An observational trial, from the patient perspective
In the week note from 10/12/15, I discuss the barriers to enrolling in a clinical trial from the patient perspective. Having never been a patient in a clinical trial, I set out to find and successfully enroll in either an interventional or observational study.
My goal was to better understand the patient perspective when participating in a clinical trial. I assumed that living in Baltimore, surrounded by a few big research institutions, and having a solid base for medical jargon, it wouldn’t take too much of my time to find plenty of options for enrollment. Two big takeaways from this search:
- My public health background and recent A+ in Medical Terminology was not a good enough base for medical (and clinical trial) jargon
- Concern: If I was having a difficult time understanding the aims and eligibility criteria of the trial, would an individual with no medical/public health background take the time to understand it?
- Eligibility criteria is a large barrier to enrollment (for the participant)
- I had initially contacted a study coordinator about an interventional trial (9/30/15), but after back-and-forth conversation between my primary care doctor and the study coordinator, I ended up being ineligible for that trial. At what point are the incentives (re: week note) outweighed by the time and effort it takes just find a trial for which one is eligible?
Fortunately, the study coordinator I had contacted was juggling a few studies for the same disease and
Principal Investigator, (PI). So I enrolled in an observational study that aimed to “advance our
understanding” of the disease and “…to establish a repository (storage site) that will help
scientists plan and achieve the research studies that may allow us to understand the complications of
[the disease] such as high blood pressure and kidney failure.”
Below is a comprehensive look into my enrollment visit, told from the patient perspective.
Enrollment (and baseline) visit on 11/12/15
The enrollment visit was nothing like I had expected. I had anticipated being left alone most of the visit – not in a room but in a hallway or waiting area with uncomfortable chairs – left to wonder how much time I had before my next test, if I was expected to be familiar with the hospital, etc., but this was not the case.
After security directed me to the appropriate floor and room, I arrived sometime around 8:25am and was greeted by a nurse and two research coordinators – all smiles. I was with these three people for the majority of the day.
The coordinators lead me to a room that had a hospital bed, couch, comfortable chair – it even had a window – and I was told that this would be my room for the duration of the visit. All of the tests, questionnaires and the patient history would be done in that room, (the equipment was brought to me), with the exception of the MRI which was in a different building.
They began by explaining the purpose of the study, why they were collecting the information and how the data would be used. They took me through the morning’s sequence of events and what to expect throughout the visit. They were very thorough and encouraged me to interrupt to ask questions which I did. There were two forms of consent, one for the study and the other for my genetic samples which, if signed, would be included in a big database for research purposes. They also covered payment, ($50), which would come in the mail about eight weeks from the enrollment visit. The whole process took about an hour. After reading, I agreed to both consent forms. Below, I have included language from the one of them, omitting some of the detailed information.
“These samples will be made available to qualified scientist for future research projects related to [disease of interest]. These samples will not be labeled with your name on them but will have a study code that will be linked to your name; this link will be available to Dr. [_________ ] and his/her study team.”
Optional Storage of Blood Samples for Future Genetic Research
“Genome-wide association studies (GWAS) look at the genetic differences that exist in the entire human genome (the complete set of human genes) and how these genetic differences relate to health conditions. As part of this study, we will be collecting information about your health and your individual genes.”
Questionnaires, blood and urine samples, & medical history
I was left alone to fill out questionnaires for physical health status, an emotional health evaluation, medication history and some administrative forms, (primary care information). Later in the visit, (after I was fed), I was given a cognitive test to measure mental function.
The co-PI then came in to introduce himself and collect a comprehensive patient/family history. He was very kind, easy to talk to and didn’t seem the least bit annoyed by my erratic answering, even though it required him to skip ahead or turn back in the questionnaire he was using to collect my medical history. He was very patient with me, taking time for me to remember dates, (e.g., age/year of diagnosis for both me and my family members with the same disease), and appreciative that I had brought all of my medical records, (for unrelated reasons, I had asked my primary care provider to copy and send them to me). The team seemed surprised and almost excited to learn that I had seven older siblings, and that six of the eight of us had inherited the disease from my father.
This took about 90 minutes and during this time, the nurse was periodically coming in to take blood samples, a urine specimen, and to bring me food. The whole team gave me the utmost confidence, privacy, and respect.
Physical Exam, Measurements, Electrocardiograph (EKG), Echocardiogram, and MRI
Next there was a physical exam and measurements were taken; this was all quick and painless. They allowed my time to eat breakfast (eggs and French toast) before the machines for the EKG and echocardiogram were brought to the room. The only discomfort I felt during these tests was that I was cold and the cool gel didn’t help but it was comfortable otherwise. They finished up and 2 of the coordinators walked me to the building where the MRI machine was located for the final exam of the day. This test was quick and actually comfortable, I think I even took a cat-nap. When the MRI was finished, the coordinators were in the waiting room and walked me back to the original building’s entrance. And that was that.
I had brought reading to keep myself occupied but the opportunity never presented itself. The team did an impressive job coordinating the order of tests and questionnaire’s based on scheduling with the co-PI and equipment specialists.
Each person I encountered was friendly and considerate, to the point where I felt appreciation for my participation in the study. Regardless of whether or not this was the case, (and whether it was intentional or unintentional), the warm dispositions of each team member added to the whole experience. While my assumption was (and still is) that any findings from the data collection would likely not impact me or my siblings, I sincerely felt like I was making a small impact on the understanding of this disease and maybe that would help researchers and future generations. I hope this sense of appreciation and impact typical for studies of this nature.
From start to finish, the total time for the enrollment visit was less than 5 hours. Every 6 months I will have a follow up phone call lasting about 15-20 minutes where I will report any serious events related to the disease, and any changes in health staus, medications, etc. My assumption here is that my time and effort to participate in this study is considered minimal when compared to the participation requirements of interventional clinical trials.
Further, I am fortunate to have access to a car, time from work, and a personal connection to the disease in question. Similarly, I am now aware of the importance of clinical trials and their need to reach a critical N. I am unsure whether I would feel as altruistic if ther were more logistical barriers to my participation (transportation, time, etc.) and I didn’t have a personal connection.
In the past and before I joined this project, I had tried to enroll in a several clinical trials. I was always dissuaded by the difficulty of getting in touch with study coordinators and not having the answers they needed to know if I fit the eligibility criteria. These are barriers before they even get you in the door – before time and transportation logistics become an issue.
Given our work, I am aware of the low publication rate of interventional clinical trials with null, negative, or uninteresting findings. Since my enrollment visit, I find myself personally invested in any outcome of their data collection. I can’t speak for other trials and other diseases, but as a participant, I partly feel as though I am owed a publication or some sort of synthesis of the findings, lessons learned, or maybe future steps. I’m not sure what it’s like to fund a study so I can’t speak for sponsors but from a patien perspective, don’t all participants deserve to see a synthesis of the data they provided?
Lastly, to briefly answer one of the questions I brought up in my week note from 10/12/15:
Given a chronic but not necessarily life-threatening condition, is it enough to know that the data provided, (i.e., blood and urine samples, imaging results, time, etc.,) could help researchers better understand disease progression, and add to a repository that will help scientists better plan future clinical trials.