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Establishment of a value assessment framework for orphan medicinal products in China.
Orphanet J Rare Dis
October 2024
West China School of Pharmacy, Sichuan University, 17 Renmin South Road (3rd Section), Chengdu, China.
Background: This study aimed to establish a suitable value assessment framework for orphan medicinal products in China based on the multi-criteria decision analysis (MCDA) method.
Methods: First, a draft framework of the MCDA criteria was built based on a systematic literature evaluation and the EVIDEM framework tools. Second, stakeholder groups were formed and expert opinions were collected through the brainstorming and expert consultation methods.
Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi-criteria decision analysis.
Haemophilia
November 2024
Àrea del medicament, Institut Català de la Salut, Barcelona, Spain.
Introduction: The value of gene therapies for haemophilia needs to be assessed holistically.
Aim: To determine the value of etranacogene dezaparvovec (ED) compared to current extended half-life (EHL) recombinant factors (rFIX), using multi-criteria decision analysis (MCDA).
Method: MCDA EVIDEM methodology adapted to orphan drugs was used, with nine quantitative criteria and four contextual criteria.
Evaluating vonoprazan and tegoprazan for gastroesophageal reflux disease treatment in Chinese Healthcare: an EVIDEM framework analysis.
BMC Gastroenterol
June 2024
Hebei General Hospital, Shijiazhuang, Hebei Province, China.
Background: In Chinese healthcare settings, drug selection decisions are predominantly influenced by the Pharmacy & Therapeutics Committee (PTC). This study evaluates two recently introduced potassium-competitive acid blockers, vonoprazan (VPZ) and tegoprazan (TPZ), utilizing the Evidence and Value: Impact on DEcisionMaking (EVIDEM) framework.
Methods: The study employed the 10th edition of EVIDEM, which includes a core model with five domains and 13 criteria.
Value of concerted and hospital hemodialysis through a multi-criteria decision analysis.
Nefrologia (Engl Ed)
February 2024
Centro Andaluz de Documentación e Información de Medicamentos (CADIME), Escuela Andaluza de Salud Pública, Granada, Spain; Consorcio de Investigación Biomédica en Red Epidemiología y Salud Pública, CIBERESP; Instituto de Investigación Biosanitaria ibs.GRANADA, Granada, Spain. Electronic address:
Objective: To evaluate the value of the provision of contracted versus hospital dialysis services for the treatment of chronic kidney disease in Spain using the multicriteria decision analysis methodology.
Method: The EVIDEM (Evidence and Value: Impact on Decision Making) evaluation framework was used to calculate the estimated value of both dialysis delivery models (arranged vs. hospital) through a virtual workshop in which different profiles participated: directors and managers, professionals and heads of units and representatives of patients and relatives.
Reporting reimbursement price decisions for onco-hematology drugs in Spain.
Front Public Health
November 2023
Departament de Farmacologia, de Terapèutica i de Toxicologia, Universitat Autònoma de Barcelona, Sabadell, Barcelona, Spain.
Introduction: Even using well-established technology assessment processes, the basis of the decisions on drug price and reimbursement are sometimes perceived as poorly informed and sometimes may be seen as disconnected from value. The literature remains inconclusive about how Health Technology Assessment Bodies (HTAb) should report the determinants of their decisions. This study evaluates the relationship between oncology and hematology drug list prices and structured value parameters at the time of reimbursement decision in Spain.
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