[refurb]

Sure, but as a result we don't have problems like thalidomide popping up every decade or so. There are benefits (and drawbacks) to having a much more stringent approval process.

And probably worth adding that in public health emergencies, the FDA is more than willing to pull back on regulations in the interest of the public good.

[barry-cotter]

You can have a stringent safety process without having any efficacy process. The FDA has no incentive to approve drugs but plenty to deny them so delay is the order of the day. Delay in approving new effective drugs costs lives.

The FDA has not shown any willingness to pull back on regulations during the current COVID crisis, nor have they historically shown any such willingness. Politicians have forced them to. They have never shown any organic willingness to relax regulations.

[vikramkr]

I'm not clear on whether you're arguing that the FDA should not be requiring efficacy? The FDA makes exceptions on cases of urgent need, and not having efficacy as a bar would reduce the reward for innovation - why would you spend all the time and effort to development a treatment for a disease if vitamins with no efficacy are already filling the market? False hope is dangerous, and healthcare is uniquely suited to attracting snake oil salesmen with cure-alls because of how serious health needs are - these regulations are written in blood

[barry-cotter]

The FDA should not require efficacy testing. If it’s safe let patients and their doctors do what they want. In practice that’s what they do anyway, see off label prescribing which is around one fifth of all prescriptions in the US. Removing efficacy testing would double or more the number of new drugs available.

Fraud is already illegal. Doctors can already be sued for malpractice. Testing for efficacy makes no sense in a world in which off label prescribing is routine.

> Assessing the FDA via the Anomaly of Off-Label Drug Prescribing —————— ——————

> Once a drug has been approved for some use, the FDA has almost no control over how that drug is actually prescribed. The prescribing of drugs for non-FDA-approved uses, called “off-label prescribing,” is widespread. Drugs prescribed off-label have not met the FDA’s requirements for proving efficacy in the off-label applications. The practice of off-label prescribing therefore raises interesting questions. Why does the FDA, in effect, require that some drugs be tested for efficacy but not others? If there are good reasons for the FDA to have strong pre-approval powers regarding efficacy, shouldn’t FDA post-approval powers be commensurate? Alternatively, if there is good reason for widespread off-label prescribing, doesn’t this call into question the FDA’s pre-approval powers?

https://www.independent.org/publications/tir/article.asp?id=...

> There are also scientific reasons to replace Phase 3. The reasoning behind the Phase 3 requirement — that the average efficacy of a drug is relevant to an individual patient — flies in the face of what we now know about drug responsiveness. Very few drugs are effective in all individuals. In fact, most are not effective in large portions of the population, for reasons that we are just beginning to understand.

> It’s much easier to get approval for drugs that are marginally effective in, say, half the population than drugs that are very effective in a small fraction of patients. This statistical barrier discourages the pharmaceutical industry from even beginning to attack diseases, such as Parkinson’s, that are likely to have several subtypes, each of which may respond to a different drug. These drugs are the underappreciated casualties of the Phase 3 requirement; they will never be developed because the risk of failure at Phase 3 is simply too great.

http://www.washingtonpost.com/wp-dyn/articles/A43257-2003Nov...

[vikramkr]

Off label use is allowed. Marketing for off label use is absolutely not allowed. Claims of off label efficacy are not remotely allowed. And the FDA has post approval powers to revoke approval for drugs not found effective. Doctor's autonomy leads to off label being allowed, and some of the legal arguments for it involve freedom of speech, not medical optimality.

Phase 3s already are no longer required, it looks like the article you linked was written in 2003? That was literally almost 17 years ago when we'd just barely sequenced the first human genome. There are entire fields of medicine - precision medicine - that literally address the whole subgroup question. Companion diagnostics are routine, rare disease approvals are up, plenty of Huntington trials in progress - that article might as well be about an alien civilization with how different things are today. Value based pricing, redefining cancer into separate genetically defined baubgroups and developing drugs for each, Gene therapy, ... It's no longer easier to get approval of a drug that's marginally successful in a large population, smaller populations with large effects make it way easier to see responses. And the 21st century cures act in 2016 evolved the regulatory landscape even more - it's like comparing Sputnik with the ISS. Yeah, they're both sattelites, but the field has come a long way in the time between the two.

[WalterBright]

Thalidomide was never approved for use in the US, and the heavy regulation came afterwards.

[jokowueu]

It's was never approved because of one person that was continually harrased if it wasn't for her it would have been approved . There were only 11 people responsible to review these new drugs . For crying out loud pcp was approved as an analgesic after using it on a few prisoners . FDA's stringent approval process is required

[WalterBright]

> FDA's stringent approval process is required

The downside of that is greatly reduced new drug development due to the expense, much more expensive drugs, and years of delay for the ones that are worked on. If you have a less popular disease, or can't wait years, you're out of luck.

[vikramkr]

Drug companies can charge whatever they want for less popular diseases. 45% of new approvals last year were for rare diseases. In the industry, rare diseases are sought out as ideal targets because of some regulatory advantages and market power. And there has been an obscene amount of money pouring into biotech and drug development over the past few years - probably one of the most active bull markets the industry has ever seen, with huge numbers of drugs in clinical trials and all sorts of fundamental new technologies under development. Industry's doing fine under the current regulatory regime, with this being one of the best times in history for development of rare disease drugs

[maxerickson]

FDA has a special process and contributes funds to developing drugs for rare diseases:

https://www.fda.gov/about-fda/office-clinical-policy-and-pro...

[ravar]

Avoiding another thalidomide would be all well if weren't for the fact that for many disorders the prognosis provided by standard practice is absolutely terrible.