scholarly journals Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development?

2018 ◽  
Vol 6 ◽  
Author(s):  
Aleksandra Baran-Kooiker ◽  
Marcin Czech ◽  
Coen Kooiker
F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 445
Author(s):  
Carina Schey ◽  
Maarten Postma ◽  
Paul Krabbe ◽  
Goran Medic ◽  
Mark Connolly

Background: There is a perception held by payers that orphan products are expensive. As a result, the current health technology assessment systems might be too restrictive for orphan drugs, therefore potentially denying patients access to life-saving medicines. While price is important, it should be considered in relation to a broader range of disease-related product attributes that are not necessarily considered by many health technology assessment agencies. To overcome these challenges, multi-criteria decision analysis has been proposed as an alternative to evaluate technologies. Methods: A targeted literature review was conducted to identify the most frequently cited attributes in multi-criteria decision analysis (MCDA) in rare diseases. From the leading attributes identified, we developed a multi-criteria decision analysis framework with which to aggregate the orphan drug values. We subsequently reviewed and plotted the relationship between single attributes and the average annual treatment costs for 8 drugs used in the treatment of rare endocrine diseases. The annual treatment costs were based on UK list prices for the average daily dose per patient. Results: The five most frequently mentioned attributes in the literature were as follows: Disease severity, Unmet need (or availability of therapeutic alternatives), Comparative effectiveness or efficacy, Quality of evidence and Safety & tolerability. Results from the multi-criteria decision analysis framework indicate a wide range of average annual per-patients costs for drugs intended for the same diseases, and likewise for diseases with a similar level of Disease severity. Conclusions: Multi-criteria decision analysis may offer a viable alternative to support discussion in reimbursement decisions for orphan drugs. The analyses can be used to inform investigations on the application of MCDAs in rare diseases.


Author(s):  
Lotte Steuten ◽  
Bert Vrijhoef ◽  
Hans Severens ◽  
Frits van Merode ◽  
Cor Spreeuwenberg

Objectives:An overview was produced of indicators currently used to assess disease management programs and, based on these findings, provide a framework regarding sets of indicators that should be used when taking the aims and types of disease management programs into account.Methods:A systematic literature review was performed.Results:Thirty-six studies met the inclusion criteria. It appeared that a link between aims of disease management and evaluated structure, process, as well as outcome indicators does not exist in a substantial part of published studies on disease management of diabetes and asthma/chronic obstructive pulmonary disease, especially when efficiency of care is concerned. Furthermore, structure indicators are largely missing from the evaluations, although these are of major importance for the interpretation of outcomes for purposes of decision-making. Efficiency of disease management is mainly evaluated by means ofprocessindicators; the use ofoutcomeindicators is less common. Within a framework, structure, process, and outcome indicators for effectiveness and efficiency are recommended for each type of disease management program.Conclusions:The link between aims of disease management and evaluated structure, process, and outcome indicators does not exist in a substantial part of published studies on disease management. The added value of this study mainly lies in the development of a framework to guide the choice of indicators for health technology assessment of disease management.


2016 ◽  
Vol 32 (4) ◽  
pp. 307-314 ◽  
Author(s):  
Hector Eduardo Castro Jaramillo ◽  
Mireille Goetghebeur ◽  
Ornella Moreno-Mattar

Objectives: In 2012, Colombia experienced an important institutional transformation after the establishment of the Health Technology Assessment Institute (IETS), the disbandment of the Regulatory Commission for Health and the reassignment of reimbursement decision-making powers to the Ministry of Health and Social Protection (MoHSP). These dynamic changes provided the opportunity to test Multi-Criteria Decision Analysis (MCDA) for systematic and more transparent resource-allocation decision-making.Methods: During 2012 and 2013, the MCDA framework Evidence and Value: Impact on Decision Making (EVIDEM) was tested in Colombia. This consisted of a preparatory stage in which the investigators conducted literature searches and produced HTA reports for four interventions of interest, followed by a panel session with decision makers. This method was contrasted with a current approach used in Colombia for updating the publicly financed benefits package (POS), where narrative health technology assessment (HTA) reports are presented alongside comprehensive budget impact analyses (BIAs).Results: Disease severity, size of population, and efficacy ranked at the top among fifteen preselected relevant criteria. MCDA estimates of technologies of interest ranged between 71 to 90 percent of maximum value. The ranking of technologies was sensitive to the methods used. Participants considered that a two-step approach including an MCDA template, complemented by a detailed BIA would be the best approach to assist decision-making in this context. Participants agreed that systematic priority setting should take place in Colombia.Conclusions: This work may serve as the basis to the MoHSP on its interest of setting up a systematic and more transparent process for resource-allocation decision-making.


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