CER Initiative begins to take shape
Scientists forge ahead with comparative effectiveness research on many fronts
When the US Congress appropriated $1.1 billion toward accelerating comparative effectiveness research (CER) as part of the American Recovery and Reinvestment Act (ARRA) in 2009, no one was quite sure what that research scenario would look like. Now that many projects arc up and running, however, the picture is becoming clearer. CER, also known as patient-centered outcomes research, is designed to inform hcalthcare decisions by providing evidence and information on the effectiveness, benefits, and harms of different treatment options. The evidence is generated from research studies that compare drugs, medical devices, tests, surgeries, or ways to deliver healthcarc in real-world settings.
“I'm very pleased with the government's investment in this research,” says Neal Meropol, MD, chair of the American Society of Clinical Oncology (ASCO)'s CER Task Forcc.” The field of oncology is probably the most strongly guided by evidence in how we practice, and oncologists welcome new methodologies to inform therapies for patients.”
Allocation of Funds
Of the $1.1 billion, $300 million was awarded to the US Department of Health and Human Services' (HHS) Agency for Hcalthcarc Research and Quality (AHRQ), $400 million was awarded to the National Institutes of Health (NIH), and $400 million was designated to be allocated at the discretion of the HHS Secretary for a variety of patient-centered outcomes research and related activities.
Members of the Engclberg Center for Health Care Reform at the Brookings Institution published an analysis of ARRA-funded CER.1 Their breakdown demonstrated that:
$442 million (46.4%) was directed toward evidence development and synthesis activities;
$375.9 million (41.3%) was spent on improving capacity to conduct CER through data, infrastructure, and methods development;
$67.2 million (7.4%) was invested in activities and tools for translation and dissemination of evidence; and
$35.3 million (3.9%) was designated for priority setting and $10 million (1.1%) for stakeholder engagement tasks.
The authors also found that within specific clinical conditions, the greatest spending was on projects related to cancer ($31.5 million or 16.6%). At the same time, they noted that few funds have been spent on understanding the genetic or genomic basis of diseases or response to treatment ($4.8 million or 2.5%).
The authors recommended that new funding include more emphasis on experimental research, the evaluation of reforms at the health system level, the identification of effects on subgroups of patients, the inclusion of understudied groups of patients, and the dissemination of results.
Off to a Good Start
Thomas Feeley, MD, spokesman for the Institute for Cancer Care Excellence at The University of Texas M.D. Anderson Cancer Center in Houston, thinks the program is off to a good start. “I think the money was spent wisely byAHRQ and the Secretary,” he says. “Answering the clinical questions from the Institute of Medicine's CER priority list is great, but that's only half the story. You have to have the infrastructure.”
In addition to funding a wide range of CER research across the country, the federal government passed a provision within the Patient Protection and Affordable Care Act of 2010 to establish the Patient-Centered Outcomes Research Institute (PCORD. This nonprofit organization is charged with better enabling health decisions by performing research that provides quality, relevant evidence on how diseases, disorders, and other health conditions can be prevented, diagnosed. treated, monitored, and managed.
PCORI's 19-member board was appointed in Septembet Among others, it includes Ellen V. Sigal, PhD, chair and founder of the Friends of Cancer Research, and Carolyn Clancy, MD, director 0fAHRQ. The board must next create an infrastructure and entity, Dr. Clancy notes.
For the first several years, PCORI will be funded by public dollars ($10 million in fiscal year FYI 2010, $50 million in FY2O11, and $150 million in FY2012). Then, a tax on insurers will fund it through 2019. The law encourages PCORI to contract with the AHRQ, NIH, and other federal agencies that focus on CER.
“Sixteen percent of the total budget will come to AHRQ to support dissemination of work funded, training, and continued improvements and methods,” Dr. Clancy adds. The dissemination of research findings is essential, and electronic health records and physician clinical decision support maybe useful tools in that area, she says. “Our perspective is that dissemination has to be much more than through media and journals. We have to exploit every possible opportunity because patients and physicians need the information in real time when they're making treatment decisions.”
Experts note that, although progress has already been made in specific CER areas, no central database for all research exists. AHRQ queried various stakeholders and learned that they would like to see a tool, similar in format to http://www.ClinicalTrials.gov, which enables easy entry of information. As a rcsult, AHRQ recently awarded a $1.6- million contract to the Lewin Group, based in Falls Church, Virginia, to develop a Web-based inventory that will categorize and catalog all federal and nonfederal CER activities.
Different Perspective on CER
A private, nonprofit organization that comes at CER from another angle is the Center for Medical Technology Policy (CMTP), based in Baltimore, Maryland, and led by Scan Tunis, MD, MSc. “There is a disconnect between the clinical research enterprise and what clinicians, patients, and payers need to know to make good decisions,” Dr. Tunis notes.
The CMTP's goal is to change the process, enabling end-users to be involved in research decisions from the start by helping determine what questions need to be addressed. Dr Tunis became aware of this challenge when he served as chief medical officer at the Centers for Medicare and Medicaid Services (CMS).
“It caused me to change the focus of Medicare's role,” he says.” As opposed to being a passive consumer of research designed by others, we agreed to pay for things only in the context of studies in which we had input. I like to think we were part of creating the wave in comparative effectiveness research.”
Dr Tunis refers to CMTP's efforts as “research diplomacy”—working closely with academic medical centers to help design studies that are relevant to end-users by engaging them and allowing them to give input. “We have a good relationship with all the stakeholders, and we get them to talk, negotiate, and reach a common vision,” he says.
One example is CMTP's work with the Southwest Oncology Group and Fred Hutchinson Cancer Research Center on their National Cancer Institute (NCI)-funded, phase 3 clinical trial that examines whether the Oncotype DX assay can predict which lymph node-positive breast cancer patients will benefit from chemotherapy, which has been effective in lymph node-negative patients. “We met for a day in Seattle and helped modify the protocol to make sure it was relevant and meaningful to patients, clinicians and payers:' Dr Tunis says. “Some of the final reservations about the trial were in part overcome by the stakeholder group, which can provide a level of assurance to the funders.”
Dr Tunis envisions the CMTP as an adviser and collaborator with PCORI and other CER-related entities, and he remains optimistic about CER's future. Many “old school” researchers have enthusiastically embraced it once they have had more experience in the area, he notes, citing Laurence Baker, DO, the chair of SWOG, as an example. “Even in the extraordinarily unlikely event that PCORI were to be dc-funded by the new Congress, CER has reached and is passing the tipping point where it's going to go forward on its own no matter what,” he says.