The design and analysis of non-randomized studies: A case study of off-label use of hydroxychloroquine in the COVID-19 pandemic.

This article focuses on several key design and analysis considerations that can improve the scientific rigor of non-randomized studies of off-label drugs. Areas covered The aim of this article is to provide an overview of best approaches that should be considered for non-randomized studies on off-label drugs. In this article, we discuss these approaches in detail and use a non-randomized study by Rivera et al. in Cancer Discovery, which analyzed factors associated with off-label treatments for COVID-19 and their potential impact on 30-day all-cause mortality among cancer and SARS-CoV-2 infected patients, as an example of methods that have been undertaken for COVID-19. Expert Opinion While non-randomized studies are inherently biased, they may be unavoidable in situations such as the COVID-19 pandemic, where researchers need to find an effective treatment quickly. We believe that a well-formed experimental design, high quality data collection, and a well-thought-out statistical and data analysis plan are sufficient to produce rigorous and credible results for making an optimal decision. PMID: 33353432 [PubMed - as supplied by publisher]
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Tags: Expert Opin Investig Drugs Source Type: research