Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.
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ABSTRACT: Background
A Multi Criteria Decision Analysis (MCDA) technique was adopted to reveal the preferences of the Appraisal Body of the Polish HTA agency towards orphan drugs (OMPs).Results
There were 34 positive and 23 negative HTA recommendations out of 54 distinctive drug-indication pairs. The MCDA matrix consisted of 13 criteria, seven of which made the most impact on the HTA process. Appraisal of clinical evidence, cost of therapy, and safety considerations were the main contributors to the HTA guidance, whilst advancement of technology and manufacturing costs made the least impact.Conclusions
MCDA can be regarded as a valuable tool for revealing decision makers' preferences in the healthcare sector. Given that only roughly half of all criteria included in the MCDA matrix were deemed to make an impact on the HTA process, there is certainly some room for improvement with respect to the adaptation of a new approach towards the value assessment of OMPs in Poland.
SUBMITTER: Kolasa K
PROVIDER: S-EPMC5922020 | biostudies-literature | 2018 Apr
REPOSITORIES: biostudies-literature
Publications
Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.
Kolasa Katarzyna K Zwolinski Krzysztof Miroslaw KM Zah Vladimir V Kaló Zoltán Z Lewandowski Tadeusz T
Orphanet journal of rare diseases 20180427 1
<h4>Background</h4>A Multi Criteria Decision Analysis (MCDA) technique was adopted to reveal the preferences of the Appraisal Body of the Polish HTA agency towards orphan drugs (OMPs).<h4>Results</h4>There were 34 positive and 23 negative HTA recommendations out of 54 distinctive drug-indication pairs. The MCDA matrix consisted of 13 criteria, seven of which made the most impact on the HTA process. Appraisal of clinical evidence, cost of therapy, and safety considerations were the main contribut ...[more]
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Project description:The need for patient engagement has been recognized by regulatory agencies, but there is no consensus about how to operationalize this. One approach is the formal elicitation and use of patient preferences for weighing clinical outcomes. The aim of this study was to demonstrate how patient preferences can be used to weigh clinical outcomes when both preferences and clinical outcomes are uncertain by applying a probabilistic value-based multi-criteria decision analysis (MCDA) method. Probability distributions were used to model random variation and parameter uncertainty in preferences, and parameter uncertainty in clinical outcomes. The posterior value distributions and rank probabilities for each treatment were obtained using Monte-Carlo simulations. The probability of achieving the first rank is the probability that a treatment represents the highest value to patients. We illustrated our methodology for a simplified case on six HIV treatments. Preferences were modeled with normal distributions and clinical outcomes were modeled with beta distributions. The treatment value distributions showed the rank order of treatments according to patients and illustrate the remaining decision uncertainty. This study demonstrated how patient preference data can be used to weigh clinical evidence using MCDA. The model takes into account uncertainty in preferences and clinical outcomes. The model can support decision makers during the aggregation step of the MCDA process and provides a first step toward preference-based personalized medicine, yet requires further testing regarding its appropriate use in real-world settings.
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| S-EPMC8033667 | biostudies-literature
Project description:Summary Objectives To combine cross-sectoral stakeholders’ preferences over interventions for knee osteoarthritis (OA) with guideline recommendations and evidence about interventions, and to investigate if these preferences differ by stakeholder group. Design A survey based on multi-criteria decision analysis was implemented whereby the stakeholders revealed the relative importance, represented as weights, of eight criteria for choosing or recommending knee OA interventions. Using data from an OA clinical guideline, 15 recommended interventions were rated on the criteria and ranked by their total scores, calculated by summing the corresponding weights. Associations between the weights and stakeholder groups were explored using regression analysis. Results Participants comprised 58 consumers with OA, 5 Māori health advocates, 79 healthcare providers, 24 policy-informants and 12 OA-researchers (N = 178; 63% female, [mean age±SD] 54 ± 13 years). Mean weights on the eight criteria, in decreasing order of importance, are: recommendation: 19.0%; quality of evidence: 17.7%; effectiveness: 15.0%; duration of effect: 13.2%; risk of serious harm: 12.8%; risk of mild/moderate side-effects: 9.4%; cost: 6.6%; and accessibility: 6.3%. For first-, second- and third-line OA interventions respectively, all land-based exercise (total score = 71.7%), NSAIDs (topical) (74.2%) and total joint replacement (74.3%) were ranked first. At all care phases, the recommended core interventions of weight management and self-management education ranked between 11th and 15th (48.0%–56.0%). Regression analysis identified only small differences in weights (≤5.7%; p < 0.01) between stakeholder groups. Conclusions Not all recommended core interventions are preferred by cross-sectoral stakeholders, which may represent a barrier to their uptake. Stakeholders’ preferences do not appreciably differ by stakeholder group.
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Project description:IntroductionRare diseases (RDs) are often chronic and progressive life-threatening medical conditions that affect a low percentage of the population compared with other diseases. These conditions can be treated with medications known as orphan drugs (ODs). Unfortunately, there is no universal definition of RDs or ODs. This systematic review (SR) will identify the quantitative and qualitative criteria and the underlying rationale used internationally to define RDs and ODs.Methods and analysisThis protocol follows the conventions for the Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols (2015 guidelines). A SR will be conducted, including a search of the following databases: PubMed, MEDLINE, EMBASE, Scopus, Web of Science, GreyLit and OpenGrey. Eligible publications will be selected based on predetermined inclusion criteria. Extracted data will be analysed using thematic and content analyses of qualitative descriptors, whereas quantitative data will be analysed descriptively and reported in the form of frequencies and percentages.Ethics and disseminationNo ethical approval is required since this SR focuses on the secondary analysis of data retrieved from the scientific literature. The outcomes of this SR will be published as part of a PhD thesis, presented at conferences, and published in peer-reviewed journals.Prospero registration numberCRD42021252701.
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Project description:Background and objectiveMulti-criteria decision analysis (MCDA) is a tool that systematically considers multiple factors relevant to health decision-making. The aim of this study was to use an MCDA to assess the value of dupilumab for severe atopic dermatitis compared with secukinumab for moderate to severe plaque psoriasis in Spain.MethodFollowing the EVIDEM (Evidence and Value: Impact on DEcision Making) methodology, the estimated value of both interventions was obtained by means of an additive linear model that combined the individual weighting (between 1 and 5) of each criterion with the individual scoring of each intervention in each criterion. Dupilumab was evaluated against placebo, while secukinumab was evaluated against placebo, etanercept and ustekinumab. A retest was performed to assess the reproducibility of weights, scores and value estimates.ResultsThe overall MCDA value estimate for dupilumab versus placebo was 0.51 ± 0.14. This value was higher than those obtained for secukinumab: 0.48 ± 0.15 versus placebo, 0.45 ± 0.15 versus etanercept and 0.39 ± 0.18 versus ustekinumab. The highest-value contribution was reported by the patients' group, followed by the clinical professionals and the decision makers. A fundamental element that explained the difference in the scoring between pathologies was the availability of therapeutic alternatives. The retest confirmed the consistency and replicability of the analysis.ConclusionsUnder this methodology, and assuming similar economic costs per patient for both treatments, the results indicated that the overall value estimated of dupilumab for severe atopic dermatitis was similar to, or slightly higher than, that of secukinumab for moderate to severe plaque psoriasis.
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Project description:BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or 'opportunity cost'-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.
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