative interviews, parameterized, and incorporated into the model. Value of implementation methods were used alongside incremental cost-effectiveness ratios (ICERs) to quantify the impact on the cost-effectiveness and net monetary benefit (NMB) of each capacity constraint and from the 3 constraints combined.ResultsEach of the 3 capacity constraints resulted in a loss of NMB ranging from £7773 (0.1% of the total) per year for localized testing to £4,907,893 (77%) for a lack of awareness about commissioning ALK testing. When combined, the constraints resulted in a loss of NMB of £5,289,414 (83%). The localization and limited pathology capacity constraints slightly increased the ICER, but the lack of commissioning awareness constraint did not change the ICER.ConclusionsCapacity constraints may have a significant impact on the NMB produced by examples of precision medicine. Value of implementation methods can be used to quantify the impact of such constraints by combining the impact of the constraints on the cost-effectiveness of the intervention with the impact on the number of patients receiving the intervention.HighlightsWhile capacity constraints may prevent the use of precision medicine in clinical practice, economic evaluations rarely account for the impact of such barriers.This study demonstrates how constraints can be identified using qualitative methods and subsequently incorporated into decision-analytic models using quantitative value of implementation methods.In addition, this article demonstrates how value of implementation methods can be used to account for the impact of capacity constraints on the costs and benefits of an intervention as well as the number of patients receiving the intervention.In the case study presented herein, a capacity constraint reducing patient access to an example of precision medicine caused the biggest loss of net monetary benefit.Health economists should consider moving beyond incremental cost-effectiveness ratios to measures of total net monetary benefit to fully capture the impact of implementing precision medicine.

Project description:Insight into current scientific applications of Big Data in the precision dairy farming area may help us to understand the inflated expectations around Big Data. The objective of this invited review paper is to give that scientific background and determine whether Big Data has overcome the peak of inflated expectations. A conceptual model was created, and a literature search in Scopus resulted in 1442 scientific peer reviewed papers. After thorough screening on relevance and classification by the authors, 142 papers remained for further analysis. The area of precision dairy farming (with classes in the primary chain (dairy farm, feed, breed, health, food, retail, consumer) and levels for object of interest (animal, farm, network)), the Big Data-V area (with categories on Volume, Velocity, Variety and other V's) and the data analytics area (with categories in analysis methods (supervised learning, unsupervised learning, semi-supervised classification, reinforcement learning) and data characteristics (time-series, streaming, sequence, graph, spatial, multimedia)) were analysed. The animal sublevel, with 83% of the papers, exceeds the farm sublevel and network sublevel. Within the animal sublevel, topics within the dairy farm level prevailed with 58% over the health level (33%). Within the Big Data category, the Volume category was most favoured with 59% of the papers, followed by 37% of papers that included the Variety category. None of the papers included the Velocity category. Supervised learning, representing 87% of the papers, exceeds unsupervised learning (12%). Within supervised learning, 64% of the papers dealt with classification issues and exceeds the regression methods (36%). Time-series were used in 61% of the papers and were mostly dealing with animal-based farm data. Multimedia data appeared in a greater number of recent papers. Based on these results, it can be concluded that Big Data is a relevant topic of research within the precision dairy farming area, but that the full potential of Big Data in this precision dairy farming area is not utilised yet. However, the present authors expect the full potential of Big Data, within the precision dairy farming area, will be reached when multiple Big Data characteristics (Volume, Variety and other V's) and sources (animal, groups, farms and chain parts) are used simultaneously, adding value to operational and strategic decision.

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A Review of the Challenges of Using Biomedical Big Data for Economic Evaluations of Precision Medicine.

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