72 research outputs found
Establishing cost-effectiveness of genetic targeting of cancer therapies
The clinical benefit of a new genomic instrument, the 70-gene signature for breast cancer patients, is being evaluated in a randomised clinical trial. The early, controlled implementation process is supported by a Constructive Technology Assessment to help decision-making in an uncertain time of development
Establishing cost-effectiveness of genetic targeting of cancer therapies\ud
The clinical benefit of a new genomic instrument, the 70-gene signature\ud
for breast cancer patients, is being evaluated in a randomised clinical\ud
trial. The early, controlled implementation process is supported by a\ud
Constructive Technology Assessment to help decision-making in an\ud
uncertain time of developmen
When to wait for more evidence?: real options analysis in proton therapy.
Purpose. Trends suggest that cancer spending growth will accelerate. One method for controlling costs is to examine whether the benefits of new technologies are worth the extra costs. However, especially new and emerging technologies are often more costly, while limited clinical evidence of superiority is available. In that situation it is often unclear whether to adopt the new technology now, with the risk of investing in a suboptimal therapy, or to wait for more evidence, with the risk of withholding patients their optimal treatment. This trade-off is especially difficult when it is costly to reverse the decision to adopt a technology, as is the case for proton therapy. Real options analysis, a technique originating from financial economics, assists in making this trade-off. Methods. We examined whether to adopt proton therapy, as compared to stereotactic body radiotherapy, in the treatment of inoperable stage I non-small cell lung cancer. Three options are available: adopt without further research; adopt and undertake a trial; or delay adoption and undertake a trial. The decision depends on the expected net gain of each option, calculated by subtracting its total costs from its expected benefits. Results. In The Netherlands, adopt and trial was found to be the preferred option, with an optimal sample size of 200 patients. Increase of treatment costs abroad and costs of reversal altered the preferred option. Conclusion. We have shown that real options analysis provides a transparent method of weighing the costs and benefits of adopting and/or further researching new and expensive technologies. The Oncologist 2011;16:1752-176
KRAS mutation testing of tumours in adults with metastatic colorectal cancer: a systematic review and cost-effectiveness analysis
__Abstract__
Background: Bowel cancer is the third most common cancer in the UK. Most bowel cancers are initially
treated with surgery, but around 17% spread to the liver. When this happens, sometimes the liver tumour
can be treated surgically, or chemotherapy may be used to shrink the tumour to make surgery possible.
Kirsten rat sarcoma viral oncogene (KRAS) mutations make some tumours less responsive to treatment
with biological therapies such as cetuximab. There are a variety of tests available to detect these
mutations. These vary in the specific mutations that they detect, the amount of mutation they detect,
the amount of tumour cells needed, the time to give a result, the error rate and cost.
Objectives: To compare the performance and cost-effectiveness of KRAS mutation tests in differentiating
adults with metastatic colorectal cancer whose metastases are confined to the liver and are unresectable
and who may benefit from first-line treatment with cetuximab in combination with standard chemotherapy
from those who should receive standard chemotherapy alone
Development and Validation of the TRansparent Uncertainty ASsessmenT (TRUST) Tool for Assessing Uncertainties in Health Economic Decision Models.
Background An increasing number of technologies are obtaining marketing authorisation based on sparse evidence, which
causes growing uncertainty and risk within health technology reimbursement decision making. To ensure that uncertainty is
considered and addressed within health technology assessment (HTA) recommendations, uncertainties need to be identifed,
included in health economic models, and reported.
Objective Our objective was to develop the TRansparent Uncertainty ASsessmenT (TRUST) tool for systematically identifying, assessing, and reporting uncertainties in decision models, with the aim of making uncertainties and their impact on
cost efectiveness more explicit and transparent.
Methods TRUST was developed by drawing on the uncertainty and risk assessment literature. To develop and validate this
tool, we conducted HTA stakeholder discussion meetings and interviews and applied it in six real-world HTA case studies
in the Netherlands and the UK.
Results The TRUST tool enables the identifcation and categorisation of uncertainty according to its source (transparency
issues, methodology issues, and issues with evidence: imprecision, bias and indirectness, and unavailability) in each model
aspect. The source of uncertainty determines the appropriate analysis. The impact of uncertainties on cost efectiveness is
also assessed. Stakeholders found using the tool to be feasible and of value for transparent uncertainty assessment. TRUST
can be used during model development and/or model review.
Conclusion The TRUST tool enables systematic identifcation, assessment, and reporting of uncertainties in health economic
models and may contribute to more informed and transparent decision making in the face of uncertainty
Immunocap® ISAC and microtest for multiplex allergen testing in people with difficult to manage allergic disease: A systematic review and cost analysis
__Background__ Allergy is a form of immune-mediated exaggerated sensitivity (hypersensitivity) to a substance that is either inhaled, swallowed, injected or comes into contact with the skin. Foreign substances that provoke allergies are called allergens. It has been claimed that multiplex allergen testing may help in diagnosing the cause of symptoms in patients with an unclear cause of allergy or who are allergic to more than one substance.
__Objectives__ To evaluate multiplex allergen testing [devices that can measure the presence of multiple immunoglobulin E (IgE) antibodies in a patient’s blood at the same time], by assessing
(1) clinical effectiveness (allergy symptoms, incidence of acute exacerbations, mortality, adverse events of testing and treatment, health-care presentations or admissions, health-related quality of life);
(2) effects on treatment (diet, immunotherapy medications, other potential testing);
(3) any additional diagnostic information provided by multiplex allergen testing; and
(4) cost-effectiveness (cost of different assessment strategies).
__Methods__ Fifteen databases were searched from 2005 to April 2015, including MEDLINE (via OvidSp), MEDLINE In-Process Citations, MEDLINE Daily Update, PubMed (National Library of Medicine), EMBASE, Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), Database of Abstracts of Reviews of Effects (DARE), Health Technology Assessment (HTA) database, Science Citation Index (SCI), Conference Proceedings Citation Index-Science (CPCI-S), BIOSIS Previews, Latin American and Caribbean Health Sciences Literature (LILACS), National Institute for Health Research (NIHR) HTA programme, and the US Food and Drug Administration (FDA); supplementary searches of conference proceedings and trials registries were performed. Review methods followed published guidance from the Cochrane Collaboration and the Centre for Reviews and Dissemination, University of York, UK. The methodological quality of included studies was assessed using appropriate published tools or a review-specific tool designed by the project team. Studies were summarised in a narrative synthesis. Owing to a lack of data on the clinical effectiveness of multiplex allergen testing, no long-term cost-effectiveness model was developed. A conceptual model structure was developed and cost analyses were performed to examine the short-term costs of various possible diagnostic pathways.
__Results__ Fifteen studies were included in the review. The very limited available data indicated that the addition of multiplex allergen testing [ImmunoCAP® Immuno Solid-phase Allergen Chip (ISAC), Thermo Fisher Scientific/Phadi
Integrating evidence on patient preferences in healthcare policy decisions
Background: Despite a strong movement towards active patient involvement in healthcare policy decisions,
systematic and explicit consideration of evidence of this research on patient preferences seems limited.
Furthermore, little is known about the opinions of several stakeholders towards consideration of research evidence
on patient preferences in healthcare policy decisions. This paper describes the protocol for an explorative study on
the integration of research on patient preferences in healthcare policy decisions. The study questions: to what
extent research evidence on patient preferences is considered in current procedures for healthcare policy decisions;
opinions of stakeholders regarding the integration of this type of evidence in healthcare policy decisions; and what
could be a decision framework for the integration of such research evidence in healthcare policy decisions.
Methods/design: The study is divided in three sub-studies, predominantly using qualitative methods. The first
sub-study is a scoping review in five European countries to investigate whether and how results of research on patient
preferences are considered in current procedures for coverage decisions and clinical practice guideline development.
The second sub-study is a qualitative study to explore the opinions of stakeholders with regard to the possibilities for
integrating evidence on patient preferences in the process of healthcare decision-making in the Netherlands. The third
sub-study is the development of a decision framework for research on patient preferences. The framework will consist
of: a process description regarding the place of evidence on patient preferences in the decision-making process; and a
taxonomy describing different terminologies and conceptualisations of ‘preferences’ and an overview of existing
methodologies for investigating preferences. The concept framework will be presented to and discussed with experts.
Discussion: This study will create awareness regarding the existence and potential value of research evidence on patient
preferences for healthcare policy decision-making and provides insight in the methods for investigating patient preferences
and the barriers and facilitators for integration of such research in healthcare policy decisions. Results of the study will be
useful for researchers, clinical practice guideline developers, healthcare policy makers, and patient representatives
Research Costs Investigated: A Study Into the Budgets of Dutch Publicly Funded Drug-Related Research
Background: The costs of performing research are an important input in value of information (VOI) analyses but are difficult to assess. Objective: The aim of this study was to investigate the costs of research, serving two purposes: (1) estimating research costs for use in VOI analyses; and (2) developing a costing tool to support reviewers of grant proposals in assessing whether the proposed budget is realistic. Methods: For granted study proposals from the Netherlands Organization for Health Research and Development (ZonMw), type of study, potential cost drivers, proposed budget, and general characteristics were extracted. Regression analysis was conducted in an attempt to generate a ‘predicted budget’ for certain combinations of cost drivers, for implementation in the costing tool. Results: Of 133 drug-related research grant proposals, 74 were included for complete data extraction. Because an association between cost drivers and budgets was not confirmed, we could not generate a predicted budget based on regression analysis, but only historic reference budgets given certain study characteristics. The costing tool was designed accordingly, i.e. with given selection criteria the tool returns the range of budgets in comparable studies. This range can be used in VOI analysis to estimate whether the expected net benefit of sampling will be positive to decide upon the net value of future research. Conclusion: The absence of association between study characteristics and budgets may indicate inconsistencies in the budgeting or granting process. Nonetheless, the tool generates useful information on historical budgets, and the option to formally relate VOI to budgets. To our knowledge, this is the first attempt at creating such a tool, which can be complemented with new studies being granted, enlarging the underlying database and keeping estimates up to date
Optimizing the use of expert panel reference diagnoses in diagnostic studies of multidimensional syndromes
__Abstract__
Background: In the absence of a gold standard, a panel of experts can be invited to assign a reference diagnosis
for use in research. Available literature offers limited guidance on assembling and working with an expert panel
for this purpose. We aimed to develop a protocol for an expert panel consensus diagnosis and evaluated its
applicability in a pilot project.
Methods: An adjusted Delphi method was used, which started with the assessment of clinical vignettes by 3
experts individually, followed by a consensus discussion meeting to solve diagnostic discrepancies. A panel
facilitator ensured that all experts were able to express their views, and encouraged the use of argumentation to
arrive at a specific diagnosis, until consensus was reached by all experts. Eleven vignettes of patients suspected of
having a primary neurodegenerative disease were presented to the experts. Clinical information was provided
stepwise and included medical history, neurological, physical and cognitive function, brain MRI scan, and follow-up
assessments over 2 years. After the consensus discussion meeting, the procedure was evaluated by the experts.
Results: The average degree of consensus for the reference diagnosis increased from 52% after individual
assessment of the vignettes to 94% after the consensus discussion meeting. Average confidence in the diagnosis
after individual assessment was 85%. This did not increase after the consensus discussion meeting. The process
evaluation led to several recommendations for improvement of the protocol.
Conclusion: A protocol for attaining a reference diagnosis based on expert panel consensus was shown feasible in
research practice
High-sensitivity troponin assays for the early rule-out or diagnosis of acute myocardial infarction in people eith acute chest pain: a systematic review and cost-effectiveness analysis
Background: Early diagnosis of acute myocardial infarction (AMI) can ensure quick and effective treatment but only 20% of adults with emergency admissions fo
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