Drug Evaluation and Decision Making in Catalonia: Development and Validation of a Methodological Framework Based on Multi-Criteria Decision Analysis (Mcda) for Orphan Drugs

Gilabert-Perramon, A; Torrent-Farnell, Josep; Catalan, Arancha; Prat, A; Fontanet, M.; Puig-Peiro, R.; Merino-Montero, Sandra; Khoury, Hanane; Goetghebeur, Mireille; Badia, Xavier

doi:10.1017/s0266462317000149

Cited by 62 publications

(78 citation statements)

References 22 publications

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“…In our country, Spain, only a few MCDAs have been published in the field of healthcare [20][21][22][23][24][25][26][27][28], although the number of healthcare-related publications that incorporate MCDA is increasing in Spain and globally.…”

Section: Disability (P=0021) 5%mentioning

confidence: 99%

“…Some MCDAs have been used in our country to compare alternative treatments for various pathologies, such as chronic dermatological inflammatory diseases, cancer, rare diseases, and cardiovascular diseases such as nonvalvular atrial fibrillation and pulmonary hypertension [24][25][26]28]. Other applications of MCDA include the development of an assessment framework for rare diseases at a regional level in Catalonia [22] and the development of a decision-support tool for drug evaluation in a pharmacy and therapeutics committee setting at the hospital level [20]. MCDA was also used to establish an algorithm for predicting price and reimbursement decisions at national and regional levels [27].…”

Section: Disability (P=0021) 5%mentioning

confidence: 99%

Section: Disability (P=0021) 5%mentioning

confidence: 99%

“…Some studies have used the EVIDEM methodology to develop the analysis [20,[22][23][24][25]28]. In those studies, the methods used to prioritize the criteria were fundamentally compositional, such as direct classification or the analytic hierarchical process [22][23][24][25][26][27][28]. Either a single-stakeholder perspective was used, such as that of patients [20,21,26] or evaluation agencies [22,27], or a multidisciplinary perspective was employed, which included patients, physicians, and other stakeholders [23][24][25]28].…”

Section: Disability (P=0021) 5%mentioning

confidence: 99%

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A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study

Andrés-Nogales

Trillo

Ruiz-Moreno

et al. 2020

PharmacoEconomics Open

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Background The clinical and economic management of retinal diseases has become more complex following the introduction of new intravitreal treatments. Multicriteria decision analysis (MCDA) offers the potential to overcome the challenges associated with traditional decision-making tools. Objectives A MCDA to determine the most relevant criteria to decision-making in the management of diabetic macular edema (DME) based on the perspectives of multiple stakeholders in Spain was developed. This MCDA was termed the MULTIDEX-EMD study. Methods Nineteen stakeholders (7 physicians, 4 pharmacists, 5 health authorities and health management experts, 1 psychologist, and 2 patient representatives) participated in this three-phase project. In phase A, an advisory board defined all of the criteria that could influence DME treatment decision-making. These criteria were then screened using a discrete choice experiment (DCE) (phase B). Next, a multinomial logit model was fitted by applying the backward elimination algorithm (relevant criteria: p value < 0.05). Finally, the results were discussed in a deliberative process (phase C). Results Thirty-one criteria were initially defined (phase A) and grouped into 5 categories: efficacy/effectiveness, safety, organizational and economic impact, patient-reported outcomes, and other therapeutic features. The DCE results (phase B) showed that 10 criteria were relevant to the decision-making process for a 50-to 65-year-old DME patient: mean change in best corrected visual acuity (p value < 0.001), percentage of patients with an improvement of ≥ 15 letters (p value < 0.001), effect duration per administration (p value = 0.008), retinal detachment (p value < 0.001), endophthalmitis (p value = 0.012), myocardial infarction (p value < 0.001), intravitreal hemorrhage (p value = 0.021), annual treatment cost per patient (p value = 0.001), health-related quality of life (HRQoL) (p value = 0.004), and disability level (p value = 0.021). Conclusions From a multi-stakeholder perspective, the selection of an appropriate treatment for DME patients should guarantee patient safety and maximize the visual acuity improvement and treatment effect duration. It should also contribute to system sustainability by being affordable, it should have a positive impact on HRQoL, and it should prevent disability.

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Section: Disability (P=0021) 5%mentioning

confidence: 99%

Section: Disability (P=0021) 5%mentioning

confidence: 99%

Section: Disability (P=0021) 5%mentioning

confidence: 99%

Section: Disability (P=0021) 5%mentioning

confidence: 99%

See 2 more Smart Citations

A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study

Andrés-Nogales

Trillo

Ruiz-Moreno

et al. 2020

PharmacoEconomics Open

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“…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%

Does MCDA Trump CEA?

Campillo-Artero

Puig‐Junoy

Culyer

2018

Appl Health Econ Health Policy

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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].

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Determining what represents value in the treatment of prurigo nodularis and its key unmet needs in Spain through Multi‐Criteria Decision Analysis

Silvestre,

Tribó,

Armario‐Hita

et al. 2023

JEADV Clinical Practice

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BackgroundPrurigo nodularis (PN) is a chronic, debilitating dermatologic disease characterised by the presence of highly pruritic nodular lesions. PN highly impacts on patients' quality of life as there are no specific treatments available in Spain.ObjectivesDetermine the main value drivers in the treatment of PN in Spain and its main unmet needs using Multi‐Criteria Decision Analysis (MCDA).MethodsLiterature review to synthesise relevant evidence in an evidence matrix based on the MCDA EVIDEM framework adapted to Spain. A multidisciplinary panel composed of dermatologists, hospital pharmacists and a patient weighted (5‐point scale; 0 minimum importance, 5 maximum importance) and scored each criterion included in the framework (from −5 to 5 or 0 to 5 depending on the criterion). Results were discussed in a reflective group session.ResultsPN was considered a severe (3.3 ± 0.7) and infrequent (2.0 ± 0.7) disease, with high unmet needs (4.2 ± 0.7) mainly due to the lack of available treatments with specific indication for PN. Current off‐label treatments were perceived to have limited efficacy/effectiveness (1.8 ± 1.1), an unfavourable long‐term safety profile (2.1 ± 0.9) and low therapeutic impact (1.7 ± 1.1). The measure of patient‐reported outcomes (2.7 ± 0.9) was perceived as important, but available tools are not specific. Although the cost of available treatments was not considered high (2.4 ± 1.5), experts agreed that PN is associated with moderately high other medical costs (3.6 ± 1.1) and indirect costs (3.1 ± 0.9). Experts considered that current guidelines and consensus (2.6 ± 0.7) are not clear on severity criteria and treatment algorithm. The quality of evidence (1.4 ± 0.5) of currently used off‐label treatments was perceived as low due to a lack of published clinical trials.ConclusionsPN was considered a severe disease associated with relevant unmet needs, including the lack of specific and effective treatments for PN, the lack of consensus on the disease definition, defined severity criteria, prevalence estimations and awareness of PN in Spain.

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Drug Evaluation and Decision Making in Catalonia: Development and Validation of a Methodological Framework Based on Multi-Criteria Decision Analysis (Mcda) for Orphan Drugs

Abstract: MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.

Cited by 62 publications

References 22 publications

A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study

A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study

Does MCDA Trump CEA?

Determining what represents value in the treatment of prurigo nodularis and its key unmet needs in Spain through Multi‐Criteria Decision Analysis

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