Presentation to the Clinical Trials Transformation Initiative Steering Committee

This visit was during a sunny day in Cleveland. If you are a Clevelander, you know how rare that is; you almost need close your eyes because you are not used to that much sunshine. However, inside the Cleveland Clinic Coordinating Center for Clinical (C5) Research, we had eye-opening meetings with the Director, Dr. Michael Lincoff, and other leadership and staff. I thank them all for their hospitality and openness to us, especially hosts Tim Crowe and Marlene Goormastic. I will limit this week note to a brief selection of important lessons learned from our visit with this Academic Research Organization.

The ever-increasing complications in the protocols and audit processes were unanimously discussed as a burden to clinical trials. These seem to stem from the overly conservative interpretations of regulatory guidelines which drive the process towards a multitude of details while derailing it from the actual purpose of conducting trials, which is bringing new, effective, safe medicines to patients. We note here that in proposing its guidelines, the regulators are challenged by two risks: the risk of adverse events because of approving unsafe medicine, and the risk of limiting access to innovative medicine because of overly stringent approval criteria. Interestingly, this is what we discussed in our week note number 7. Another interesting finding was the disconnect between protocol designers and practice: “The perfect study is designed in an ivory tower,” as the C5 Regulatory and Quality Assurance Manager, Kelly Breznia, put it. It seems some studies are designed with scientific data collection in mind, without paying enough attention to what is actually feasible in clinical settings. It also appears that true representative participation from patients is yet to be observed because, on average, about 10% of patients would agree to participate in research. This ratio, of course, goes up when patients have a rare disease or a disease that has progressed considerably.

In this visit we were also introduced to some very novel ideas. Dr. Lincoff stated that we need a cultural change on how we perceive research, a paradigm shift in which research and medical practice are truly integrated. In my view, this seemingly simple, yet powerful idea needs to be promoted and implemented. Think about it. Why do we perceive clinical research as something separate, when in reality, medical practice ultimately depends on the results of research. Another important idea is “zero-based budgeting,” in which we begin our projects with a budget of zero, and we add budget lines only if we find these additions absolutely necessary, and not because they appeared in a previous project.

I would like to close with emphasizing that we cannot passively benefit from findings of research; we as a patient population should also take part in research. This is yet another cultural change, where in the current system, individual benefits trumps societal benefits.

The picture below shows how sunny that day was, and also shows our enjoyment on reflecting on our day at the Cleveland Clinic.

Mehdi Jalalpour