Cost of developing new drugs may be lower than industry claims: trial

The cost of developing drugs is high because of all the drugs that fail, after requiring lots of money to develop. You can't just look at the successful drug and say that's the cost. There are drugs that don't get to the trial stage, so also you can't just look at trials, even if you include failed ones.

The researchers cited in this article seem to be promulgating the fallacy that we need only look at the cost of a successful drug trial, and that's the cost. The drugs magically appeared out of nowhere, for free, and equally magically, they are working drugs, so we already know our trial will succeed. It's just a charade we have to go pay for to get the government's rubber stamp, and then it's all good!

I used to be in the academic side of the so-called industry and I can tell you that most drugs fail because of horrendously bad decisions and hypotheses. Most companies make decisions on what drugs to take to trial on stupidly misleading preclinical data and have been graciously rewarded for their mistakes with no consequences.

There is no "academic side" of the pharmaceutical industry. There is just academia.

And the goal of academia (publishing) are very different than the industry (identifying a new drug).

Agree. I don't think there is any signal from the "academic side" of this discussion. All of the detailed relevant information is very proprietary. I know multiple large players in the industry that share huge amounts of data with academics regularly, but not that data. Decision makers are informed by internal experts with just as many bona fides (and more) than academic peers. Having been in both worlds, and admittedly in my opinion only, academics do well to move their technical fields forward into new and exciting and useful science medicine and economics but do little (well nothing really) in delving into or even accessing the actual data that directly informs clinical trial decisions. That said, I can totally understand why Pharma clinical trial decisions are baffling to academics. E.g., why did aduhelm go forward to (conditional) market approval? That one is baffling to me, but I don't have the data:)

[edit] I'm going to add something here. The grandparent refers to "stupidly misleading pre-clinical data." Pre-clinical data informs very inexpensive (relatively) Phase I studies. And there is a ton of it. You might have potentially misleading pre-clinical data that suggests the possibility of an entirely new therapeutic mechanism of action for a huge unmet need coupled with massive amounts of pre-clinical data that demonstrates a near certainty that the intervention will be safe. That's the entire point of moving forward to a Phase I or Ib (or maybe even II) study. The 1000x-10,000x more expensive phase 3's are not relying on pre-clinical studies.

Then what are the phase 3s relying on? Why is it so hard for such a bunch of supposedly smart people to figure out why their expensive trials are not successful often? Here’s a simple answer: it doesn’t actually matter to the individuals. They still get paid.

I’m from the academic side in the sense have talked to many folks who quit industry to start jobs and come from a lab that did get a drug into the market eventually. I might not know this “secret data” you speak of but given the results are crap, I think they speak for themselves anyway.

Because it’s unbelievably hard to

1. Produce a molecule that does what you want it to in the body

2. Figure out how to get that molecule where it needs to be in the body

3. Prevent it from doing all sorts of other gnarly things

4. Convince people you’ve succeeded in doing 1, 2, and 3 to the degree they’re willing to volunteer themselves to put this new molecule inside their bodies — without incentivizing them in any way

5. Convince enough people to do that and collect enough data about them to know for a fact that your drug does X and does not do Y

6. Convince regulators that not only have you done 1, 2, 3, 4, and 5, but that it’s also better on some dimension than the currently available treatment

In short: because it’s ridiculously hard. If it’s easy you should go give it a shot, help millions or billions of suffering people, and make billions of dollars in the process. Why wouldn’t you?