Originally Posted by
wiggin
Lewk, I am giving you the benefit of the doubt on this one because I assume you do not have first hand knowledge of how drugs and devices are developed and regulated. So instead I'll educate you.
Making a new drug or device is indeed a typically slow process. For drugs, you first need to identify a worthwhile target (which itself might take decades of basic science research). Once you have a target but don't actually have a way of hitting said target, we typically start the 'clock' on drug development. Scientists have a variety of methods of developing drugs that appropriately modulate the targeted cellular process, but typically it involves very large scale screens against libraries of compounds (think hundreds of thousands) followed by sophisticated chemical modification of the best candidates to improve their safety and efficacy profiles. So let's say you've been very fortunate and it only took you 3 or 4 years to find a drug candidate that you think - based on cell studies and maybe some small animal studies - that it might do what you want it to do. Then you need to check a lot of boxes - can you manufacture the compound? How will it be delivered? Does it need a special formulation (excipients, etc.)? Once you have something in a form similar to what you'd want to use in a human - again, if you're lucky, you're maybe 5 years into your development process - only then do regulatory agencies really get involved.
Generally there is a relatively small and fairly straightforward set of tests that must be done before a regulatory agency will be comfortable starting a first in human trial - some basic toxicity/etc. tests on cells and small animals, and they might also want to see some sort of efficacy data in a good animal model. Essentially, they want some level of assurance that you aren't likely to kill your patients and that your therapy will, in the future, have some potential upside for the patient population. These tests can be done quite quickly - on the order of months - and are by and large tests you would want to do before putting it in a human anyways. Efficacy testing in animals might take a lot longer, depending on the indication they're going for, but that's a function of biological reality, not FDA bureaucracy.
Okay, so you've spent a few extra months (and maybe a million bucks) to check your boxes, and you go for a phase I trial. Phase I trials are intended to be very small numbers of patients testing the drug (or device) for safety/toxicity alone, with little to no consideration of efficacy in treating an indication. Depending on the technology in question, patients may even be healthy or otherwise not likely to benefit from the drug. It's really just to determine a safe dosing range and as a sanity check against any unforeseen consequences. Phase I trials, thus, tend to have short endpoints and relatively modest recruitment time and cost. Depending on the indication they might be completed in a year or much less. Again, this is something you'd probably want to do at any rate.
Then you typically carry out phase II and phase III trials - both of which look at efficacy in addition to safety, but with different scales. Phase II trials look at modestly larger numbers of patients, so while they're interested in following efficacy, it's also a way to see if any safety issues crop up that weren't seen in the very small Phase I setting. Phase III trials are even larger and are intended to provide a definitive answer about efficacy and safety for an FDA submission. These trials are typically where the biggest cost comes in (after initial R&D/discovery work) and also take the most time. But again, for an ethical scientist who wanted to accurately determine if their technology was safe and effective for use, there isn't much you'd change about trial design with or without the FDA.
The only big delay happens after you finish your phase III trials. You have to put all of your data - from the earliest preclinical tox and validation studies all the way through to the final phase III trial - into a big application and send it to a regulatory agency like the FDA. They then need to look at all of your data, analyze it themselves, discuss risk/benefit tradeoffs, and arrive at a decision. Typically this process takes about a year. It's certainly possible to make this shorter (likely be increasing funding for the FDA) but it's really the only major delay/cost the FDA adds to the process. And during the review period, typically companies are working on gearing up commercialization strategy and manufacturing and the like, so it's not obvious that they'd be able to go to market immediately if the FDA, say, halved the review period.
Perhaps more telling is that the FDA is well aware that this is costly for companies to wait for review, so they provide a number of different fast track designations that allow for expedited applications and review. For example, if a device is considered 'substantially similar' to a previous device (i.e. it's essentially an iteration of a basic and proven design), you can apply for approval through the '510k' process that is much faster, cheaper, and easier in terms of what you need to show in terms of clinical safety and efficacy data. You can get humanitarian exemptions for fast track approval of treatments that don't have good alternatives. You can get orphan drug status for drugs targeting tiny patient populations, which effectively limit what kind of clinical testing you can do. Generics and biosimilars have shorter approval cycles. Etc.
There are lots of ways to speed the approval process, and the FDA actively participates in them. Typically they encourage device/drug firms to meet with them early and often through so-called 'presubmission meetings' to make sure that the preclinical and clinical research plan looks reasonable and will likely pass muster. Companies will often carry out clinical trials in other countries where it is cheaper to operate (though still operating under strict IRB and regulatory controls, of course) in order to lessen the cost of clinical trials. I even was talking to an executive at one massive pharma company who said that they leverage their global operations to reduce the amount of time between closing a clinical trial and submitting regulatory paperwork by having their regulatory experts work on the submission around the clock, handing the documents off to offices around the globe.
I have spent a lot of time recently immersed in the world of preclinical and clinical testing and the subsequent FDA approval process. While it's certainly laborious and mind-bogglingly expensive, I've been continually impressed with how fundamentally reasonable the FDA has been most of the time. If there's a standard assay for a specific outcome that isn't optimal for your technology because of a solid scientific reason, you can just explain your logic, present an alternative, and they'll typically accept it. If you have clinical data that's okay but not amazing, if it meets their predetermined criteria for success, they'll often err on the side of approval in balancing risks and benefits to patients. And if you have an entirely new class of therapy that they're unfamiliar with, they'll work closely with you to assure that you can adequately demonstrate the safety and efficacy of your technology without an undue burden.
Working in a highly regulated industry is no fun, obviously. And there are times when you are frustrated by the bureaucracy or risk aversion involved (though one consolation is that you often get to insulate your technology a bit from competition because of the barrier to market entry). But by and large they're doing something we would want to do anyways in order to provide the best care for patients, and they're doing it in a broadly reasonable, fair, and sophisticated manner. It's not hard to get approval from someone (doesn't need to be the FDA) to test our a new drug (or vaccine) for a phase I trial - you really need to show pretty basic data. The 'scientists' who decided to do this trial without even an IRB were simply being lazy, unethical, and were probably bad at the science part to boot.
It has gotten harder to get approval for therapies, that is true. Partly that's because learning from past failures gives us better and more numerous tests to eliminate problematic compounds. Partly it's because a lot of the low hanging fruit has already been plucked - it's a lot harder to make a safe and effective drug to substantially improve on current clinical practice than it used to. Partly it's because new therapies tend to be so complex and so prone to unexpected challenges that it takes time to truly evaluate them. And part of it is that they were far too lax in the past, mostly because they didn't know any better. But I don't see a lot of evidence that it's just pointless bureaucracy holding back our champions of capitalism.