Orange Book: also known as the Approved Drug Products with
Therapeutic Equivalence Evaluations, the Orange Book contains detailed
information on all approved drugs and must list all extant patents.
Orphan Drug: a drug that treats a disease that affects less
than 200,000 Americans or for which there is no reasonable expectation
that the cost of research and development will be recovered from sales
in the United States. The Orphan Drug Act provides special incentives
for producers of orphan drugs.
Phase I: clinical trial designed primarily to determine the
safety of an experimental drug.
Phase II: clinical trial that evaluates an experimental
drug’s safety, assesses side effects, and establishes dosage
Phase III: clinical trial designed to verify the safety and
effectiveness of an experimental drug. Success in Phase III trials can
lead to marketing approval.
Phase IV: post-approval clinical trials used to monitor safety
and efficacy or examine additional applications of drugs.
Pioneer (brand-name) drug: the patented version of a drug.
Contrast with generic drugs, the competing versions produced when
pioneer patents expire.
Preclinical studies: studies that test a drug on animals and
nonhuman test systems. Safety information from such studies is used to
support an investigational new drug application (IND).
Salami slicing: filing for multiple orphan drug designations
on the same drug.
Small-molecule drug: a drug produced using defined chemical
synthesis or extraction. Contrast with biologics, drugs produced by
Surrogate marker: an indirect measure of effectiveness, such
as a laboratory test or tumor shrinkage, used to show a strong potential
for effectiveness in accelerated drug approval.
Alliance: agreement between two or more companies to cooperate
in some way.
Angel investor: wealthy individual who personally provides
startup capital to very young companies to help them grow.