Dataset Information

A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma.

ABSTRACT: Drug regulators around the world make decisions about drug approvability based on qualitative benefit-risk analysis. In this work, a quantitative benefit-risk analysis approach captures regulatory decision-making about new drugs to treat multiple myeloma (MM). MM assessments have been based on endpoints such as time to progression (TTP), progression-free survival (PFS), and objective response rate (ORR) which are different than benefit-risk analysis based on overall survival (OS). Twenty-three FDA decisions on MM drugs submitted to FDA between 2003 and 2016 were identified and analyzed. The benefits and risks were quantified relative to comparators (typically the control arm of the clinical trial) to estimate whether the median benefit-risk was positive or negative. A sensitivity analysis was demonstrated using ixazomib to explore the magnitude of uncertainty. FDA approval decision outcomes were consistent and logical using this benefit-risk framework.

SUBMITTER: Raju GK

PROVIDER: S-EPMC7418461 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature

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A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma.

Raju G K GK Gurumurthi Karthik K Domike Reuben R Kazandjian Dickran D Landgren Ola O Blumenthal Gideon M GM Farrell Ann A Pazdur Richard R Woodcock Janet J

Clinical pharmacology and therapeutics 20171120 1

Drug regulators around the world make decisions about drug approvability based on qualitative benefit-risk analysis. In this work, a quantitative benefit-risk analysis approach captures regulatory decision-making about new drugs to treat multiple myeloma (MM). MM assessments have been based on endpoints such as time to progression (TTP), progression-free survival (PFS), and objective response rate (ORR) which are different than benefit-risk analysis based on overall survival (OS). Twenty-three F ...[more]

PMID: 28901535

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Project description:The selection and assessment process of appropriate robots became a more complex and complicated task due to various available alternatives and conflicting attributes which must take into consideration. Also, uncertainty which exists usually in the selection process is an unavoidable component that needs to be thoughtfully measured and traditional multi-attribute decision-making approaches failed to deal precisely with it. Since almost all decisions originate from subjective ordinal preferences, handling uncertainty using linguistic variables is also not enough. Thus, the objective of the current study is to present a new extended ordinal priority approach in the neutrosophic environment for the first time to select an appropriate robot. Since neutrosophic is one of the most effective and accommodating tools for handling uncertainty, thus, this method goes to transform linguistic information into triangular neutrosophic numbers using a new presented scale. This scale was used to determine the importance degree of attributes and alternatives regarding experts' opinions. Also, the score function of the triangular neutrosophic number is used for prioritizing attributes and alternatives. The experts in our proposed method have the same degree of importance, since each expert is a person with special skills and knowledge representing mastery of a particular subject. To measure the applicability and efficiency of the proposed approach, an experimental case study has been established for the robot selection problem of a new pharmaceutical city in Egypt for the first time. The source of data in this case study is experts, interviews, and questionnaires. Also, sensitivity and comparative analysis are further made for verifying the power of the proposed approach. The outcome of this study shows that the suggested approach for robot selection is quite helpful and has a great performance under uncertainty over classical and fuzzy ordinal priority approaches. Also, the suggested approach is less consumption of time and simpler than the fuzzy ordinal priority approach. Therefore, we recommend firms and governments to apply it for increasing product quality, hence the profitability of manufacturing industries and decrease needless costs.Supplementary informationThe online version contains supplementary material available at 10.1007/s40747-022-00721-w.

Project description:Value-based decision-making involves trading off the cost associated with an action against its expected reward. Research has shown that both physical and mental effort constitute such subjective costs, biasing choices away from effortful actions, and discounting the value of obtained rewards. Facing conflicts between competing action alternatives is considered aversive, as recruiting cognitive control to overcome conflict is effortful. Moreover, engaging control to proactively suppress irrelevant information that could conflict with task-relevant information would presumably also be cognitively costly. Yet, it remains unclear whether the cognitive control demands involved in preventing and resolving conflict also constitute costs in value-based decisions. The present study investigated this question by embedding irrelevant distractors (flanker arrows) within a reversal-learning task, with intermixed free and instructed trials. Results showed that participants learned to adapt their free choices to maximize rewards, but were nevertheless biased to follow the suggestions of irrelevant distractors. Thus, the perceived cost of investing cognitive control to suppress an external suggestion could sometimes trump internal value representations. By adapting computational models of reinforcement learning, we assessed the influence of conflict at both the decision and learning stages. Modelling the decision showed that free choices were more biased when participants were less sure about which action was more rewarding. This supports the hypothesis that the costs linked to conflict management were traded off against expected rewards. During the learning phase, we found that learning rates were reduced in instructed, relative to free, choices. Learning rates were further reduced by conflict between an instruction and subjective action values, whereas learning was not robustly influenced by conflict between one's actions and external distractors. Our results show that the subjective cognitive control costs linked to conflict factor into value-based decision-making, and highlight that different types of conflict may have different effects on learning about action outcomes.

Dataset Information

A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma.

Publications

A Benefit-Risk Analysis Approach to Capture Regulatory Decision-Making: Multiple Myeloma.

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