Weighting the OrPhar-SEFH MCDA Framework for Its Use in Ultrarare Kidney Diseases
Author(s)
Fernando Caravaca, MD1, Constantino Fernández, MD2, Daniel Gallego, Mr.3, Pedro Gómez, Mr.4, Alicia Herrero Ambrosio, PharmD, PhD5, Silvia Manrique, PharmD, PhD6, Elena Román, MD7, Josu Aguirre, PhD8, Pau Vesa, MSc8, Raquel Fernández, Ms.9, Sofía Villafranca, Ms.9.
1Hospital Universitario 12 de Octubre, Madrid, Spain, 2Complejo Hospitalario Universitario de La Coruña, La Coruña, Spain, 3Federación Nacional ALCER, Madrid, Spain, 4Economista de la Salud. Ex Subdirector General de Farmacia. Ex Secretario General de la ONT. Funcionario del Ministerio de Sanidad en excedencia, Madrid, Spain, 5Hospital Universitario La Paz, Madrid, Spain, 6Hospital General Universitario Gregorio Marañón, Madrid, Spain, 7Hospital Universitario y Politécnico La Fe, Valencia, Spain, 8IQVIA, Barcelona, Spain, 9Sobi, Madrid, Spain.
1Hospital Universitario 12 de Octubre, Madrid, Spain, 2Complejo Hospitalario Universitario de La Coruña, La Coruña, Spain, 3Federación Nacional ALCER, Madrid, Spain, 4Economista de la Salud. Ex Subdirector General de Farmacia. Ex Secretario General de la ONT. Funcionario del Ministerio de Sanidad en excedencia, Madrid, Spain, 5Hospital Universitario La Paz, Madrid, Spain, 6Hospital General Universitario Gregorio Marañón, Madrid, Spain, 7Hospital Universitario y Politécnico La Fe, Valencia, Spain, 8IQVIA, Barcelona, Spain, 9Sobi, Madrid, Spain.
OBJECTIVES: To determine the importance of evaluation criteria to assess the value of targeted therapies for the treatment of the ultra-rare kidney diseases Complement 3 Glomerulopathy (C3G) and primary Immune complex-mediated Membranoproliferative Glomerulonephritis (IC-MPGN) through Multi-Criteria Decision Analysis (MCDA).
METHODS: An adapted EVIDEM framework, developed by the Rare Diseases and Orphan Drugs Working Group of the Spanish Society of Hospital Pharmacy (Orphar-SEFH) for orphan drug evaluation in Spain, was weighted according to the relative importance of its constituent criteria, considering its usefulness in the assessment and decision-making process for the treatment of the ultra-rare kidney diseases C3G and primary IC-MPGN in Spain. Each quantitative criterion was scored on a scale of 1-5 (1=very unimportant, 5=very important) by a panel of 7 experts: 2 hospital pharmacists, 1 national payer, 3 nephrology experts and 1 patient representative, with a posterior discussion.
RESULTS: Comparative efficacy/effectiveness was rated the most relevant criterion, mean score (4.9) followed by the unmet needs (4.4). Disease severity, comparative safety/tolerability and therapeutic impact were also perceived as highly relevant criteria (4.3 each). Patient Reported Outcomes (PRO) and quality of the evidence were scored with a 3.9; cost criteria, including other medical costs and non-medical/indirect costs, received a score of 3 and 2.9, respectively.
CONCLUSIONS: The Orphar-SEFH MCDA framework has been weighted for its potential use in the evaluation of specific treatments for C3G and primary IC-MPGN. The relative importance of criteria was consistent across profiles, with efficacy/effectiveness, unmet needs and disease severity always being among the most relevant criteria, and cost-related criteria among the least, likely reflecting difficulties of capturing accurate cost estimates.
METHODS: An adapted EVIDEM framework, developed by the Rare Diseases and Orphan Drugs Working Group of the Spanish Society of Hospital Pharmacy (Orphar-SEFH) for orphan drug evaluation in Spain, was weighted according to the relative importance of its constituent criteria, considering its usefulness in the assessment and decision-making process for the treatment of the ultra-rare kidney diseases C3G and primary IC-MPGN in Spain. Each quantitative criterion was scored on a scale of 1-5 (1=very unimportant, 5=very important) by a panel of 7 experts: 2 hospital pharmacists, 1 national payer, 3 nephrology experts and 1 patient representative, with a posterior discussion.
RESULTS: Comparative efficacy/effectiveness was rated the most relevant criterion, mean score (4.9) followed by the unmet needs (4.4). Disease severity, comparative safety/tolerability and therapeutic impact were also perceived as highly relevant criteria (4.3 each). Patient Reported Outcomes (PRO) and quality of the evidence were scored with a 3.9; cost criteria, including other medical costs and non-medical/indirect costs, received a score of 3 and 2.9, respectively.
CONCLUSIONS: The Orphar-SEFH MCDA framework has been weighted for its potential use in the evaluation of specific treatments for C3G and primary IC-MPGN. The relative importance of criteria was consistent across profiles, with efficacy/effectiveness, unmet needs and disease severity always being among the most relevant criteria, and cost-related criteria among the least, likely reflecting difficulties of capturing accurate cost estimates.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA358
Topic
Health Technology Assessment
Topic Subcategory
Value Frameworks & Dossier Format
Disease
Rare & Orphan Diseases