SayUncle wonders why with all the press about miracle cures, life is still pretty much putting up with horrible diseases until finally one kills you. I actually most like this explanation for how these stories end up in the media, from the comment section over at Uncle’s. I don’t blog much about work topics, mostly because they pay me to make computers solve drug discovery problems, and I don’t like mixing work and hobby. But I can speak on this topic a bit.
The short answer is that taking breakthrough scientific discoveries and turning them into a pill your doctor can prescribe you is a very difficult, long path that spans more than a decade typically, and that assumes you’re successful in the end, which you probably won’t be. As much as libertarians will want to blame the FDA for this, the FDA isn’t really entirely responsible for this state of affairs. That’s not to say the FDA is blameless, but, for the most part, the problem is rooted in the fact that most of the easy drugable targets have been hit already, and what the industry is left with are harder problems.
Moreover the current industry paradigm for discovering drugs is poorly suited to more difficult targets. The best way I can put it is that if our industry built airplanes, we’d throw thousands of workers at the problem, without too much of a plan, assembling parts and hurtling them up in the air to see if they flew. Do that enough, eventually you’ll probably get some hastily assembled hunk of metal to fly for a bit. But it’s not very efficient at getting a final product. When the industry was hugely profitable, and easy targets were plentiful, this was a successful model. When the problem got harder — we not only need planes that can glide for a bit, we need jetliners — that method no longer works. The problem is, the industry is just starting to figure this out, but they don’t have a paradigm to replace it yet. We still don’t really know how to build airplanes in a systematic way, going back to the analogy.
There have been companies that have developed a more systematic way to discover new compounds that can hit more difficult targets. I currently work for a company that is trying to do drug discovery using supercomputers (which is where I come in). But even doing things this way just offers you a better chance at success. It doesn’t automatically make getting a pill your doctor can prescribe you an easy problem. In the mean time, the industry is in the process of imploding, as patents run out and pipelines dry up. There aren’t enough new drugs to replace what’s going off patent, and that is going to have an effect on research into new drugs.
So where does the FDA come in? The FDA approval process is a significant reason why investigational new drugs fail. Most of the times drugs fail this process, it’s for good reasons, like a really poor side effect profile, which is a nice way of saying the drug slowly cooks your liver, or damages your heart (think Vioxx). Other reasons are that they aren’t efficacious. And having watched this process happen, I can tell you if the FDA approval process, or something like it, weren’t in place, the industry would put drugs on the market that kill people. Not because we’re evil, but because it’s relatively easy to convince yourself of things that aren’t necessarily true, fail to do the right tests, and overlook things. The problem with the FDA is they’ve take their primary role and taken it way beyond basic safety and efficacy. The joke is you couldn’t get Tylenol approved today (toxic to the liver in doses not much higher than the therapeutic dose) nor could you get Aspirin (promotes gastrointestinal hemorrhaging) approved, even though both are generally regarded as safe by the FDA. To me the FDA’s role is essentially to prevent fraud — if you’re marketing a drug to do X, and saying it’s safe and effective, you need to prove that first. Obviously a drug that fries your liver shouldn’t be acceptable. But there are many cases where the FDA is taking their mission way beyond what’s good for society as a whole, and are erring way too much on the side of caution. That’s good for covering the asses of bureaucrats, but not too good for getting life altering and life saving treatments into people’s hands.
