Challenges of Enrolling Children in Clinical Trials

Barry Kreiswirth, PhD, founding director, Public Health Research Institute Tuberculosis Center, professor of medicine at Rutgers University, discusses the challenges of conducting clinical trials as the threat of antibiotic resistance increases.

Interview Transcript (slightly modified for readability)

“We try to make an antibiotic so [when] you have a bacterial infection, we treat to kill that bacterial infection. The other idea of this, especially among patients who don’t have a good immune system, is maybe we give a drug to the patient who basically now has an enhanced immune system to fight off the bacteria. That approach, in combination with new antibiotics, is really the path to go. The problem is to try to make the host more active to fight an infection is not a trivial idea; it’s a real fine balance to try to make the host more robust and sometimes it has deleterious effects.

The strategy people are taking, is that if you work both at the antibiotic side and [at] the host side, maybe together they’ll synergize, but again, you’re still back to, [when] try to do those clinical trials, to get patients to enroll. I’ll give you an example that I learned recently which was really to my horror. You now have a new antibiotic that you want to give to neonates, so these are infants who basically are sick-born babies, and you now have to a clinical trial. You have to now go to parents and say, ‘Your son Johnny, I know he’s very sick, [and] we have a new drug that we want to test and this drug probably won’t help Johnny, but we want to see if Johnny will enroll in the study just so we’ll know, we’ll have data, so maybe two years from now, another baby could be helped by this drug.’ You have to now convince a parent who’s worried about the health of their newborn [to enroll the baby] into a trial that’s not going to have any bearing whatsoever on this child. You also have to recognize that this child may get a placebo and not even this drug. So how do you do these things? I mean, how do you convince these young parents or old parents (regardless) with a newborn baby, who they’re worried about as [to] whether they’ll make it, to put [that baby] into a clinical trial? The more you hear about these things, because I don’t deal with pediatrics at all and I don’t deal with patients, just the thought that these are the challenges. One of the concerns of developing a new antibiotic is, you have to do clinical trials; you have to enroll patients; you need enough data to go to the FDA. It is not only really expensive, it’s really hard to do just [because of] these factors.

I really have a lot of appreciation for people who do this, actually at Duke I learned that the only people who are allowed to actually get involved in these clinical trials as physicians, are physicians who have children, so [that] when they talk to the parents, they can actually say in honesty, ‘I have a child and this is what I [would do]’ because the parents would say, once they even think about doing it, [is] ‘What would you do?’ A physician [who] doesn’t have children can’t even put [himself/herself] (I don’t know how anyone can) in that position, but you can imagine.

These are some of the challenges [that] the public doesn’t understand and this is why clinical trials are millions and millions of dollars, because it takes so long to enroll patients, to get them to be in the study, just to generate enough data to say, ‘This drug’s okay but it’s not as good as this drug and it’s not going to make it.’ People don’t recognize [that] this is a rough game and the more we have resistance, the more the challenges rise.

I do a little of clinical trial work and I’m always astounded [that the public doesn’t understand] why it’s so hard to make drugs. I think the pharmaceutical [companies] can do a better job at educating the public, because all [that] we hear about is the pharmaceutical company just ripping off people in terms of the costs. Well, they don’t understand all of the factors, and the fact that most of the drugs don’t make it— they don’t get approved. You go down this path, fail, fail, fail, fail, you finally get a successful drug, you’re going to charge a lot of money to pay back all the failures, but the public doesn’t recognize that. Even as scientists and as patients, we don’t recognize that, too, but that’s what’s underlying this and I don’t know why the pharmaceutical company doesn’t do a better job on educating the public; all we see is the sides of the EpiPen, right? Not that I think that’s great, I think that’s horrible, but [people] don’t understand the other business part of it, which really should be discussed; I think it’s an important role that the pharmaceutical company just completely misses in dealing with the public.”