An orphan drug is one which no one wants to take responsibility for.

Features that can result in a drug being considered an orphan:

(1) rare disorder with not enough people affected to make development cost effective

(2) cost of development greater than possible return

(3) risk of liability greater than possible return


FDA criteria for an orphan drug:

(1) one that targets a disease affecting < 200,000 people in the US

(2) one that will take > 7 years to be profitable for a company


Groups that may refuse to take responsibility for orphan drug development:

(1) pharmaceutical companies

(2) governments


Because only a small number of people may be affected, clinical trials as conducted for common disorders may be difficult to conduct. It may be necessary to change the requirements for drug approval with this in mind.


Since most countries will face the same problems, a country may need to modify drug importation laws to allow access to these drugs.


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