Intro to Regulatory: Drug & Device Discovery; the U.S. Regulatory Process
Question 1 (no more than 2 typed pages)
You are developing a drug for the treatment of Parkinson Disease. You want to use a biomarker. Discuss a trial design employing a biomarker. When is a biomarker a surrogate? How could you establish a surrogate marker?
Question 2 (no more than 3 typed pages)
You are working with a Dr. Reiser, Chairman of Medicine at the university, developing a device that removes suPar from the circulation that prevents the development of membranous glomerular sclerosis. Pre-clinical studies show the device effective in removing suPar from circulation. What are the steps needed for development – regulatory, ethical and trial design(s)? Describe your pivotal study for regulatory approval. Could this device go through the 510K pathway for approval in U.S.?
Question 3 (limit two pages)
Describe and discuss the steps involved in drug development – stages, the trial designs often used, number of patients in each phase, their duration and the average drugs success in each phase. What steps are necessary to receive FDA approval for initial study, clinical trial initiation, approval and marketing?
Question 4 (limit two pages)
You want to use Bretylium – a neurotransmitter depleting agent used in cardiology as an anti-arrhythmic drug to treat severe manic patients. The drug is no longer available to prescribe, but you found a supply available for $140/100 gms from Sigma Aldridge, a chemical company. What steps do you need to do to initiate studies in manic patients with this chemical? Can you relate the potential problems to experiences of other investigators in the literature that lead to significant issues?
Question 5 (limit 3 pages)
You are advising a company on the development of a novel therapy for forgetfulness. The company asked you if the Phase 2 studies should be parallel or crossover design and for you to explain why you are recommending one over the other. Then they want to know if the Phase 3 study should be crossover or parallel and explain your reasoning on the pros and cons of the different study designs? Finally, there is a question raised from the pre-clinical evaluation that the drug’s effects may dissipate over time. What study design may be best to evaluate this potential problem and why?
What would be the role of an enrichment design and a randomized placebo withdrawal study?