GMS 6847 – Translational Research & Therapeutics

Fall 2023

Course Title: GMS 6847 (Section 28C7) Translational Research and Therapeutics: Bench, Bedside, Community, & Policy

Tuesdays, 12:50-2:45 pm & Thursdays, 12:50-1:40 pm, Academic Research Building (ARB) R5-265

Fall 2024 schedule coming soon!

Course Directors:  Gemma Casadesus, PhD, and Wayne T. McCormack, PhD

Section Leaders:

Intro & TBL:  Wayne T. McCormack, PhD & Gemma Casadeus, PhD

T0 Preclinical Research:  Jeffrey Harrison, PhD

T1 Clinical Research:  Brian Law, PhD

T2 Clinical Research:  Tom Rowe, PhD

T3 Implementation Science:  Elizabeth Shenkman, PhD

T4 Public/Population Health:  Kelly K. Gurka, MPH, PhD

Course Administrator:  Michele Duval-Martin

Office hours: By appointment

Course Objectives

This course is for basic science students to gain an insight into clinical research and clinical application, for clinical science students to gain insight into the basic science required to reach clinical trials, and for all students to gain insight into the importance of industry, regulation, marketing, and acceptance into medical practice, which constitutes the endpoint of translational research.  The course meets during the Fall semester for three hours per week, and includes guest lecturers with varied expertise in the principles of translational research.  The course will emphasize understanding and applying the experimental approaches of the multiple phases of translational research to a significant health problem.  It will utilize multimodal teaching technique combining didactic lectures, reading assignments, and multidisciplinary team­based learning (TBL).

This course will introduce and explore in-depth the multi-T-phase concept of translational research.  It will emphasize moving knowledge and discovery gained from basic science research at the bench to its application in clinical and community settings, and finally into national health policy.  This course will explore the breadth of preclinical research in the identification of opportunities and approaches to health problems as it relates to small molecules, biologics, diagnostics, and devices.  It will elucidate the important aspects of clinical research as it relates to Phase I-III clinical trials that take observational studies to evidenced-based guidelines for treatments.  Finally, the course will explore the implementation and public health phases of translational research, which include dissemination and implementation of research from clinical trials to health practices, evaluation of their impact, and the potential integration into population health policy.

General Course Learning Objectives:

  1. Describe the process of identifying potential connections between scientific discoveries and human conditions (aging, injury, fatigue, pain, etc.) or diseases (diabetes, cancer, hypertension, infection, etc.).
  2. Describe the process of identifying an unmet medical need (e., review knowledge of human conditions/diseases and their treatment and vice versa; this should include a discussion of benefit/risk/limitations of current treatments).
  3. Identify how scientific knowledge or discoveries might be used to improve upon existing therapies (compare benefit and risk of current versus proposed therapies).
  4. Describe the major steps in the process where basic scientific discoveries are applied towards solving medical problems in patients and society.
  5. Discuss the different types of knowledge or expertise that are required to move between translational phases (e., formal sciences vs. natural sciences vs. social sciences; a priori knowledge vs. post priori knowledge) and the importance of team science.

Specific Course Learning Objectives by Section

T0 – Preclinical Research:  Identification of opportunities and approaches to health problems:  Envisioning a Path Between Bench and Bedside

  1. Describe how small molecules, biologics, devices, and diagnostics can be used to treat human conditions or diseases.
  2. Discuss how different model systems are used to approximate molecular, cellular, or physiologic aspects of the human environment (e., in vitro biochemical or cellular models, animal models, etc.) and the advantages/limitations of each approach.
  3. Discuss what type of benchmark findings need to be obtained before preclinical studies can be moved into human trials (e., standards underlying preclinical evaluation, regulatory requirements).
  4. Define therapeutic modalities and describe how they can be used.
  5. Discuss other real-world considerations that influence the impact of a healthcare innovation including cost, insurance coverage, its accessibility to patients, and market size.

T1 – Clinical Research (Phase 0-I Clinical Trials):  Discovery of Candidate Health Applications – The Journey from Laboratory to the Clinic

  1. Explain how intellectual property is protected and why this is important.
  2. Recognize governmental and institutional requirements which must be met to initiate clinical studies.
  3. Discuss the purpose and design of Phase 0 and I clinical trials (what type of information is obtained from these studies and how is this used to guide future translational studies).
  4. Identify and surmount roadblocks frequently encountered in Phase I trials and how these might be avoided by the implementation of Phase 0 trials.

T2 – Clinical Research (Phase II-III Clinical Trials):  Health Application to Evidence­Based Practice Guidelines – From the Bedside Back to the Bench

  1. Discuss the purpose and categories of clinical trials.
  2. Distinguish between Phase I, II, and III clinical trials.
  3. Know and discuss the important elements that make up a clinical trial study protocol.
  4. Discuss the major types of clinical trial designs.
  5. Describe important regulatory steps associated with Phase II and III clinical trials.

T3 – Implementation Science:  Practice Guidelines to Health Practices

  1. Describe the disseminating of new discoveries to communities and populations.
  2. Identify examples of evidence-­based clinical best practices that when implemented result in known health disparities.
  3. Describe the steps to implement a new health benefit (e., device, drug) and track population-level access.
  4. Discuss issues related to quality of life measurement and patient reported outcomes.

T4 – Public/Population Health:  Practice to Population – Health Impact

  1. Discuss the factors and interventions that influence the health outcomes of populations.
  2. Describe how to measure the health outcomes of populations.
  3. Describe the social determinants of population health.
  4. Identify the steps to evaluate public health program and interventions.

Course Schedule (August 24th – December 7th)

Translational Research & Therapeutics: Bench, Bedside, Community, & Policy
2-hour sessions (12:50-2:45 pm) 1-hour sessions (12:50-1:40 pm)
Thu, Aug 24 Drs. Casadeus & McCormack

Intro to Translational Research Phases; Unmet medical need (BRCA)
Homework: Unmet medical need worksheet

Intro Tue, Aug 29 Drs. McCormack & Casadeus

Intro to Team Science; Team formation

Thu, Aug 31

Dr. Elias Sayour – Biologics Case Study

T0
Pre-clinical
Tue, Sep 5

Dr. Jeff Harrison – Intro to Preclinical Res I & II

Thu, Sep 7

Dr. Lee Sweeney – Small Molecule Case Study

Tue, Sep 12

T0 TBL Session

Thu, Sep 14

Team meeting: apply T0 to unmet medical need

Tue, Sep 19

Brief T0 Team Reports*

Thu, Sep 21

Dr. Brian Law – Intro to Clin Res (Phase 0-I)

T1
Clinical(phase 0-I)
Tue, Sep 26 (1:00-3:00 pm, 3256 CTRB)

Short Talks:  HealthStreet, CTSI Resources

Tour CTSI Clinical Research Facilities

Thu, Sep 28

Dr. Abhisheak Sharma – Metabolism in Clinical Trials

Tue, Oct 3

i2b2 OneFlorida Integrated Data Repository Workshop

Thu, Oct 5

Dr. Coy Heldermon – First-in-Human Trials

Tue, Oct 10

T1 TBL Session

Thu, Oct 12

Brief T1 Team Reports*

T2
Clinical (phase II-III)
Tue, Oct 17

Dr. Tom Rowe – Intro to Clin Research (Phase 2-3)

Dr. Jennifer Mulligan – Phase 2 & 3 Clinical Trials

Thu, Oct 19

Dr. Manuela Corti –  Therapy Trials in Gene Therapy

Tue, Oct 24

T2 TBL Session

Thu, Oct 26

Brief T2 Team Reports*

T3
Imple-mentation Science
Tue, Oct 31

Dr. Thomas George – Phase IV Clinical Trials

Dr. Elizabeth Shenkman – Intro to T3 Translational Research, Dissemination and Implementation Science

Thu, Nov 2

Dr. Elizabeth Shenkman – Comparative Effectiveness Research and Pragmatic Clinical Trials

Tue, Nov 7

T3 TBL Session

Thu, Nov 9

Brief T3 Team Reports*

T4
Public/ Population Health
Tue, Nov 14

Dr. Kelly Gurka – Intro to T4 Translational Research; Practice to Population; Evaluation of Public Health Programs & Health Policy

Thu, Nov 16

Dr. Sarah McKune – Global Health

Tue, Nov 21

Dr. Tom Pearson – Precision Public Health

Thu, Nov 23 – Thanksgiving Holiday – No Classes
Tue, Nov 28

T4 TBL Session

Thu, Nov 30

Team Meetings, Course Evaluations

Tue, Dec 5

Final Team Reports*

   *Submit PPT file to Michele Duval-Martin (mduvalmartin@ufl.edu) by 10:00 am

Methods of Evaluation and Grading

Students will be evaluated based on their performance on TBL readiness assurance tests (50% of grade) and on team presentations (50% of grade).

Exams (50% of grade):  There will be five team-based learning sessions, each of which will include an individual readiness assurance test (IRAT) and team readiness assurance test (TRAT), consisting of the same questions.  These tests will consist of multiple choice type questions based on content in assigned reading materials and immediately preceding lectures.  The relative weight of the IRAT and TRAT will be determined by student voting.  Team appeals for the TRAT must be sent by e-mail to the section leader within 24 hours (i.e., by 3:00 pm the following day).

Team Activities and Presentations (50% of grade):  Student participation in the team activities and presentations will be assessed by the course faculty as outlined below.

Four Brief Team Reports          5% each                 20%

Final Team Report                                                     25%

Participation/Attendance                                          5%

The following grading scale will be used for this course:

A    93­100%                      C         74-­76%

A-  90­92%                        C-        70-­73%

B+ 87-­89%                      D+       67­69%

B    84­86%                       D         64­66%

B-  80­83%                        D-        60­63%

C+ 77-79%                        F          < 60%

At the end of the semester the final letter grade cut-offs may be adjusted lower, but will not be adjusted higher.

Attendance: Requirements for class attendance and make-up exams, assignments and other work in this course are consistent with university policies that can be found at:  https://catalog.ufl.edu/ugrad/current/regulations/info/attendance.aspx

 Accommodations for students with disabilities:  Students requesting classroom accommodation must first register with the Dean of Students Office. The Dean of Students Office will provide documentation to the student who must then provide this documentation to the Instructor when requesting accommodation.

Required and recommended textbooks:  Lecture materials and reading assignments will be provided in PDF format in Canvas. There is no required textbook.

Information on current UF grading policies:  Please consult the following website: https://catalog.ufl.edu/ugrad/current/regulations/info/grades.aspx

Course evaluation process:  Students are expected to provide professional and respectful feedback on the quality of instruction in this course by completing course evaluations online via GatorEvals. Guidance on how to give feedback in a professional and respectful manner is available at https://gatorevals.aa.ufl.edu/students/. Students will be notified when the evaluation period opens, and can complete evaluations through the email they receive from GatorEvals, in their Canvas course menu under GatorEvals, or via https://ufl.bluera.com/ufl/. Summaries of course evaluation results are available to students at https://gatorevals.aa.ufl.edu/public-results/.

Materials, Supplies, and Fees:  Not applicable.