Overview of Clinical & Translational Research @ Penn townsend@upenn.edu September 6 2018 8:00-8:30

What do I need to do human research? Training Mentor Vocabulary Structure/Space Ideas Subjects Datasets Regulatory Resource Locator Protected Time Statistical Support Funding Knowledgelink Collaborators/Community Coaching/Writing Passion Persistence

Mentor Choose well – Knowledgeable Has Time for U Around OK to have a Team

What is Clinical Research? Clinical Research is Research with human subjects that is: Patient-oriented research. Research conducted with human subjects (or on material of human origin such as tissues, specimens, and cognitive phenomena) for which an investigator (or colleague) directly interacts with human subjects. Excluded from this definition are in vitro studies that utilize human tissues that cannot be linked to a living individual. It includes: mechanisms of human disease therapeutic interventions clinical trials development of new technologies Epidemiological and behavioral studies. Outcomes research and health services research. Studies falling under 45 CFR part 46.101(b) (4) (Exemption 4) are not considered clinical research by this definition.

What is translational research? 5 What is translational research? “Translational research means different things to different people, but it seems important to almost everyone” Stephen Woolf, JAMA, 2008 Commonly used terms: clinical research, patient-oriented research, translational research NIH roadmap: Zerhouni, Science 2003 “Bench to bedside”

6 “the transfer of new understandings of disease mechanisms gained in the laboratory into the development of new methods for diagnosis, therapy, and prevention and their first testing in humans.” T1 translation “the translation of results from clinical studies into everyday clinical practice and health decision making.” T2 translation: Sung et al., JAMA 2003: Institute of Medicine Clinical Research Roundtable report

T3 and T4? T3: Dissemination and implementation research 7 T3 and T4? T3: Dissemination and implementation research Community-based participatory research Can occur in “practice-based research networks” Westfall et al., JAMA 2007 Key questions: Is a treatment currently being used? What are gaps in care or barriers to care? Accuracy or efficacy when implemented in real- world settings? T4: Public policy research

Structure 1 Dulles PCAM 4 South PPMC Mutch Building 1st Floor

Subjects & Data & Datasets

Resource Locator www.itmat.upenn.edu

EPIC CALL SERVICE

Biostatistics Andrew Cucchiara, PhD (10:30 – 10:50; STAY TUNED!)

$$$ Funding $$$

Intramural Funding

Extramural Funding

https://www.citiprogram.org/Default.asp?

Collaborations – Network - Resources ITMAT Bioinformatics Core Clinical Research Computing Unit (CRCU) Biostatistics Core Biochemistry Core Lab Center for Clinical Epidemiology and Biostatistics (CCEB) Research Nurse Core Kinetics Modeling and Simulation (KMAS) Bionutritional Research Core Clinical Translational Research Center (CTRC) Biomedical Lab Post-Doctoral Fellow Investigational Drug Service Biostatistics, Bioinformatics, Pharmacometrics Animal Facilities ITMAT Clinical Trials Unit Human Subject Protection Animal Testing Research Plan Basic Research Physician-Scientist Clinical Research Research Plan Biomedical Mass Spectrometry Industry Administration / Office of Human Research (OHR) Institutional Animal Care and Use Committee (IACUC) Regulatory Science LC-MS/MS Core in mice Institutional Review Board (IRB) Development of unbiased markers in humans CTRC Advisory Council Office of Research Services (ORS) Sample Analysis Food and Drug Administration (FDA) External Scientific Adviser and Industry

Coaching/Writing

Passion

Persistence

Foundations of good research Good idea Important topic $$ Capable Coordinators Recruitment

Why do we need clinical research? New Drugs Devices Procedures Old Drugs with new uses …

Why No matter how well you alter (or correct) the pathophysiology in an animal disease model, you wont know whether it will help people until you do it in people Without clinical and translational research our observations in the basic sciences, in observational cohort studies, and in our clinical practice(s) will simply consume our time but serve no ultimate benefit

Recap WHAT IF MY HOW LONG IS THIS STUDY IS GOING TO TAKE? NEGATIVE? WE’VE GOT THIS IDEA… ETHICS REGULATORY TRAINING IS IT WORTH IT? FINDING SUBJECTS FUNDING STORING THE INFORMATION PUBLISHING STATISTICS PRESENTING LIFESTYLE WHO IS GOING TO DO THE WORK?????

Where to find help www.itmat.upenn.edu http://www.itmat.upenn.edu/ctrc/index.shtml http://www.med.upenn.edu/clinrestraining/

Overview of Clinical & Translational Research @ Penn townsend@upenn.edu September 6 2018 8:00-8:30