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Determining value in the treatment of activated PI3Kδ syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders.
Abad, María Reyes; Alerany, Carmen; Alsina, Laia; Granados, Eduardo López; Neth, Olaf; Poveda, Jose Luis; Rivière, Jacques G; Rodríguez-Gallego, Carlos; Tutein Nolthenius, Joanne B; Figueiredo, Raquel; Labazuy, Silvia Subías; Gil, Alicia.
Afiliación
  • Abad MR; Pharmacy Department, H.U. Miguel Servet, Zaragoza - Spain.
  • Alerany C; Pharmacy Department, H.U. Vall d' Hebron, Barcelona - Spain.
  • Alsina L; Clinical Immunology and Primary Immunodeficiencies Unit, Pediatric Allergy and Clinical Immunology Department, H. Sant Joan de Déu. Institut de Recerca Sant Joan de Déu. Universitat de Barcelona, Barcelona - Spain.
  • Granados EL; Immunology Department, H.U. La Paz, Madrid - Spain.
  • Neth O; Paediatric Infectious Diseases, Rheumatology and Immunology Unit H.U. Virgen del Rocío, Seville - Spain.
  • Poveda JL; Pharmacy Department H.U. i Politècnic La Fe, Valencia - Spain.
  • Rivière JG; Paediatric Infectious Diseases and Immunodeficiencies Unit, H.U. Vall d'Hebron University Hospital, Barcelona - Spain.
  • Rodríguez-Gallego C; Infection and Immunity in Pediatric Patients Research Group, Vall d'Hebron Institut de Recerca (VHIR), H.U. Vall d'Hebron, Barcelona - Spain.
  • Tutein Nolthenius JB; Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiencies, Medical University of South Carolina, Barcelona - Spain.
  • Figueiredo R; Department of Immunology, H.U. of Gran Canaria Dr. Negrin, Las Palmas de Gran Canaria, Gran Canaria - Spain.
  • Labazuy SS; Pharming Technologies B.V., Leiden - The Netherlands.
  • Gil A; Pharming Technologies B.V., Leiden - The Netherlands.
Glob Reg Health Technol Assess ; 11: 124-130, 2024.
Article en En | MEDLINE | ID: mdl-38784663
ABSTRACT

Introduction:

Activated phosphoinositide 3-kinase (PI3K)δ syndrome (APDS) is an ultra-rare inborn error of immunity (IEI) combining immunodeficiency and immune dysregulation. This study determined what represents value in APDS in Spain from a multidisciplinary perspective applying multicriteria decision analysis (MCDA) methodology.

Methods:

A multidisciplinary committee of nine experts scored the evidence matrix. A specific framework for orphan drug evaluation in Spain and the weights assigned by a panel of 98 evaluators and decision-makers was used. Re-evaluation of scores was performed.

Results:

APDS is considered a very severe disease with important unmet needs, including misdiagnosis and diagnostic delay. Current management is limited to treatment of symptoms with off-label use of therapies supported by limited evidence. Therapeutic benefit is partial, resulting in limited disease control. Haematopoietic stem cell transplantation (HSCT), the only potential curative alternative, is restricted to a reduced patient population and without evidence of long-term efficacy or safety. All options present a limited safety profile. Data on patients' quality of life are lacking. APDS is associated with high pharmacological, medical and indirect costs.

Conclusions:

APDS is considered a severe disease, with limited understanding by key stakeholders of how treatment success is assessed in clinical practice, the serious impact that has on patients and the associated high economic burden. This study brings to light how MCDA methodology could represent a useful tool to complement current clinical and decision-making methods used by APDS experts and evaluators.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Glob Reg Health Technol Assess Año: 2024 Tipo del documento: Article Pais de publicación: Italia
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Glob Reg Health Technol Assess Año: 2024 Tipo del documento: Article Pais de publicación: Italia