3 ms·

Other points not covered. We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the mod

Other points not covered. We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. The problem of tainted and ineffective generics confounds the analysis. A recent investigation shows the fda's proceedure of unannounced inspections only in the us means that 75 percent of indian and Chinese generics makers failed inspections during the few years when unannounced inspections were done. Now they have moved back to announcing. It is not sure that each failure meant there are bad drugs out there, but a majority of failures were 100 percent faked test data regarding the chemicals inside the drugs.

eitally 7y ago
Can you point me to reference material supporting those numbers? I'd like to read more detail (and share with my partner, who works in pharma).
klipklop 7y ago
As somebody that benefits from using a drug 'off-label' I strongly disagree. There are many cases where off-label drug use gives a minority of people tremendous benefit. Take a look at the people with various brain disorders using Ambien off-label. It can literally bring people out of coma-like states temporarily. Other examples off-label use: * Propranolol for anxiety. * Guanfacine for blood pressure issues, now used on label for ADHD.
- DanBC 7y ago
  > Propranolol for anxiety. Is that off-label? https://bnf.nice.org.uk/drug/propranolol-hydrochloride.html https://bnf.nice.org.uk/drug/propranolol-hydrochloride.html
  - awalton 7y ago
    As far as I'm aware there's no beta blocker that's approved for performance anxiety in the US. Paperwork for Propranolol in the US has on-label usages listed as: Hypertension, Angina Pectoris, Atrial Fibrillation, Myocardial Infarction, Migraine, Essential Tremor, Hypertrophic Subaortic Stenosis or Pheochromocytosis.
maxxxxx 7y ago
I work in medical devices and without off label use by doctors it would be really hard and expensive to innovate and explore new indications. Maybe there should be some registry and published information but I don’t see an alternative.
klodolph 7y ago
> We need to end off label use. How would you propose doing that? Under the current system, we would simply be redirecting people to on-label prescriptions, which are higher priced but not better. > Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. That's not what off-label is. Off-label just means that it's not an approved indication for the drug. The approved indications are what the drug companies are allowed to market / recommend. > One might as well not have fda approval. This is a fairly absurd comment to make, so I'm not even sure how to respond to it. Surely you are already aware that the FDA approval process requires studying the safety, contraindications, etc for a medication, and that these do not need to be redone from scratch for new applications? Off-label use is just cutting through a small bit of regulatory box-ticking, box-ticking that doesn't add much value. Off-label use is common and standard of care often involves off-label use. There are simply not enough resources to research the combination of every drug with every indication, not enough to provide the level of care that we can provide with off-label use.
dillondoyle 7y ago
Disagree. Yes, I am more libertarian here where I think anyone should have choice to consume ANY drug legal or not. But even from a more mainstream viewpoint, there are lots of examples of for-profit medicine where a drug company pays for an on-label use via a study where off-label use is cheaper and similarly effective. For example Ketamine and Esketamine. One is very cheap and widely available one is very expensive and not widely available. I hope we can address the fundamental problems with for profit medicine, I think this is just one negative externality of our crazy system.
apsec112 7y ago
For less common diseases, sometimes all medications are off-label, because the market is small enough that nobody will fund studies to get a new indication approved. There are approximately 100,000 human diseases known to science. Who is going to pay for new approvals (costing tens of millions each) for every single one? Should every patient with one of these diseases be yanked off their existing medication indefinitely, even if it works for them?
wl 7y ago
> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. The approval process itself is what's crazy. Drugs are approved for treating disease X, but drugs don't work that way. They are chemicals that modify processes in the body to produce collections of effects that help treat some medical issue. What does prednisone treat? It's an immunosuppressant, an anti-inflamatory, and it reduces blood calcium levels. That's useful in treating a range of conditions like heart failure, cancer, autoimmune diseases, and many, many others. One example of the insanity of this regime is the HPV vaccine. What does the drug do? It helps the immune system fight off certain strands of HPV. What was it approved for? Preventing cervical cancer. Since men don't have cervixes, it wasn't FDA-approved for men and any administration of that vaccine was strictly off-label. It took an additional approval and many years to get it approved for men, even though there was no reason to believe the vaccine would work in women but not in men.
DanBC 7y ago
> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. Everyday in England 30,000 people with learning disability take a psychotropic medication when they don't have the condition that the medication is licensed for. NHS England has a programme to stop this misuse of medication. It's called STOMP - STop Over Medicating People with LD. https://www.england.nhs.uk/learning-disabilities/improving-health/stomp/ https://www.england.nhs.uk/learning-disabilities/improving-h... This medication is often off-label use of anti-psychotic medication, used for a "sedative" effect. People with learning disability are at increased risk of constipation (sensory issues around food; reduced range of food being eaten; not much say about what they eat; reduced access to exercise; etc etc), and these meds often have constipation as a side effect. This combination causes harm. It sometimes kills people. There's variable awareness of constipation as a side effect of clozapine. Most doctors know about agranularcytosis, and they're aware constipation can be a problem. But there are some doctors who don't know that the constipation side effect kills more people than agranularcytosis. I agree. It's really important to stop misuse of medication.
awalton 7y ago
> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. This is patently ridiculous. The FDA just proved the drug was safe in therapeutic doses and that it treated what it said it treated. They didn't say that the drug wasn't safe to treat other things, and they didn't say the drug was generally unsafe. If doctors are prescribing non-therapeutic doses, yeah, that might be a problem, but there are entire clades of diseases that would otherwise be untreatable without off-label use. (And the frank alternative - labeling all of the drugs as being usable for all of the various conditions - can't keep up with reality. The FDA is slow and meticulous, and if we forced that on off-label used drugs, there'd be a lot more suffering for literally no good reason. And it probably wouldn't do anything for actually stopping off-label usage - doctors would just start lying and overclassifying their patients as having diseases to give them the meds. E.g. there'd be a whole lot of autoimmune patients with various "overlap syndromes" that suddenly have lupus and/or rheumatoid arthritis, despite not meeting the current diagnostic criteria, just so they could keep taking adalimumab, methotrexate and/or hydroxychloroquine. It's just red tape they don't need nor care about.) We've done actual wonders in the world by simply scanning through approved drug combinations to find treatments for ailments. An example of this is in cancer treatment: it's fairly common one cancer drug will treat multiple classes of cancer, especially in combinations with other drugs (even ones not designed to fight cancer specifically). It's so commonly the case that this is now its own entire enterprise: high throughput scanning of drug combinations to fight tumors. https://www.biorxiv.org/content/10.1101/051698v1.full https://www.biorxiv.org/content/10.1101/051698v1.full It's also been done to treat viruses and autoimmune diseases. I'm not a super libertarian or anything, but I believe if a doctor has a reasonable belief that a drug will treat a condition, and therapeutic trial evidence bears it out, there's not a good reason not to prescribe it, whether or not it says on the bottle that that's okay. I've benefited from this in the past and I will continue to in the future.

eitally 7y ago

Can you point me to reference material supporting those numbers? I'd like to read more detail (and share with my partner, who works in pharma).

klipklop 7y ago

As somebody that benefits from using a drug 'off-label' I strongly disagree. There are many cases where off-label drug use gives a minority of people tremendous benefit. Take a look at the people with various brain disorders using Ambien off-label. It can literally bring people out of coma-like states temporarily. Other examples off-label use: * Propranolol for anxiety. * Guanfacine for blood pressure issues, now used on label for ADHD.

DanBC 7y ago
> Propranolol for anxiety. Is that off-label? https://bnf.nice.org.uk/drug/propranolol-hydrochloride.html https://bnf.nice.org.uk/drug/propranolol-hydrochloride.html
- awalton 7y ago
  As far as I'm aware there's no beta blocker that's approved for performance anxiety in the US. Paperwork for Propranolol in the US has on-label usages listed as: Hypertension, Angina Pectoris, Atrial Fibrillation, Myocardial Infarction, Migraine, Essential Tremor, Hypertrophic Subaortic Stenosis or Pheochromocytosis.

maxxxxx 7y ago

I work in medical devices and without off label use by doctors it would be really hard and expensive to innovate and explore new indications. Maybe there should be some registry and published information but I don’t see an alternative.

klodolph 7y ago

> We need to end off label use. How would you propose doing that? Under the current system, we would simply be redirecting people to on-label prescriptions, which are higher priced but not better. > Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. That's not what off-label is. Off-label just means that it's not an approved indication for the drug. The approved indications are what the drug companies are allowed to market / recommend. > One might as well not have fda approval. This is a fairly absurd comment to make, so I'm not even sure how to respond to it. Surely you are already aware that the FDA approval process requires studying the safety, contraindications, etc for a medication, and that these do not need to be redone from scratch for new applications? Off-label use is just cutting through a small bit of regulatory box-ticking, box-ticking that doesn't add much value. Off-label use is common and standard of care often involves off-label use. There are simply not enough resources to research the combination of every drug with every indication, not enough to provide the level of care that we can provide with off-label use.

dillondoyle 7y ago

Disagree. Yes, I am more libertarian here where I think anyone should have choice to consume ANY drug legal or not. But even from a more mainstream viewpoint, there are lots of examples of for-profit medicine where a drug company pays for an on-label use via a study where off-label use is cheaper and similarly effective. For example Ketamine and Esketamine. One is very cheap and widely available one is very expensive and not widely available. I hope we can address the fundamental problems with for profit medicine, I think this is just one negative externality of our crazy system.

apsec112 7y ago

For less common diseases, sometimes all medications are off-label, because the market is small enough that nobody will fund studies to get a new indication approved. There are approximately 100,000 human diseases known to science. Who is going to pay for new approvals (costing tens of millions each) for every single one? Should every patient with one of these diseases be yanked off their existing medication indefinitely, even if it works for them?

wl 7y ago

> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. The approval process itself is what's crazy. Drugs are approved for treating disease X, but drugs don't work that way. They are chemicals that modify processes in the body to produce collections of effects that help treat some medical issue. What does prednisone treat? It's an immunosuppressant, an anti-inflamatory, and it reduces blood calcium levels. That's useful in treating a range of conditions like heart failure, cancer, autoimmune diseases, and many, many others. One example of the insanity of this regime is the HPV vaccine. What does the drug do? It helps the immune system fight off certain strands of HPV. What was it approved for? Preventing cervical cancer. Since men don't have cervixes, it wasn't FDA-approved for men and any administration of that vaccine was strictly off-label. It took an additional approval and many years to get it approved for men, even though there was no reason to believe the vaccine would work in women but not in men.

DanBC 7y ago

> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. Everyday in England 30,000 people with learning disability take a psychotropic medication when they don't have the condition that the medication is licensed for. NHS England has a programme to stop this misuse of medication. It's called STOMP - STop Over Medicating People with LD. https://www.england.nhs.uk/learning-disabilities/improving-health/stomp/ https://www.england.nhs.uk/learning-disabilities/improving-h... This medication is often off-label use of anti-psychotic medication, used for a "sedative" effect. People with learning disability are at increased risk of constipation (sensory issues around food; reduced range of food being eaten; not much say about what they eat; reduced access to exercise; etc etc), and these meds often have constipation as a side effect. This combination causes harm. It sometimes kills people. There's variable awareness of constipation as a side effect of clozapine. Most doctors know about agranularcytosis, and they're aware constipation can be a problem. But there are some doctors who don't know that the constipation side effect kills more people than agranularcytosis. I agree. It's really important to stop misuse of medication.

awalton 7y ago

> We need to end off label use. Saying a drug is approved for one diagnosis and then allowing it for any other diagnosis is, in the modern age, simply crazy. One might as well not have fda approval. This is patently ridiculous. The FDA just proved the drug was safe in therapeutic doses and that it treated what it said it treated. They didn't say that the drug wasn't safe to treat other things, and they didn't say the drug was generally unsafe. If doctors are prescribing non-therapeutic doses, yeah, that might be a problem, but there are entire clades of diseases that would otherwise be untreatable without off-label use. (And the frank alternative - labeling all of the drugs as being usable for all of the various conditions - can't keep up with reality. The FDA is slow and meticulous, and if we forced that on off-label used drugs, there'd be a lot more suffering for literally no good reason. And it probably wouldn't do anything for actually stopping off-label usage - doctors would just start lying and overclassifying their patients as having diseases to give them the meds. E.g. there'd be a whole lot of autoimmune patients with various "overlap syndromes" that suddenly have lupus and/or rheumatoid arthritis, despite not meeting the current diagnostic criteria, just so they could keep taking adalimumab, methotrexate and/or hydroxychloroquine. It's just red tape they don't need nor care about.) We've done actual wonders in the world by simply scanning through approved drug combinations to find treatments for ailments. An example of this is in cancer treatment: it's fairly common one cancer drug will treat multiple classes of cancer, especially in combinations with other drugs (even ones not designed to fight cancer specifically). It's so commonly the case that this is now its own entire enterprise: high throughput scanning of drug combinations to fight tumors. https://www.biorxiv.org/content/10.1101/051698v1.full https://www.biorxiv.org/content/10.1101/051698v1.full It's also been done to treat viruses and autoimmune diseases. I'm not a super libertarian or anything, but I believe if a doctor has a reasonable belief that a drug will treat a condition, and therapeutic trial evidence bears it out, there's not a good reason not to prescribe it, whether or not it says on the bottle that that's okay. I've benefited from this in the past and I will continue to in the future.