The National Organization for Rare Disorders wants FDA to be a bit more rigorous in communicating its regulatory standards. The agency has always been flexible in determining how much evidence of safety and efficacy is necessary when it comes to orphan drugs; sponsors, investors and patients would benefit if that is more clearly spelled out.

Former MedImmune CEO David Mott discusses the key regulatory and reimbursement risks in drug development. While he sees plenty of venture capitalist money for truly innovative products, there are several areas he tends to avoid at all costs: vaccines, antibiotics, diabetes and-over the longer term-oncology drug development.

Tim Coté has been a tireless advocate for greater involvement by Big Pharma in orphan drug research since taking over as head of FDA's Orphan Products Office two years ago. We talked to him about the response he's heard so far.