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Both patient participation in clinical trials and compassionate use of experimental treatments are fairly common for cancer patients, with various accessibility barriers. (One issue with the latter, for example, is that the incentives aren't lined up for companies to provide unapproved drugs to dying patients, you're way more likely to get a horrible complication that leads to bad press than a miraculous recovery).

Here's an insightful blog series about Jake Seliger's experience participating in clinical trials. He was a regular HackerNews user who passed away in 2024: https://bessstillman.substack.com/p/please-be-dying-but-not-...

by amelius4 hours ago|

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What is the success rate of a clinical trial? Just to see things in perspective.

by throwup2384 hours ago|

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It's around 10-15% for the whole drug I-III flow (13.8% according to [1]), but that varies dramatically based on therapeutic area. On the order of a third of infectious disease vaccines might be approved but only maybe 5% of oncology therapies because the latter often have a different standard for approval so it's cheaper to run trials.

[1] https://pmc.ncbi.nlm.nih.gov/articles/PMC6409418/

by amelius3 hours ago|

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That's interesting, but I was talking about the success rate of someone with a terminal illness going the clinical trial route. Sorry, I now see that my question was not so precise.

by throwup2383 hours ago|

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For cancer, it doesn't seem to impact survival odds at all [1]. In other fields it may improve metrics a small bit but that's largely because in clinical trial patient selection, they're very careful to exclude anyone with an even remotely confounding factor (like weight/BMI).

[1] https://www.science.org/content/article/joining-cancer-trial...

by tyre6 hours ago|

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In the US, the FDA has a Compassionate Use exemption to clinical trials for exactly this circumstance!

There must be informed consent, no reasonable alternatives (which, in cases we deem terminal, is often the case), and some evidence pointing to the treatment possibly being helpful. It's an excellent ethical program that gives patients a choice and advances science.

by throwup2386 hours ago|

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In my experience most legitimate biotech companies working on promising drugs and therapies don’t want to touch the exemption with a 30 foot pole. Since they raise most of their money from the public to fund clinical trials, a single bad reaction could generate enough bad PR to derail fundraising and kill the drug. Sticking to clinical trials allows them to control that blast radius so even though the FDA approves >95% of applications, in practice very few drugs are available that way.

The biggest exception is oncology. Since everyone knows that chemotherapy is hell, cancer drugs tend to get a pass and pre-approval companies are (slightly) more willing to work with compassionate use exemptions.

by contingencies4 hours ago|

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Both of my parents have benefited from access to early medical trials. One is currently very late stage IV cancer. Access to trials is usually proxied through respected doctors/oncologists affiliated with major hospitals rather than offered broadly. I assume for reasons of experimental protocol and integrity the overseeing doctors are typically not the same as the conceiving research team.