How do clinical drug trials work?

Jan 10, 2014

Every drug in the pharmacy has to go through clinical trials before it is approved for use in the United States. But how do clinical trials work? Lorraine Rapp and Linda Lowen, hosts of WRVO's health and wellness show Take Care, recently spoke with Dr. Lindsay McNair, a leading pharmaceutical physician and researcher about who participates in these trials and why.

Lorraine Rapp: Clinical trials play a huge role in whether or not a new drug get approved in the United States. Would you describe the process for us?

Dr. Lindsay McNair: In phase one of clinical research, the drug is tested for the first time in a small number of volunteers to primarily look at the safety of the drug, and figure out what is expected to be the target range of the dosage. In phase two of testing, the drug is tested in a larger number of volunteers. And in addition to getting more data about safety and trying to focus the dose and dose regimen even more, it’s also looked at for efficacy in those studies. So those tests are usually done in people who have the disease or the condition that the drug is intended to treat or improve. Phase three of drug development is done in the largest number of subjects. And those studies are really done primarily to get good data on the efficacy of the drug, often compared to the available current therapies, if there are any, and also to get more information on the safety. And then the combination of the data that came from all of those studies is used to determine whether or not it should be approved for people to be able to use.

Linda Lowen: I would imagine that the process is not inexpensive. So who funds these studies?

Dr. McNair: Well the funding can come from a lot of sources. It can come from the person who owns the drug that’s being tested, and sometimes that’s the pharmaceutical company, sometimes it’s a hospital or medical center, sometimes it’s even the doctor who invented the drug themselves. The funding can also come from organizations, like the National Institutes of Health or the National Cancer Institute, which give grants to support clinical trials for new drug development, as well. And then there are a number of private organizations like advocacy groups that can also help support clinical trial work.

Lorraine Rapp: I’m curious to get a little information about why people would choose to participate in a clinical trial and what’s required of them.

Dr. McNair: A lot of people participate in these trials because they want the information that they can contribute to help people in the future. A lot of people who have illnesses or conditions that are not being adequately treated by the medications that are available to them participate in clinical trials because they hope that the new drug might give them some relief that their current therapies didn’t. And then for some clinical trials, especially the first clinical trials, the phase one trials, that are done in a small number of people who actually (are) healthy volunteers who don’t have the disease being studied, and some of those people participate in studies because they get compensated for their time.

Lorraine Rapp: If you did have a disease that was being studied, and you were offered a clinical trial, do you ever have to worry that maybe you’re going to get a placebo?

Dr. McNair: There are very strict ethical guidelines for when a placebo is acceptable to be used. And those include situations like where there is no available, effective therapy for the (disease). Or for when a delay in treating the condition will not cause a worsening of the condition or increase in symptoms during the time of the delay while that person is taking the placebo

More of this interview with Dr. Lindsay McNair can be heard on Take Care, Sunday evening at 6:30 p.m. Support for this story comes from the Health Foundation for Western and Central New York.