Ten Commandments for Randomized Trials of Pharmacological Therapy for COPD and Other Lung Diseases

We describe the concept of the counterfactual principle as it applies to the treatment of patients and to the randomized trial. We then present ten methodological tenets for the design and statistical aspects of randomized controlled trials evaluating the effectiveness of drugs used in the treatment of several respiratory diseases. They include eight study design and two statistical analysis principles: 1) Study question; 2) Intervention; 3) Study population; 4) Blinding; 5) Run-in period; 6) Follow-up; 7) Outcome; 8) Safety; 9) Intent-to-treat; 10) Covariate adjustment. These tenets are described using mainly examples from trials of pharmacological treatments for COPD, as well as some from asthma and idiopathic pulmonary fibrosis, conducted over the last 30 years. The careful application of these principles in the conduct of randomized trials will provide rigorous studies and improve the validity of results. The ensuing clearer interpretation of findings will permit their well-founded contribution to treatment guidelines and optimal clinical management.PMID:34468248 | DOI:10.1080/15412555.2021.1968816
Source: COPD: Journal of Chronic Obstructive Pulmonary Disease - Category: Respiratory Medicine Authors: Source Type: research