23rd on book tv on cspan2. Up next, the conversation from the Bipartisan Policy Center about Patient Centered research. How to provide the most effective medical treatment. Well hear from doctors of the Veterans Affairs department, John Hopkins University and heartfelt patient. [inaudible] [inaudible] good morning. It is great to see olivia here today. Im the chief medical advisor and i want to welcome all of you to todays event. For those of you who are new to bbc. We want to come by the best ideas from both political ideas to promote health and security for all. Todays event is being streamed live online and on cspan2. A recording will be available later this week. We invite you to interact with us on twitter about todays event using the bpc live. Well be taking questions after the panel discussion. If you are watching online and have a question for our panelist or opening speaker you can use bpc live and we will try to get to those during the q a. Over the next 90 minutes will be discussing Patient Centered comparative Effective Research. Essentially what our topic will be focusing on today is identifying how best to provide optimal care for patients based on science and based on patient priorities. As a physician for many years i recall instances conversations with patients when there was evidence in support of a particular course of treatment over another. I als recall other instances whn theres a lack of evidence. It is important for Healthcare Providers and patients so they can make the best Treatment Choices together. We call this, shared decisionmaking area today swarm is the first of two Educational Forums bpc plans to host on this topic. This morning we hope to learn how and why this research is conducted, how the results are used by patients, Healthcare Providers and other stakeholders and how this process is currently funded. Our second form will go deeper into the future scope and direction of comparative Effectiveness Research and its role in improving outcomes and effects on how care spending. Im sure the panel will touch on this today. Were honored to be hosting a fantastic group of speakers and leaders from the public and private sectors. We are thrilled to have a patient joining us today to provide her thoughts on this very important topic. Let me start by introducing doctor jody siegel who will be our opening speaker. You have jodys bio in front of you. She is a professor of medicine at Johns Hopkins university. A National Expert on comparative Effective Research and she will help by providing an overview of our topic. This will be followed by a moderated discussion with our panel in a discussion with all of you. Thank you. [inaudible] [inaudible] [inaudible] [inaudible] [inaudible] thank you very much for the invitation to be here today to join you and teach about this important topic before we hear from the panel of experts. I aim it for you to understand the goals of comparative Effective Research. But we hope to accomplish with this research and what fits the Translational Research pathway. As is fitting for Patient Centered research, lets start with the patient. Some of you may have asked this question to your doctors, doctor, should i be taking aspirin to prevent a heart attack . I know that i have some worries and risk factors. Should i . Clearly as clinicians we all want to get the right treatment to the right patient at the right time. Comparative Effective Research it generates evidence to inform the decisions that we make as clinicians but are made as pairs and as patients make. I wanted to start verbatim with the definition of comparative Effectiveness Research from the institute of medicine in 2009. Comparative Effectiveness Research is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor clinical condition or to improve the delivery of care. The purpose of cer is to assist all stakeholders, clinicians, purchasers, policymakers to make informed decisions. This is about making decisions that will improve healthcare at the individual and Patient Healthcare levels. What are these questions that we need to make decisions about . What do Patients Want to know . Should i take aspirin . Should i start mammography at age 40 . Should i have my cancerous prostate remover in my safe waiting a bit . Should i take one of the newer anticoagulants to treat my blood clot . What do doctors want to know certainly some of the same things, but also, should i use the robot in this hysterectomy or should i use the usual open procedure . Should i recommend to my patients a colonoscopy or the new dna based stool cards adequate . Are the new medicines for diabetes better than metformin which i was prescribed . What does medicare or other pairs want to know . Should we cover implantable defibrillators . Should we cover Home Care Services after hip replacement . How often should we cover jerry jerry nutrition for those in nursing homes. Lets take a look at where we are in the terms of the pathway. A lot of people look at translational pathway moving left to right, basic research. Research that understands the mechanism of disease, the chemistry, the biology, physiology, genetics of how disease occurs. Clinical research is often early testing in humans. Testing treatments and devices to see if its safe in humans. Publisher based research is often Testing Research that is at a certain level. Like access to food or exercise. And then the Healthcare Delivery system, what is the best way to deliver care. Even for the right on this picture i would put dissemination and implementation research. What is the best way to actually get evidence into the hands of the people who need to implement the evidence. What is the best way to get the information to doctors or to payers . I was put comparative Effective Research to the right of the transitional pathway. The population base, the clinical comparative research, the Health Services research, and certainly the dissemination and implementation research. I misys Research Important . Many important healthcare decisions have little scientific evidence. Sorry to say that it is improving for the past 30 years that we still make lots of clinical decisions based on Expert Opinion or tradition, or i just think this is what is best for my patient. Certainly the quality and value of the care we deliver is uncertain. There is variation in practice, regionally across the country. You have to assume that some of the care delivered is not benefiting patients. There are tremendous economic implications of rising healthcare costs, new and very expensive treatments and diagnostic tests. They very rapidly going to practice, perhaps before theres evidence to suggest its usual for the whole population. Their slow translation into evidencebased practices. Some things we know what to do but its hard to get that into practice. What should be study . The institute of medicine in 2009 was tasked with considering priorities for comparative Effective Research. The iom panel solicited input and prioritized 100 research questions. Here is an example of a few. One is compare the effectiveness of Management Strategy into earlystage breast cancer. Establish a registry to compare the effectiveness of treatment strategies for low back pain. You can imagine these answer questions that are very important to clinicians and patients. Among the hundred top priorities, half were about Healthcare Delivery system which was a little surprising to many people. But emphasizing that understanding the comparative Effective Research. Who funds this type of research . Many different funders have some part in comparative Effective Researched to varying degrees. I want to highlight to that do the bulk of funding and that would be the agency for Healthcare Research and quality since 1999. Its a federal agency with the sole purpose of producing evidence to make healthcare safer, higher quality, more accessible, equitable and affordable. There also committed to training the next generation of researchers including comparative Effectiveness Researchers. Patient centered outcomes Research Institute since 2010 Funds Research that will help patients choose Treatment Options that best meet their needs. For this research that advances the quality and relevance of evidence of how disease can be effectively diagnosed, treated, and monitored. This is not to say that others dont also fund this research. These agencies have these as the primary mission. Let me highlight some comparative Effectiveness Research. You can get the research and see its impact. This is a fairly wellknown example from doctor peter, my colleague at hopkins. So they invested in the comprehensive unit based Safety Initiatives in 2003. The dr. Asked, is there a better way to prevent central line infections than what we are currently doing . Central line to catheter in the to give fluids, food and medicine. This program in the first 18 months they do 1500 lives in nearly 200 million just in michigan. The intervention was a very systematized checklist of processes that should have happened before central line is placed and while it is place. A very lowtech intervention that was highly impactful. Now, more than 1100 hospitals in 1800 teams have participated in initiatives like this. They have now spread out to do other things besides prevent central line infections. The evidencebased Practice Centers funded by hr q have produced more than 500 comprehensive systematic literature reviews. These literature reviews are used as the evidence to support the u. S. Preventative Services Task force recommendations. To support professional cited guidelines like American College of physicians, used to inform the nih consensus conference and informed cms decisions. This is about treating symptoms of diabetic neuropathy. Decisions for cancer treatment. Treatment of muscle invasive bladder cancer. As i said, these comprehensive reviews are used as guidelines. This came out just this month. These were guidelines about noninvasive treatments for low back pain. These are the u. S. Preventative Services Task force for screening for colon cancer. This is about treatment and management of gout. Again, these are all based on the systematic reviews ended by hr q. Im a pause for a moment to talk about the key methodologies that we use. We talked about evidence synthesis. That is taking the evidence that exists in the literature and comprehensively synthesizing it so it is useful. Certainly we also do evidence generation. So making new evidence. Sometimes this uses experimental methods like trial. Sometimes it uses observational methods like data from the Patients Health record or data from the administrative claims, billing data from patients and applying very rigorous and sometimes complex message to glean evidence from this data. Stakeholder engagement methods are part of cr as the dissemination and implementation methods. Other examples of important results for medical practice includes a study for the virginia surgical improvement program. So, these researchers asked, his bariatric surgery, weight loss surgery more effective at preventing deaths than usual care which is no surgery in morbidly obese veterans . So, they identified 2500 veterans, mostly men who had bariatric surgery and compared them to similar patients have not had the surgery. As these lines the show, that they diverge. The surgical patients had a much lower rate of mortality over the 12 years of followup than the non surgically treated patients. Rigorously done informs suggesting perhaps bariatric surgery is appropriate for these patients. Another is funded by its about inter venous versus oral antibiotics for children with bone infections after hospital discharge. So they asked are all into biotics as good as intravenous antibiotics . Yes, children treated with antibiotics did not have more treatment failures and it was much more satisfying to the patients, the parents and had fewer trips to the emergency room for comp locations of the catheters. That was the trial. That was a pragmatic trial. It was conducted in the usual care settings of these patients. The Patient Centered outcomes Research Institute has made major advances in establishing networks for the conduct of pragmatic clinical trials. These are different than the trials conducted by perhaps tried companies that are tightly controlled and the patients all look alike and everybody gets tight followup. These are trials conducted in the patients usual care setting and cared for by their usual doctors or surgeons. So we have set up a cornet with Clinical DataResearch Networks which are networks of Health Systems and patient powered networks which are driven by patients. For example, john hopkins is part of the past Clinical Network which is other Health Symptoms in pennsylvania and utah. So the first trial that theyre involved in is answering this question, what is the best dose of aspirin to prevent heart attacks and strokes. Its hard to believe this question has not been answered. Aspirin has been used since the 1990s for stroke prevention. Should patients get 81 milligrams or 325 milligrams of aspirin . This is a big trial embedded in the usual healthcare system. Should enroll 20000 patients across the six sites. What outcomes are important when we conduct cer . This is Patient Centered research. Therefore the outcomes that we look at should be important to patients. Clinical trials often dont measure outcomes that are important to patients. They may be important to the fda if they are approving a product and saying that its safe, but theyre not always that relevant to patients. So Patient Centered Outcome Measures are measures that assess the impact of the disease and treatment of patients for example, pain. A very patient relegated outcome. Patients do not want pain. Depression. Theres lots of different measures and scales that are validated to measure outcomes that are important to patients. Like the hamilton rating scale for depression. Others might be survival or even time to work so that researchers can access these tools. May find the evidence, i might go to the clearinghouse which is a store the prevention of cardiovascular disease. In fact that guideline was based on a review funded by hr q and 2015. In fact, that review became the u. S. Preventative Services Task force background for the recommendations. So for my man patient, i would say there is be level evidence, yes, i think you will benefit for the use of aspirin to prevent heart attack and stroke. For my older woman patient i would say the evidence is unclear. Not strong evidence, im not sure which way to go. I want to finish by saying this is not exactly new. Cer and cr Type Research has been described in literature since the 1950s comparing different medicines. In fact, pragmatic trials were first described in the late 1960s by french epidemiologist and did not gain focus until the past decade. The virginia has been doing Health ServicesResearch Since the 1970s. The growing tension in the 1980s with appreciation for what is evidence in the rising healthcare cost, the establishment of arc in 1999 and more recently nearly has focused the funding on cer and has done a lot to advance the methodologies we use to do the research. I do think it is now recognized as an essential late part of the translational pathway to improve patient outcomes, hopefully in a sustainable healthcare system. Thank you for your attention. [applause] thank you for that excellent presentation in providing some level setting for the audience. I would like to invite our moderator and panelists to the stage. Our moderator for this morning is gail lewinsky, many of you know gail who is a senior fellow at project had been former administrator of the Health Care Financing administration. Gail is also serving as one of four cochairs along with former Senate Majority leaders tom daschle and bill fritsche. And also andy on a new Bipartisan Group of Health Policy experts to identify a path forward on health care reform. Gil, thank you for yo