ABSTRACT
As drug development costs began to rise in the late 1960s and 1970s with new Food and Drug Administration requirements for demonstrating relative safety and efficacy, manufacturers of drugs, biologicals, and diagnostic agents faced a dilemma. An essential interplay developed between patent protection and regulatory requirements. Patent protection became essential for emerging products developed by research-intensive, vertically integrated firms that looked to a few major market winners to survive in this era of increased development costs. Those orphan drugs that command high prices have generated intense controversy over whether the market exclusivity provision is creating an unnecessary monopoly, keeping prices artificially high. A 1996 National Institutes of Health technology assessment panel addressed issues in diagnosis and treatment of the disease and concluded that despite the success of enzyme therapy, treatment is limited by the cost. It is a sign of progress that for some orphan drugs, accessibility rather than availability is now the challenge requiring creative solutions.
