RBM Pilot Trial
A famous saying tells us that “experience often comes after one needs it”. Risk-based monitoring (RBM) is a major development and often fails in implementation due to its additional complexity and the variety of opinions of various stakeholders. In order to gain experience before one needs it, the RBM consortium advises that a pilot trial be run and a staging approach chosen, instead of doing the “big bang” strategy

[1]. This article will help you to select the right trial from your portfolio so that you can minimize potential hazards and pitfalls.

When a CRO or a sponsor decides to begin embracing RBM, a question usually arises: what trial best suits the pilot? Bearing in mind that RBM introduces its own risks [2], it is essential to understand that the choice of a clinical trial could be decisive for the success or failure of an RBM initiative.

What to ensure before RBM?

The TransCelerate group provides basic advice on what should be prepared before launching the RBM and centralized monitoring [3].

  1. It is advisable to determine that well-defined processes exist for conducting monitoring away from sites.
  2. Enabling technologies for RBM, aimed on the translation of data into information that can result in appropriate actions, are available and your staff should be trained to use them.
  3. Ensure that roles(s) are clearly defined—who should be conducting central monitoring and RbM activities—and that the skills and capabilities needed to fulfil these roles are well understood.
  4. Ensure that organization alignment for those conducting central monitoring activities has been established.
  5. The path from risk assessment to risk identification to risk management must be clear and agreed by all stakeholders.
  6. Ensure that well-defined critical success factors have been established for the implementation of central monitoring.
  7. Ensure that corporate risk tolerance (RT) in the context of the study is clearly defined. RT is the corporate’s risk appetite within a trial. Are you ready to test certain new monitoring procedures?

When the above mentioned points have been checked, the choice of a suitable pilot trial becomes an important question on the corporate agenda.

  1. RbM early adopters tend to concentrate their efforts on phase III studies [3]. However, from the RT point of view, phase IV also suits the requirements of an RbM pilot well.
  2. The usual time horizon of a trial RbM pilot is around 1–1.5 years. It may happen that a trial goes on further, and the RbM lessons become obvious.
  3. Straightforward protocol design. Unnecessary complexity in a clinical protocol is never a good characteristic, and in an RbM pilot it is worse. During the establishment of RbM one experiences additional workload; therefore, it is particularly important to have a simple and straightforward protocol design. Try to select a clinical trial with only one or two primary endpoints, with only a few secondary endpoints, and with a simple visit schedule.

An RbM pilot gives your team a chance to gain experience, to learn how to react on risk indicator escalation and to be able to take actions based on these escalations accordingly. It gives you a chance to learn the lessons and avoid certain issues in the future by rolling out the RbM for the whole portfolio. Besides, it provides inspiration for success stories, which are to be communicated to further RbM sceptics within a company or outside.

It is a fact that there is much homework needed before RbM can take place and, like any major change, RbM deserves a sound change management plan. Ensure the team’s open-mindedness and readiness to try RbM and to learn from the experience, then start small and try again and again until success.

References

[1] M. Alsumidaie, M. Proupín-Pérez, A. Andrianov, B. Widler, P. Schiemann, and J. Schenk, “RbM Guidance Document: Ten Burning Questions about Risk-Based Study Management.” Available from: http://www.appliedclinicaltrialsonline.com/rbm-guidance-document-ten-burning-questions-about-risk-based-study-management. [Accessed: 26 Jan 2015].

[2] A. Andrianov, PhD, “Can RBM Influence the Data Quality and Patient Safety Inversely?” Applied Clinical Trials, 31 Mar 2015.

[3] J. Gough, B. Wilson, M. Zerola, P. Wallis, L. Mork, D. Knepper, and H. Achenbach, “Defining a Central Monitoring Capability Sharing the Experience of TransCelerate BioPharma’s Approach, Part 2,” Therapeutic Innovation & Regulatory Science, vol. 50, no. 1, pp. 8–14, Jan. 2016.