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How the FDA Could Change the Way It Approves Drugs

fivethirtyeight.com

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Re: How the FDA Could Change the Way It Approves Drugs

#11

Post by eitally »

streptomycin wrote: post #6

Another 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…

Precisely. Here's a specific example: infantile spasms. I was helping my wife research this condition yesterday (for work -- thankfully neither of our children were affected). I found this[1] really great summary article from 2006 that synthesized the findings of numerous trials over the past 50 years and presented the data in a very clear and understandable way.

If you want to medicine to build on the foundations of history, you need to be able to reference the history.

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1464162/

Re: How the FDA Could Change the Way It Approves Drugs

#12

Post by danieltillett »

rayiner wrote: post #7
streptomycin wrote: post #6

Another 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.…

Drugs die for all sorts of reasons other than being ineffective. A major one is the cost of getting a drug approved is now so expensive that you can’t make money off it. The vast majority of diseases are effectively beyond treatment because the market for them is not large enough to support the cost of developing a new drug for them.

The other major reason for failure at Stage I/II is that the preclinical models we are using are not a good match for human disease. This means that a drug that works in a mouse model can’t ever work in humans because the mouse model does not reflect the human disease (Alzheimer's is the classic for this). We are using preclinical models we know are useless and are then surprised when the drugs found using them don’t work in humans.

Re: How the FDA Could Change the Way It Approves Drugs

#13

Post by danieltillett »

streptomycin wrote: post #6

Another 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…

Of course you can - what you do is grant approval conditional on you showing that they are effective once on the market (this is already done with Stage IV trials). With technology it would be much easier to track all patients using a conditionally approved drug and we would get far more useful data too.

Re: How the FDA Could Change the Way It Approves Drugs

#14

Post by danieltillett »

ikeboy wrote: post #9

On 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?

There aren’t any. The FDA and it’s related regulatory authorities around the world are responsible for killing more people than any other organisation that has ever existed (conservatively in the hundreds of millions).

You might wonder how this could be. Basically the cost of getting a drug approved is such that large sections of the human population (poor people especially) have diseases for which we have no treatment or no cheap treatment. We need to get the cost of drug development down to the level that pharmaceutical companies can make a profit developing and selling medicines to poor people or people with what are considered rare diseases.

Re: How the FDA Could Change the Way It Approves Drugs

#15

Post by danieltillett »

Alex3917 wrote: post #8
refurb wrote: post #2

Instead, 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.

It is rather hard to take a drug if the company won’t give it to you.

Pharmaceutical companies are very reluctant to even give drugs to desperate patients because the FDA requires them to report all adverse side-effects even though they have no control over the conditions in which the drug is used. You can actually kill a totally fantastic drug by letting people use it outside of a controlled clinical trial.

Re: How the FDA Could Change the Way It Approves Drugs

#16

Post by ikeboy »

danieltillett wrote: post #14
ikeboy wrote: post #9

On 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?

There aren’t any. The FDA and it’s related regulatory authorities around the world are responsible for killing more people than any other organisation that has ever existed (conservatively in the hundreds of millions). You might wonder how this could be. Basically the cost of getting a drug approved is such that large sections of the human population (poor people especially) have diseases for which we have no treatme…

Why hasn't anything been done since those studies came out? Is there just not awareness?

Re: How the FDA Could Change the Way It Approves Drugs

#17

Post by pinaceae »

danieltillett wrote: post #14
ikeboy wrote: post #9

On 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?

There aren’t any. The FDA and it’s related regulatory authorities around the world are responsible for killing more people than any other organisation that has ever existed (conservatively in the hundreds of millions). You might wonder how this could be. Basically the cost of getting a drug approved is such that large sections of the human population (poor people especially) have diseases for which we have no treatme…

refuting your BS would take far longer than you vomiting this utter, utter nonsense into HN.

just please never take any drug approved by the FDA ever.

Re: How the FDA Could Change the Way It Approves Drugs

#18

Post by danieltillett »

ikeboy wrote: post #16
danieltillett wrote: post #14

Earlier quoted context omitted.

There aren’t any. The FDA and it’s related regulatory authorities around the world are responsible for killing more people than any other organisation that has ever existed (conservatively in the hundreds of millions). You might wonder how this could be. Basically the cost of getting a drug approved is such that large sections of the human population (poor people especially) have diseases for which we have no treatme…

Why hasn't anything been done since those studies came out? Is there just not awareness?

It is a very interesting question. The reason is what is known as regulatory capture. This is normally thought of as the regulators being captured by the industry they are trying to regulate, but it is really more a symbiotic relationship between both sides. Through regulation the large pharmaceutical companies can keep out the competition from any small pharmaceutical companies, while the regulators gain a nice job and importance.

The only losers here are the public since we miss out on cheap effective drugs because the regulations make them too expensive to produce for most diseases and people.

Re: How the FDA Could Change the Way It Approves Drugs

#19

Post by danieltillett »

pinaceae wrote: post #17
danieltillett wrote: post #14

Earlier quoted context omitted.

There aren’t any. The FDA and it’s related regulatory authorities around the world are responsible for killing more people than any other organisation that has ever existed (conservatively in the hundreds of millions). You might wonder how this could be. Basically the cost of getting a drug approved is such that large sections of the human population (poor people especially) have diseases for which we have no treatme…

refuting your BS would take far longer than you vomiting this utter, utter nonsense into HN. just please never take any drug approved by the FDA ever.

Glad to see reason is alive and well on HN. Don’t both trying to provide an argument, just hurl personal abuse.

Since I live in Australia I don’t ever take any drug that is approved by the FDA (this is the job of the TGA). Even if I did live in the USA, I fail to see me advocating for less regulation of the drug approval process somehow prevents me using a drug that has been approved in the past. I am advocating for more drugs, not less.

Re: How the FDA Could Change the Way It Approves Drugs

#20

Post by ccw9302 »

ikeboy wrote: post #9

On 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?

Without FDA, consumers would be flooded with misleading drug ads and waste lots of money on ineffective drugs.

US is really lucky to have FDA.

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