All Times EDT
Keywords: Platform trials, Alzheimer's
Background: Drug development trials have demanded more and more resources over time, in terms of patients, investigator time, calendar time and money. This is particularly a problem in Alzheimer’s dementia, where prevention studies take many years and require many patients in order to demonstrate a delay in the disease development. Within oncology, one has started using more complex trial designs, also covered by the term master protocols. Time is now ripe for expanding these designs into other disease areas. Objective: To create a proof-of-concept platform trial within Alzheimer’s dementia. Methods: Complex trials challenge the classical viewpoint that a clinical trial studies a single drug in a single disease. One such type of design is platform trials, also known as a trial machine, where multiple drugs are tested in the same disease, but in such a way that treatments enter when they become available and exit when enough knowledge has been gathered, showing either futility or efficacy. Besides the operational efficiency, a platform trial allows for sharing the placebo group, which reduces the number of patients receiving an inactive treatment. Results and Conclusions: This will be illustrated with the EPAD project. EPAD is a public-private partnership running two trials. The first trial studies untreated healthy participants. This study serves as feed-in to the second study, which is a proof-of-concept platform trial for treatments that are supposed to prevent the development of Alzheimer’s disease. The trial setup was developed so that it was ready to start, but no new investigational drugs entered so the trial never really started. The pro’s and con’s of platform trials will be discussed in this light.