Perspectiva de los pacientes y variables económicas como objetivo en los ensayos clínicos, valores añadidos a los parámetros clínicos / Patients’ perspective and economic variables as objectives in clinical trials: added value to clinical parameters

Objetivo. Las medidas de salud autopercibidas complementan los parámetros clínicos para medir la eficacia. En este estudio se evalúa el uso de cuestionarios para la medida de salud desde la óptica del propio paciente como parámetro clínico, incluyendo aquellos que miden la calidad de vida relacionada con la salud (CVRS), y la frecuencia de inclusión de medidas relacionadas con los costes directos e indirectos en el diseño de los ensayos clínicos y de los estudios postautorización. Además, se mide la validez interna de los protocolos y si existe relación entre la calidad metodológica en el diseño del estudio y la inclusión de resultados notificados por el paciente (RNP). Material y métodos. Estudio observacional retrospectivo de los protocolos pertenecientes a ensayos clínicos (EC) y estudios postautorización aprobados por el Comité Ético de Investigación Clínica del centro (2008-2010). Se recogieron datos relativos a: la especialidad médica, patología, calidad metodológica (cuestionario de Jadad, 0-5), inclusión en los objetivos de RNP y/o de costes directos o indirectos. Para aquellos EC que incluían medidas de CVRS, se analizó el tipo de cuestionario utilizado. Para aquellos que no la analizaban, se consideró la pertinencia de su inclusión. Resultados. Se analizaron un total de 70 protocolos (59 EC y 11 estudios postautorización), 37 (52,8%) de los cuales incluyeron resultados notificados por el paciente, 3 (4,3%) como variable primaria. El análisis de datos por áreas terapéuticas puso de manifiesto que endocrinología, neurología, digestivo y cardiología fueron las áreas donde los RNP fueron analizados en mayor proporción. La puntuación media de la validez interna fue de 2,8, observándose una mayor calidad metodológica en los protocolos con mayor inclusión de RNP. Únicamente 13 (22%) EC y 2 (18,2%) estudios postautorización incluyeron variables económicas. Conclusiones. La aparición de variables económicas en los ensayos clínicos y estudios postautorización evaluados ha sido baja, en cambio, en más de la mitad de los protocolos revisados se han incluido instrumentos para medir resultados notificados por los pacientes, lo que refleja la importancia de estos parámetros en la valoración de la eficacia de los tratamientos con medicamentos, si bien su uso todavía no es sistemático(AU)

Objective. Patient-reported outcome (PRO) measures complement traditional biomedical outcome measures. The purpose of this study was to evaluate the use of PRO measures including health-related quality of life (HRQoL) questionnaires as a measurement of efficacy and the frequency of inclusion of economic variables related to direct and indirect costs in the design of clinical trials and phase IV observational studies. Moreover, for the trials quality score were measured, and if there were any relationship between the quality study design score and the PRO inclusion. Material and methods. Retrospective observational study of the clinical trials and phase IV observational studies approved by a Clinical Research Ethics Committee (2008-2010). We gathered data concerning general aspects including medical specialty, pathology, methodological quality based on Jadad scale (0-5), inclusion of PRO and economic variables. For clinical trials including HRQoL measurements, we analysed the type of questionnaire in use. Where there were no HRQoL measurements, we analysed if their inclusion would have been proper or not. Results. A total of 70 protocols (59 CTs and 11 phase IV observational studies) were analysed; 37 (52.8%) included PRO measures, and 3 protocols (4.3%) used them as a primary endpoint. Data analysis by therapeutic area showed that PRO measures were most commonly studied in the fields of endocrinology, neurology, digestive diseases, and cardiology. The average quality score for the trials was 2.8. The trials with more PRO inclusion in their end points had a significantly higher quality score. Only 13 (22%) clinical trials and 2 (18.2%) phase IV observational studies included economic variables. Conclusions. The emergence of economic variables in clinical trials and phase IV observational studies evaluated was low, however, more than half of the revised protocols have included PRO measures, reflecting the importance of these parameters in the assessment of the effectiveness of drug treatments, although its use is still not systematic(AU)

[Patients' perspective and economic variables as objectives in clinical trials: added value to clinical parameters]. / Perspectiva de los pacientes y variables económicas como objetivo en los ensayos clínicos, valores añadidos a los parámetros clínicos.

OBJECTIVE: Patient-reported outcome (PRO) measures complement traditional biomedical outcome measures. The purpose of this study was to evaluate the use of PRO measures including health-related quality of life (HRQoL) questionnaires as a measurement of efficacy and the frequency of inclusion of economic variables related to direct and indirect costs in the design of clinical trials and phase IV observational studies. Moreover, for the trials quality score were measured, and if there were any relationship between the quality study design score and the PRO inclusion. MATERIAL AND METHODS: Retrospective observational study of the clinical trials and phase IV observational studies approved by a Clinical Research Ethics Committee (2008-2010). We gathered data concerning general aspects including medical specialty, pathology, methodological quality based on Jadad scale (0-5), inclusion of PRO and economic variables. For clinical trials including HRQoL measurements, we analysed the type of questionnaire in use. Where there were no HRQoL measurements, we analysed if their inclusion would have been proper or not. RESULTS: A total of 70 protocols (59 CTs and 11 phase IV observational studies) were analysed; 37 (52.8%) included PRO measures, and 3 protocols (4.3%) used them as a primary endpoint. Data analysis by therapeutic area showed that PRO measures were most commonly studied in the fields of endocrinology, neurology, digestive diseases, and cardiology. The average quality score for the trials was 2.8. The trials with more PRO inclusion in their end points had a significantly higher quality score. Only 13 (22%) clinical trials and 2 (18.2%) phase IV observational studies included economic variables. CONCLUSIONS: The emergence of economic variables in clinical trials and phase IV observational studies evaluated was low, however, more than half of the revised protocols have included PRO measures, reflecting the importance of these parameters in the assessment of the effectiveness of drug treatments, although its use is still not systematic.