Moving Clinical Trials Forward

First, my criterion for success in the drug development enterprise is to “fail quicker.” By that I mean deciding at the earliest phases that a compound is either not working or to too toxic. The key ingredient in this scenario is to develop discriminating bio-markers that will predict success in a larger confirmatory trial. The investment of people, time, and money for large, pivotal, global trials is enormous, and so finding out early on that it’s not going to work allows you to put it on the shelf and move on to something else. Secondly, drugs that we develop at Genzyme for rare genetic diseases, cancer, or immune mediated diseases are going to be used in all parts of the world. To that end, Genzyme has a very large global footprint for a biotech company. Global development and strategic pricing are complex, but critical to our culture and mission.

Maximizing performance
I'm sure we could all do a better job of implementing clinical trials. Developing new medicines is a tough business though. At Genzyme, we do our work in teams. It’s a people and process balance, and so we try to match the right people with the right team. Each individual team member brings their own personality and functional content expertise to the effort. If you study the flow process diagrams that have been created for clinical development, with all the things that have to occur in parallel, it's just mind-boggling to think that we actually pull it off once in a while.

Driving forward
In order to drive the industry forward we need to embrace and enhance our performance in two key areas. Patient safety is the most important issue that confronts the drug development enterprise today. We must understand the safety profile of our drug before it gets to market, or at least that we put in place procedures to systematically evaluate the rare safety events of the drug after it's approved. Most clinical trials may test 500 or 1000 patients for efficacy, but you need many, more exposures in order to see the rarer safety signal. That said, a drug can never be 100 percent safe, and we have to live with that; it’s the risk-benefit ratio that is key to determining the ultimate value of a new drug to society and to individual patients. 

Secondly, the pharmaceutical industry is not in a good place right now. Society and governments are skeptical of our motives. What I believe is that we focus on the patient; everything else will follow. We must turn around this trust issue. We must help people understand what we do and why we have a value to society.

About the contributor

Richard Polisson joined Genzyme in 1997. He manages the corporate clinical trials for programs in osteoarthritis, cartilage repair, tissue engineering, surgical wound repair, transplantation, immune mediated diseases, diagnostics, and neurologic disease. Polisson has formal training in the quantitative methods of clinical investigation, and has 28 years of experience in patient-orientated and translational research in academia, industry and at the National Institutes of Health. Before joining Genzyme, he was Clinical Director of the Arthritis Unit at Massachusetts General Hospital and Associate Professor of Medicine at Harvard Medical School