scholarly journals The application of multi-criteria decision analysis to inform in resource allocation

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 445
Author(s):  
Carina Schey ◽  
Maarten Postma ◽  
Paul Krabbe ◽  
Goran Medic ◽  
Mark Connolly
Keyword(s):  
Decision Analysis ◽  
Health Technology Assessment ◽  
Disease Severity ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Orphan Drugs ◽  
Analysis Framework ◽  
Multi Criteria Decision Analysis ◽  
Annual Treatment

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.

OP100 How Health Technology Assessment Is Adapting To Orphan Drugs In Canada – Not!

2017 ◽  
Vol 33 (S1) ◽  
pp. 45-46
Author(s):  
Durhane Wong-Rieger ◽  
Ferg Mills
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Unmet Need ◽  
Health Technology ◽  
Orphan Drugs ◽  
Evaluation Framework ◽  
Lymphocytic Leukemia ◽  
Primary Analysis ◽  
Small Patient Population

INTRODUCTION:Some countries have distinct pathways for drugs for rare diseases (DRDs) (1). In May 2014, the Canadian Agency for Technologies in Health (CADTH) rejected the option of a separate review pathway for DRDs, reiterating that “pharmacoeconomic analyses are critical for all types of drugs”. While the gap between positive recommendations for common and rare drugs may have narrowed, the rejection for DRDs is still proportionally much higher (2). The default has been to provincially negotiate drug access, for patient populations, subgroups or individuals. Still not wishing to create a separate pathway, in March 2016, CADTH produced a revised evaluation framework for “uncertain clinical and pharmacoeconomic evidence” and other considerations representing “significant unmet need” including rarity and difficulty to study because of small patient population”(3). This study analyzes recommendations for DRDs following the two CADTH revisions.METHODS:Methods used were: synthesis of previously conducted analyses of CADTH recommendations for rare and non-rare drugs, primary comparative analysis of CADTH recommendations for DRDs from 2004 to 2016, and qualitative analysis of two drugs submitted for both rare and non-rare conditions: everolimus (breast cancer, pancreatic neuroendocrine tumours, and tuberous sclerosis complex) and ibrutinib (chronic lymphocytic leukemia, small lymphocytic lymphoma, and Waldenström's Macroglobulinemia).RESULTS:Previous analyses found that DRDs received more negative recommendations than did non-rare drugs; both clinical and economic evidence were differentiating factors. The primary analysis provided an additional understanding of reasons for negative recommendations. There is low consistency across assessments and across the two CADTH review committees. The case studies illustrated the challenges for DRDs to overcome barriers of cost-effectiveness and certainty of clinical evidence, even with the revised framework.CONCLUSIONS:This research challenges the premise that Health Technology Assessment for all drugs can result in fair and equitable recommendations for DRDs. Moreover, assessments based on “significant unmet need” do not appear to provide consistent or equitable guidelines for addressing the issues specific to rare diseases.

TESTING MULTI-CRITERIA DECISION ANALYSIS FOR MORE TRANSPARENT RESOURCE-ALLOCATION DECISION MAKING IN COLOMBIA

2016 ◽  
Vol 32 (4) ◽  
pp. 307-314 ◽  
Author(s):  
Hector Eduardo Castro Jaramillo ◽  
Mireille Goetghebeur ◽  
Ornella Moreno-Mattar
Keyword(s):  
Decision Making ◽  
Resource Allocation ◽  
Decision Analysis ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Social Protection ◽  
Health Technology ◽  
Resource Allocation Decision ◽  
Allocation Decision ◽  
Multi Criteria Decision Analysis

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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