by Sydney Anderson
Participation of Cystic Fibrosis (CF) patients in clinical trials and research studies is crucial for innovation and progress towards a cure, but there are many barriers that can prevent or disincentivize participation. I spoke about this issue with Karoline Servier, who is a research coordinator at The Johns Hopkins Hospital in Baltimore, Maryland and works within the Cystic Fibrosis clinic. To get a better idea about the relevance of the topic that I discussed with Karoline, I also spoke with a 21-year-old woman living with CF to better understand her personal experience with participating in studies and trials.
I asked the CF patient: 1) How often she had been offered to participate in a research study or clinical trial, 2) if she ever participated, 3) why she did or did not participate, 4) if she was interested participating in studies, and 5) if she knew where to look/who to ask for regarding open studies and trials. The CF patient answered that she did get offered to participate in studies often, had never participated in any studies or trials, but had considered it, and the reasons were due to her busy schedule and financial restraints on travel costs. Further, the CF patient expressed an interest in participating in clinical trials and studies but did not know who to ask or where to find information regarding open studies. My insights from this conversation informed my discussion with Karoline about her perspective on barriers to clinical trial enrollment.
Karoline explains some potential solutions to overcome barriers to clinical trial participation like utilizing new technological developments, getting research staff more involved with patients, educating patients more often about ongoing studies, and encouraging patients to ask their CF care centers about clinical trial information. She also highlights the importance of being open and honest about the personal barriers to studies, because the clinic may offer support or helpful resources.
The conversation below has been edited for length and clarity.
Sydney Anderson: I want to ask you about clinical trial and research study participation in the CF community. I have CF, and I was offered to do quite a few clinical trials or studies when I was young and I never did them because I was always busy with school, or my family always lived about an hour away from a clinic. I have three other siblings, so my family was always very busy in general. Actually, the first study I’d ever agreed to do was the RESCUe one I participated in just recently at Johns Hopkins.
Karoline Servier: Yeah it was an easy one, so that’s understandable. [Participation] is definitely a big thing that we’re working on as a whole research team at Johns Hopkins, and how to get more participants to be involved in research, because there are a lot of barriers. When you reached out about this, I was like, “Oh, this is great,” because I feel like this is on the top of a lot of our minds, and there is a lot of research out there, and doctors have multiple research trials that they want to close out on and hit their max.
SA: What would you say is the percentage of yeses you get regarding participation versus nos?
KS: It’s really going to depend on the type of study that you’re looking at. I think that, for example, the study that you participated in, RESCUe, I’ve had a majority of yeses, because it did not require much extra effort. I think there are 30 people on the trial right now. So 30 people said “yes” to that study, and three people told me “no,” and the main reason they told me “no” was because they were unable to produce any sputum anymore because of CFTR modulators. (Editor’s note: This study only required a sputum collection the morning before clinic, another in-clinic and then another collection the day after that the participants had to mail within 24 hours.) The drug trials are a lot smaller, and you definitely get a lot more nos, but you only need a small handful of people. Then, the ones that involve just doing online surveys, I would say get 95% yeses.
SA: Do you find that there is a specific age group that tends to participate? Or patients with other demographic information, like their insurance, their economic situation, etc?
KS: Younger adults, 20s to 30s, are more willing to participate. The trials that you’re compensated more for are definitely more appealing to young people. The younger generation is more inclined to participate in the interventional ones too, like the drug trials. I’ve had people who are older, like 60s or 70s, who do these research trials, and they tell me they don’t want to be paid. I’m like, “I appreciate that, but legally I have to pay you.” Then, [there’s] insurance. Anything that we do as research will be paid for no matter your insurance, so that’s not really a factor. I have had patients come back to me and say they don’t think their insurance is going to cover it, but I let them know it’s covered and all funded through the CF Foundation.
SA: Typically, how many participants do you aim to get for the drug trials? Because I would think the more people that agree, the more robust the results will be.
KS: It really depends. A lot of these drug ones are very specific. Sometimes those trials are only looking for, maybe, 10 participants, but it also depends on what the qualifications are. Sometimes, participants have to be a certain genotype, or they can’t be on certain medications, or have been on X CFTR modulator for a certain amount of time. For the study I’m working on currently, the participant has to grow a certain bacteria within X amount of months. I would say the drug trials usually range from five to 20. There are definitely ones that are bigger populations, like 50, but it’s not as typical. Also, I don’t know if you’re aware, but a lot of the studies are branched off to other hospitals. Other hospitals are participating in recruiting patients, because it’s hard to get all patients from one hospital to participate in the study. It helps speed up the study because usually there is a deadline. For one study that we are doing at Johns Hopkins, we have not been able to recruit someone in the past three months, but there are so many other hospitals participating that helps balance it out.
SA: What do you think are some of the most common barriers to people not wanting to participate?
KS: I would say the biggest barrier is when a study involves extra visits, because a lot of these patients come from far away, and it’s a hassle for them to even come to regular CF appointments. Another barrier is whether the financial compensation feels like it’s worth the time. If it is an interventional study, that is an overall barrier, because there are more risks involved with it. It makes people more anxious. Also, the compensation. In most studies, but not all, the participants are paid, and I think participants have to decide whether the compensation feels worth the effort.
SA: Has there been any discussion on a solution to this issue, maybe virtual options when possible?
KS: [We’re] trying to maximize research when patients are already in the clinic. We can do consents virtually or over the phone. [We’re] also trying to maximize what we can do online, like a lot of e-consents and surveys to make it more appealing. Education is a big part of it. There’s some disconnect between what we’re telling patients and what the patients are understanding. A lot of [patients] don’t fully comprehend how beneficial their help could be down the line. I also think that comes from having patients more comfortable with research staff. We are trying to get research people more involved with the patients, because they’re more inclined to say yes when they’re talking to someone they’re comfortable with. Then, [there’s] transportation. This barrier goes pretty unexpressed but I do think it is a big one, and I just don’t think patients realize that the clinic offers resources that the patient can use for transportation.
SA: I tend to only hear about studies when I’m in the clinic and a research coordinator approaches me. What is a way that CFers who might be interested in doing studies, but just haven’t been in a clinic for a while, can be informed about the current trials and studies?
KS: We use a listserv. We have one for our patients and one for family members. It’s basically CF announcements, whether it’s a study or our newsletters. We’re also trying to create a list of all studies that we have ongoing with a brief summary of what they are about and give it to patients when they’re at the clinic. The CF Foundation website is always a resource to use for looking up studies as well.
SA: Ok great, so CF patients should ask their research coordinator at their clinic how trials are advertised because I’m sure every hospital will be different.
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Sydney Anderson is a 25-year-old evening law student at American University Washington College of Law. She has an undergraduate degree from Texas A&M University and works full-time for an employment litigation firm in Washington D.C.
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