[MUSIC] Welcome back to Introduction to Translational Science. This is segment three, Translation to Patients. In this segment, you're going to hear about the amazing work of Dr. Raymond Dorsey and Dr. Marvin Doyley, both translational scientists at the University of Rochester Medical Center. Dr. Doyley studies imaging and new ways to look at the human body. Dr. Dorsey studies new ways to perform translational research in patients with neurologic diseases and how to use technology. Really cool, Dr. Dorsey on my phone, an app that he invented. In this segment, we're going to talk about T2 Research, Translation to Patients. At this level of research, investigators test new interventions under controlled environments to figure out is this care effective. Findings from T2 studies yield knowledge about the efficacy of the interventions, and form the basis of clinical application. Taking these treatments in to the clinic outside of controlled studies. These types of studies involve asking the question, does this treatment really benefit patients? We're no longer about discovery, we're at the phase of trying to figure out implementation. T2 study designs include phase III clinical trials, observational studies, evidence synthesis-based studies, and development of guidelines. Let's dive a little deeper into this process of translation. In the last segment, we spoke about basic research and translation into humans, the T0, T1 transition. In this segment we're going to talk about translation from T1 now into T2, Translation to Patients. Phase III Clinical Trials are an integral part of translation to patients. They start with a new treatment that's already passed the phase I and phase II clinical trials level, and they move into testing in a larger group of individuals. The goal of these trials is to really dig in and find out if the new treatment is better than a standard treatment. Or if a standard treatment does not exist, if the new treatment really confers an advantage, does it improve the health of the person receiving that treatment. These trials are bigger, they usually include a large number of patients, 300 or more, sometimes a few thousand patients, and they take a number of years to finish. Not every discovery makes it to a phase III clinical trial, and thus the discoveries that do make it to these trials show even greater promise for efficacy and for providing new therapies, diagnostic methods, and treatments. The next type of study is observational. Observational studies focus on the causes, preventions, and treatments for a disease. They also may differ from experimental design studies because the investigator only observes the subjects, they don't really intervene, except to gather data. So subjects in observational studies are not receiving active therapies. Observational studies can be used to develop diagnostic methods, to study the progression of certain medical conditions or diseases. To look retrospectively and observe subjects and occurrences that have already happened in the past, and use that data in records and self-reports to make new observations into advanced knowledge. Observational studies designs can look at a group of people, a cohort. They can be case-controlled where they look at individuals across a time spectrum. They can look at a cross-section of an entire group of people at specific time points. And they can look at an entire population, an ecology, and look at how those individuals are interacting with their environment. And examine the relationship between, for example, exposure to a toxic environment and an outcome by looking at how the population responds at the level of data rather than the level of an individual. The next type of study is called an Evidence Synthesis study. These studies look at the entire magnitude of evidence, perhaps across a variety of smaller studies. And they use advanced statistical techniques to synthesize that knowledge and to ask okay, now that we've done a dozen different studies on this therapy, what do all those studies combined show? The collection of studies is often as effective or more effective than a much, much larger controlled studies that might only apply to a small number of people. A type of these studies is called a meta-analysis. And this study design uses statistical methods to combine study findings across a number of different studies. And really, the ultimate goal of a meta-analysis is to look at how strong is the effect of a therapy, or how accurate is a diagnosis. A different type of study is a synthesis of evidence from systemic reviews. This type of study design looks at a set of clearly specified, and rigorous, and reproducible methods, and looks to analyze the results from all the relevant studies on a topic. The goal of a systemic review is to identify what is known and not known about a selected topic. It's to look at the gaps in our knowledge and to figure out where to go next. Finally, a clinical guidelines development study looks to develop guidelines that translate best evidence into best practice. Of course, when we go to our health care providers, we want them to be acting on the latest guidelines and provide us with the best care. Those guidelines have to be developed somewhere. Good clinical guidelines promote quality, they reduce variations in how health care is delivered. They improve diagnostic accuracy and they discourage the use of ineffective, or even interventions that we have found to be potentially harmful. The steps in developing these guidelines include identifying and refining the subject area, getting together a group of experts and other practitioners to wait the evidence. And then to develop and translate that into recommendations that can be adopted as a clinical practice guideline for all practitioners to use. External review of the guideline is usually the last step. So in the next segment, you're going to hear first from Dr. Ray Dorsey from the University of Rochester Center for Human and Experimental Therapeutics. And I'm not going to steal Dr. Dorsey's really cool app and research details, I'll let you hear from him. He's changing the way scientific his research is done. It used to be that you had to go to the research center, to be a research subject. Dr. Dorsey's goal is that the research center comes to you. And then you're going to hear from Dr. Marvin Doyley, who's associate professor of Electrical and Computer Engineering and Biomedical Engineering at the University of Rochester. Dr. Doyley is helping us imagine new ways of looking at the human body and imaging to allow us to see things that we never were able to see before. I hope you enjoy this vision of our research future and translation. [MUSIC]
