So we have another whopper of a piece of health care legislation before us in the 21st Century Cures Act. I believe that this one runs over 1,000 pages, so anyone who tells you that they’re on top of the whole thing is probably bluffing. Bills like this are giant piñatas, and all kinds of stuff falls out of them when they’re whacked. Here’s an attempt, based on what I know about it, to see what we’re in for, because at the moment the bill looks reasonably likely to pass in its current form. Here’s a rundown from Stat on the bill, one from NPR, a largely negative one from The American Prospect,
First off, there’s plenty of money being spread around. Over ten years, nearly $5 billion is allocated for the Precision Medicine Initiative, the BRAIN Initiative and Biden’s “Cancer Moonshot”. This is basically a large dose of funding for the NIH, which is probably not a bad thing, although NIH funding can be hard to judge. Its budget increased substantially during the G.W. Bush’s first term, although there was a hangover afterwards when it didn’t keep going up in the same manner. As I understand it, these current funding provisions would be subject to a yearly congressional vote (or at least some of them are), which is much different than a solid ten-year chunk of funding. The first four fiscal years, though, would see money going into the “NIH and Cures Innovation Fund”, which (according to IP Watchdog) is to be spent this way:
At least $500 million of these NIH funds would be used for an Accelerating Advancement Program to partner with national research institutes and national centers to accomplish biomedical research objectives. Of the remaining $1.25 billion or less, at least 35 percent will be allocated to early stage investigators which have earned at most one NIH competitive grant; at least 20 percent allocated for “high-risk, high-reward research”; and at most 10 percent reserved for intramural research. The other $110 million left in the yearly budget will be allocated to a cures development program and will fund drug manufacturing studies, health software and obtaining data on disease history.
The NIH is also directed to set up an “Innovation Prizes Program” to try that out as an incentive for research, and I’ll be interested to see how that goes (that is, what they’re going to direct these to and whether the prizes themselves will be sufficient). One provision directs the NIH to specifically set aside funding for “high risk, high reward” research, which is something that I’m in favor of. There’s a lot of other stuff about establishing councils and collaborative bodies that I’m going to skip over, since I’m never sure if these things are useful or not. In other funding, one billion goes to the states directed towards opioid addiction.
The FDA gets increases in the maximum pay for its employees, under the heading of trying to recruit (and keep) better staff, and this surely reflects the problem that their expertise can be more valuable in the private sector than it seems to be at the agency. But the main news on the regulatory side are some potentially big changes in the drug approval process as well, and these are getting a lot of attention. For one thing, the agency is supposed to “establish processes under which patient experience data may be considered in the risk-benefit assessment of a new drug”. And I’m not quite sure what that means, to be honest, since you’d think that the data from clinical trials might well be called “patient experience data”. Unfortunately, my guess is that this is more like directing the agency to take into account the testimony of people who show up before the hearings regardless of what the actual clinical data said. We’ll see. The actual language of the bill says that it’s “data collected by patients, parents, caregivers, patient advocacy organizations, disease research foundations, medical researchers, research sponsors, or other parties determined appropriate by the Secretary that is intended to facilitate or enhance the Secretary’s risk-benefit assessments“. That could go in a lot of different directions, with that “deemed appropriate” clause as the gatekeeper.
Risk-benefit seems to be at the heart of what a lot of people like about the bill and what a lot of people hate about it. In general, it seems to loosen up FDA approval standards in the service of getting things through faster, by several means. This is particularly noticeable in the many provisions in the bill about medical devices, but it shows up some directed towards antibiotics and regenerative therapies as well. That last one is thought by some be a favor from Mitch McConnell to some donors in the business. There’s no way to satisfy everyone here. There are people who seem convinced that the FDA is this huge, agonizingly slow lump sitting in the way of medical progress, and in my own experience, the same people also tend to believe that regulatory agencies in the rest of the world zip right along, approving things that we don’t see in the US for years. The truth, from what I can see, is closer to the opposite. The FDA approval process has been sped up several times over the last couple of decades, and now is generally faster than than Europe or Japan.
And there’s some fundamental confusion here. I really don’t think that there’s this huge backlog of wonderful therapies piling up behind a big FDA roadblock. FDA approval is not what shows that a drug works; clinical data do that. You can instruct everyone to collect less data, but then you will approve – and ask people and their insurance companies to pay for – more things that don’t actually work. How we are supposed to spend less on health care by approving expensive medications that don’t do much good escapes me. I realize that there’s room to argue about this sort of thing, of course – requiring everyone to run nine Phase III trials would ensure that only really, really efficacious therapies make it to market, but there would be damned few of them (and they’d cost the earth). But I think that it’s a basic principle of how things work that just because you can screw up in one direction doesn’t mean that you can’t screw up in the opposite. Too tight is bad, and too loose is bad, too, and the argument here is whether we’ve reached “too loose” or not. (I should note that there are those who say that the FDA has already been directed to do things like this, with inconsistent and contradictory results).
On the drug-company side of things, the bill originally had provisions that would exclude reporting requirements on some continuing medical education efforts, and that really had the potential to not look good. Indeed, this part of the bill was controversial enough that Sens. Warren and Grassley demanded its removal before they could vote for it, and that happened at the last moment. I’m not sure if that removes their last objections, though, and I believe that Sens. Durbin and Sanders have also come out against the bill, last I heard. As far as I know, the only way that the legislation can clear the Senate in time during this session would be by unanimous consent, so issues like this matter a great deal. In general, it would be hard to vote against a bill that increases cancer funding and efforts against opioid addiction, and a lot of other popular things, but if you can characterize it as an unconscionable giveaway to the drug companies you could probably swing it. So we’ll see this week how that shakes out. I would think that attempts at this point to modify the bill would almost certainly shove it into the next congress, and at that point who knows what happens.