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PCORI Forms its First Four Advisory Panels and Selects Dr. Alvin I. Mushlin

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Dr. Alvin I. Mushlin, the Nanette Laitman Distinguished Professor and chairman of the Department of Public Health at Weill Cornell Medical College and public health physician-in-chief at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, was selected for one of four new advisory panels of the Patient-Centered Outcomes Research Institute. PCORI received 1,295 applications for panel membership and selected 84. Dr. Mushlin is the first faculty member based at Weill Cornell Medical College to serve PCORI in an official capacity as a member of a standing PCORI board or committee.

PCORI was authorized by Congress in 2010 as part of the Affordable Care Act. As an independent, non-profit organization, PCORI's mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions and to speed the implementation of patient-centered knowledge into practice.

Dr. Alvin I. Mushlin
Photo credit: John Abbott

Dr. Mushlin will be a member of the Advisory Panel on Assessment of Prevention, Diagnosis and Treatment Options. This panel will examine research on the comparative effectiveness of alternative strategies for prevention, treatment, screening, diagnosis and management of diseases. The other new panels will focus on patient engagement, improving health care systems and addressing disparities.

The new panels will advise PCORI on critical research questions for possible funding and on ways to ensure that PCORI's work remains patient-centered. Members will help PCORI identify and prioritize research questions in their respective areas and will provide ongoing feedback on evaluating and disseminating resulting research. Each panel has 21 members representing different sectors across the health care community, including researchers, clinicians, patients, caregivers, insurers, employers and industry. The panels are critical to PCORI's ongoing effort to ensure that stakeholder input helps to refine its research portfolio and other activities. We asked Dr. Mushlin to tell us more about the importance of the PCORI panels and his own work in comparative effectiveness research.

How has your own research prepared you to be an active player in this field?

I have always thought that we can make medical care more effective and efficient, and thereby help more people and do a better job for our patients. Although I recognize the need for more and better biomedical research, I have thought that just having more knowledge about diseases and what causes them was not enough. While basic science insights are critically important, in order to determine how to optimize care and achieve the best outcomes, we need research methods and approaches from the public health and social sciences. Studies to help understand the effectiveness of diagnostic tests, treatments and clinical strategies, as well as how to deliver care most efficiently, are necessary. One of my early mentors helped me to learn about such efforts in the fields of clinical epidemiology and health services research, and I entered a training program that included graduate education to gain those research skills.

During my career I have studied and evaluated a number of important clinical strategies and technologies, including diagnostic tests and radiologic procedures such as magnetic resonance imaging (MRI). My work has always focused on new and significant technologies, but it has also included the development and testing of new research methods that help to improve our understanding of their effectiveness and efficiency in the future. I was involved in the first randomized controlled trials evaluating automatic implantable cardioverter-defibrillators (ICDs) for patients with coronary artery disease at risk for fatal arrhythmias (the MADIT trials), and led the economic analyses alongside these trials. The methods we developed still represent a model for how to perform such studies.

What is the current focus of your research?

My major research interest now is to develop further the methods and capability to improve our knowledge base for informing clinical decision making, thereby enhancing the value of medical care. Examples include the development and use of patient registries and large observational data sets to track outcomes and assess the effectiveness and safety of medical devices, tests and technologies. I am also still involved with projects that use decision analysis to help define clinical use recommendations. Through these and related approaches we can do more to inform the intelligent use of medical resources and to reduce the future costs of medical care, particularly in the United States.

How successfully do you think comparative effectiveness research is currently being translated into policy and practice worldwide?

I have always thought that when the evidence was clear and compelling, doctors would use it to change what they are currently doing, and that hospital and health care systems would follow their lead. My thinking now is changing a bit, as it seems increasingly clear that this is not always the case. I recently completed a study with a young cardiologist colleague here on the use of medications as compared with stents or angioplasties for patients with coronary artery disease following the results of the COURAGE trial, which showed that drugs were basically as effective as angioplasty or stents for many such individuals. We found that a substantial percentage of patients were not receiving the recommended medical therapy before getting stents. As a result, we are now systematically tracking the diffusion and impact of research findings from comparative effectiveness research (CER) in order to better understand how to make medicine more evidence-based. Although this is critically important, the main problem remains that there is still not enough CER being done to inform and influence care in a major way, and some of the CER that is out there is not solid and convincing enough to bring about change.

How do you think comparative effectiveness research can be improved?

First, we need more and better CER. It's more than a lack of funding and lack of research opportunities that is the problem. We also need better methods, study designs and statistical techniques to carry out this kind of research.

We need to find feasible alternatives and complements to randomized controlled trials (RCTs) for the evaluation of new drugs, devices or approaches in medicine. While RCTs are recognized as producing the most reliable data, they can be very expensive to conduct and take a long time to produce results, and in some situations it may not be possible or ethical to do them. We need to learn how to use observational data sets (i.e. electronic health records, registries, hospital data and administrative data) for these evaluations. Innovations in study design and statistics, as well as better methods of record-keeping, could make existing data the keys to important medical research in the future. Significant progress along these lines, mostly through electronic medical records, has been made in the last several years, but we need to do even more. There is still too much skepticism about our ability to inform clinical practices from such observational studies. That must change; however, even the best evidence will not alter behavior until we have better strategies to influence thinking and to change health care systems — that will always require a mix of science and politics.

Why is outcomes research so important to health care today?

The ACA has its aim to make high quality health care available and accessible to all. Therefore, determining the most effective, efficient and cost-effective care is essential. Developing and refining methods to make CER truly credible and capable of influencing clinical practice and health policy is of the utmost importance. We need to accumulate a sizable and robust research portfolio of studies to demonstrate that CER can deliver and improve the quality and efficiency of medical care. We should not underestimate the challenge that this represents or lose track of this important dimension as we proceed over the next few years.

Why is outcomes research integral to the ACA?

The goal of the ACA is to enhance access to quality medical care for all Americans. For this to become a reality, we must find ways to inform the practice of medicine with practical information about which interventions work best in medical care, and thereby to use our resources most effectively to enhance the health of our patients. This is the purpose of patient-centered outcomes research, the words that form the name of this institute. Also envisioned in the ACA are changes to the ways we insure and finance medical care that shift the incentives generally toward these same goals. However, unless we, as physicians and patients, have the information to make optimal decisions, the financial incentives alone will not assure the results we all want. Outcomes and comparative effectiveness research are essential to advance the goals of the ACA. I can't see any way that our country would be able to proceed along this path without some way of encouraging and enhancing this type of research. I also think that, by doing so, we are reestablishing our lead in the world in the quality and delivery of medical care.

How do you see the panel contributing going forward?

The Institute of Medicine and Department of Health and Human Services have already released priority lists of general topics for CER. The new panels established by PCORI will play a significant role in advising which specific CER projects should be encouraged and funded. Of course, we all think that as long as studies include health-related outcomes that CER will be useful and have importance to patients. But really understanding patients' concerns and the elements of care that are critical to them and to their physicians, and how these can be incorporated into priority setting for research funding, is new territory. I anticipate that the panel will give the inclusion of the patient perspective and the likelihood that research will influence clinical decisions significant emphasis in its work and that the priorities it develops will thereby break new ground and be of real importance for medical research, writ large, going forward.

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