From Pharmalot, a post describing BIO's lobbying efforts with the Senate and FDA to expand the use of accelerated review in the FDA for drugs to diseases that do not have any approved cures. This would be applied not only to drugs for Orphan conditions but likely to cancers or AIDs drugs as well.
Under accelerated approval, a drugmaker can conduct shorter trials based on the measured effect of the drug rather than on an actual clinical outcome. Sen. Kay Hagan (NC-Dem) would also ask that Phase II data could be used for drugs where data can't be "ethically, feasibly or practicably generated."
Presumably, this lobbying effort is being done in part as a consequence of criticism that the FDA's approval process has become too unpredictable in these type of clinical areas. The FDA responded that the agency has approved 85 rare-disease treatments since 2006, and that 75 percent were based on limited clinical trials.
That observation would tend to undermine industry's claims and make them sound as if they are sounding a "false alarm". Notwithstanding, I would still tend to favor expedited approval on drugs meeting the criteria above if there is also strong post-approval surveilance. Based on analysis of that data, all parties involved should be willing and prepared to pull drugs from the market that do not live up to their pre-approval performance predictions when exposed to larger patient groups.
Posted by Bruce Lehr Nov 16th 2011.