3 ms·

JAMA: 24 of the 37 drugs the FDA approved in 2022 were based on a single study

19 points by hammock 3y ago

duskwuff 3y ago
> Expedited Programs for Serious Conditions: CDER has four broadly applicable programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions: Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval. In 2022, 24 of the 37 of CDER’s novel drug approvals (65%) used one or more of these expedited programs. -- https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022 https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-... I'm not certain if those are the same 24 drugs, but it seems likely -- in which case, this researcher has just empirically established that these fast-track programs exist. :)
icegreentea2 3y ago
Two points. Might be easy to miss, but I think the equally (if not more) concerning result of this study is that "Results were posted for 103 studies (25%) and, in 24 (6%) studies, results were first posted within 6 months after approval.". Now, clearly the FDA has the unposted results to make their decision, but I do agree that it's incredibly concerning that these results are being posted promptly. Second... ugh, I wish they just published their scraped dataset along with this letter. It's only a 413 row spreadsheet. Come on. I know there's a note saying "How to access data: We will provide access to the data on a Stanford University website"... but come onnnnn (I went poking a tiny bit, I couldn't find it yet)
dekhn 3y ago
I had to read the paper to understand that this means each of the 24 drugs was approved by a single study- that is, one drug, one study. Not 24 drugs, one study. Running clinical trials is absurdly expensive and challenging. It's often not possible to run multiple trials.
- duskwuff 3y ago
  Precisely. It's particularly difficult to run multiple trials of drugs for rare diseases -- there's a limited pool of patients available for these studies, and splitting them up between multiple studies makes it much more difficult for each of those studies to reach a statistically significant conclusion.
- jfengel 3y ago
  Would adding more studies even do anything more than one proper study? More data is always better, to be sure, and it would be great to run multiple designs. But one "gold standard" study should suffice, and demanding more just feels like moving the goalposts.
  - dekhn 3y ago
    The largest studies are composed of multiple sites of participants. This has multiple advantages; for example, it makes it easier to recruit a diverse population, reducing trial failures due to population-specific effects. Also, it helps randomize some aspects of treatment, so you don't depend too much on one site's special magic. If you can't recruit a large enough N of patients in one site, you might be able to recruit your N across multiple sites. There are downsides- overhead (each site has its own IRB that must approve the study) and additional complexity (the statistical modelling needs to be carefully managed). It's not uncommon in Cancer trials where there are multiple regional centers that specialize in cancer treatments, who see all the patients with specific types of cancer in a geographical region, and the prevalence of the cancer is high in the population. Also, if you want to change the usage for a drug, IE you were approved for lung cancer but now you want to use it for liver cancer- you might need to run another study. Not multi-site, but just a completely new study with the same medication, but new patients, new protocol (dosage and timing), new site location.
- _Microft 3y ago
  The title made me think that there was one study that included 24 drugs which I found concerning. In the end I have flagged the submission now because of its editorialized and misleading title.

19 points by hammock 3y ago

> Expedited Programs for Serious Conditions: CDER has four broadly applicable programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions: Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval. In 2022, 24 of the 37 of CDER’s novel drug approvals (65%) used one or more of these expedited programs. -- https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022 https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-... I'm not certain if those are the same 24 drugs, but it seems likely -- in which case, this researcher has just empirically established that these fast-track programs exist. :)

icegreentea2 3y ago

Two points. Might be easy to miss, but I think the equally (if not more) concerning result of this study is that "Results were posted for 103 studies (25%) and, in 24 (6%) studies, results were first posted within 6 months after approval.". Now, clearly the FDA has the unposted results to make their decision, but I do agree that it's incredibly concerning that these results are being posted promptly. Second... ugh, I wish they just published their scraped dataset along with this letter. It's only a 413 row spreadsheet. Come on. I know there's a note saying "How to access data: We will provide access to the data on a Stanford University website"... but come onnnnn (I went poking a tiny bit, I couldn't find it yet)

dekhn 3y ago

I had to read the paper to understand that this means each of the 24 drugs was approved by a single study- that is, one drug, one study. Not 24 drugs, one study. Running clinical trials is absurdly expensive and challenging. It's often not possible to run multiple trials.

duskwuff 3y ago
Precisely. It's particularly difficult to run multiple trials of drugs for rare diseases -- there's a limited pool of patients available for these studies, and splitting them up between multiple studies makes it much more difficult for each of those studies to reach a statistically significant conclusion.
jfengel 3y ago
Would adding more studies even do anything more than one proper study? More data is always better, to be sure, and it would be great to run multiple designs. But one "gold standard" study should suffice, and demanding more just feels like moving the goalposts.
- dekhn 3y ago
  The largest studies are composed of multiple sites of participants. This has multiple advantages; for example, it makes it easier to recruit a diverse population, reducing trial failures due to population-specific effects. Also, it helps randomize some aspects of treatment, so you don't depend too much on one site's special magic. If you can't recruit a large enough N of patients in one site, you might be able to recruit your N across multiple sites. There are downsides- overhead (each site has its own IRB that must approve the study) and additional complexity (the statistical modelling needs to be carefully managed). It's not uncommon in Cancer trials where there are multiple regional centers that specialize in cancer treatments, who see all the patients with specific types of cancer in a geographical region, and the prevalence of the cancer is high in the population. Also, if you want to change the usage for a drug, IE you were approved for lung cancer but now you want to use it for liver cancer- you might need to run another study. Not multi-site, but just a completely new study with the same medication, but new patients, new protocol (dosage and timing), new site location.
_Microft 3y ago
The title made me think that there was one study that included 24 drugs which I found concerning. In the end I have flagged the submission now because of its editorialized and misleading title.