RESUMEN
BACKGROUND: Centralized administrative databases enable low-cost pragmatic randomized trials (PRTs) of drug effectiveness and safety. We simplified the PRT strategy by using designed delays (DD) to evaluate drug policies. OBJECTIVES: To reassess our DD trial of a cost-saving nebulizer-to-inhaler conversion policy and a proposed DD trial of reduced restrictions on Cox-2 inhibitors. RESEARCH DESIGN: We randomized 52 pairs of communities and clusters of physician practices to the policy either on time or after a 6-month delay. Our 2-stage qualitative reassessment comprised: (1) applying criteria for reporting PRTs and (2) assessing DD trials in 3 domains of responsibility: policymakers' decisions, researchers' decisions, and joint decisions involving negotiation. MEASURES: A draft checklist of 22 Consolidated Standards of Reporting Trials (CONSORT). Researchers' recollections of their degree of influence on decisions. RESULTS: DD trials deviated from ideal PRTs in the policymakers' domain: the policies affected mixtures of drugs, users, and illnesses, and implementation was not by strict protocol. Aspects negotiated by researchers and policymakers also deviated from ideal: length of delay; size and location of control group; unit of randomization; additional data collection; and communications to physicians. The DD trials complied better with CONSORT in the researchers' domain of analysis and interpretation. CONCLUSIONS: DD trials can be negotiated with policymakers. Low cost and simplicity of DD trials partly compensate for some limitations for evaluating drug safety and effectiveness. The ethics question of whether a DD is routine evaluation or research depends on its purpose and generalizability.
Asunto(s)
Evaluación de Medicamentos/métodos , Seguro de Servicios Farmacéuticos , Formulación de Políticas , Ensayos Clínicos Controlados Aleatorios como Asunto/métodos , Proyectos de Investigación , Colombia Británica , Análisis Costo-Beneficio , Inhibidores de la Ciclooxigenasa 2/administración & dosificación , Inhibidores de la Ciclooxigenasa 2/economía , Toma de Decisiones en la Organización , Costos de los Medicamentos , Evaluación de Medicamentos/economía , Evaluación de Medicamentos/ética , Humanos , Seguro de Servicios Farmacéuticos/economía , Seguro de Servicios Farmacéuticos/estadística & datos numéricos , Relaciones Interprofesionales , Programas Nacionales de Salud/economía , Programas Nacionales de Salud/organización & administración , Programas Nacionales de Salud/estadística & datos numéricos , Nebulizadores y Vaporizadores/economía , Ensayos Clínicos Controlados Aleatorios como Asunto/economía , Ensayos Clínicos Controlados Aleatorios como Asunto/éticaRESUMEN
Medical conduct relies on the efficacy and tolerability of a given pharmacotherapy. On the basis of an experimental comparison of different treatment options, a clinical trial provides the foundation for an evidence-based medicine that complies with state-of-the-art scientific practice. The quality of the data assessment determines the clinical relevance of the study results. Clinical trials are classified into Phases I-IV to ensure the development of an effective and safe pharmaceutical. The investigator is responsible for being experienced in the relevant area of expertise, for conducting the study according to the investigational plan, and for complying with ethical guidelines and drug safety.