Exploring Values of Health Technology Assessment Agencies Using Reflective Multicriteria and Rare Disease Case

Goetghebeur, Mireille; Wagner, Monika; Samaha, Dima; O’Neil, William M.; Badgley, Danielle; Castro-Jaramillo, Hector; Abrishami, Payam; Sarría‐Santamera, Antonio; Cleemput, Irina; Tringali, Michele

doi:10.1017/s0266462317000915

Cited by 17 publications

(21 citation statements)

References 21 publications

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“…Because the authors used the perspective of an OMP manufacturer, several principles were different from the other publications, e.g., the principle not to perform EMA's assessment of therapeutic benefit again during HTA on the national level. Goetghebeur et al ( 57 ) conducted a questionnaire (based on EVIDEM) among health authority representatives from 8 countries to collect feedback on the mandates and values of HTA agencies, to examine ethical underpinnings of the HTA values and to explore trade-offs.…”

Section: Resultsmentioning

confidence: 99%

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

Baran-Kooiker

Czech

Kooiker³

2018

Front. Public Health

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Background: Multi-criteria decision analysis (MCDA) is a decision-making tool that can take into account multidimensional factors and enables comparison of (medical) technologies by combining individual criteria into one overall appraisal. The MCDA approach has slowly gained traction within Health Technology Assessment (HTA) and its elements are gradually being incorporated into HTA across Europe. Several groups of scientists have proposed MCDA approaches targeted toward orphan drugs and rare diseases by including criteria specific to rare diseases. The goal of this article is to provide an overview of the current state of knowledge and latest developments in the field of MCDA in HTA for orphan drugs, to review existing models, their design characteristics, as well as to identify opportunities for further model improvement.Methods: A systematic literature search was conducted in January 2018 using four databases: MEDLINE (Pubmed), EBSCO HOST, EMBASE, and Web of science to find publications related to use of MCDA in the rare disease field (keywords: MCDA/orphan drug/rare disease and synonyms). Identified MCDA models were analyzed, e.g., structure, criteria, scoring, and weighting methodology.Results: Two hundred and eleven publications were identified, of which 29 were included after removal of duplicates. 9 authors developed own MCDA models, 7 of which based on literature reviews intended to identify the most important and relevant decision criteria in the model. In 13 publications (8 models) weights were assigned to criteria based on stakeholder input. The most commonly chosen criteria for creation of the MCDA models were: comparative effectiveness/efficacy, the need for intervention, and disease severity. Some models have overlapping criteria, especially in the treatment cost and effectiveness areas.Conclusions: A range of MCDA models for HTA have been developed, each with a slightly different approach, focus, and complexity, including several that specifically target rare diseases and orphan drug appraisal. Models have slowly progressed over the years based on pilots, stakeholder input, sharing experiences and scientific publications. However, full consensus on model structure, criteria selection and weighting is still lacking. A simplification of the MCDA model approach may increase its acceptance. A multi-stakeholder discussion on fundamental design and implementation strategies for MCDA models would be beneficial to this end.

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Section: Resultsmentioning

confidence: 99%

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

Baran-Kooiker

Czech

Kooiker³

2018

Front. Public Health

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“…This can be elicited informally or formally through an interpretive scoring scale capturing judgment (see Tool Adapt and pilot, Additional file 2 ). The conclusion of this individual reflection: the balancing act of these considerations is facilitated by the interpretive grid, which serves as a reminder us of the underlying ethical imperative associated with each criterion [ 18 ]. …”

Section: Resultsmentioning

confidence: 99%

“…The conclusion of this individual reflection: the balancing act of these considerations is facilitated by the interpretive grid, which serves as a reminder us of the underlying ethical imperative associated with each criterion [ 18 ].…”

Section: Resultsmentioning

confidence: 99%

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Can reflective multicriteria be the new paradigm for healthcare decision-making? The EVIDEM journey

Goetghebeur

Cellier²

2018

Cost Eff Resour Alloc

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BackgroundMultiple technologies, procedures and programs call for fairly-based decisions for prioritization of healthcare interventions. There is a diversity of perspectives of what constitutes a legitimate decision, which depends on both the process and the reasoning applied. Current approaches focus on technical aspects while methods to support alignment of decisions with the compassionate impetus of healthcare systems is lacking.MethodsThe framework was developed based on an analysis of the foundations of healthcare systems, the reasoning underlying decisions and fair processes. The concept of reflective multicriteria was created: it assumes that decisionmakers guided by a generic interpretative frame rooted in the compassionate impetus of healthcare systems, can sharpen their reasoning, raise awareness of their motivation and increase legitimacy of decisions. The initial framework was made available through a not for profit organization (the EVIDEM Collaboration, 2006–2017) to stimulate its development with thought leaders and stakeholders in an open source philosophy. Development was tailored to the real-life needs of decisionmakers and drew on several domains of knowledge including healthcare ethics, evidenced-based medicine, health economics, health technology assessment and multicriteria approaches.ResultsThe 10th edition framework builds on four dimensions: (1) the universal impetus of healthcare systems, (2) reasoning, values and ethics, (3) evidence and knowledge on interventions, and (4) a transformative process. Mathematical aspects of the framework are designed to help clarify, express and share individual reasoning; this non-conventional use of numbers requires a cultural change and needs to be phased in slowly. The framework includes four tools for easy adaptation and operationalization: (a) concepts and operationalization, (b) adapt and pilot, (c) evidence matrix, (d) mathematical representation of reasoning. Application is useful throughout all types of healthcare interventions, for all levels of decision, and across the globe.ConclusionBy clarifying their reasoning while keeping decisionmakers aware of the impetus of healthcare systems, reflective multicriteria provides an effective approach to increase the legitimacy of decisions. Beyond a tool, reflective multicriteria pioneered by EVIDEM is geared to transform our vision of the value of healthcare interventions and how they might contribute to relevant, equitable and sustainable healthcare systems.Electronic supplementary materialThe online version of this article (10.1186/s12962-018-0116-9) contains supplementary material, which is available to authorized users.

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“…The discussion below builds on the commentaries of Culyer in particular regarding his reflection on the need to learn by doing, changes needed to enhance HTA, the embedding of ethics in the HTA process, the authority of the exercise and the collective thinking it fosters. 3,4 Although the EDP builds on the framework accountability for reasonableness (A4R), 5 which was set forth more than 20 years ago to address procedural ethics, through four conditions that can enhance legitimacy, 6 there is no mention in the Oortwijn paper of ethics, nor on the alignment of processes with the mission and values of agencies. However, HTA are currently struggling with core issues central to their legitimacy such as:…”

Section: T He Article By Oortwijn Et Al Provides Important Andmentioning

confidence: 99%

Deliberative Processes by Health Technology Assessment Agencies: A Reflection on Legitimacy, Values and Patient and Public Involvement Comment on "Use of Evidence-informed Deliberative Processes by Health Technology Assessment Agencies Around the Globe"

Goetghebeur

Cellier

2020

Int J Health Policy Manag

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Legitimacy of deliberation processes leading to recommendations for public financing or clinical practice depends on the data considered, stakeholders involved and the process by which both of these are selected and organised. Oortwijn et al provides an interesting exploration of processes currently in place in health technology assessment (HTA) agencies. However, agencies are struggling with core issues central to their legitimacy that goes beyond the procedural exploration of Oortwijn et al, such as: how processes reflect the mission and values of the agencies? How they ensure that recommendations are fair and reasonable? Which role should be given to public and patient involvement? Do agencies have a positive impact on the healthcare system and the populations served? What are the drivers of their evolution? We concur with Culyer commentary on the need of learning from doing what works best and that a reflection is indeed needed to "enhance the fairness and legitimacy of HTA."

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Exploring Values of Health Technology Assessment Agencies Using Reflective Multicriteria and Rare Disease Case

Abstract: Reflective multicriteria are useful to explore substantive values of HTAs, reflect how these values and their ethical underpinnings can be operationalized into criteria, and explore the ethical reasoning at the heart of the healthcare debate.

Cited by 17 publications

References 21 publications

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

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

Can reflective multicriteria be the new paradigm for healthcare decision-making? The EVIDEM journey

Deliberative Processes by Health Technology Assessment Agencies: A Reflection on Legitimacy, Values and Patient and Public Involvement Comment on "Use of Evidence-informed Deliberative Processes by Health Technology Assessment Agencies Around the Globe"

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