Of course, the big show will be the suit against the FDA. The drugmaker has accused the agency of messing with the incentives provided under the Orphan Drug Act and selectively applying policy when it comes to enforcing the law concerning compounding.
The typical clinical trial model may not be best suited for generating evidence that a drug is safe and effective against diseases where patient enrollment can be difficult and diseases progress slowly and unevenly over a long period of time, according to Steven Grossman, who worked on the original Orphan Drug Act in 1983 and is now a policy and regulatory consultant.