Back in 2013 and again in 2016, I wrote about Catalyst Pharmaceuticals, a South Florida company whose business plan I found repellant. That’s because said plan involved running an old generic compound (3,4-diaminopyridine) through the FDA modern approval process, which under the agency’s rules would grant them market exclusivity. I have railed about this process before; I think it’s too generous a reward for too little work and too little risk. In fact, there’s basically no risk whatsoever: a compound in this position is already being given to human patients and has been given to human patients for years (decades, in fact). You know it works. You know the liabilities and you know that they’re manageable. You already know everything that a clinical development program is supposed to tell you. So all you have to do is walk up to the tree, pluck off that sweet juicy low-hanging piece of fruit, and start enjoying yourself.
That’s what Catalyst is doing. 3,4-diaminopyridine has been given out for free by a company called Jacobus Pharmaceuticals under a compassionate use program, but that’s because Jacobus didn’t understand the modern world. The modern world is where people thought that Catalyst might charge $100,000 for a drug that they didn’t discover, that no one involved has taken any discovery risks for whatsoever, and that was already helping patients for free. But guess what? They’re listing it at $375,000. Because they can. We’ve all been watching this story as it has been coming slowly over the horizon, and here it is. So if for some reason you were hoping for some more bad press about evil, greedy drug companies, this is your lucky day. If you have the neuromuscular disease that 3,4-DAP treats, though, then not so much.
There is no reason that exploitation of a regulatory loophole should allow for this sort of thing. Rewards should be earned. But the FDA thinks so highly of its plan to take long-used generics and re-bless them that they make this possible. So although I have contempt for Catalyst Pharmaceuticals and all the other little fly-by-night outfits who are taking advantage of this same process, let’s put the blame where it belongs: on the FDA. I have defended the agency vigorously in the past, but I can’t defend this. This is stupid, this is costly, and this is wrong.