What I learned week 5

There were several things this week that I learned about clinical trials and drug development, as well as how to take this knowledge and impress future businesses I apply to with terminology I've learned. The biggest thing career wise I learned this week is to really stay current with what's happening within the pharmaceutical world. This mean reading articles, even subscribing to pharmaceutical magazines. This also means getting to know the companies I plan to apply too. Knowing about the culture of a company is very important, because it directs how they do business. Dr. Priefer acknowledged that if you can throw out some terms when on an interview such as LD50, the amount of an ingested substance that kills 50 percent of a test sample, for example, this already puts you ahead of the other applicants because its shows you understand the drug development process and a general knowledge of how things work; even if you know just the basics. So that takes care of the career knowledge taken away this week; the educational studies that both Dr. Priefer and Dr. Siwale focused on were drug development and clinical drugs, drug chemistry and biology and the phases of clinical investigation. Both speakers went into detail about the phases of clinical investigation so I thought this would be important to learn to memory. There are three main phases of clinical trials, and an optional fourth. Phase I tests for possible harm, immediate side effects and works out the correct dosage. This phase usually consists of young healthy individuals, about 50 or so. Phase II tests whether treatment is effective in patients, this also tests for more side effects and lastly tests against the placebo drug. This group consists of around 500 individuals who are affected by the disease. The last formal phase is phase III. This phase tests whether treatment is still effective in patients, it also tests over longer periods over different countries and tests against possible alternative treatments. This is the largest phase, consisting of up to thousands of patients.  There three phases combined take ten to fifteen years to complete, and like is aid before there is an optional phase IV. Phase IV tests over longer periods of time, in different groups of people and / or combines with other treatments. Dr. Siwale also educated us about the chemistry and biology of drugs. I recalled some information from my low-level science classes such as biology, chemistry, and disease and drug therapy. I learned terms such as dissolution rate, partition coefficient, and drug solubility which are all terms/concepts on how the drug enters and effects the body and how fast it effects the bloodstream. Both Dr. Priefer and Dr. Siwale talked about the effective median dose (ED) as well as the median toxic dose (TD), which I believe I now can teach to someone else. The last thing I took away from Dr. Siwale's presentation was on the topic of Orphan drugs. I remember reading about Orphan drugs from my industry report but I didn't know in detail about them. Basically, orphan drugs are drugs for rare disease that usually affect less than 200,000 people in the united states. There is no reasonable expectation when developing orphan drugs that research and developments costs will be recovered, so I questioned why create them? So, I looked online. In fact, the FDA Office of Orphan Products Development (OOPD) provides incentives, such as cash vouchers for sponsors to develop products for rare diseases. The program has successfully enabled the development and marketing of over 600 drugs and biologic products for rare diseases since 1983. Other financial benefits and incentives include:

· Annual grant funding to defray the cost of clinical testing

· Tax credits for the costs of clinical research

· Assistance in clinical research study designs

· Seven-year period of exclusive marketing after an orphan drug is approved

· Waiver of Prescription Drug User Fee Act (PDUFA) filing fees (over $1,000,000 per application for FY 2009)

And this is what I learned this week!

Sources: https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/ucm2005525.html