The Patient-Centered Outcomes Research Institute issued its heavily anticipated first report on national priorities Monday, but stopped short of specifying any diseases or conditions that many hoped or feared the $4.5 billion agency would target for comparative effectiveness research.
Rather, the brief, 22-page "Draft National Priorities for Research and Research Agenda, Version 1," merely carries general statements, which allude to five broad categories the 21-person Board of Governors believes address deficits in methods of research, rather than research on drug or procedure efficacy.
For example, there is no mention of any issues outlined in the Institute of Medicine's report, 100 Initial Priority Topics for Comparative Effectiveness Research from 2009.
"Well, you may say, why didn't you list a few?" Harlan Krumholz, MD, one of PCORI governors, rhetorically asks in an interview with HealthLeaders Media after the report's release Monday.
"Rather than say, well, you know people with A-fib, they have a more important decision than people with breast cancer or people with multiple sclerosis have a more important opportunity than people with Parkinson... We didn't want to exclude particular patient groups or particular questions."
Rather, the panel said, it was more important to "define the boundaries" and "define the types of questions that we want to invest in," says Krumholz, a cardiologist who directs the Yale-New Haven Hospital Center for Outcomes Research and Evaluation.
Eventually, says Krumholz, the commission wants to look at how patients make choices and how they have access to information to make informed decisions. And it wants to understand how to conduct research with patients to test different approaches.
"Take Joe Paterno (former Penn State football coach who died Sunday after developing complications from chemotherapy)," Krumholz says. "I don't know how informed he was about how an 85 year-old with advanced lung cancer, what those kinds of options are and the likelihood of dying from chemotherapy might be versus taking a more palliative care approach, versus some other option that he might have had.
"Most people in that situation are baffled by their choices and lack personalized estimates of what their risks and benefits are."
"We had no shortage of urgent questions that needed to be addressed," Krumholz says, adding that "the circumscribing of an agenda around a small number of them, however important they may be, would lead to the exclusion of others."
Tanisha V. Carino, senior vice president of the independent health advisory firm Avalere Health, said she was initially surprised by the report's lack of specificity about the burden of disease, such as heart failure or pneumonia.
"I thought they would go the route of the Institute of Medicine...which was very specific down to the actual research."
But when she read the report, she began to see a different picture, she explains. "They're trying to carve a different niche for themselves, and that may be where we have to start, is to begin asking people ... how to actually integrate the end users of the information into the actual research infrastructure.
More specific areas will likely come later, Carino says, when the payer community, instead of just federal appropriations, starts to pay PCORI's bills.
The institute is budgeted to spend $4.51 billion between 2010 and 2019. But starting in fiscal year 2013, in addition to $150 million per year from general appropriations, PCORI's funding will come from a $1 annual per member charge to health plans with another $1 per Medicare beneficiary coming from the Medicare Trust Fund. Funding goes up for health plan members and Medicare beneficiaries to $2 for each starting in 2014.
The five broad areas the institute listed are:
1. Comparative Assessment of Options for Prevention, Diagnosis, and Treatment
This includes studies that compare effectiveness of strategies for prevention treatment, screening, diagnosis or surveillance that have not been adequately studied against alternative options to better support decision-making and studies that compare risk-stratification and prognostication tools with attention to demographic, biological, clinical, social, economic and geographic factors that influence outcomes.
2. Improving Healthcare Systems
This area is designed to focus more on how care is delivered rather than what care is delivered. For example, the agency wants to study such delivery modalities as disease management, telemonitoring, telemedicine, integrative health practices, care coordination, performance measurement and incentives, protocols. This is said to include alternative system approaches, such as methods of communication and documentation in electronic health records.
It also is to examine "new and extended roles for allied health professionals" such as pharmacists, nurses, physician assistants, dentists, patient navigators and volunteers."
3. Communication and Dissemination Research
How should the healthcare system increase clinician and/or patient shared decision making and awareness of options is the focus of this targeted area for research. This area includes research in how the healthcare system can provide information using understandable language in a variety of settings.
4. Addressing Disparities
The institute wants to reduce or eliminate disparities by studying differences in patient preferences or differences in response to therapy across socioeconomic, demographic and other patient characteristics.
5. Accelerating PCOR and Methodological Research
This area is designed to address research on better ways to conduct comparative effectiveness research, such as better ways to engage patients and those at risk, "particularly those who have been historically hard-to-reach," and assure their validity.
The agency asks the public to comment on this draft report until March 15.