Perelman School of Medicine at the University of Pennsylvania

Case Studies in Translational Research

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Guidelines & Outline | Fall 2017

Course description

A student-led, faculty-supported exploration of the role of physician-scientists in bench to bedside translational medicine. We will examine 5 case studies of translational research using investigators from Penn and industry as preceptors. The course design, which was pilot-tested last year and revised for this year, requires active involvement by all of the students on all of the case studies, working in small teams in which students will rotate as team leader. The course directors and case preceptors have chosen the topics, but the members of each week’s lead team will be responsible for selecting and guiding deliverables. Team roles are described below.


Course goals

Physician-scientists are especially well-suited for guiding the development of new therapeutic and diagnostic modalities from the research setting to the clinic, but may not be trained in all of the skills needed to accomplish this. The challenges attendant with accomplishing this successfully are diverse and in some respects very different from the day to day issues of running a basic science research lab (or a PhD thesis project). The goal of CSTR2.0 is to teach you about these challenges and how to overcome them in the context of translational science.

Among the useful skills that we will focus on in this course are: 1) Setting research goals and meeting milestones in the setting of clinical research. 2) Selecting outcomes and producing deliverables. 3) Protecting intellectual property and managing conflict of interest. 4) Performing preclinical and clinical studies. 5) Organizing, leading and working in teams that include people with different skills. 


Lead and Support Team organization

This course is intended to be part of the second year MD/PhD curriculum. There will be a sign-up sheet by David in Maggie’s office as of Monday August 22. By Friday August 26, please sign up to be on one of the 5 teams, each of which will have either 4 or 5 team members. Each team will be the Lead Team on one of the 5 translational cases, and a support team on each of the other 4.


Course organization

The class will meet 14 times in the Fall semester:  an introductory session on August 22nd, a field trip to SPARK therapeutics ( on August 24th, two didactic sessions, and 5 double sessions (one double session for each case study).

August 22 Team sign-up (Maggie’s office)
August 22 Intro/Guidance session
August 24 SPARK therapeutics (
August 29 Didactic Session
Aug 31 & Sept. 7 Lead team A  
Sept. 14 Didactic Session
Sept. 21 & 28 Lead team B
Oct 5 & 12 Lead team C
Oct 19 & Nov 9 Lead team D
Nov 14 & 30 Lead team E



Team roles

Each team acts as the lead team for one case study and as a support teams for each of the other 4 case studies. One way to do this is to have each member of the lead team act as temporary leader for one of the support teams, helping to select the deliverable chosen for that support team. A list of possible deliverables is shown below.

Each double session requires at a minimum a slide or chalk talk by the lead team plus at least one deliverable from each support team. Work by the lead team in advance of Week 1 should include selection of the appropriate primary articles (basic and translational) with the help of the preceptor and the course directors. Week 1 class time will be used for an overview presentation by the lead time, selection of the deliverable and initial team work on the deliverables. Week 2 will be used for presentation and discussion by each of the teams under the guidance of the lead team.


Preceptor’s roles

Provide the topic and a suggest a clinical and basic article. Meet with the Lead Team in advance of Week 1 at least twice. Advise on additional papers and on deliverables. Attend Week 1 and Week 2.


Some possible deliverables (check with the course directors if you want to add to this list)

Deliverables should reflect the goals of the course and the skills listed above. Examples include: design of relevant preclinical studies, design of relevant clinical studies, IRB (human studies) and IACUC (animal) protocols, presentations to the FDA, device development, patent application(s), designing a translational path from an initial discovery, conducting translational research within University of Pennsylvania regulations for faculty involvement, recognizing and avoiding conflict of interest when translating your own work to the clinical space, obtaining funding from venture capital sources as well as federal sources, statistical analysis of clinical data, designing checkpoints within clinical trials.


Evaluation and feedback

This course places an emphasis on regular evaluation and, if needed, modification as we go along. Therefore, the last 10 min of each Week 2 session is reserved for whole class feedback and evaluation by the course directors.



This is an official BGS course and, as a consequence, graded ABCDF. Since the course design is entirely team-based and there will not be a test, grades will be based solely on participation. Last year everyone received an A.  Attendance will be taken. Notify Lauren in advance if you have to miss a class.