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Pragmatic clinical trials: Testing treatments in the real world

Pragmatic clinical trials: Testing treatments in the real world

Do you know the difference between an explanatory and a pragmatic clinical trial? Many researchers are familiar with explanatory clinical trials, which look at the effectiveness of a particular intervention to improve health in a controlled setting. But what makes a clinical trial pragmatic?

Pragmatic trials may test the same intervention as an explanatory trial, but they are conducted in real-world clinical practice settings, with typical patients and by qualified clinicians, who may not, however, have a research background. Often, the positive results from explanatory trials have been found to be less effective in practice than they were in the lab, because several factors not present in the controlled setting can affect the eventual outcomes.

What makes pragmatic trials different?

With older people, for example, it's often difficult to recruit the required number of participants who fit strict eligibility requirements. Many older people have, or are at risk for, conditions such as high blood pressure or heart disease. These conditions are likely to exclude them from explanatory trials. In pragmatic trials, though, researchers often relax eligibility requirements and accept participants with these conditions and control for it in their analysis.

Another feature of pragmatic trials is how participants are randomized to treatment and control groups. In an explanatory trial, participants may be randomized in the order that they are recruited. In pragmatic trials, participants are often randomized at the group level. One group of participants treated in a setting such as a hospital, nursing home, clinic, or physician's practice, might receive the intervention, while people matched to be treated in a similar setting might serve as the control group. Using multiple health systems as the trial setting increases the likelihood that the results of the research can be widely applied.

Popularity is increasing

Pragmatic trials are becoming increasingly popular because they're well suited for studies of how to get health services out into wider practice. In addition to testing a particular intervention, pragmatic trials can also illustrate the differences in how the intervention works in different health care settings, such as hospitals, clinics, or physician practices. Pragmatic trials also seem likely to allow greater participation of older adults.

As health care has become more integrated, the system itself has an increasing influence on care. Pragmatic trials are taking advantage of these settings and conducting research that is specific to the practitioners and patients involved in these complex systems. They may use the full apparatus of a health system, including an electronic health record, patient reminder systems, telephone-based care, and sometimes group visits.

NIA and pragmatic trials

NIA recently joined with other NIH Institutes to fund studies using a pragmatic clinical trial approach. One relevant RFA (RFA-RM-16-019) is using the NIH Common Fund's Health Care Systems Research Collaboratory, which currently coordinates the work of nine pragmatic trials.

NIA is particularly interested in conducting additional pragmatic trials. Some of the studies now in progress include:

  • PROVEN – a cluster-randomized control trial of an advance care planning video program for nursing home patients in 359 facilities within two health care systems.
  • STRIDE Study – a cluster-randomized trial to determine the effectiveness for fall prevention of an evidence-based, patient-centered, multifactorial intervention that combines elements of practice redesign; a multifactorial, individually tailored intervention; and practice guidelines offered by the CDC and other organizations.
  • Shed MEDS – a randomized controlled trial to evaluate the effects of an intervention to reduce medication use among hospitalized older adults discharged to skilled nursing facilities.
  • TRAIN-AD – a cluster randomized controlled trial to evaluate a multicomponent intervention to improve infection management for suspected urinary and lower respiratory tract infections among advanced dementia residents in 24 Boston area nursing homes.

The National Advisory Council on Aging recently approved a concept for a funding opportunity announcement for support of additional pragmatic clinical trials. We'd love to hear your ideas for trials with real-world settings and real-world solutions. Please contact me or comment below.

9 Comments
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Posted by Jeff Borghoff on Jun 07, 2017 - 1:42 pm

Marcel, do these pragmatic trials include trials that can be conducted for older people with Alzheimer's and related dementias?

Posted by Marcel Salive on Jun 16, 2017 - 3:58 pm

Yes, some of these pragmatic trials are studying the services or treatments for persons with Alzheimer’s disease and related dementia. Further such trials would be welcome for submission to the NIA.

Posted by Lynne on Jun 07, 2017 - 1:56 pm

As more attention is paid to efficacy and feasibility of interventions in real-world settings, I hope funding agencies will prioritize those proposals that include not just stated commitment from community partners, but also feature sharing of resources and decision-making with community partners. This is the only way to build community capacity for research, as well as increase probability for real-world changes after the funding ends.

Posted by Marcel Salive on Jun 16, 2017 - 3:59 pm

Yes, I agree. We have in some cases encouraged the investigators to outline in detail the role and contributions of their community partners in the delivery of the intervention.

Posted by LuayneSmith on Jun 08, 2017 - 11:29 am

Cyber communication is used more often causing frustration and confusion with elderly and many baby boomers. Many people prefer the written word, but find we are charged a fee if we ask for written bills, appointment reminders and even bank statements. Moreover, those who are trying to get used to the cyber system are knocked off line because they are not typing fast enough for the computer at the receiving end has been programmed without options to "go slower." Just one of many thoughts from mom, daughter, caregiver for dimentia patients and nurse with over 30 years experience.

Posted by Ronald"Bozo"McLain on Jun 08, 2017 - 11:34 am

Do any of the studies actually help anyone? Or is it just someway for someone who wants to make some money possiable?

Posted by Marcel Salive on Jun 16, 2017 - 4:00 pm

The intention of pragmatic trials is to improve health, and so we believe that would be helpful. The evaluation of the trial results is typically based on whether or not health is improved.

Posted by Darla on Jun 08, 2017 - 11:28 pm

The theory behind genetic MTHF 677 and how medications adversely affect treatments and how to educate physicians on this genetic disorder to better serve patients

Posted by Marcel Salive on Jun 16, 2017 - 4:01 pm

Thank you to all the commenters for these thoughtful responses. I look forward to other comments and would be happy to discuss research ideas by telephone as well.

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