Concordancia entre las expectativas y preferencias de los pacientes y los criterios de evaluación de la Agencia Europea del Medicamento / Concordance between expectations and preferences of patients and evaluation criteria of the European Medicines Agency

RESUMEN

Objetivo: Los criterios de autorización de comercialización de medicamentosde la Agencia Europea del Medicamento se reflejan en losEuropean Public Assessment Reports. El objetivo es describir las expectativasy preferencias de nuestros pacientes externos oncohematológicoscon respecto a sus tratamientos orales, y evaluar la concordancia con losresultados de los European Public Assessment Reports.Método: Se elaboró una encuesta sobre las expectativas y preferenciasde los pacientes oncohematológicos respecto a la supervivencia globaly calidad de vida, con tres ítems: expectativas sobre el tratamiento, preferenciasde beneficio y disposición a recibir tratamientos novedosos conresultados inmaduros. Se revisaron los European Public Assessment Reportsde los fármacos indicados. Se utilizó el índice kappa (κ) para evaluar laconcordancia entre las expectativas y preferencias de los pacientes respectoal beneficio en supervivencia global y calidad de vida descrito enel European Public Assessment Report correspondiente. La concordanciaentre la disposición de los pacientes a recibir nuevos tratamientos y losresultados de los European Public Assessment Reports se evaluó mediantela concordancia absoluta (Ao). Resultados: Se incluyeron 29 participantes y se consultaron 19 EuropeanPublic Assessment Reports diferentes.

ABSTRACT

Objective: The European Medicines Agency’s marketing authorisationcriteria for drugs are reflected in the European Public Assessment Reports.The objective is to describe the expectations and preferences of our oncohematologicaloutpatients with respect to their oral treatments, and toevaluate the concordance with the results of European Public AssessmentReports.Method: A survey of onco-hematological patients’ expectations andpreferences about overall survival and quality of life was developed, withthree items: expectations on treatment, preferences of benefit and willingnessto receive novel treatments with non-definitive results. European PublicAssessment Reports of the indicated drugs were reviewed. Kappa index(κ) was used to assess the agreement between patients’ expectations andpreferences respect to the benefit in overall survival and quality of lifedescribed in the corresponding European Public Assessment Report. Concordancebetween willingness of patients to receive novel treatments andEuropean Public Assessment Reports results was evaluated by absoluteagreement (Ao). Results: There were 29 participants, and 19 different European PublicAssessment Reports were consulted. Patients’ expectations about theirtreatment: 82.1% expected improvement in overall survival and qualityof life; the κ value between expectations and results of European PublicAssessment Reports was 0.091 (confidence interval 95%: –0.025 to0.207). Patients’ preferences about benefit of their treatment: 92.6% preferredquality of life; the κ value was 0.016 (confidence interval 95%:–0.127 to 0.160). Willingness to receive novel treatments: 82.1% participantsdemanded benefit in overall survival or quality of life; exigenceswere met in Ao = 53.6% of patients.