Patricia Devitt Risse, PharmD, President, Precision for Medicine, Oncology and Rare Disease, looks at how implementing new technology will change how clinical trials are run and predicts how studies will look in 10 years.
Utilizing “real-world data” in clinical trials
The single activity that will change clinical trials the most in the next 10 years will be the move of patient medical information into electronic format, enabling the utilization of “real-world data” in powerful ways, including to implement synthetic trial arms.
Currently, randomized clinical trials comparing “new” vs “standard treatment” are the gold standard for assessing drug effect. This requirement forces the total size of the trial to increase to allow for enrollment into a control arm therefore adding significant time and cost by mandating enrollment into an arm to demonstrate the impact of standard therapy.
Historical controls are more efficient and economical, but have not been widely applied due to a concern for bias of the selected information sources. By establishing and confirming robust eligibility requirements, defining general criteria of comparable endpoints, and applying data from a large and varied patient source, the introduction of selection bias should be effectively mitigated. Eliminating this patient pool from the enrollment target will speed the development process and dramatically increase our capacity to test new therapies, making the “system” more efficient and accelerating the pathway of novel agents to proof of concept and approvals.
Virtual trial challenges
Virtual trials bring study screening, recruitment, and participation directly to patients at home. This should allow for faster enrollment across a broad source of patients which can accelerate overall development timelines. The convenience of virtual participation should also improve retention with fewer drop-outs and replacements required. The power of technology platforms, virtual investigator networks and smart devices enable this to occur, but key challenges have yet to be addressed for broader application.
From a technology compliance point of view, regulatory requirements for the remote data capture tools must be clearly defined and articulated, including the assurance of privacy and protection of individual patient data. The issue of selection bias must also be considered since patients have varying skills sets as well as accessibility to the technology. Remote scanning, sampling, and telemonitoring techniques must be confirmed and validated to ensure appropriate assessment of treatment impact and potential toxicities. These challenges will take years to overcome, with assurance of alignment of the regulatory authorities, before any significant shift will occur in trial approach.
Clinical trials in 10 years
Clinical trials will be personalized approaches, with individualized and data-driven precision products to more accurately direct therapy based on unique, patient-centric molecular profiles. Real-world data and telemedicine will be leveraged to identify and communicate with targeted patients with unique mutations or diagnoses to help facilitate engagement and enrollment, with improved freedoms for participation with enhanced technology applications.
Robust biomarker platforms will drive patient selection to help optimize therapeutic impact and overall patient benefit. Monitoring of clinical data will largely be remote with limited need for on-site visits and inspections because of the electronic source record anonymous data transfer to the clinical database. Products with unique targets that can provide larger benefit to fewer patients will outnumber “me too” approaches.
The regulatory agencies will remain focused on leveraging technology and translational data to support shorter development timelines and approvals, to speed delivery of benefit to patients in need, and to improve overall efficiencies and cost control of conducting trials.