Résumé
OBJECTIVE:We refer to the effectiveness (known as pragmatic or real world) and efficacy (known as explanatory or desired or ideal world) of interventions. However, these terms seem to be randomly chosen by investigators who design clinical trials and do not always reflect the true purpose of the study. A pragmatic-explanatory continuum indicator summary tool was thus developed with the aim of identifying the characteristics of clinical trials that distinguish between effectiveness and efficacy issues. We verified whether clinical trials used the criteria proposed by the indicator summary tool, and we categorized these clinical trials according to a new classification.METHOD:A systematic survey of randomized clinical trials was performed. We added a score ranging from 0 (more efficacious) to 10 (more effective) to each domain of the indicator summary tool and proposed the following classifications: high efficacy (<25), moderate efficacy (25-50), moderate effectiveness (51-75), and high effectiveness (<75).RESULTS:A total of 844 randomized trials were analyzed. No analyzed trials used the criteria proposed by the indicator summary tool. Approximately 44% of the trials were classified as having moderate effectiveness, and 43.82% were classified as having moderate efficacy.CONCLUSIONS:Most clinical trials used the term “efficacy” to illustrate the application of results in clinical practice, but the majority of those were classified as having moderate effectiveness according to our proposed score. The classification based on the 0-100 score is still highly subjective and can be easily misunderstood in all domains based on each investigator’s own experiences and knowledge.
Sujets)
Humains , Essais contrôlés randomisés comme sujet/statistiques et données numériques , Résultat thérapeutique , Bibliométrie , Essais cliniques comme sujet/classification , Plan de recherche , Essais contrôlés randomisés comme sujet/classificationRésumé
Estudos de não-inferioridade são modelos experimentais desenvolvidos com o objetivo de determinar se um novo tratamento ou procedimento não é menos eficaz que outro já estabelecido e considerado como controle. São de grande importância no estudo de tratamentos em que o uso de placebo é inviável. Requerem metodologia diferente dos estudos clássicos, chamados de estudos de superioridade, especialmente no planejamento e análise estatística. O presente artigo é uma revisão dos principais elementos diferenciais entre estudos de não-inferioridade e estudos tradicionais. Existe ainda em nosso meio um considerável grau de desconhecimento sobre o uso correto dessa metodologia, o que certamente compromete a validade de alguns estudos clínicos.
Non-inferiority trials are experimental models designed to determine whether a new treatment or procedure is not less effective than an established one, which is considered as standard. They are especially important in the assessment of treatments in which the use of placebo is impracticable. They differ substantially from the classical superiority trials and require different approach, especially in the planning and the data analysis. This paper is a review of the key differences between non-inferiority and traditional clinical studies. There is a considerable amount of misunderstanding on the correct use of this experimental design, which certainly compromises the credibility of some clinical assessments.
Sujets)
Humains , Essais cliniques comme sujet/classification , Placebo/classificationRésumé
Disciplina situada na confluencia das ciencias medicas, humanas, biologicas e fisicas, a homeopatia necessita de uma abordagem diversificada. Sua avaliacao nao tem sentido a menos que ela seja adaptada a sua realidade pratica, ao seu metodo e aos seus conceitos de base. Ela necessita, alem de criterios proprios da farmacologia classica, criterios especificos: sensibilidade de sistemas biologicos, protocolos clinicos adaptados a uma terapeutica individualizada, estudo biofisico de interacoes soluto-solvente e aprofundamento dos problemas conceituais