White Paper

Master Protocol Studies: Technology Considerations For The 'Never Ending' Clinical Trial


The underlying goal of using master protocols is to increase efficiency and expedite drug development for critical unmet need. Now that the FDA is encouraging creativity in trial designs, it is important for all stakeholders (sponsors, CROs, vendors, etc.) to adapt processes to follow the science and ultimately bring medicines to patients, faster. This white paper addresses the complexities that come with these trial designs, and how technology – specifically randomization and trial supply management (RTSM) solutions – are critical to run flexible, robust and quality driven trials.

As defined by the FDA, a master protocol is “a protocol designed with multiple sub-studies which may have different objectives and involves coordinated efforts to evaluate one or more investigational drugs in one or more disease subtypes within the overall trial structure.” Simply put, a master protocol acts like a map showing many routes to get to a destination where the sponsors don’t know in which direction their study will evolve, until the trial is under way. There are two types of trial designs that fall under Master Protocols: Basket Trials and Umbrella Trials.

A basket trial involves a single investigational drug or drug combination that is studied across multiple cancer populations defined by disease stage, histology, number of prior therapies, genetic or other biomarkers, or demographic characteristics. An umbrella trial is designed to evaluate multiple investigational drugs administered as single drugs or as combination drugs in a single disease population.

There is an understanding that master protocol studies will look dramatically different in few years (i.e., 20+ new cohorts, etc.). These trials need to be designed in a way that can support multiple protocols, amendments, patient populations and supply chains, all while maintaining quality and patient safety.

What is needed to disrupt this process and help streamline the execution of master protocol studies? While there are many aspects of a master protocol that can be streamlined, the focus on this white paper is on the use of technology, specifically Randomization and Trial Supply Management (RTSM).

Master protocols can become incredibly complex as sponsors are following where the science leads. Traditional RTSM systems were not built to support the level of flexibility and agility needed from a technology standpoint. Therefore, a robust, modern, 100% configurable RTSM is critical to enable the goals of these studies – increasing overall study efficiency and expediting drug development.

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