Making an Argument to Revitalize Clinical Trials

March 3, 2011

Researchers are pushing for a new system of clinical trials in which different companies team up and share the costs to test new therapies and devices.

With the current clinical trial process in the United States on shaky ground due to smaller groups of willing patient participants, skyrocketing costs, and expanding regulatory requirements, a group of researchers are proposing a new model to revitalize the research engine.

Collaborative trials—in which different companies team up and share the costs to test new therapies or devices—will ensure safe and effective treatments become available more quickly and at a lower cost than they do today, wrote Daniel Ryan, MD, and colleagues from the University of Rochester Medical Center in a column published in the New England Journal of Medicine.

“The process of approving drugs for clinical use is progressively reaching an impasse in certain areas, and the problem will only be made worse with discoveries relating to personalized medicine, because there will be more drugs but smaller patient populations to test them in,” said Ryan, who is chairman of the Department of Pathology and Laboratory Medicine at URMC. “This is a way to restructure the system that will help us bring more good drugs forward for patient care.”

A collaborative clinical trial could involve similar therapies from different companies, such as two drugs to lower blood pressure. Or, the trials could test a combination of different therapies from different companies, with each therapy designed to target a unique pathway or mechanisms involved in a more complex disease.

There are many advantages to collaborative trials, including easier patient enrollment, especially when a disease affects a small number of patients, or a therapy can only be tested in a small subgroup of patients with a specific genetic mutation. Competition for patients can slow the enrollment process and ultimately the time it takes to get results.

“Rather than two or three separate clinical trials competing for patients, things can get done faster by companies coming together and conducting a single trial,” said Arthur J. Moss, MD, a cardiologist at the Medical Center. “You can also use a single control group of patients, which cuts the number of participants needed and subsequently time and costs.”

Another major advantage is the ability to directly compare several new therapies, or new versus old therapies, since they are all being tested in the same patient population, with the same set of rules, measuring the same set of outcomes.

“Treatment A may work compared to control in one trial, and treatment B may also work compared to control in another trial, but we wind up with no information on how to compare A and B,” said Charles Francis, MD, a professor in the Departments of Medicine and Pathology and Laboratory Medicine at URMC.

“In the end, if we can come up with a more robust trial design that actually compares the effectiveness of different treatments, we are right in line with President Obama’s healthcare initiative, which calls for providers and researchers to look at the things we’re doing and study whether or not they are effective,” added Ryan.

Source: University of Rochester Medical Center