Multiple Criteria Decision Analysis for Health Care Decision Making--Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force.

Health care decisions are complex and involve confronting trade-offs between multiple, often conflicting objectives. Using structured, explicit approaches to decisions involving multiple criteria can improve the quality of decision making. A set of techniques, known under the collective heading, multiple criteria decision analysis (MCDA), are useful for this purpose. In 2014, ISPOR established an Emerging Good Practices Task Force. The task force's first report defined MCDA, provided examples of its use in health care, described the key steps, and provided an overview of the principal methods of MCDA. This second task force report provides emerging good-practice guidance on the implementation of MCDA to support health care decisions. The report includes: a checklist to support the design, implementation and review of an MCDA; guidance to support the implementation of the checklist; the order in which the steps should be implemented; illustrates how to incorporate budget constraints into an MCDA; provides an overview of the skills and resources, including available software, required to implement MCDA; and future research directions.

Multiple Criteria Decision Analysis for Health Care Decision Making--An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force.

Health care decisions are complex and involve confronting trade-offs between multiple, often conflicting, objectives. Using structured, explicit approaches to decisions involving multiple criteria can improve the quality of decision making and a set of techniques, known under the collective heading multiple criteria decision analysis (MCDA), are useful for this purpose. MCDA methods are widely used in other sectors, and recently there has been an increase in health care applications. In 2014, ISPOR established an MCDA Emerging Good Practices Task Force. It was charged with establishing a common definition for MCDA in health care decision making and developing good practice guidelines for conducting MCDA to aid health care decision making. This initial ISPOR MCDA task force report provides an introduction to MCDA - it defines MCDA; provides examples of its use in different kinds of decision making in health care (including benefit risk analysis, health technology assessment, resource allocation, portfolio decision analysis, shared patient clinician decision making and prioritizing patients' access to services); provides an overview of the principal methods of MCDA; and describes the key steps involved. Upon reviewing this report, readers should have a solid overview of MCDA methods and their potential for supporting health care decision making.

The identification of benefit in medical intervention: an overview and suggestions for process.

Benefit and risk assessments are not only important to regulatory authorities but also important to the providers, patients, pharmaceutical industry, and payers. In order for patients and providers to continue to have access to new innovative medicines, which have some level of inherent risk, it is critical to have a systematic and balanced focus on understanding the safety risks and benefits to the patient during drug development, at the time of approval and postmarketing. There has been a significant amount of activity around efforts to improve the ability to assess risks in the postmarketing environment. However, there is no widely accepted, systematic approach or process for the ongoing evaluation of benefit. This article introduces 4 critical components in the process of identifying and assessing benefit with a goal of providing a framework that is transparent, comprehensive, applicable to various perspectives, and simple to communicate and implement. We propose the development of a catalog applied to a particular disease to identify the optimal data sources and methods to address the interests of a given perspective. Two key resources will need to be developed to support the catalog development: (1) a summary of benefit measures and preferences by disease and from various perspectives and (2) an investment in a simple visual communication mechanism with minimal statistical language. As the emphasis is on transparency, relevance, applicability, and communication, this approach to assessing benefit should maximize the impact of these data to all stakeholders and decision makers.

A quantitative approach to benefit-risk assessment of medicines - part 1: the development of a new model using multi-criteria decision analysis.

PURPOSE: One of the most important uses of benefit-risk assessment pertains to approval of new medicines by regulatory authorities and the subsequent review of these products during their life-cycle when new safety and/or efficacy data becomes available. At present, there exist no validated, well-accepted models for benefit-risk assessment that have the appropriate degree of sophistication, and as a consequence no models are widely used by regulatory authorities or industry. The aim of the study was therefore to develop a new model for benefit-risk assessment of medicines using multi-criteria decision analysis (MCDA). METHODS: The MCDA methodology was used for a systematic approach to assess the benefit risk ratio of medicines. The reasons for adopting this approach were (1) taking multiple benefit and risk criteria into account, (2) making a judgement on the evidence and potential uncertainty because of the incompleteness of evidence, and (3) making trade-offs of the benefits against risks. RESULTS: It was demonstrated through a seven-step approach how MCDA is used to construct the model. Ten benefit and ten risk criteria were identified to form a value tree. Then fixed scales were established for all criteria and options on the criteria were scored. Weights were assigned for each criteria using swing-weighting. Finally sensitivity analysis was carried. CONCLUSIONS: This novel approach based on MCDA has the potential for being applied as a new tool for judging and deciding on the benefits and risks, thereby helping regulators and industry in the development and approval of new medicines and their adequate use.

A quantitative approach to benefit-risk assessment of medicines - part 2: the practical application of a new model.

PURPOSE: Decisions leading to drug approval demand careful attention with respect to balancing benefits and risks. The new benefit-risk assessment model provides reassembling of the pieces of decision-making information using computer software to present a coherent overall picture for decision-makers. The aims of the study were to evaluate content validity of the new model and examine its practical application. METHODS: The expert panel (Annex 1) drawn from regulatory agencies in USA and Europe, academia and pharmaceutical industry, were encouraged to frank and open discussions about the way in which important, far-reaching decisions are taken within both companies and regulatory agencies and the potential shortcomings of such processes. The main component of the meeting was an interactive demonstration of the application of the technical model MCDA. The model was discussed using a hypothetical scenario based on safety and efficacy data relating to an atypical antipsychotic agent that was clinically tested against placebo and a comparator compound. RESULTS: The expert panel unanimously agreed that the methodology had great potential and it was recommended that it should be explored further as an adjunct to the decision-making process for both companies and regulatory agencies. An important part of this would be to undertake retrospective testing using actual case studies. CONCLUSIONS: The outcome of the workshop underpins practical utility of the new benefit-risk assessment MCDA model and provides evidence of its content validity. It is hoped that this encourages acceptability by regulatory agencies and the industry for its use in real scenarios.