Orphan Indications: Streamlined Approval PotentialFor those interested, you can read up on orphan drug status here:
https://en.wikipedia.org/wiki/Orphan_drug
Here's some great information from that Web page:
"Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokineticsand pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. 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.
Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable for pharmaceutical companies, government intervention is often required to motivate a manufacturer to address the need for an orphan drug....
The intervention by government on behalf of orphan drug development can take a variety of forms:
- Tax incentives.
- Enhanced patent protection and marketing rights.
- Clinical research financial subsidization.
- Creating a government-run enterprise to engage in research and development (see Crown corporation).
Currently there are more than 400 orphan designated drugs in clinical trial process."