3 ms·

Perhaps we need a third level of FDA approval. In addition to 'approved' and 'not approved', we could have 'safe but not demonstrated to be effective'. Drugs in

Perhaps we need a third level of FDA approval. In addition to 'approved' and 'not approved', we could have 'safe but not demonstrated to be effective'. Drugs in the third category would be legal to prescribe but Medicare and insurance companies would not be required to pay for them. Although in practice I guess this would probably not be very different from 'not approved', in which case it seems right for the FDA to reject ineffective drugs. In any case, one thing we definitely do not want is to incentivize the creation of ineffective drugs, especially expensive ones.

dougmwne 5y ago
What on earth would be the point of that? Why should we encourage the development of new snake oil? Why encourage drug companies, which currently must provide evidence their drugs work, to pivot into snake oil? Do you think we are an enlightened people immune from the snake oil our ancestors used to fall for? Recent pandemic events would beg to differ!
- s1artibartfast 5y ago
  I think the point would be "safe but efficacy TBD" and be coupled with extensive monitoring until efficacy is either confirmed or denied. I think this is the general goal of the approval, but our reimbursement system is not set up to handle it because it doesn't take the level of benefit into account. Federal reimbursement is set at what the company wants, even if the product provides better or worse treatment than the alternative.
  - dougmwne 5y ago
    "Safe but Efficacy TBD" is a phase 3 clinical trial. And measuring out the level of benefit vs. the cost to medicare is a totally different issue, I think. At that point, we know it works, and it deserves an approval. I think that's where these laws around publishing prices come in. No individual is going to be able to interpret them, and not even doctors were aware of what their treatments cost. But having the prices published gives advocacy organizations a chance to run the numbers of effect size vs treatment cost and point out areas where the cost is extortionate. It could also be pretty awesome if there was a labeling requirement on all FDA approved drugs for effect size, from a red "not very effective" to a yellow "moderately effective" to green "highly effective".
    - s1artibartfast 5y ago
      >"Safe but Efficacy TBD" is a phase 3 clinical trial. I think you miss the point of the parent post. The point would be to allow drug companies to sell drugs that are reasonably safe, but efficacy is unknown. This would allow drugs and products to come to patients quicker, in a way that is decoupled from reimbursement. >It could also be pretty awesome if there was a labeling requirement on all FDA approved drugs for effect size, from a red "not very effective" to a yellow "moderately effective" to green "highly effective". You are in luck! The FDA has what are literally called "Labeling Requirements" for all approved drugs, which includes a description effect sizes and side effects. You can find the effect sizes by searching "Prescribing information [Drug Name]". It doesn't include a color code, but does have "X% of patients had Y effect, Z% of patients had N effect, ect"
      - dougmwne 5y ago
        I am definitely missing the point of allowing nonfunctional drugs to be sold and advertised as curing anything. What keeps me from selling magic molecule pills and advertising that they are approved for safety and in trials to cure cancer? And even if you disallow advertisements for drugs not yet proven effective, what's to stop my magic molecule pills from going viral and making me a whole lot of money on something I know is extremely unlikely to treat any disease, because I literally put a random inert molecule in there?
        s1artibartfast 5y ago
        The problem that this could address is the FDA is slow and has an all or nothing approach. This means: 1) Timing: There are people who suffer and die while products are held up in lengthy phase 3 approvals. 2) Human Impact: There are drugs& devices which are safe, but have weak efficacy data. For some like aducanumab, if they work, they could help millions of people today. This puts the FDA in a tough spot weighing a big potential benefit to society against things medicare costs for a low/no efficacy drug. Im personally not advocating for throwing open the gates to any snake oil. I think a reasonable proposal would be to require robust data on safety, a strong argument for why it should work, and require monitoring of efficacy after approval. If time tells that your magic pills dont work, they get pulled from the market. If time tells that they do work, you get your gold star The first link in this post explains it better than I can. https://news.ycombinator.com/item?id=28620621 https://news.ycombinator.com/item?id=28620621

dougmwne 5y ago

What on earth would be the point of that? Why should we encourage the development of new snake oil? Why encourage drug companies, which currently must provide evidence their drugs work, to pivot into snake oil? Do you think we are an enlightened people immune from the snake oil our ancestors used to fall for? Recent pandemic events would beg to differ!

s1artibartfast 5y ago
I think the point would be "safe but efficacy TBD" and be coupled with extensive monitoring until efficacy is either confirmed or denied. I think this is the general goal of the approval, but our reimbursement system is not set up to handle it because it doesn't take the level of benefit into account. Federal reimbursement is set at what the company wants, even if the product provides better or worse treatment than the alternative.
- dougmwne 5y ago
  "Safe but Efficacy TBD" is a phase 3 clinical trial. And measuring out the level of benefit vs. the cost to medicare is a totally different issue, I think. At that point, we know it works, and it deserves an approval. I think that's where these laws around publishing prices come in. No individual is going to be able to interpret them, and not even doctors were aware of what their treatments cost. But having the prices published gives advocacy organizations a chance to run the numbers of effect size vs treatment cost and point out areas where the cost is extortionate. It could also be pretty awesome if there was a labeling requirement on all FDA approved drugs for effect size, from a red "not very effective" to a yellow "moderately effective" to green "highly effective".
  - s1artibartfast 5y ago
    >"Safe but Efficacy TBD" is a phase 3 clinical trial. I think you miss the point of the parent post. The point would be to allow drug companies to sell drugs that are reasonably safe, but efficacy is unknown. This would allow drugs and products to come to patients quicker, in a way that is decoupled from reimbursement. >It could also be pretty awesome if there was a labeling requirement on all FDA approved drugs for effect size, from a red "not very effective" to a yellow "moderately effective" to green "highly effective". You are in luck! The FDA has what are literally called "Labeling Requirements" for all approved drugs, which includes a description effect sizes and side effects. You can find the effect sizes by searching "Prescribing information [Drug Name]". It doesn't include a color code, but does have "X% of patients had Y effect, Z% of patients had N effect, ect"
    - dougmwne 5y ago
      I am definitely missing the point of allowing nonfunctional drugs to be sold and advertised as curing anything. What keeps me from selling magic molecule pills and advertising that they are approved for safety and in trials to cure cancer? And even if you disallow advertisements for drugs not yet proven effective, what's to stop my magic molecule pills from going viral and making me a whole lot of money on something I know is extremely unlikely to treat any disease, because I literally put a random inert molecule in there?
      - s1artibartfast 5y ago
        The problem that this could address is the FDA is slow and has an all or nothing approach. This means: 1) Timing: There are people who suffer and die while products are held up in lengthy phase 3 approvals. 2) Human Impact: There are drugs& devices which are safe, but have weak efficacy data. For some like aducanumab, if they work, they could help millions of people today. This puts the FDA in a tough spot weighing a big potential benefit to society against things medicare costs for a low/no efficacy drug. Im personally not advocating for throwing open the gates to any snake oil. I think a reasonable proposal would be to require robust data on safety, a strong argument for why it should work, and require monitoring of efficacy after approval. If time tells that your magic pills dont work, they get pulled from the market. If time tells that they do work, you get your gold star The first link in this post explains it better than I can. https://news.ycombinator.com/item?id=28620621 https://news.ycombinator.com/item?id=28620621