So today is when FDA Commissioner nominee Scott Gottlieb goes before the Senate committee, and it’s time for some thoughts on him, and some more thoughts on the FDA under the new administration. I purposely did not write about all the names that were mentioned for the post, because I just didn’t feel like spending the energy trying to catch all the trial balloons, but it seems clear that there was quite a bit of relief when Gottlieb’s name finally came up. He’s definitely not the FDA head that you’d have seen under a Hillary Clinton administration, but compared to some of the touted candidates, he came across as noticeably sane and reasonable. And that’s all we can expect these days.
Steve Usdin at Biocentury has a good piece on Gottlieb leading off their April 3 issue. He emphasizes that the man is going to be in a tough position, because there are a number of vocal, powerful constituencies that want contradictory things from him. I’m going to stipulate right off that Gottlieb will be approved. I think that enough Democratic senators were relieved that he was chosen, versus some of the more. . .colorful. . .possibilities, and enough of them fear what could come their way if they were to block Gottlieb’s nomination, that he’ll have more than enough votes to be approved. That’s actually one of the Trump administration’s more potent weapons (“Just think whom we could have chosen!”), and although it’s not a particularly honorable one, it has a long tradition in politics and I’m not surprised at all to see it being used.
So there are going to be ritual expressions of horror and pledges of I Cannot In Good Conscience Vote For This Person, but in the end, I fully expect that Gottlieb will be running the FDA. What happens then? Here’s Usdin:
Once he’s confirmed, Gottlieb will probably spend more time dodging friendly fire from the White House than deflecting attacks from congressional Democrats. The White House budget proposals indicate that FDA will not be exempt from the Trump administration’s project to dismantle the administrative state.
Most people who care about FDA, including members of Congress, biopharma CEOs and patient advocates, think the agency is on the right track. . .Trump, however, has spoken often and loudly about FDA, always to criticize the agency. He has said the agency is slow and burdensome and too often dashes the hopes of desperate patients. He has proposed rewriting medical product user fee deals. And he has promised to appoint a commissioner who will unleash a flood of new drugs by wiping a huge number of regulations o the books.
This puts Gottlieb in a tight spot because FDA regulations aren’t holding back huge numbers of great new therapies.
Exactly. I’ve said so at length myself, and most people who have experience in drug development say it, too. From the outside, it looks like FDA approval must be the rate-limiting step for drug approval, but it isn’t. The mental picture of a giant dammed-up lake of wonderful therapies, held back by a huge concrete bureaucracy that allows only a mocking trickle to get through at any one time – is wrong. There is indeed a huge lake of potentially great stuff out there, but the dam that’s holding it back is our lack of understanding of human biology. That seems kind of unbelievable to people without biomedical experience – I mean, we know so much – but believe me, we know so much on the relative scale of what we used to know, but we know so little compared to what we need.
Investigational drugs fail because they don’t work, and they fail because they cause too much harm. Those two reasons account for the great majority of all clinical failures, and 90% of all investigational drugs still fail in the clinic. Neither of them represent situations that you’d want to hand on to the public. I know that there are many people who believe that a less restrictive FDA would bring drug prices down, but I don’t see it: if you let through a lot of stuff that just doesn’t do anyone any good (or outright causes them harm) then you have caused patients (and their insurance companies) to pay for things that have not advanced human health at all. How that saves money is beyond me.
Yes, the thought is that if trials are less onerous, that they’ll cost less and those savings will be passed on to the customers, but that’s not how it works. Drugs aren’t priced the way that they are just because of the cost of trials – they’re priced the way that they are mainly because people (and their insurance companies) will pay those prices. Just like any other good on the market, although it’s true that drugs aren’t quite like many of those other goods. Anyway, the contribution of drug trials to the cost of drugs isn’t that the trials are so long and expensive, as much as the fact that so many of those trials tell us that our drugs don’t work, and we have to eat those costs. Ah, but what if those trials are cheaper? Won’t the failures sting less, then, financially? But if those trials are cheaper, which means faster and smaller, then they won’t even be powerful enough to even tell us if the drugs worked or not, or if they’re safe or not. What then has been gained?
Steve Usdin’s sources tell him that Gottlieb was picked over strong objections from some of Trump’s advisors, who wanted a more burn-it-down approach, and there will still be pressure to do as much of that as possible. (This piece at Stat seems to confirm that). I’ve never envied the job that the FDA and its commissioner have to do, and I envy Gottlieb even less than usual. He’s going to have to deal with fights about user fees, regulatory reform, budget cuts, the opioid epidemic, and more, and quite likely do do some of those under fire from his own administration. Good luck to him, and good luck to us.