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How Neurology Can Keep Patients Front and Center in Research Outcomes — Embracing a National Initiative

Moran, Mark

doi: 10.1097/01.NT.0000414603.38144.38
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The AAN and its members discuss ways in which neurology can embrace a federal initiative to focus on patient-centered research and outcomes.



Communication with patients and dissemination of evidence-based best practices to facilitate patient participation in health care decisions — one of five areas designated as research goals by a federal initiative, the Patient-Centered Outcomes Research Institute (PCORI) — will be a priority for the AAN.



In a March 5 letter written in response to the draft priorities for research released in January by PCORI, AAN President Bruce Sigsbee, MD, said that the goal of communication and dissemination, outlined in the PCORI report, is “most concordant” with the current efforts of the AAN and has the organization's support as a highest priority.

This is an area where not only is the gap large, but there are few other federal dollars dedicated to address this priority,” Dr. Sigsbee wrote. “The private sector is incentivized to address ‘communication and dissemination’ when there is financial gain. This is at variance with the ‘comparative effectiveness’ motivation, which is expected to lead to utilization of the least expensive resources needed to attain effectiveness. PCORI needs to focus on developing usable end products for dissemination and application by clinicians and patients.” [For more on the AAN response, see “AAN Responds to Mandate from PCORI.”]



PCORI was established as part of the Patient Protection and Affordable Care Act. The initiative intends to encourage research that provide patients with a better understanding of the prevention, treatment and care options available, and the science that supports those options.

In its January report, the PCORI outlined national research priorities including five broad areas about which it is seeking public comment — the assessment of prevention, diagnosis, and treatment options; improving health care systems; communication and dissemination; addressing disparities; and accelerating patient-centered and methodological research. (For a summary of the five priority areas see “PCORI: Five Priorities for Research.”)

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In an interview with Neurology Today, Richard Dubinsky, MD, chair of the Practice Improvement Subcommittee of the AAN Practice Committee, said dissemination of recommended best practices has been a continuing challenge.

“This is an important gap identified by PCORI that needs to be addressed,” he said. “It appears that many health care providers are slow to adopt recommendations in our practice parameters unless they are encouraged to do so by financial incentives or disincentives that are part of reporting systems. But we would rather have buy-in from clinicians based on the science behind our recommendations than have external factors influencing clinical practice.”

Dr. Dubinsky also highlighted, as especially important for neurology, PCORI's fifth priority area — accelerating patient-centered and methodological research. “In studies of neurological treatment, outcomes have been fairly simplistic and not always patient-oriented,” he told Neurology Today.

“Studies of multiple sclerosis, for instance, have traditionally looked at the number of new lesions over a period of time with the assumption that the number of lesions correlates with disease burden,” he said. “But a functional outcome measure based on patient and family member input might have more validity than simply counting lesions. Similarly, in headache research there has been a tendency to simply count the number of headaches without incorporating the fact that there can be tremendous variability in how disabling headaches can be for different patients.

“These are lofty goals that PCORI has set out as their objectives, but this is a field of medicine that is in its infancy,” Dr. Dubinsky continued. “It is not certain to me how quickly this field of patient-centered research can mature to achieve these objectives.” He noted that at this stage PCORI is seeking public comment on how best to allocate their first round of grant dollars to best pursue the large research objectives set out in its report.

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Neurologists who spoke with Neurology Today agreed that the research priorities outlined by PCORI are largely non-controversial and that all of them are relevant to neurology. For example, with regard to prevention of stroke, S. Claiborne Johnston, MD, said that carotid endarterectomy is probably not offered to all the people who would benefit while others are receiving it who shouldn't.

“Understanding attitudes about risk and also predictors of stroke risk with and without treatment could allow us to make better recommendations,” he said. “Similarly, treatments for unruptured aneurysms could be applied much more rationally.”



Dr. Johnston, director of the Clinical and Translational Science Institute and associate vice chancellor of research at the University of California, San Francisco, added: “We also definitely need to work on better care coordination models. There are lots of chronic and debilitating diseases in the neurosciences and traditional physician-centric models of care are not necessarily the best. Some models for care of dementia are pretty remarkable, relying much more heavily on nurses and training of patients' families and other caregivers. Neurological diseases are increasingly burdening society so we need to be front and center in studying better ways of caring for our patients not just when they are acutely ill.”

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Barbara Vickrey, MD, MPH, professor and vice chair of the department of neurology at the University of California, Los Angeles (UCLA), noted that increasing attention is being paid to health disparities.

“There is a lot of interest in the use of new technology to improve delivery of health care services and to give individuals tools to help manage their own chronic condition or to use in health promotion, for example, the application of handheld mobile devices to help people with medication reminders or to track their activity level,” she told Neurology Today.

“Yet, some groups may be disadvantaged in being able to access and use this technology due to low income or to language or cultural barriers. So, comparative effectiveness research that compares these new health technology strategies to usual care must include these disadvantaged populations, or else health disparities might be aggravated because of lack of access to or applicability of technologies found effective in more educated or wealthy groups.”

She noted that there are four stages of research on disparities: surveillance; research on causal or contributing factors; testing interventions; and dissemination of effective interventions. “In neurology, with a few exceptions, we are still at the first and second stages of disparities research, in which there may be anecdotal evidence of the existence of disparities but little data on the extent or on contributing factors, which are typically needed to base applications that fall within PCORI's mission,” she said.

The most outstanding exception, she said, is stroke, for which it is well-documented that there are substantial differences in the occurrence and outcomes of stroke across minority groups relative to whites, and at least a partial body of knowledge on mechanisms driving these differences.

Dr. Vickrey cited as an important example of disparities research a 2010 study in the journal Neurology showing a much higher age of death from muscular dystrophy for whites compared to blacks in the US.

“These findings were not anticipated by many advocates and researchers in this area, and the data are now driving further research to figure out what are the determinants of this problem, and research on how to redress it,” she said.

“There is an enormous potential opportunity through PCORI for research to address disparities in health and health care for neurological conditions, but for many neurologic conditions, we need the surveillance and mechanisms research for investigators to be able to show the need for studies that PCORI might rate as a high priority.”

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  • Assessment of Prevention, Diagnosis, and Treatment Options. Research should focus on 1) clinical options with emphasis on patient preferences and decision-making, 2) biological, clinical, social, economic, and geographic factors that may affect patient outcomes.
  • Improving Health Care Systems. Research should focus on 1) ways to improve access to care, receipt of care, coordination of care, self-care, and decision-making, 2) use of non-physician health care providers, such as nurses and physician assistants, and the impact on patient outcomes, 3) system-level changes affecting all populations, diseases, and health conditions.
  • Communication and Dissemination. Research should focus on 1) strategies to improve patient and clinician knowledge about prevention, diagnosis and treatment options, 2) methods to increase patient participation in care and decision-making and the impact on health outcomes, 3) communication tools that enhance decision-making and achieve desired outcomes, 4) ways to use electronic data (“e-health records”) to support decision-making, 5) best practices for sharing research results.
  • Addressing Disparities. Research should focus on 1) ways to reduce disparities in health outcomes, 2) benefits and risks of health care options across populations, 3) strategies to address health care barriers that can affect patient preferences and outcomes.
  • Accelerating Patient-Centered and Methodological Research. Research should focus on 1) ways to improve the quality and usefulness of clinical data in follow-up studies, 2) methods to combine and analyze clinical data that follow patients over time, 3) use of registries and clinical data networks to support research about patient-centered outcomes, including rare diseases, 4) strategies to train researchers and enable patients and caregivers to participate in patient-centered outcomes research.

The entire report is online at

Mark Moran

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In the March 5 letter to the Patient Centered Outcomes Research Institute (PCORI), AAN President Bruce Sigsbee, MD, urged that funding of PCORI priorities avoid duplication of existing efforts in both research and infrastructure. “For example, where adequate alternative funding sources are available, such as for developing options for prevention, diagnosis, and treatment, PCORI should support the utilization of existing sources and focus its unique funds on its other priorities,” he wrote.

And he emphasized that PCORI needs to have an ongoing evaluation plan to measure how it is meeting its goals. Dr. Sigsbee also proposed that the following items be incorporated into its five priority areas:

  • Quality of care in smaller, rural hospitals versus tertiary care large hospitals from the perspective of stakeholders (patients, providers, insurers).
  • Value of high-priced interventions in health care from the perspective of stakeholders, especially patients and caregivers.
  • Definition of outcomes and determination of how selected outcomes apply to specific diseases, patient populations, provider type, and structure.
  • Emphasis on improved communication between health care providers and patients/caregivers.
  • Payment and reimbursement structures and strategies to ensure that physicians and other health care providers are not dis-incentivized to participate in patient- and outcome-oriented practice. PCORI must ensure that clinicians are not alienated or ignored and that patient-physician communication research agendas support this fundamental concept.
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• Kenneson A, Vatave A, Finkel R. Widening gap in age at muscular dystrophy–associated death between blacks and whites, 1986–2005. Neurology 2010; 75:982-989.
    ©2012 American Academy of Neurology