Pragmatic Clinical Trials Leverage Valuable Informatics Tools

Oct. 5, 2017
As the Patient-Centered Outcomes Research Institute funds more comparative clinical effectiveness research studies, informatics researchers are developing new tools to identify and recruit potential patients and gather data.

As the Patient-Centered Outcomes Research Institute (PCORI) funds more comparative clinical effectiveness research studies, informatics researchers are developing new tools to identify and recruit potential patients and gather data.

On Sept. 12, the Patient-Centered Outcomes Research Institute (PCORI) board approved spending $97.9 million to fund 11 studies comparing different approaches to improving care for a range of health conditions.

These comparative clinical effectiveness research studies rely heavily on informatics infrastructure to recruit patients and gather data.

In a recent presentation at Duke University, Russell Rothman, M.D., the vice president for population health research at Vanderbilt University Medical Center, described some of the informatics infrastructure of one PCORI-funded study, ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness).

ADAPTABLE is a $14 million, three-year pragmatic clinical trial that is comparing the effectiveness of two different daily doses of aspirin widely used to prevent heart attacks and strokes in individuals living with heart disease. Its goal is to enroll 20,000 patients. Pragmatic trials are designed to reflect “real-world” medical care by recruiting broad populations of patients, embedding the trial into the usual healthcare setting, and leveraging data from health systems to produce results that can be readily used to improve patient care.

The Duke Clinical Research Institute is the coordinating center for ADAPTABLE.

Dr. Rothman, who serves as the co-chair of the steering committee of the study, is also the principal investigator of the PCORI-funded Mid-South Clinical Data Research Network (CDRN), centered at Vanderbilt University Medical Center (VUMC). The CDRN includes academic health centers, community hospitals and a range of outpatient practices including primary care, specialty practices and safety net facilities that serve diverse populations across the Southern United States.

The Mid-South CDRN has data on 9 million patients, Rothman said. “We have data going back to as early as 2004 in the network,” he added.

It encompasses three large health systems:

 • The Vanderbilt Health System, which currently includes electronic medical records for over 2 million patients;

• The growing Vanderbilt Healthcare Affiliated Network (VHAN), which currently includes 45 hospitals, 300 ambulatory practices, and will cover over 3 million patients in the mid-South;

•  A partnership with Greenway Health and other national organizations to provide access to over 1,600 practices and 16 million patients across the country; and

•  The Carolinas Collaborative, which includes University of North Carolina at Chapel Hill, Duke University and Health Sciences South Carolina and reaches over 9 million patients.

The CDRN also has been working on linking EHR data to claims data such as an all-payer claims database in Tennessee to get more complete pictures about patients, which Rothman said is key to studying outcomes.

The Mid-South CDRN has developed tools and capacities to support comparative effectiveness research and pragmatic research. It has methods to develop and validate computable phenotypes for identification of particular patient groups.  It can extract clinical data from electronic health records, claims, and other data sources.  It also has developed tools for electronic identification, contact, consent and recruitment of patients. 

Based on his presentation and details from the CDRN website, here are some tools developed to support pragmatic clinical trials:

DATA

The Mid-South CDRN uses the PCORnet Common Data Model Version 3 as the main syntactic and semantic standard. It leverages standard terminologies and coding systems for healthcare to enable interoperability with and responsiveness to evolving data standards.

They have taken EHR data from health systems and transformed it into the common data model to run queries against. There are 80 such data marts nationwide. 

The CDRN also used REDCap, a secure, web-based application that allows users to build and manage online databases and surveys.

REDCap uses a study-specific data dictionary to eliminate all programming requirements for the creation of electronic case report forms and participant survey instruments for individual studies.

PHENOTYPING

The Synthetic Derivative Record Counter (RecordCounter) is a web-based tool that provides exploratory data figures for research planning purposes and feasibility assessment. The RecordCounter allows the user to input basic medical data such as demographic information, ICD 9 and CPT codes, medications, labs, keyword document searches, as well as searching for available genetic data on subjects.

The Phenotype Knowledgebase website, PheKB, is a collaborative environment for building and validating electronic phenotype algorithms. PheKB has tools to enable cross-site collaboration for algorithm development and validation. These include electronic discussion and phenotype update notifications for a given phenotype; the ability to post algorithm validation details and tools for validation of algorithms including chart review forms and automated calculation of validation statistics such as specificity and sensitivity; and an integrated data dictionary and dataset validation tool used to ensure consistent formatting and coding of data sets to be shared.

RECRUITING

ResearchMatch, launched in 2009, is a disease-neutral, online volunteer-researcher matching service. Currently, over 100,000 volunteers, over 4,000 researchers, and 128 institutions are using ResearchMatch to streamline the connection between researchers and volunteers.

The studies are taking advantage of Electronic Recruitment via email.  Vanderbilt has a variety of patient portals including MyHealth and MyResearch at Vanderbilt that can be used for patient recruitment.  (Rothman said 30,000 patients have signed on to MyResearch at Vanderbilt, agreeing that they would be willing to be contacted directly about any study that fits their conditions.)

Additionally, patients with an encounter in Epic can be recruited electronically.

Electronic-Consent (e-Consent) is a newly evolving platform for consenting patients either on site or at home using a computer-based consent form rather than traditional paper documentation.  Consent forms can be implemented in REDCap, and patients can access consent forms via computer, mobile phone or tablet. Vanderbilt can recruit patients in REDCap via voice calls and SMS text messages. With this module enabled, participants can take automated surveys by phone or text message.

“We were able to survey over 10,000 patients in less than six months by leveraging these informatics approaches,” Rothman said.

ADAPTABLE is the first large pragmatic clinical trial in PCORnet. Researchers identified patients with coronary artery disease through phenotype and consented them electronically. Patients are being randomized into low-dose or regular aspirin groups and followed electronically for two years by pulling their electronic health record data and surveying patients as well. The goal is to see what the optimal dose of aspirin is. “We have been recommending aspirin for decades and still don’t know what the optimal dose is. We are excited to be answering that question in a pragmatic way.”

“PCORnet is a powerful network for pragmatic research,” Rothman said. “Informatics alone is insufficient to conduct pragmatic trials, but informatics approaches can help to identify, retain and follow patients.”

Among the projects the PCORI board approved on Sept. 12 was an $8.8 million study on opioid use among patients with chronic pain, assessing whether cognitive behavioral therapy or pain self-management therapy is more effective at alleviating pain and reducing opioid use.

The board also approved nearly $38 million to fund five studies to improve treatment of multiple sclerosis and an additional $18 million to fund two projects related to treatment for young patients with sickle cell disease who are transitioning to adult care.

Finally, the board approved $32.8 million for three studies that aim to generate real-world evidence through pragmatic study designs:

• An $8.5 million project comparing two types of therapy to lessen problems with swallowing in patients undergoing radiation therapy for head and neck cancer. One group of patients will proactively receive therapy while undergoing radiation, and the other group will receive therapy only if difficulty with swallowing occurs after radiation. 

• An $10.8 million study involving patients undergoing surgery to fix a broken bone in a limb that will determine which of two commonly available preoperative antiseptic skin solutions is better for reducing hospital-acquired surgical-site infections.
 

• A $13.4 million study to compare the effectiveness of two cavity-prevention techniques—a simpler treatment involving fluoride varnish and silver diamine fluoride, and a more-complex treatment involving sealants and fluoride varnish. The study is comparing the two techniques when delivered in school-based programs to Latino and Hispanic children in areas with a scarcity of dental care providers and clinics.

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