ALEXANDRIA, VIRGINIA—“There are three ways to try to show that your drug has a treatment benefit,” Frank J. Sasinowski, MS, MPH, JD, said at NORD’s Rare Diseases and Orphan Products Breakthrough Summit. “There’s the 1962 law’s standard of two adequate, well-controlled studies; there’s the 1997 statutory amendment that says one adequate, well-controlled study with confirmatory evidence; and there’s the 1998 FDA guidance document that specifies one study providing statistically persuasive evidence and where a second study would be difficult to conduct on practical or ethical grounds.”
Mr. Sasinowski advised sponsors, researchers, and investors seeking regulatory approval of a rare disease therapy to choose a path from the outset. “If you’re an investor, you should demand that [the sponsor] tell you which path it is pursuing. If you are going into your pre–Investigational New Drug meeting, you should be talking to the FDA about which path from the very beginning so that then later you don’t accuse the FDA of changing its mind and redirecting you.”
Being clear from the start will help accelerate development, Mr. Sasinowski advised. “Regardless of which path you pursue, know that there can be an accelerant to that road to approval if you are either pursuing a drug for a serious condition where there is an unmet medical need (Subpart H) or pursuing an orphan drug.” Mr. Sasinowski’s analyses of FDA’s Subpart H approvals and approvals for orphan drug therapies establish that the FDA has exercised regulatory flexibility for these two categories of therapies.
“You should have a clear, fixed idea of what you are going into from day one” and be aware of what sort of flexibility that path warrants, Mr. Sasinowski advised.
—Glenn S. Williams