In 2015, I came to work as a research assistant for the HDSA Center of Excellence at Rocky Mountain Movement Disorders and fell in love with both the research and the involvement with patients. My 10-year anniversary is coming up soon, and I’m very pleased to share my perspectives on the patient’s experience in trials and how we, as clinical coordinators, can best support both the patient and the trial sponsor.
I will surprise no one when I say it isn’t easy. It’s a tightrope with danger on the one side of creating “our-way-or-the-highway” relationships with patients, and on the other side, over-accommodating and weakening the data integrity in the process. My job, and that of my two clinical coordinator colleagues, is to attend to the nuances of this walk, such that the study succeeds in its goals and there is no patient regret. A tall order, but that is the goal.
Becoming a Selected Site
Site participation begins with an industry representative or other sponsor reaching out to us with a feasibility questionnaire, while also giving us a peek into their study, as we all try to understand how practical it would be for our site. Do we think we have patients who would fit this criteria? Do we see any hurdles for finding enough patients to be involved?
If we are selected as a site, our regulatory coordinator takes care of all the start-up paperwork and compliance pieces. At this point, our job as coordinators is to start thinking of which patients we think would fit and how, logistically, we are going to operate in this trial, including what tools we will use to keep track of everything and how we will assure consistent adherence to the protocol.
Filtering Candidates for Inclusion
We use EnrollHD as a gateway to help sort candidates. There are a lot of scores we capture in Enroll-HD that correlate with inclusion/exclusion criteria for upcoming interventional trials.
Many of our patients participate in Enroll-HD even before they are eligible for a clinical trial of an HD treatment. Having gone through this step with them makes patients a little bit more apt to hear me out on the more intensive trials, because they already know me well.
Using a master database, we can sort through candidates to find criteria we need to look at for the particular drug trial. These include medication history and proxy measures for total functional capacity, total motor scores and symbol digit modality test scores — all used to try to create a facsimile of their HD-ISS score.
At this point we will reach out to potential participants by phone to inform them about the trial. We’ll always send them the consent forms beforehand, so they and their families have adequate time to consider the study and ask questions.
We start with the big hitters, like why are we doing this study, how long will it be, what is required of the participants, and the logistics of participating. We also discuss the safety profile, potential side effects or adverse events with this medication, telling them what we know, but reminding them that it’s still research, and we’re going to learn as we go
Through these conversations and questionnaires, we organize the variables on a shared platform with my team, which includes two other coordinators. The three of us call ourselves the tricycle — we’re all equal parts, equally important, and somewhat interchangeable in our roles.
To Trial or Not to Trial
The biggest factor I see that affects a patient’s willingness to participate is the goal of the medication — symptomatic treatment versus potential disease modification. There’s an excitement when people anticipate symptom-relief in its own right, but assenting to a disease-modifying treatment is more sobering. The “takers” here are generally more deeply educated about risks and opportunities. Some of the hardest conversations I have are about gene therapy trials and others where the patient may be unlikely to see good outcomes in their lifetime.
Another concern is over whether participating in a certain trial will make them ineligible for a future trial, which is a real issue. They will have been exposed to an investigational medication and that can become a confounder for other trials. The fact that they may be putting all their eggs in one basket needs to be really well-articulated to patients prior to entry in a trial that, for example, makes a permanent genetic change.
Sometimes the route of administration can be a really important differentiator for people in determining which trial makes the most sense for them: one may be a pill, another a drink, another an intrathecal injection, and yet another may be brain surgery.
I try to dispel some misconceptions about lumbar punctures, and remind them that the procedure is safe and they will be numbed prior to insertion of the needle. Even if it isn’t the route for administration of a drug, lumbar punctures are still part of collecting safety data in most studies.
Another hard conversation is talking to patients who ask, “What do you think I should do?”
Always, the message is clear that it is 100% their decision. They have to decide if the potential benefit to them or to future people living with HD outweighs potential headaches or other side effects, or potentially being ineligible for future trials.
Accommodating Participants’ Lives and Schedules
With the shift in testing treatments earlier in the disease, participants are more likely to be working. Some people live far away, while many, nevertheless, find a way. In fact, a recent participant, who is a busy professional, worked on their laptop throughout an infusion!
Some of the sponsoring companies have modified things somewhat to accommodate people better, while still accomplishing what they need to for the trial. Examples are reimbursement for long-distance travel and meals, car rental service, or travel agents to help with flights.
Some of the logistical pieces of patients’ special requests can be trickier to navigate. Perhaps they have an additional person they want to bring, or they want to rent a car and extend their stay. Sometimes people will ask to get their labs drawn or their MRI done closer to home. This is understandable, but is generally not allowed as the labs and MRI need to be completed in a consistent fashion for comparison. This isn’t always possible, and one of the trickiest parts of educating people about the trial is trying to articulate why not.
Sometimes you have to take a step back and remember that what’s so second nature to you as a coordinator, isn’t for the participant. You’re trying to give them a Research 101 course, while talking about the particular study as well.
Bumps in the Road
Patients who experience complications usually need to come see us quite a bit more often. We had a patient who lives far away who had to fly back in for a blood draw, and then fly home. So, it felt very dramatic, but they understood the importance of it and were willing to accommodate us.
Sometimes there is a dosing pause, sometimes we need extra vigilance in watching their laboratory values as a safety measure. In these cases, a good network and good communication, built into the infrastructure of research, enables us to shore up the whole of the patient’s health so they can hopefully continue in the trial. To triage such situations, we collaborate closely with the sponsor, the primary investigator and the medical monitor, who all are all familiar with the compound and the known intricacies of its effects.
Inconveniences for participants naturally occur, but the beauty of this patient population is that they are, by and large, more than willing to accommodate.
Navigating a Negative Trial
It is not uncommon for a trial to be halted because the data doesn’t support the overall continued development of a medication. If a participant feels a treatment has been helpful for them, it can be really difficult to explain that it’s not feasible for them to continue to get it.
I always want to be sure I’m setting realistic expectations, that having the trial stopped, or adverse events or side effects, are all possibilities. We talk about how being part of a study, whatever the outcome, still enables the participant to get a lot of medical attention — they’re getting a lot of blood work done, and getting a close eye from their physician, all of which should enhance care.
Selflessness Prevails in Enrollment Decisions
We find in our HD patients this heightened sense of altruism. It’s a family disease, and so they’ve had to watch their parents go through this, and are trying to pick a different course for themselves. And then, of course, they likely have other loved ones, like children, who might be impacted.
Further, we often find this response that, even if it might not help me, it might help others unrelated to me who share this genetic anomaly. And that deep commitment to research for the larger community seems to me to be unique and special among HD patients.
Patients may tell me they feel like this research gives them the opportunity to be proactive, which I think is a beautiful thing. Even if maybe we don’t learn what we want to, we will always learn something from these trials.
