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

Background The use of Multi-Criteria Decision Analysis (MCDA) in health care has become common. However, the literature lacks systematic review trend analysis on the application of MCDA in health care. Aim To systematically identify applications of MCDA to the areas of health care, and to report on publication trends.

https://www.onlinelibrary.wiley.com/doi/pdf/10.1111/hex.12287

Application of multicriteria decision analysis in health care: a systematic review and bibliometric analysis.

Healthcare decision making is usually characterized by a low degree of transparency. The demand for transparent decision processes can be fulfilled only when assessment, appraisal and decisions about health technologies are performed under a systematic construct of benefit assessment. The benefit of an intervention is often multidimensional and, thus, must be represented by several decision criteria. Complex decision problems require an assessment and appraisal of various criteria; therefore, a decision process that systematically identifies the best available alternative and enables an optimal and transparent decision is needed. For that reason, decision criteria must be weighted and goal achievement must be scored for all alternatives. Methods of multi-criteria decision analysis (MCDA) are available to analyse and appraise multiple clinical endpoints and structure complex decision problems in healthcare decision making. By means of MCDA, value judgments, priorities and preferences of patients, insurees and experts can be integrated systematically and transparently into the decision-making process. This article describes the MCDA framework and identifies potential areas where MCDA can be of use (e.g. approval, guidelines and reimbursement/pricing of health technologies). A literature search was performed to identify current research in healthcare. The results showed that healthcare decision making is addressing the problem of multiple decision criteria and is focusing on the future development and use of techniques to weight and score different decision criteria. This article emphasizes the use and future benefit of MCDA.

Making Good Decisions in Healthcare with Multi-Criteria Decision Analysis: The Use, Current Research and Future Development of MCDA.

The 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. The 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. A 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. The 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. Our 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.

/pdf/value-based-reimbursement-decisions-for-orphan-drugs-a-r4857kkgi0.pdf

Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.

Current orphan drug policies are unsatisfactory when viewed from almost all perspectives. Patients find that, although therapies are available for many rare conditions, access to care is sometimes restricted. Pharmaceutical manufacturers have responded to the incentives for research embodied in orphan drug legislation, only to find that funds are not made available to pay for therapies once developed. Those funding health care find that most orphan drugs do not justify funding based on standard value for money criteria, yet that they face political problems if they fail to provide funding for therapy.
 A literature review was conducted in order to determine the precise nature of the problems and to suggest potential solutions. Current orphan drug policies are not fit for the purpose and initiatives need to be taken in the areas of (1) clarifying society’s views about the priority to be given to orphan drugs, (2) revising the arrangements for pricing and reimbursement of orphan drugs, (3) defining the priorities for research into rare diseases and (4) developing ‘joined up’ policies to deal with these issues. Without changes in the current policies, pharmaceutical companies will eventually cease responding to the incentives to develop orphan drugs, because they will increasingly be uncertain whether the drugs, if developed, will be reimbursed.

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Orphan drugs policies: a suitable case for treatment

A pilot study of multicriteria decision analysis for valuing orphan medicines.

Objectives: The purpose of this research is to identify the attributes to include in an orphan medicinal product (OMP) value framework, determine their relative importance via a multi-criteria decision analysis (MCDA) process, and test whether an MCDA approach can practically support decision making. 
Methods: The project included literature searches on the natural history and burden of 40 rare diseases and how payers assess treatment value as well as three workshops. Workshops also were held: the first with GlaxoSmithKline managers working on OMPs, the second with EU clinical and health economics experts, and the third with representatives of rare diseases patient groups in the European Union. Participants refined the attributes, weighted them, scored two case study OMPs in terms of those attributes, and tested the sensitivity of the overall ratings to changes in weights and scores. 
Results: Eight non-monetary attributes were agreed: four concern the disease being treated and four the treatment itself. Workshop participants agreed consensus weights for the attributes. The patient group representatives and the clinical and health economics experts both attributed about half of the weight to attributes reflecting the disease being treated, and half to attributes of the treatment. Patient group representatives gave greater weight than the experts to patients’ quality of daily life and less weight to clinical factors. The weighted attributes were readily applied by workshop participants to two example OMPs and yielded distinct ratings of their respective values. 
Conclusions: An OMP value framework with agreed attributes and weights is a viable proposition using an MCDA approach, and could improve clarity and transparency in decision making about the value of OMPs.

/pdf/multi-criteria-decision-analysis-to-value-orphan-medicines-4exu2az5w8.pdf

Multi-Criteria Decision Analysis to Value Orphan Medicines

The purpose of this research is to identify the attributes to include in a value framework for orphan medicinal products (OMPs), determine their relative importance using a multi-criteria decision analysis (MCDA) process, and test whether an MCDA approach can support decision making in practice. The project included literature searches and three workshops. The latter involved pharmaceutical industry R&D managers, clinical and health economics experts, and rare disease patients' groups. Participants refined the attributes, weighted them, scored two case study OMPs on those attributes and tested the sensitivity of the overall ratings to changes in weights and scores. Eight non-monetary attributes were agreed, four about the disease being treated and four about the treatment itself. The weighted attributes readily were applied by workshop participants to two example OMPs and produced distinct value ratings. The project showed both that an OMP value framework with agreed attributes and weights can be created using an MCDA approach and that this could increase clarity and transparency in decisionmaking about the value of OMPs. A revised version of this paper has been published in Value in Health and can be downloaded from - https://www.clinicalkey.com/#!/content/playContent/1-s2.0-S1098301513043568?returnurl=null&referrer=null Please cite as - Sussex, J., Rollet, P., Garau, M., Schmitt, C., Kent, A. and Hutchings, A., 2013. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value in Health, 16(8), pp.1163-1169.

P. Rollet

Papers

A pilot study of multicriteria decision analysis for valuing orphan medicines.

Multi-Criteria Decision Analysis to Value Orphan Medicines

Multi-Criteria Decision Analysis to Value Orphan Medicines

PHP100 Defining Elements of Value for Rare Disease Treatments

PHP102 From Value to Price: What Should Be the Path for Orphan Drugs?