Biotech Values

[iwfal]

Disease Charities misfocus (one more follow up to my BMRN AdComm review post)

The FDA asked for CINRG natural history data that was not delivered with the NDA (and the FDA couldn’t get it either).

It amazed me that they couldn't get the data from CINRG - a group dedicated to DMD. Obviously I don't know the reason, but as a general rule I think the charities almost always seem to focus on new treatments and very little on detailed understanding of the natural history and the best clinical markers and prognostics. This is not a small or nuanced problem. It probably delays drug approvals by years - e.g. by forcing bigger/longer trials or just plain more trials. (Note that smarter orphan drug biotechs tend to start spending substantial money collecting natural history early - because they know it increases the odds and lowers cost and time to market)