Perelman School of Medicine at the University of Pennsylvania

Case Studies in Translational Research

Topics | Fall 2021



Topics for CSTR include: 

Working with the FDA

Approval process for medicines 
-   Investigational New Drugs 
-   Orphan disease designation 
Approval process for devices 
-   Investigational Device Exemption 
Consulting with the FDA before you apply 

Institutional review boards (what they do and how to get approval) 

Informed consent 

Good manufacturing practice (GMP) 

What is this? 
Who sets the rules and who regulates the GMC facility? 

Human clinical trials 

Phase I, Phase II, Phase III, and Phase IV trial design 
Trials in children (How does this differ from adult trials) 
First in human trials 
Preclinical data needed to begin a human trial 
Data safety monitoring board (what is this and do you need one)? 
Ethical considerations in trial design 

Health related software development 

How is this regulated? 
How is it tested? 
How do you sell a health related app? 

From academia to industry 

Balancing academic appointments as you start a company 
Deciding to leave academia for industry 
Returning to academia once you leave 

Working with industry as an academic physician scientist 

Consulting 
Contracting 
Licensing 

Intellectual property 

Patenting your ideas 
When do you file a patent? 
Who files a patent? 
What is a disclosure and how does this affect patents? 

Licensing your patented ideas 

Why would you do this? 
Who gets the money?

Starting a company 

How do you do this? 
Advantages and disadvantages 

Raising money  

How much do you need to start a company? 
Where does that money come from? 
What can be supported by the NIH or other grant agencies? (SBIR, R01, other grants) 
Evaluating market size 
Venture capita

Please also consider the impact of racial disparities, socioeconomic disparities, and vulnerable populations

Clinical trial design
Drug targeting, dosing, marketing, and pricing
Equity in the availability of therapies (local, regional, global)
Medical bioinformatics, decision making, and apps
Informed consent (language choices, cultural considerations)

Conflicts of interest