Factors affecting efficiency of Adaptive Platform Trials (APTs)

Clinical trial activity is growing globally and represents significant expense to achieve potential patient benefits. New designs such as adaptive platform trials (APTs) offer promise. APTs are complex and costly to establish requiring extensive coordination, legal and ethical considerations, and significant design investments. There are however many potential benefits to patients, industry, trialists, governments and other funders. Some of these benefits include: economies of scale, the ability to evaluate more than one intervention and treatment domain, more efficient sample size design including smaller control groups, faster evaluation timeframes, the flexibility to add/drop and adapt interventions and to prioritise interventions with greater initial promise. There is importantly also an opportunity to strengthen engagement and ongoing relationships with patients and community which is essential to trial success. However quantitative evidence on the costs, efficiency, and cost-effectiveness of running APTs compared to running conventional randomised controlled trial (RCT) is limited. There is a need to understand in which contexts APTs are a favourable design choice. It is also essential with new innovative trial designs that we harness data collected to understand more about how to refine and improve trials and ultimately outcomes for patients.