2012 Jointly Presented KCALSI/Frontiers Research Symposium takes on the bench-to-bedside continuum
When it comes to translational research, the numbers can be overwhelming. It currently takes a new discovery an average of 13 years to make it to market. Ninety-five percent of discoveries never get that far and those that do carry an extraordinary cost – averaging more than $1 billion.
Although the numbers are sobering, they are also a source of inspiration. KCALSI’s May 31 Research Symposium, co-hosted with Frontiers – the Heartland Institute for Clinical and Translational Research, brought together some of the country’s top experts in translational medicine to explore how we can work together to shorten timelines, lower costs and improve the quality of patient care in the United States and around the world.
Opportunities to improve translational medicine exist at every stage of the lengthy journey that new therapies take from bench to bedside. Some of these opportunities are: doing more with data that has already been generated; improving the way we conduct clinical trials; increasing collaboration across departments and institutions; accelerating the adoption of innovative clinical advancements and thinking outside of the box. This year’s research symposium explored a number of exciting opportunities for improvement across the spectrum, reminding attendees that together we can improve translational research. And more than that, together we must.
300 Billion Points of Data, Unlimited Opportunities
If you ask Atul Butte, MD, PhD, Division Chief and Associate Professor of Systems Medicine in the Department of Pediatrics at Stanford University School of Medicine, many of the answers sought by modern medicine already exist. The key to unlocking them is learning to ask the right questions.
Dr. Butte is an advocate for the power of translational bioinformatics: applying tools to convert more than 300 billion points of molecular, clinical and epidemiological data – measured by researchers and clinicians over the past decade – into diagnostics, therapeutics and new insights into disease.
The availability of publications containing massive amounts of cross-disciplinary data has contributed to Dr. Butte’s success. He estimates that we are just a few weeks away from having one million publicly available RNA microarrays. DNA data is nearly as accessible – thanks to the National Institutes of Health’s database of Genotypes and Phenotypes (dbGaP). The availability of this data creates enormous opportunities.
“We used to generate a hypothesis and generate data to answer that question. Today, there is so much data available, the new magic is in figuring out what questions we want to ask,” said Dr. Butte.
Does the availability of this data mean that the interesting work in science is complete? Absolutely not, says Dr. Butte. But it does mean that in some cases, the critical data has already been generated.
“It’s just sitting there, waiting for you and your questions.”
Rethinking Clinical Trials
No drug can come to market without first being tested in clinical trials. Unfortunately, that is an increasingly expensive undertaking. According to Pfizer, the cost of conducting trials is now 60 percent of total drug development costs – that is a 30 percent increase since the 1980’s. The increasing cost of trials poses major challenges to NIH’s mission and the viability of the clinical research enterprise in the U.S. Moreover, traditional approaches to clinical trials are not providing the evidence base to inform the diagnostic and treatment decisions that clinicians must make in routine practice situations.
According to Gary E. Rosenthal, MD, Professor of Internal Medicine and Health Management and Policy and Director of the Institute for Clinical and Translational Science at the University of Iowa, the growing cost of clinical trials makes the U.S. less competitive worldwide. Dr. Rosenthal is leading the effort to rethink how we conduct clinical trials in the U.S. He is driven by a simple idea.
“How do we develop the evidence that is needed to make sure that the new drugs and devices actually benefit populations?”
He advocates for a pragmatic approach to clinical trials based on a handful of fundamental tenets: designing studies to more closely mimic routine clinical practice; abandoning the use of placebos; and focusing on effectiveness instead of efficacy.
This approach may require patients to remain involved in the trial long-term in order to track the real life outcomes of the study on patients. For this reason, the approach may be better suited for studying chronic conditions that receive treatment over many years. In the end, the results are more accurate and generated at a lower cost – a critical improvement in the way we conduct clinical trials.
Institutionalizing Collaborative Innovation
When you bring great minds together, great things happen. As intuitive as that may seem, the traditional structure of academic research environments often make it difficult for great minds to cross departmental and disciplinary boundaries to work together.
That is no longer the case at the University of Chicago where Julian Solway, MD serves as the Walter L. Palmer Distinguished Service Professor in Medicine and Pediatrics; Associate Dean for Translational Medicine, Biological Sciences Division; Chair for Research in the Department of Medicine; and Chair of the Committee on Molecular Medicine.
Dr. Solway and colleagues are working together to use Clinical & Translational Science Awards (CTSAs) synergistically to develop clinical and translational scientists and to catalyze the formation of multidisciplinary research teams. The effort has made it possible to recruit investigators from a broad range of departments, and across the career spectrum, to collaborate together in ways that were not possible before.
The initiative focuses on infrastructure improvements (like a new Biomedical Informatics program) and on cultivating a culture of collaboration, but in the end, it is the connections between great minds that makes the difference.
“Putting people together across the translational research spectrum is very effective,” said Dr. Solway.
Inspiring Physicians to Adopt Data-Driven Practices
There is no shortage of information available to physicians – in fact, staying up-to-date on new research and guidelines is a major part of the job. But being aware of new data and implementing that data consistently are sometimes two different things.
John A. Spertus, MD, MPH, FACC, Director of Health Outcomes Research at Saint Luke’s Mid America Heart Institute and Professor and Daniel J. Lauer Missouri Endowed Chair in Metabolism and Vascular Disease Research at the University of Missouri-Kansas City is working to turn that around.
Dr. Spertus is one of the developers of ePrism, a novel, web-based technology that executes multivariable risk-prediction models with individual patient data to support the use of individualized, evidence-based risk estimates, which will support a better process of care, improved outcomes and lower costs.
The ePrism system is part of a patient care strategy that begins with the implementation of comparative effectiveness research, which informs appropriate use criteria, allowing physicians to apply risk modeling, and build predictive models that improve routine care.
The model enables personalized medicine to be delivered in routine clinical care to support a safer process of care, improved patient outcomes, reduced cost and better patient experiences.
“We can work together to create the toolkit that can really transform American medicine,” said Dr. Spertus.
Thinking Outside of the Dish
Organ and tissue printing may sound like science fiction, but the technology is taking huge leaps at the University of Missouri under the leadership of Gabor Forgacs, PhD, George H. Vineyard Professor of Biological Physics at the University of Missouri; Executive Director at the Shipley Center for Innovation at Clarkson University; and Scientific Founder of Organovo, Inc. and Modern Meadow, Inc.
Organ and tissue printing makes it possible to produce human or animal tissue with the help of a computer and a 3-D jet printer.
In addition to fascinating opportunities, such as producing meat and leather (initiatives of Dr. Forgacs’ Modern Meadow, Inc.), tissue printing holds potential for translational research.
“What if you could engineer a little piece of liver from your own cells to test a treatment for yourself?” asked Dr. Forgacs.
The answer: a new path to the bedside and more personalized treatments for patients everywhere.
Reinventing Translational Medicine
The Research Symposium culminated with a panel discussion that brought the themes of the day together around a single topic: “The Reinvention of Translational Medicine.”
The perspectives and experiences of the day’s individual speakers were complemented by a panel of nationally-renowned experts on the topic of translational medicine. Moderated by Lesa Mitchell, Vice President at the Ewing Marion Kauffman Foundation, the panel featured:
Joseph M. Smith, MD, PhD, FACC, Chief Medical Officer and Chief Science Officer at West Wireless Health Institute
Anna Barker, PhD, Director of the Transformative Care Network and Co-Director of the Complex Adaptive Systems Initiative at the Arizona State University
Scott J. Weir, PharmD, PhD, Director of the Institute for Advancing Medical Innovation at the University of Kansas Medical Center
Although panelists represented distinct perspectives on translational medicine, they shared a fundamental belief that to improve translational medicine, we must reduce dependency on the National Institutes of Health to fund and direct research. Instead, we must empower citizens to take a more active role in solving today’s healthcare challenges, in part, by facilitating greater access to personal medical data.
For one panelist, these issues represent a critical shift in the U.S. healthcare system.
“This is not so much about solving an incremental problem in healthcare, but around solving a larger issue in the delivery of healthcare,” said Dr. Smith. “There is a desperate need for innovation, but it isn’t just around the gadget. It is around a care delivery model that is affordable.”
Dr. Barker picked up on themes of collaboration and the availability of data from earlier in the day.
“I believe that knowledge is built by teams. Knowledge is additive from individuals, but teams of people actually create knowledge,” she said. “We are at an inflection point in medicine, where the data needs to be available for everyone.”
Dr. Weir has seen the impact of this collaborative, multidisciplinary approach to addressing leukemia lymphoma-related issues at the Institute for Advancing Medical Innovation.
“We’re able to accelerate projects by using innovative approaches and we’re advancing projects through at a much lower cost,” he said.
And for Dr. Weir, there is a simple recipe for success.
“It’s about finding the right partners and committing to putting the partnership above the interest of the individuals.”
In this approach, we begin to see the potential to reinvent translational research and to fulfill the Symposium’s theme, “Better, Smarter, Faster: The Power of Collaboration in Translational Research.”