Life sciences companies are simultaneously facing three separate, but related forces that will shape the way that they collect and monitor data on the patient/caregiver experience in clinical trials.
First, in accordance with the 21st Century Cures Act, the US Food and Drug Administration (FDA) is developing a series of guidance documents to address the use of patient/caregiver data in regulatory decision making. Thus, Sponsors are actively working to represent the voice of the patient in their regulatory submissions and increasingly are collecting electronic clinical outcomes assessments (eCOA) and/or electronic patient-reported outcomes (ePRO) in their clinical trials.
Second, ICH E6 R2 which was adopted in December 2016 has encouraged sponsors to adopt a more proactive approach to study design, risk management and study monitoring, particularly risk based monitoring. Many regulators have produced guidance documents, including the FDA who has most recently published a Question-and-Answer document intended to facilitate Sponsors’ implementation of Risk-Based Monitoring (RBM). This Q&A further supports the agency’s belief that “risk-based monitoring is an important tool to allow sponsors to identify and address issues during the conduct of clinical investigations.”
Third, the International Council for Harmonization (ICH) has published draft revisions to ICH E8 (R1) to address challenges with designing and executing trials with diverse designs and data sources. The revision emphasizes that the quality of clinical research — required to protect patients and generate reliable and meaningful data — relies on both good design and good execution. This revision also stresses the importance of Quality by Design (QbD) approach to clinical trials, and the need to rapidly identify and address systemic issues.
As the industry moves to adopt patient-centric approaches to clinical trials, there is an opportunity to adopt the best practices of QbD and risk-based methodologies to ensure that patient reported data are effectively being monitored.
In this paper, we discuss the value of using advanced technology — specifically Centralized Statistical Monitoring technology such as - Medidata Detect to oversee subject safety and data quality/integrity in a risk-based manner. Such monitoring is particularly relevant for the monitoring of large data sets, such as eCOA/ePRO data as all regulators agree that a risk based approach including Centralized Statistical Monitoring of data in real time may enable sponsors to be able to identify the reasons for missing data, inconsistent data, protocol violations, or protocol deviations etc. and take corrective actions to minimize the likelihood of these occurring during the remainder of the clinical investigation.