Clinical Trials: What’s Under the Hood?
Stefan Tarnawsky Nov 19, 2018
Camp Fire (in red) shows active forest fires near Paradise, CA with San Francisco shrouded in smoke in the low-middle.
“So why would anyone want to participate in this trial?”
Clinical trials are the foundation of medical knowledge; they’re how we improve outcomes for our patients. Later in my career, I plan to participate in medical research and contribute to evidence-based practice. The Introduction to Clinical Research course has introduced me to the intricacies of studies with patients. I’ve met with IRB members, charge nurses, dietitians, tissue bankers, biostatisticians, data managers, quality and safety officers; the oiled gears that enable clinical research to run smoothly.
Today, I’m in Goodman Hall, working alongside Scott – the study coordinator for an international, multi-centered clinical trial of Alzheimer’s. Scott’s role is to recruit patients, consent them, care for the patient during each visit, and keep track of all patient data for the trial. Like all studies, this one is voluntary, and patients can stop participating at any time. However, while enrolled, they agree to an arduous schedule: 5 visits in the first year and 1-3 in each subsequent year. Each visit includes memory and cognitive testing, bloodwork, MRIs, PET scans, and lumbar punctures. The study is observational. Whereas some of these results will be available to clinicians to guide patient care, no experimental treatments will be offered. As such, given a compensation of $75-150/year (or ~ $5/hr), I was puzzled why a patient would agree to participate in this trial.
I was taught in medical school that patients enrolled in clinical trials have better outcomes compared with those who are not. The reasoning was as follows: patients on a trial are monitored more closely and have more interactions with healthcare professionals. Presumably, this heightened oversight has a positive effect irrespective of whether the patient gets the experimental therapy or the placebo.
This rationale is sound; but is it true? The issue has been extensively studies. Most meta–analyses suggest that there is no objective benefit from enrolling in a trial. However, some disagree. The Catch-22 is: how to run a study that compares outcomes between those who are on vs. off a study? After all, the selection of patients for a trial is not random; there are very specific inclusion and exclusion criteria that intentionally limit the scope of each study. As such, patients selected for clinical trials may be less sick than those who are not on a trial and do objectively better for this reason alone.
In sum: the objective benefit from enrolling in a trial is negligibly small. So why participate? My day with Scott demonstrated two reasons. First, many patients want to contribute to medical knowledge; especially for diseases that are poorly understood, such as Alzheimer’s. The patient that I saw was still quite functional and cognitively aware. He was eager to learn more about his diagnosis and to help his physicians learn more as well. Albeit he understood that he would not be getting any special therapeutic intervention, he was nevertheless eager to participate in order to help posterity.
The second reason to participate in a trial is the increase in exposure to the health system. My patient spent 6 hours with Scott, other RNs, and MDs during his visit. That is a lot of opportunity to ask all the questions that weigh on his mind. The answers may not lead to better outcomes – as noted above – but they provide a great comfort to the patient. Scott’s diligence, kindness, and inexhaustible patience visibly calmed the patient and his family. They left wearing great large smiles. True, my patient’s objective outcomes – overall survival, the MMSE, the PHQ-9 – may not improve. But the significance of those smiles and cannot be overstressed. Indeed, perhaps our current outcome measures fail to capture the most important patient benchmarks.