Guidelines & Outline | Fall 2018
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.
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.
Topics to be covered
CSTR is about human studies and the development of new therapeutics and devices. Relevant topics that should be examined this Fall in your presentations include the following. Not every topic needs to be included in every presentation, but we should aim to cover them all before the course is complete. Take this list into account when you meet with your preceptor and design your presentations. The members of Team A will be the official list managers for the entire course - which means checking off topics as they are covered in all of the presentations, not just their own.
• Intellectual property (IP) and patents: Why do you need them, why will investors want them and what do they do for you? Who owns the patents if you work at Penn?
• Human studies approvals: When do you need it, how do you obtain it from an IRB (institutional review board) and when do you need to request modifications?
• Informed consent: When do you need it and who can or can’t give it?
• First-in-human trials: What type of data do you need to proceed from cell culture and animal studies to humans? How do you know you have enough data to proceed?
• Clinical trials in pediatrics: When are you allowed to do trials on kids and who can give consent for their participation?
• Conducting an Investigational New Drug (IND) or Investigational Device Exemption (IDE) study in academia: What are some of the key responsibilities of academic clinical trial sponsors? What are some of the key responsibilities of academic clinical trial investigators?
• Dealing with the FDA: What role does the FDA play in new drug and new device development and commercialization? What does “safe and effective” mean and what is the role of the FDA in evaluating it?
• Human trial design: What are the differences between and goals of phase 1, 2 and 3 clinical trials? How large do they have to be? What are the right controls and when do you need them?
• Conflict of interest (COI): What does this term mean and how does it affect your ability to do new drug and device development and testing?
• Orphan diseases: What is this designation mean and how does it affect drug development?
• Good manufacturing practice (GMP): What does this term cover, how does it affect the development of new drugs and biologicals? Who sets the rules?
• Device development: How is it different than drug development? How is it regulated by the FDA?
• Health-related software development: How is it regulated and tested? When can you sell a health-related app?
• Evaluating market size: how do you decide whether it is worth proceeding to commercialization?
• Paying for it: What role do the NIH, venture capital and other potential sources of funds play in the development and commercialization of new drugs, biologicals and devices? What is an NIH SBIR? How do you obtain funding through these mechanisms and what, if anything, will you have to give up to obtain it?
• Safety in human trials: What is the DSMB and what is the role of an independent safety and trial monitoring board in clinical trials?
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.
The class will meet 13 times in the Fall semester: an introductory session on August 23rd, a field trip to SPARK therapeutics (http://www.sparktx.com) on August 24th, two didactic sessions, and 5 double sessions (one double session for each case study).
|August 23||Intro/Guidance session|
|August 24||SPARK therapeutics (http://www.sparktx.com)|
|Sept. 5 & Sept. 12||Lead team A|
|Sept. 26 & Oct. 1||Lead team B|
|Oct. 3 & Oct. 8||Lead team C|
|Oct. 15||Information Session with guest speakers|
|Nov. 5 & Nov. 7||Lead team D|
|Nov. 12 & Nov. 14||Lead team E|
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.
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.