13 ms·

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 o

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.

HarryHirsch 7y ago
nor have they historically shown any such willingness You are uncritically repeating libertarian propaganda. The first Google hit for "FDA fast-track" is a press release from FDA for the approval of azidothymidine. Anyone who remembers the early days of the AIDS epidemic will know what kind of a wonder drug zidovudine was. Approval was in 1987; they discovered the HIV virus in 1984. 3 years from early discovery to market. A record, but AIDS was a public health emergency. Please do not repeat lies.
- WalterBright 7y ago
  > AIDS was a public health emergency. I.e. there was enormous political pressure put on the FDA to do something. If you suffer from a rare disease that is not on the 6 o'clock news every day, that's not likely to happen. (I remember those days - everyone was talking about AIDS, everyone was terrified of it.)
  - HarryHirsch 7y ago
    There is much more: https://www.fda.gov/drugs/nda-and-bla-approvals/fast-track-approvals https://www.fda.gov/drugs/nda-and-bla-approvals/fast-track-a... Just last year: triclabendazole for liver flukes. That's emphatically not political. From 2018: apalutamide, an androgen receptor antagonist. Prostate cancer doesn't nearly get the attention it deserves, not political but sorely needed. 2017: secnidazole, for bacterial vaginosis. Strange to see that there, given that there are sufficient nitroimidazoles at market already. No political pressure. Please, no reflexive regurgitation of libertarian propaganda directed against the FDA. They actually do their job.
vikramkr 7y ago
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 7y ago
  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=240 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/A43257-2003Nov14.html http://www.washingtonpost.com/wp-dyn/articles/A43257-2003Nov...

HarryHirsch 7y ago

nor have they historically shown any such willingness You are uncritically repeating libertarian propaganda. The first Google hit for "FDA fast-track" is a press release from FDA for the approval of azidothymidine. Anyone who remembers the early days of the AIDS epidemic will know what kind of a wonder drug zidovudine was. Approval was in 1987; they discovered the HIV virus in 1984. 3 years from early discovery to market. A record, but AIDS was a public health emergency. Please do not repeat lies.

WalterBright 7y ago
> AIDS was a public health emergency. I.e. there was enormous political pressure put on the FDA to do something. If you suffer from a rare disease that is not on the 6 o'clock news every day, that's not likely to happen. (I remember those days - everyone was talking about AIDS, everyone was terrified of it.)
- HarryHirsch 7y ago
  There is much more: https://www.fda.gov/drugs/nda-and-bla-approvals/fast-track-approvals https://www.fda.gov/drugs/nda-and-bla-approvals/fast-track-a... Just last year: triclabendazole for liver flukes. That's emphatically not political. From 2018: apalutamide, an androgen receptor antagonist. Prostate cancer doesn't nearly get the attention it deserves, not political but sorely needed. 2017: secnidazole, for bacterial vaginosis. Strange to see that there, given that there are sufficient nitroimidazoles at market already. No political pressure. Please, no reflexive regurgitation of libertarian propaganda directed against the FDA. They actually do their job.

vikramkr 7y ago

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 7y ago
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=240 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/A43257-2003Nov14.html http://www.washingtonpost.com/wp-dyn/articles/A43257-2003Nov...