Open AccessBeyond Placebo: Alternative Options to the Randomized Control Trial Design in Rare Disease Studies
Author(s) -
Preethi Sriram,
AUTHOR_ID
Publication year - 2020
Publication title -
clinical trials and practice: open journal
Language(s) - English
Resource type - Journals
ISSN - 2771-7380
DOI - 10.17140/ctpoj-3-110
Subject(s) - clinical trial , protocol (science) , clinical study design , medicine , randomized controlled trial , placebo , disease , intensive care medicine , research design , alternative medicine , rare disease , orphan drug , medical physics , surgery , bioinformatics , pathology , social science , sociology , biology
Unique difficulties and challenges can arise for rare diseases and orphan disease indications within a clinical trial. Some of the challenges encountered by trials that are working on rare diseases can include recruitment and enrollment. One of the explanations for the challenges that are encountered in rare disease trials is because of the trial design of study, where the investigational product is being compared to the placebo. This review paper discusses the usage of placebo and when other options could be taken into consideration while creating the protocol, specifically with consideration to rare disease studies. It is essential for the investigators and those designing the protocol to consider alternative options from the standard randomized controlled trial. The aim of this paper is to review alternative trial design options. The trial designs discussed in this review paper include crossover trials, single arm studies and historical data, and n-of-1 trials.