Orphan Drugs for Rare Diseases

1. Go to:http://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm

a. In the start date, type 01/01/2010.

b. In the “Search Results”, pull down “Only Approved Products”.

c. In the “Sort results”, pull down to show “Most Recent to Earliest”.

d. In the “Records per Page”, type 100.

e. Press “Run Search”.

f. Pick one drug whose row number is the same as the last 2 digits of your student number. You may need to click on the second page to see your number. Write your student number across your name in your submission.

g. From the table, obtain info on the orphan designation or disease/indication

h. Use Pubmed (http://www.ncbi.nlm.nih.gov/pubmed/?querykey=4%26dbase=pubmed%26querytype=eSearch%26) to find references on the drug and indication (disorder), market size (number of people affected) and discuss the rare disease or condition for which the drug is being or will be investigated, the proposed use of the drug, and the reasons why such therapy is needed. If not much information is available in PubMed about your drug or disease, pick a different drug to work on that is closest to your student ID.

i. Provide a description of the drug, and include the identity of the active moiety if it is a drug composed of small molecules, or of the principal molecular structural features if it is composed of macromolecules; its physical and chemical properties, if they are available.

j. Discuss the scientific rationale or a medically plausible basis for the use of the drug for the rare disease or condition, including all relevant data from in vitro laboratory studies, preclinical efficacy studies conducted in an animal model for the human disease or condition, and clinical experience with the drug in the rare disease or condition, if available.

k. Reference section is required for this homework (Follows citation style for journal that is found in: http://jb.asm.org/site/misc/journal-ita_org.xhtml#02)