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American Reinvestment and Recovery Act

ARRA Funds Support Comparative Effectiveness Research in Cancer

Improving Patient Outcomes and Healthcare Value

A relatively new but increasingly important concept, comparative effectiveness research (CER) promises to help make healthcare more personalized, safer, efficient and effective. And researchers at the NCI are leading the way in this rapidly emerging field.

CER, also called Patient-Centered Outcomes Research, seeks to evaluate the impact of different options for diagnosing, treating or managing a given condition in a particular patient group. Comparative effectiveness researchers are looking beyond the tightly controlled conditions and often homogenous populations found in clinical trials to determine how diagnostics and treatments are being used—and what impact they are having—in real world settings among diverse patients.

Critics of CER often worry that comparing different treatment options head-to-head could ultimately limit treatment choices, and prevent physicians from determining a course of care based on expertise and  a particular patient’s needs. But proponents explain that this misses the point: the fundamental purpose of CER is to uncover objective information that will allow health care providers and their patients to make the best individualized decisions possible.

The American Recovery and Reinvestment Act (ARRA) gave CER a tremendous boost, providing $1.1B in funding for CER, $400 million of which went to the National Institutes of Health.

The NCI and its network of cancer centers and researchers has deep experience, scientific expertise and a nationwide infrastructure to put CER funding to good use. To date, ARRA funds enabled NCI to support approximately $70 million in research, including $50 million in Grand Opportunities and $13 million in Challenge Grants. Grantees/Researchers are examining a broad spectrum of cancer-related issues, including smoking cessation trials, cancer screening methods, risk behavior interventions, surgery and treatment options, community cancer surveillance and genomic medicine. Remaining NIH ARRA funds for CER will be used to support a variety of initiatives, including training the next generation of CER researchers and improving CER methods.

CER Grants in Action: University of Virginia Charlottesville

One Grand Opportunity grant recently awarded by the NCI went to the University of Virginia Charlottesville to create a genome-enabled electronic medical record. According to one of the grant’s principal investigators, Dr. William Knaus, this will provide user-friendly, standardized information about a patient’s genome and family history. He said this technology will help patients and physicians develop personalized paths to cancer prevention and treatment.  Their research helps form the basis for future independent CER projects, including an effort to understand the value of referring patients to genetic counselors for evaluation and possible testing.

“We want to help make prevention efforts very individual and specific,” said Dr. Knaus. “Currently there is a great deal of confusion and controversy, for example around generalized breast screening guidelines. We think that if you can provide individualized preventive profiles, every woman will begin receiving mammograms when it is appropriate for them—based on a wide variety of personal factors such as family health history, genetics, environment and lifestyle.”

ARRA funds awarded through this grant have helped create three new positions at UVA—a project director, genetic counselor and a research assistant—and has helped to preserve two research faculty positions.

“Right now we don’t do an adequate job of using family medical history and emerging genetic tests to provide optimal preventative and curative medicine to many Americans,” said Dr. Knaus. “As emphasized by Francis Collins, now is the time for genomics and personalized medicine to begin to influence and enhance medical practice and delivery. By getting the right information about what works best to the right people at the right time, we can begin to address the rapidly escalating costs in our health care system.”

CER Grants in Action: Group Health Research Institute

Dr. Diana Buist from the Group Health Research Institute in Seattle is currently involved with three new CER projects funded by NCI Grand Opportunities grants.

One project, called “SEARCH: Screening Effectiveness and Research in Community Based Healthcare”, leverages data from the Cancer Research Network—a network of research centers across the U.S.—to improve colorectal and cervical cancer screening in real-world settings.

“Cancer Research Network institutions are geographically, racially and ethnically diverse and provide comprehensive care to patients,” said Dr. Buist. “This gives us a complete palate of data throughout the continuum of care—from etiology through prevention, treatment and ultimately, outcomes.” According to Dr. Buist, SEARCH is creating an infrastructure to link data from a variety of sources, allowing researchers to quickly and efficiently examine the effectiveness of various screening and prevention methods, and translate these findings directly into medical practice.

Other grant projects underway at Group Health in collaboration with the University of Washington and the Fred Hutchinson Cancer Research are exploring cancer screening and diagnostics. One study, with the Breast Cancer Surveillance Consortium is researching how to best design and deploy breast screening programs in specific populations, while another study known as “ADVICE” is evaluating the effectiveness of various diagnostic technologies across multiple cancer types.

“There’s very, very little evidence around the use of cancer diagnostics at the time of workup,” said Dr. Buist. “For example, we know that CT scanning is happening a lot, but we don’t know whether or not it has any impact on outcomes. To answer that question, we need to pool data in large samples with a fair amount of population variability.”

In addition to paving the way for future groundbreaking research studies, through these Grand Opportunity grants participating institutions have created more than 20 positions, including computer programmers, project managers, research associates and biostatisticians.

“These stimulus funds have allowed researchers an opportunity to really think outside of the traditional box—and we need to be doing that to move the science forward,” said Dr. Buist. “My hope is that we, the scientific community, can really educate folks around the importance of comparative effectiveness research. What types of questions can and should be addressed, and why these questions are important to healthcare delivery as well as public health.”