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Equal Parts Caregiver and Advocate: Clinical Trials from the Caregiver’s Point of View

“There are only four kinds of people in the world: Those who have been caregivers, those who are currently caregivers, those who will be caregivers, and those who will need caregivers.”

— Roslyn Carter

Each November, National Family Caregivers Month recognizes and honors all those who have dedicated themselves to caring for ailing loved ones. It also helps raise awareness of the often overwhelming issues they face, so that we can work together as a society to alleviate them. This year, Premier Consulting is highlighting one of our own — Andrea Miller, Manager of Life Science Solutions — who cared for her husband, Tom, throughout his battle with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. In this Q&A, Andrea discusses the experience, especially as it relates to the two clinical trials Tom participated in.

How was your husband diagnosed with ALS?

I believe that Tom self-diagnosed six months before his official diagnosis, but just didn’t tell anyone. In the spring of 2015, he was having cramping in his hands, but more importantly, he was having twitching in his upper arm.

We couldn’t get any answers. Finally, he took an EMG (electromyography). That was Friday, May 8. We got the diagnosis that day. My husband’s response was, “I couldn’t have ALS; I don’t even like baseball.”

The first thing we did was go to a specialist at the Mayo Clinic for a second opinion. Then we chose the University of Cincinnati ALS Clinic as the place Tom would be getting his care, because it’s close to home; we didn’t want to drive an hour and half each way. Going to a clinic is already an emotionally trying, exhausting day, and, after all, we weren’t really getting treatment at that point. We were getting tools to help manage our lives and medicines to manage Tom’s symptoms.

So you pursued other options in addition to routine care?

We wanted to participate in a clinical trial. I’ve been in pharma for over 20 years, and I’ve searched many times for many other reasons. But our original doctor doesn’t work with clinical trials — it’s just not his thing.

When you get a diagnosis of a terminal illness, you are busy coping with what it means. Can he work? Should he work? What do we tell the kids? When? You aren’t thinking about treatments and trials. But with ALS, the median survival is two to five years. So, if you are going to participate in a clinical trial, you have to start immediately; otherwise, the exclusion criteria will lock you out.

The problem is, physicians who are involved in clinical trials will be promoting their own, and those who are not probably won’t even discuss the concept of a trial. So it’s up to the caregiver either to do the research or to find someone who can.

You spoke of the need to research trials immediately. Do you think sponsors should rethink their exclusion criteria?

In some cases, I do. Obviously, sponsors don’t want patients who can’t finish the study, but diseases like ALS progress at different rates. In those cases, it might make sense to look at individuals. I know a woman who has been diagnosed for eight years, and she still runs and homeschools her three little kids. But she can’t participate in a clinical trial because of the exclusion criteria — most ALS studies disqualify anyone diagnosed more than 18 months before the start of the trial.

You did the research on clinical trials being conducted?

I became the advocate; Tom became the patient, so he could focus on his well-being. They say with ALS you live longer if you’re happy, so my goal was to keep him safe, secure, and loved. We closed his law practice, and I went back to work. And I started researching trials. With ALS, there is not a lot of, “We know this works.” That’s where the trial comes in.

About 10 months after Tom’s diagnosis, I found a trial for a repurposed drug that seemed promising.

What was the trial like for you?

We went once every six weeks, then eight weeks, then three months, and it was an eight- to 10-hour day, with me taking the day off work. Sometimes his parents or our friends could help, but as Tom’s disease progressed, that became more difficult. Since they weren’t helping him on a daily basis, they just didn’t have the confidence and knowhow to manage his needs.

It was like going to a clinic — they did all the same tests. But much of it was very disorganized, and ultimately, Tom was thrown out of the trial because of a scheduling error. That was very upsetting, since I really felt like it was helping him. Of course, you don’t know whether you are on the placebo or not, but having something to look forward to can help prolong life: If you think you are getting better, it helps. And when months count, that’s a big deal.

But you found a second trial?

Yes. We found one that didn’t have the same strict exclusion criteria as most others. But it was tougher to participate — by then, Tom couldn’t use his arms. There was no way the trial coordinator and the phlebotomist could have performed the testing by themselves, so I became an active participant in the trial. I would get him out of his chair. If he had to stand, I held him. If he needed to lie down, I would get him there. If he were hungry or thirsty, I would feed him. That takes an experienced caregiver.

The trial was for a drug that is normally given as an IV to help lung function during surgery. They had made it an oral product. Ultimately, we withdrew, for two reasons. First, the study was very draining on both of us. Second, Tom experienced a 40 percent decrease in lung function after four years of strong lung function tests up to that point; I was afraid they didn’t have the dosing right. (We were probably in the dose-finding portion of the study.) We had to weigh the risk against the reward.

How could sponsors better support patients and caregivers?

It’s tough taking an entire day off work, and it’s a long, emotional, exhausting day. There might be a way of decentralizing some of the trial, to eliminate the travel time and added days off for the caregiver. They were measuring the same things at clinic as they were at the trial site, and we were already logging the medications, the lot number, and when the meds were taken. And there are ways to manage the other logistics involved in a decentralized clinical trial.

On-site, it would be nice to have more trained support. So much of what a caregiver does is specific to each patient that I don’t know if more training would even be effective. Those who haven’t faced a particular situation before, even professionals, may not know how to deal with that situation when it arises. But when you live with a disease like this every day, you are often thinking, “How are we going to fix this? How can we avoid a pressure sore? How can I lift someone who is 230 pounds?” And then figuring it out.

Finally, sponsors can ensure that someone experienced is engaging with the patients and caregivers and actively collecting the data — because the data drives the outcome for your label, for your approval, for meeting your endpoints.

Any final thoughts for other caregivers?

The biggest thing about being a caregiver is that you have to be an advocate as well. You have to step in and ask the questions and do the research, because people don’t know about clinical trials. Most caregivers also don’t understand how to utilize palliative care or fight for the coverage. We did palliative care for two years, and when I ran into something I was having trouble with, the nurse often had a suggestion from dealing with similar issues with other patients. That’s invaluable.