Friday, September 09, 2011

Is it time to change the clinical monitoring practice in clinical trials?

In industry, the current monitoring practice relies on ‘on-site monitoring’ and 100% source data verification (on all data fields). This process is very costly and is one of the main reasons that the clinical trials now become so expensive. This process is really the most conservative interpretation of ICH E-6 guidance on
Guideline for Good Clinical Practice and the 1988 FDA’s “Guidance for the Monitoring of Clinical Investigations”. These guidance only require “the sponsor should ensure that the trials are adequately monitored” and leave the door open in terms of the frequency of the monitoring and the approaches of the clinical monitoring. In industry, the conduct of the clinical trials are highly regulated. Sponsors are usually take the most conservative approaches no matter how costly these approaches are.

Will ‘on-site monitoring’ be really effective? Will 100% source data verification really be needed? Should we identify the new ways to conduct the cost-effective clinical monitoring?

Last month, FDA withdrew its 1988 guidance on “Guidance for the Monitoring of Clinical Investigations” and issued its draft guidance “Oversight of Clinical Investigations - A Risk-based Approach to Monitoring”. The newly issued guidance suggested it is acceptable to use alternative approaches (such as remote monitoring, centralized monitoring, risk-based monitoring). The guidance also suggested that the source data verification should be focused on critical fields (key efficacy and safety variables) and less than 100% source data verification on less important fields may be acceptable. The guidance gives a clear signal that the Sponsors are encouraged to explore the cost-effective ways to conduct the clinical monitoring instead of solely relying on the on-site monitoring.

If this guidance gets implemented, we may expect the increasing role of statisticians in clinical monitoring, especially the centralized monitoring. Currently, statisticians will identify the issues in the late stage of the clinical trials when statisticians or statistical programmers start to perform the data analyses. The new guidance says:

“…notably, the advancement in EDC systems enabling centralized access to both trial and source data and the growing appreciation of the ability of statistical assessments to identify clinical sites that require additional training and/or monitoring.”

“Centralized monitoring is a remote evaluation carried out by sponsor personnel or representatives (e.g., data management personnel, statisticians, or clinical monitors) at a location other than the site(s) at which the clinical investigation is being conducted.”

2 comments:

Carl Anderosn said...

I agree that more statisticians will be necessary in monitoring clinical trials. This is not only necessary for centralized monitoring, but also for what FDA has been calling, "compliance monitoring." This can mean looking at different trends to determine if data outliers indicate research misconduct or serious noncompliance. I am convinced that biostatisticians at FDA find as much fraud as the inspectors, if not more.

Web blog from Dr. Deng said...

During DIA conference, one officer from BIMO (BIoresearch MOnitoring program) in FDA stated that FDA will require the sponsor to provide an analysis data set containing the site-level summary results (for example the treatment difference for each site). This data set will facilitate the agency to identify any outliers or any issues for auditing. This request is in line with FDA's position "Prioritization of Clinical Trial Inspections" as indicated in their website.

http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/ucm226306.htm

"Prioritization of Clinical Trial Inspections. FDA Centers are developing new approaches for improving the process for selecting clinical investigators and other entities for inspection, both at the pre-approval stage and earlier in the product development process. The Center for Drug Evaluation and Research (CDER) is piloting a tool that uses a sponsor-submitted clinical analysis dataset and assesses clinical sites associated with an application for inspection using a risk-based prioritization process model. This tool also has automated features to streamline the inspection request process, and is expected to provide a more efficient, analytical, and timely approach to clinical site inspection and site monitoring. Similarly, the Center for Devices and Radiological Health (CDRH) and the Center for Biologics Evaluation and Research (CBER) have early intervention programs whereby ongoing studies are identified for inspection using risk-based criteria. "

However, this request will add additional burden to the sponsor side and the request data set may not be very useful if the # of subjects at each site is small.

CBER representative later clarified that this request for site-level summary data set was only for CDER division.