![]() The intervention by government on behalf of orphan drug development can take a variety of forms: Critics of free market enterprise often cite this as a failure of free market economic systems. Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable, government intervention is often required to motivate a manufacturer to address the need for an orphan drug. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. However, some statistical burdens are lessened in an effort to maintain development momentum. Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. 5.2 Keck Graduate Institute Center for Rare Disease Therapies.2.2 Homozygous familial hypercholesterolemia.
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