Millions of Americans still live with pain due to few treatment options and scarce data on their efficacy. In reflecting on this issue, Stanford Pain’s Dr. Vafi Salmasi had to wonder: “how can we gather more people and more data to help pain patients faster?” His recent study findings are promising: a new patient-centric research method called “Pragmatic Comparative Effectiveness Trials (CETs)” may be the ticket to better pain care.
“These novel designs study how treatments work in real world application,” he explains. “When well-embedded in routine clinical care, we can study a large number of patients at a much lower cost compared to more traditional explanatory clinical trials.”
What are Pragmatic CETs, and why do we need them?
How do pragmatic CETs make it easier to study more people, less expensively? In a traditional randomized control Trial (RCT), all aspects of a clinical trial have to be tightly controlled, like a carefully choreographed symphony. Researchers have to make sure every participant gets exactly the same treatment, follow strict protocols, and monitor everything closely. This requires a lot of resources—time, money, and manpower—to make sure everything goes according to plan.
Pragmatic CETs are a bit more relaxed. Instead of supervising every little detail, PCTs are more interested in how treatments work in real-world settings. This means researchers can use existing data, broaden eligibility requirements, and use resources already available in clinics and hospitals. Using study findings from multiple health systems ensures that the conclusions include data from a wide population of patients. This broader diversity makes results applicable to a greater number of people.
While RCTs are often considered the “gold standard" in clinical research, they may not fully capture how well a treatment will work in someone’s actual life.
Salmasi asserts that long consent forms are not as necessary for PCE trials, because already existing treatments and medications are being used. “Should patients really need to fill out a 16-page consent form to join a simple trial?” he questions, adding that longer consent forms are still important for RCTs, because new medications and treatments are studied.
Research indicates that some patients are open to a shorter informed consent procedure in the name of more research, as long as they remain involved in all personal data use and clinical care choices. Because pain research is impossible without willing participants, the need to make it less burdensome to participate is beneficial to all.
"None of the studies we do can be completed without these generous individuals who participate and advance science."
“I want participants to know how valuable their contribution is,” Salmasi says. “None of the studies we do can be completed without the help of generous individuals who participate in order to advance our knowledge of the science surrounding pain.”
Overcoming Hurdles of Pragmatic CETs
While pragmatic CETs hold tremendous promise, they are also not without challenges. Unlike objective measures commonly used in other medical specialties, pain management often relies on subjective assessments of patients' experiences.
“Applying these designs in pain medicine has distinct challenges because most outcomes are patient-reported,” he says. “We believe Stanford Pain Management Center is in a unique position to use these novel designs with our efficient learning healthcare system, CHOIR.”
CHOIR software acts as a communication hub where providers can store information like treatment best practices, patient survey data, and research findings. For example, in a large, multi-center trial where multiple healthcare systems participate in a Pragmatic CET, CHOIR will allow all the data from that trial to track the progress of each participant and coordinate patient care in one spot. This is game-changing for researchers conducting a large-scale trial.
A Shift Towards Pro-Patient Care
Pragmatic CETs represent a paradigm shift towards a pro-patient approach in healthcare. By embedding trials into routine clinical care, informing patients about these trials, and obtaining informed consent, these studies become accessible to a diverse range of patients. They are done during the process of regular clinic visits, with no extra visits required. Moreover, these new trials track long-term treatment data about medication benefits for those who need it most: pain patients.
By embracing these methodologies and harnessing the potential of pragmatic CETs, Salmasi believes Stanford can pave the way for more evidence-based care for individuals living with chronic pain. He hopes these strides in pain research give patients a sense of hope.
“Sharing knowledge about their condition in a study is one way for patients to make life better for themselves and others with chronic pain.”
“I hope we can make meaningful advances in understanding and better treating chronic pain,” he shared. “Sharing knowledge about their condition in a study is one way for patients to make life better for themselves and others with chronic pain.”
