Timothy R. Coté, MD, MPH (Principal & CEO of Coté Orphan)
You may ask “Unwritten Rules” what does that mean. The Unwritten rules are recognized as rules that are not available for public comment except in the occurrence that the director happens to mention them at an open forum or it is acknowledged by staff members that have worked within the office to become accustomed to that respective unwritten rule.
My first example of an unwritten rule is for Pediatrics. The pediatrics unwritten rule is classified as “a disease among children is considered its own disease, not considered a subset of the whole disease”. This is vital because you need to know whether or not you should invoke the sub setting issues. Children with a disease are not a subset of all people with the disease. The disease occurring among children is considered its own disease. That is why you can obtain orphan designation for diseases that occur in children such as Chron’s disease, Ulcerative Colitis, Schizophrenia or Aids because it’s considered its own disease. If it was considered a subset of a disease you would have to demonstrate that the product does not work outside of that subset. There is a great chance that will not occur due to the fact that a drug that works against Aids in children will probably work in HIV infections among adults as well which outlines the distinction. If you look at the data set of all designations you will see that pediatrics are considered a separate disease and not a subset of the whole disease. In the compendium of what is passed down from director to director, information such as this you will not find anywhere because it is viewed as a passed down unwritten rule.