Blazing a shorter trail between the lab and patients

“This is a very expensive and slow process, which we as an institution are trying to carry out more effectively,” says M. D. Anderson President John Mendelsohn, M.D., who has served on a number of national committees sponsored by the Institute of Medicine of the National Academy of Sciences to improve the clinical trial process.

“There are three main areas the country needs to address in which M. D. Anderson can provide leadership,” Mendelsohn says.

First, we need to better assess the risk-benefit balance of a potential new drug. “Over the past decade or so I think there has been tremendous emphasis on avoiding risk, sometimes at the expense of exploring benefits,” Mendelsohn says.

“A person who has advanced lung cancer, and without a miracle only has a few months to live, might be willing to accept certain risks to try something new. But that may be very different from a person who has diabetes and expects to live 30 or 40 years longer, who might be more cautious about the risks of taking a new drug.”

A variety of veto points to avoid risk are built into the clinical trial approval process. The U.S. Department of Health and Human Services oversees all three of the federal agencies that conduct reviews — the National Cancer Institute, U.S. Food and Drug Administration and Office for Human Research Protections. These should consolidate their reviews into a single, seamless process, Mendelsohn says.

Second, we need to adequately value clinical research at all levels. Once a pharmaceutical company gets a drug approved for one type of cancer, the drug can be prescribed for others, reducing the company’s incentive to conduct additional trials. In addition, companies often lack interest in comparing new drugs and in studying combinations of new therapies.

That often leaves the NCI or individual cancer centers to conduct the trials.

“The NCI budget to pay for the costs of clinical investigations is not nearly enough to cover these costs,” Mendelsohn says. The NCI often covers less than half the cost of a trial. “We need to place a higher value on good clinical research and then fund it adequately.”

Third, we need to demand excellence and innovation in deciding which trials to carry out. “Clinical trials are getting more expensive and they are getting more sophisticated,” Mendelsohn says. “They often involve laboratory study on the patient’s tumor specimen to choose the drug that is most likely to work.

“But we may be trying to do too many clinical trials. We need to focus our faculty’s time and energy and our precious funds on those sophisticated trials that have the greatest chance of improving the care of a patient. All clinical divisions are looking to preserve our most precious institutional resource, the time and effectiveness of our faculty and staff.

“The future is very exciting,” Mendelsohn says. “New approaches are changing the practice of oncology to work in a more rational and targeted way. Cancer drug development is making tremendous progress, but we want it to be even faster and more effective. We at M. D. Anderson are doing everything we can to help accomplish that.”