A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan Medicines

Sussex, Jon; Rollet, Pierrick; Garau, Martina; Schmitt, Claude; Kent, Alastair; Hutchings, Adam

doi:10.1016/j.jval.2013.10.002

Cited by 102 publications

(132 citation statements)

References 9 publications

(15 reference statements)

Supporting

Mentioning

110

Contrasting

Unclassified

Order By: Relevance

“…The most apparent problem here before public health authorities is what reimbursement criteria to use and in which manner to implement them in realworld settings. Concentrating on a limited number of indicators seems more functional (15). However, it usually leads to the marginalization of other relevant considerations and affected groups (16).…”

Section: Discussionmentioning

confidence: 99%

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Искров

Стефанов

2016

Balkan Med J

View full text Add to dashboard Cite

Background: During times of fiscal austerity, means of reimbursement decision-making are of particular interest for public health theory and practice. Introduction of advanced health technologies, growing health expenditures and increased public scrutiny over drug reimbursement decisions have pushed governments to consider mechanisms that promote the use of effective health technologies, while constraining costs. Aims: The study's aim was to explore the current rationale of the drug reimbursement decision-making framework in Bulgaria. Our pilot research focused on one particular component of this process -the criteria used -because of the critical role that criteria are known to have in setting budgets and priorities in the field of public health. The analysis pursued two objectives: to identify important criteria relevant to drug reimbursement decision-making and to unveil relationships between theory and practice. Study Design: Cross-sectional study. Methods: The study was realized through a closedended survey on reimbursement criteria among four major public health stakeholders -medical professionals, patients, health authorities, and industry. Empirical outcomes were then cross-compared with the theoretical framework, as defined by current Bulgarian public health legislation. Analysis outlined what is done and what needs to be done in the field of public health reimbursement decision-making. Results: Bulgarian public health stakeholders agreed on 15 criteria to form a tentative optimal framework for drug reimbursement decision-making. The most apparent gap between the empirically found preferences and the official legislation is the lack of consideration for the strength of evidence in reimbursement decisions. Conclusion: Bulgarian policy makers need to address specific gaps, such as formal consideration for strength of evidence, explicit role of efficiency criteria, and means to effectively empower patient and citizen involvement in public health decision-making. Drug reimbursement criteria have to be integrated into legitimate public health decision support tools that ensure the achievement of national public health objectives. These recommendations could be expanded to all Eastern European countries who share common public health problems.

show abstract

Section: Discussionmentioning

confidence: 99%

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Искров

Стефанов

2016

Balkan Med J

View full text Add to dashboard Cite

show abstract

“…Current discussions on financing ODs deal with principles of priority setting based on distributive justice, involvement of stakeholders along the evaluation process, 3 and value-bearing factors and their combination for a comprehensive assessment [22,[33][34][35] .…”

Section: Comprehensive Value Assessmentmentioning

confidence: 99%

“…Although there is agreement in considering a broader approach to set the value of pharmaceuticals in general, including their societal value, and regardless of some pilot experiences which are currently under development, there is no internationally agreed-on taxonomy for valuebearing criteria or measuring instruments [26,32,33,[41][42][43] . Despite these limited advances, different factors have been considered for value-bearing pharmaceuticals, including the disease and patient characteristics, the possibility of improvement either in health (effect size for efficacy and safety) or non-health outcomes (procedural attributes) [44] , and the increasingly recognised value of innovation [45,46] .…”

Section: Comprehensive Value Assessmentmentioning

confidence: 99%

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Mincarone

Leo²,

Sabina³

et al. 2017

Public Health Genomics

View full text Add to dashboard Cite

The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the “regulatory capture” effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

show abstract

“…EVIDEM recommends a test criterion of ''mutual independence'', but despite this, still includes clearly interdependent criteria like comparative outcomes, type of benefit and knowledge of intervention. Though few in number, applications of MCDA to drugs provide more examples of blurred definition, such as manufacturing complexity and public health interest, which overlap and lack mutual independence, as with level of research undertaken, knowledge of the intervention, level of uncertainty of effectiveness, survival and life-saving [9,14,[36][37][38][39][40][41][42].…”

Section: Further Double Counting In Evidem and Elsewherementioning

confidence: 99%

“…The main arguments are that (1) MCDA includes a comprehensive and explicit list of value criteria not captured by the traditional methods of economic evaluation; (2) since it allocates quantitative weights to the different evaluation criteria, their relative importance is incorporated explicitly in the evaluation, thus making values and elicited preferences more consistent and transparent; and (3) the participation of all agents involved in assessing the value of alternatives increases the legitimacy of the process [1][2][3][4][5][6][7][8][9][10][11][12][13][14]. Arguments against MCDA include the inadequate treatment of opportunity cost, its vulnerability to double counting, and the fallacious attribution of deficiencies to methods of economic evaluation (EA) like cost-effectiveness analysis (CEA) in HTA [13][14][15][16][17][18][19][20][21][22][23][24]. We do not consider further the third argument for MCDA, because such participation seems to be a standard element of good practice in CEA [25][26][27][28][29][30][31][32].…”

mentioning

confidence: 99%

Does MCDA Trump CEA?

Campillo-Artero

Puig‐Junoy

Culyer

2018

Appl Health Econ Health Policy

View full text Add to dashboard Cite

A growing amount of literature claims that multicriteria decision analysis (MCDA) is superior to economic evaluation in health technology assessment (HTA). The main arguments are that (1) MCDA includes a comprehensive and explicit list of value criteria not captured by the traditional methods of economic evaluation; (2) since it allocates quantitative weights to the different evaluation criteria, their relative importance is incorporated explicitly in the evaluation, thus making values and elicited preferences more consistent and transparent; and (3) the participation of all agents involved in assessing the value of alternatives increases the legitimacy of the process [1][2][3][4][5][6][7][8][9][10][11][12][13][14]. Arguments against MCDA include the inadequate treatment of opportunity cost, its vulnerability to double counting, and the fallacious attribution of deficiencies to methods of economic evaluation (EA) like cost-effectiveness analysis (CEA) in HTA [13][14][15][16][17][18][19][20][21][22][23][24]. We do not consider further the third argument for MCDA, because such participation seems to be a standard element of good practice in CEA [25][26][27][28][29][30][31][32].

show abstract

A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan Medicines

Abstract: The multicriteria decision analysis approach piloted works and could be developed for use by payers and health technology assessment bodies.

Cited by 102 publications

References 9 publications

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Criteria for Drug Reimbursement Decision-Making: An Emerging Public Health Challenge in Bulgaria

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Does MCDA Trump CEA?

Contact Info

Product

Resources

About