How the FDA Could Change the Way It Approves Drugs
fivethirtyeight.com
How the FDA Could Change the Way It Approves Drugs
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Re: How the FDA Could Change the Way It Approves Drugs
#2Instead, they say, the standards should take into account the severity of the diseases that the drugs seek to address.
The FDA already does this. Take a look at the Duchenne's muscular dystrophy drugs currently up for review. One of them fail to show a statistically significant difference from the control group and the other one only had a single trial of 10 patients.
The likelihood is that one or both of the drugs will be approved since no treatments currently exist.
Then take a look at diabetic drugs. Any hint of cardiovascular side effects and the FDA slaps a black box warning on the package and asks for a 10,000 patient follow-up trial. This makes sense because drugs already exist to control diabetes.
This article seems to lay out a more mathematically driven method for statistical testing, but it's a bit ingenuous to say this is something new for the FDA.
Re: How the FDA Could Change the Way It Approves Drugs
#3[deleted]
Re: How the FDA Could Change the Way It Approves Drugs
#4The real source of the problem is at the preclinical stage. The requirements here are so strict that almost no drugs make it out of mice and dogs and into humans for actual testing.
Re: How the FDA Could Change the Way It Approves Drugs
#5It's a do more harm than good question.
Re: How the FDA Could Change the Way It Approves Drugs
#6Another concern with approving drugs more easily: you might then never find out if they really work or not.
The reason we need very large, expensive, and strict clinical trials is because it's hard to see if a drug actually works or not otherwise - too many confounders. So if we pass drugs through with a higher error rate, we'll likely never know if some of them actually work or not. Maybe not a problem for a disease with no treatment at all, but imagine you have 10 treatments and you suspect that 7 of them don't actually work, but you don't know which 7. Is that better than just having 3 available treatments where only 1 of them doesn't actually work?
Re: How the FDA Could Change the Way It Approves Drugs
#7Another concern with approving drugs more easily: you might then never find out if they really work or not. The reason we need very large, expensive, and strict clinical trials is because it's hard to see if a drug actually works or not otherwise - too many confounders. So if we pass drugs through with a higher error rate, we'll likely never know if some of them actually work or not. Maybe not a problem for a disease…
This is the heart of it. People like to paint this as a regulatory problem--the FDA keeping all these potentially useful drugs off the market due to its bureaucracy. But he fact is that 85-90% of drugs that hit early Stage I clinical testing don't make it through approval. That means the science is bad at predicting what will work and what won't work, and spectacularly so. That's a science problem, not a regulatory.
People bring up the "more harm than good" issue, but they're only thinking of one kind of harm: drugs that actually hurt people. But having ineffective drugs on the market also has a cost, and it's probably the bigger one. The market for effective drugs is almost certainly not efficient given the difficulty of ascertaining efficacy and the information asymmetries involved. Thus, there is a real risk of people dying because the inefficient market selects for drugs with good marketing over ones that work.
Re: How the FDA Could Change the Way It Approves Drugs
#8Instead, they say, the standards should take into account the severity of the diseases that the drugs seek to address. The FDA already does this. Take a look at the Duchenne's muscular dystrophy drugs currently up for review. One of them fail to show a statistically significant difference from the control group and the other one only had a single trial of 10 patients. The likelihood is that one or both of the drugs w…
Keep in mind also that as a patient you're allowed to take basically any drug you want, companies just aren't allowed to sell those drugs to other people and claim they treat a specific disease without FDA approval. So this whole article is basically just shilling for even less stringent requirements to market a drug as being effective, even though that hurdle is already trivially easy to overcome in these cases.
Re: How the FDA Could Change the Way It Approves Drugs
#9On a related topic, http://www.fdareview.org argues that much of the FDA is more harmful than useful; in particular the efficacy requirement. Does anyone know good counterarguments?
Re: How the FDA Could Change the Way It Approves Drugs
#10> A patient with a seriously life-threatening disease like lung cancer is perhaps more willing to gamble on a risky drug in pursuit of a cure, while someone with a disease that has a high survival rate such as diabetes presumably cares more about avoiding adverse side effects.
If he's more willing to gamble, then why not just let him do it? The FDA's job should be to enforce truth-in-labelling (which means drugs with no beneficial use should be labelled as such, and those with horrible side effects should be labelled as such), and patients working in concert with their physicians should determine which drugs to take.
For this to work, we may need to limit the ability of drugs to be advertised. I'm okay with limiting the civil right to free speech of pharmaceutical companies in order to respect the fundamental human right to ingest whatever one wishes of patients.