A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain

Authors

  • Néboa Zozaya Weber, Madrid - Spain https://orcid.org/0000-0003-4618-6894
  • Teresa Caballero Servicio de Alergología, Hospital Universitario La Paz; Hospital La Paz Institute for Health Research (IdiPaz), Madrid and Center for Biomedical Research Network on Rare Diseases (CIBERER U754), Madrid - Spain https://orcid.org/0000-0003-3005-9858
  • Teresa González-Quevedo Unidad de Referencia de Angioedema para Andalucía, Unidad de Alergología, Hospital Universitario Virgen del Rocío, Sevilla - Spain
  • Pedro Gamboa Setien Unidad de Alergología, Hospital Universitario de Cruces, Bizcaia - Spain https://orcid.org/0000-0002-7125-8709
  • M. Ángeles González Servicio de Farmacia, Hospital Universitario La Paz, Madrid - Spain https://orcid.org/0000-0001-7678-8904
  • Ramón Jódar Servicio de Farmacia, Hospital Universitario Bellvitge, Barcelona - Spain
  • José Luis Poveda-Andrés Servicio de Farmacia Hospitalaria, Hospital Universitari y Politècnic la Fe, Valencia - Spain
  • Encarna Guillén-Navarro Sección de Genética Médica, Servicio de Pediadría, Hospital Clínico Universitario Virgen de la Arrixaca, IMIB-Arrixaca Universidad de Murcia, CIBERER-ISCIII, Murcia - Spain
  • Agustín Rivero Cuadrado Hospital Universitario La Paz, Madrid - Spain https://orcid.org/0000-0002-0911-1669
  • Álvaro Hidalgo-Vega Fundación Weber, Madrid and Universidad de Castilla-La Mancha, Toledo - Spain

DOI:

https://doi.org/10.33393/grhta.2022.2333

Keywords:

Drugs evaluation, Hereditary angioedema, Lanadelumab, Multicriteria decision analysis (MCDA), Rare disease

Abstract

Introduction: Hereditary angioedema (HAE) is a rare genetic disease that impairs quality of life and could be life-threatening. The aim of this study was to apply a multicriteria decision analysis to assess the value of three long-term prophylactic (LTP) therapies for HAE in Spain.

Methods: A multidisciplinary committee of 10 experts assessed the value of lanadelumab (subcutaneous use), C1-inhibitor (C1-INH; intravenous), and danazol (orally), using placebo as comparator. We followed the EVIDEM methodology that considers a set of 13 quantitative criteria. The overall estimated value of each intervention was obtained combining the weighting of each criterion with the scoring of each intervention in each criterion. We used two alternative weighting methods: hierarchical point allocation (HPA) and direct rating scale (DRS). A reevaluation of weightings and scores was performed.

Results: Lanadelumab obtained higher mean scores than C1-INH and danazol in all criteria, except for the cost of the intervention and clinical practice guidelines. Under the HPA method, the estimated values were 0.51 (95% confidence interval [CI]: 0.44-0.58) for lanadelumab, 0.47 (95%CI: 0.41-0.53) for C1-INH, and 0.31 (95%CI: 0.24-0.39) for danazol. Similar results were obtained with the DRS method: 0.51 (95%CI: 0.42-0.60), 0.47 (95%CI: 0.40-0.54), and 0.27 (95%CI: 0.18-0.37), respectively. The comparative cost of the intervention was the only criterion that contributed negatively to the values of lanadelumab and C1-INH. For danazol, four criteria contributed negatively, mainly comparative safety.

Conclusion: Lanadelumab was assessed as a high-value intervention, better than C1-INH and substantially better than danazol for LTP treatment of HAE.

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Published

2022-01-25

How to Cite

1.
Zozaya N, Caballero T, González-Quevedo T, Gamboa Setien P, González M Ángeles, Jódar R, Poveda-Andrés JL, Guillén-Navarro E, Rivero Cuadrado A, Hidalgo-Vega Álvaro. A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain. Grhta [Internet]. 2022 Jan. 25 [cited 2022 Sep. 26];9(1):14-21. Available from: https://journals.aboutscience.eu/index.php/grhta/article/view/2333

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Original Research Articles