For decades, clinical trials – critical links in bringing new discoveries into clinical practice – have revolutionized our understanding of how best to treat patients. The Institute for Transformative Clinical Trials is dedicated to improving health outcomes of patient populations by conducting novel trials that generate evidence about the effectiveness and safety of therapeutic interventions. By focusing on trials that address important clinical and public health questions, trial findings should change clinical practice as well as generate new scientific knowledge.
The Institute, which grew out of the International Center for Health Outcomes and Innovation Research (InCHOIR), offers the interdisciplinary expertise to design, conduct and analyze innovative clinical trials that span the translational spectrum from early phase to pragmatic, comparative effectiveness trials. The Institute offers these capabilities to translational and clinical researchers within and outside the Icahn School of Medicine at Mount Sinai. Building upon our long-standing expertise in serving as a Coordinating Center for Multi-center Clinical Trials and Clinical Trial Research Networks, we welcome partnering with other researchers to serve as the DCC or CCC for other new federally-supported clinical research team efforts. Moreover, the Institute is enthusiastic about continuing to collaborate with the private sector to help design and conduct early phase, confirmatory and pragmatic trials of drugs, biologicals and devices.
Medical research is generating new science at an unprecedented rate, which is reflected in a steady stream of novel drugs, biologicals, devices, and procedures that have the potential to improve patient lives and address unmet health needs. However, translating these innovations into practice requires a robust clinical trials enterprise, and there is growing recognition that the nation’s infrastructure is inadequate to meet the needs of 21st century medicine.
Nearly half of recent Phase III trials have failed to demonstrate therapeutic effectiveness because of flaws in the design of earlier phase human trials. The vast majority of trials have significant difficulty in recruiting patients. It is disconcerting that only about 10% of all adult patients participate in clinical trials in this country, and this recruitment percentage is even lower among the elderly, women and minorities. As a consequence, the results of many trials have limited applicability to the care of important segments of our population, contributing greatly to the variability of healthcare quality, and is often the case when rigorous evidence is absent.
Another compelling reason for revamping the clinical trial enterprise is the inadequacy of the evidence for making every-day clinical decisions involving commonly used diagnostic and therapeutic modalities. Pragmatic trials, which differ in design from the confirmatory trials used for FDA registration, compare existing alternative treatments (rather than experimental treatments to placebo), use a broader range of clinical outcomes than in early phase or confirmatory trials, and take place within in the setting of real-world conditions. As such, their results are directly relevant to the complex clinical decisions faced by physicians and patients alike, and help guide improved effectiveness and efficiency of care delivery.
To address these crucial needs, Mount Sinai has created the Institute for Transformative Clinical Trials, which has as its mission the design, conduct and analysis of innovative clinical trials that span the spectrum from early phase to confirmatory to large-scale pragmatic trials.
Our primary measure of success is that our trials generate insights that change treatment patterns, enhance patient-centered clinical decision making, reduce disparities in health care, and shape health care policies. In conducting these trials, we hope to achieve the following:
- Accelerate the introduction of new diagnostic and therapeutic interventions into practice
- Improve the evidence base for current practices
- Develop new analytical approaches and methodologies for clinical trials
- Expand the use of novel patient-centered outcomes
- Employ novel data collection approaches using apps and wearable devices
- Increase the diversity of patients participating in clinical trials
- Streamline data collection through the use of registry and electronic health record data
- Train and educate future generations of clinical researchers