Patient Retention: Quality of Life Is Key

One reason the dropout rate for Phase III clinical trials may top 30% is due to the burden they can put on patients — medically, financially, and timewise. See ways to boost retention, including providing trial results to patients and taking into account participants’ quality of life.

According to the National Research Council, the dropout rate for Phase III clinical trials can sometimes be more than 30 percent.1 There are many factors influencing that number, but the overarching theme is the burden clinical trials can put on patients — medically, financially, and timewise.

Jenn McNary

Jenn McNary is co-founder and principal, Canary Advisors, a boutique patient advocacy consulting firm with a focus on regulatory and access patient engagement. She is also co-founder of One Rare, a nonprofit forum designed to meet the needs of young adults living with rare diseases and chronic conditions. Her son Austin Leclaire is a member of the One Rare board who’s living with Duchenne muscular dystrophy.

McNary says one thing she would like to see happen more often with clinical trials is returning the data to the patient — and that can be an incentive to stay in a trial.

“A lot of times with our lives, you’re participating in a clinical trial for a decade or more, especially with these gene therapy studies, especially with studies that have long-term observations first in human, et cetera,” she says. “So, things like cardiac MRIs and pulmonary function testing and functional assessments are being taken at these trial visits. I’m a huge supporter of returning that data to the families so they don’t have to go repeat those data endpoints at their clinic.

“A lot of times care during a clinical trial is one of the perks,” she adds. “So, if you don’t have insurance that covers a cardiac MRI, but you're getting one as part of the study, you’ve got to give that data back to the patient’s own cardiologist so they can make care and treatment decisions based on that, too.

“If you’re asking us to come to a study site every couple of months, then getting a clinic visit in two, three, four times a year, that burden is doubled,” she continues. “But if you can offer that, ‘hey, we’re going to do these assessments at the study site and provide that back to your care team,’ that’s an added benefit and that will keep you in the study. That will be a compliance thing. It is really an incentive to say, ‘Hey, we are going to cover these tests throughout the years as long as we have the data for our study.’”

Obtaining quality of life data

Austin Leclaire

Another motivator for patients to stay in trials is if it’s improving their quality of life. “That’s really hard to quantify, but I think quality of life is important,” Leclaire says. “If the study to get that data is taking away from my everyday life and not bringing quality to it, then I think that’s not very valuable.”

To truly improve quality of life, trials should be measuring real-world outcomes. Yet, many trials fall short in this regard, choosing to train the lens on clinical endpoints instead of adopting a view that encompasses life outside the clinic.2

McNary says it’s important to collect and share patient reported outcomes about quality of life with decision-makers in the clinical trial process.

“We [at Canary] just launched a program where we’re collecting via qualitative interviews patient reported outcomes for studies, and that is directly working with patient families,” she says. “Sometimes it’s parents, sometimes it’s patients themselves to collect those quality of life measures.

“We know the FDA is really focused on how a patient feels and functions as part of the review of the data,” she continues. “We know that Congress mandates them to include patient experience data into their review and hopeful approval. They have advisory committee hearings, they have mechanisms like PFDD meetings throughout the drug development process that patients can weigh in. But what is better than interviewing and talking with families who are participating in a study to find out if this study indeed changed their quality of life outside of the clinic?”

McNary gives an example from her own experience working with a team to design a patient reported outcomes or qualitative interview study for patient experience.

“We found out that not only were these kids walking a little better as part of the study, walking a little further, maybe in six minutes going up the stairs better, but that also meant that these people, these families didn’t have to move to houses that were one floor,” she says. “They didn’t have to change schools. Maybe some of the kids were at parochial schools where there were no elevators. So, the fact that they could still use stairs meant they might complete their years there at that school. They were traveling more, and they were able to enjoy life as a family a little bit more because not needing a wheelchair to travel and being able to walk was a huge benefit to these families.”

Those aren’t things that would be discovered during a clinical trial with traditional clinical endpoints, McNary says. “So, it really is kind of that additional color,” she says. “And I think to the patient, especially the adult patient, if they’re going to have the burden of being part of a study on the other end, they want it to improve their quality of life. That’s what they’re interested in overall. So, we do have to capture that if we’re going to be able to show it.”

The power of the ‘patient hat’

McNary says when designing a trial, it’s important for sponsors to put on their patient hat.

“We’ve all been or will be patients at some point,” she says. “So what I would say is today, look at what you are working on, whether you’re designing some communications that are going to be in the hands of a trial participant, whether you are planning that trial, you’re designing a protocol, you’re thinking about how many injections this child has to have or how many times they have to visit a site. Wear your patient hat today as you’re navigating your role in the company and try to make one change that lessens the burden or makes something clearer or makes something more patient-centric.”

McNary says she doesn’t like the phrase “patient centric” because she thinks it’s overused and under-demonstrated.

“But really think about what’s one thing you can do to make a patient’s life better in the role that you are filling at your sponsor company and do that. I think that action is incredibly important,” she says.