Small Victories

One of the quant-ish memes in these endeavors is its use in the field of pharma. Should NHST be used? Should Bayes be used? Should single arm trials be allowed? And so on. But one of them has to do with the gaming of the system. There are as many ways to attempt that as there are the stars in the sky. Over the last decade or so, one of the most popular has been Orphan Drug designation.

You can read up the wiki article for all the gory details, but the summary is that Orphan Drug designation blesses the sponsor with lots of additional profit making opportunities. One of the more subtle, and not necessarily obvious, during the application process: can/will the drug, if approved, be easily moved into a wider population than the orphan indication? The orphan benefits, by current law, follow the drug into such indications. Some mainstream pundits have kvetched about that and about the number of potential patients which define orphan, currently 200,000. That's a lot.

Well, today's briefing.com brings some good news. If you think spiking the cheating is good news:

6:01 am CytoDyn announces that its application for Orphan Drug Designation was not granted by the Office of Orphan Products Development of the FDA 'because PRO 140 appears to have the potential to treat more than just the subset of multi-drug resistant HIV patients for which the designation was requested'

I wonder how much they'll send along to Jared or one of the Wonder Kinder to have FDA change its mind?