3 ms·

I'm in medical and one major issue with Compassionate Use programs [1] is there often just isn't the supply yet to divert doses/devices to non-trial patients. B

I'm in medical and one major issue with Compassionate Use programs [1] is there often just isn't the supply yet to divert doses/devices to non-trial patients. By definition they are pre-release treatments, which means manufacturing is still ramping up and is often only available in small quantities. We would love to be able to provide access to our device to everyone (its quite promising), but we are barely able to meet demand for the clinical trial. With those restrictions, the call was made that we had to end compassionate use treatments for the foreseeable future - with limited supply they had to be devoted solely to the clinical trial, which is designed to give the maximum possible data about efficacy, not just anyone it might help. 1) https://www.fda.gov/news-events/public-health-focus/expanded-access https://www.fda.gov/news-events/public-health-focus/expanded... - The FDA is actually pretty good about allowing these treatments

hinkley 3y ago
Sounds a lot like Mythical Man Month territory as well. More patients means more techs and more doctors to offer advice/test for side effects, and you can’t just wave a wand and get those. You have to invest in people to roll out a product.
- civilitty 3y ago
  Exactly. Patient selection in clinical trials is already notoriously difficult even with phase I trials where it's a few dozen healthy people at most. Finding the right patients so your control and test groups don't have a lot of confounding factors is a field unto itself. Doing so with terminal patients, who often have a lot of things going on including just the psychology of dying, is even more so. Almost everyone hires out to contract research organizations that have entire departments dedicated to the process with years long relationships with major hospitals and patient groups that serve as "sales funnels" for their clinical trials.
spoonfeeder006 3y ago
Could they increase supply in anticipation of dying people wanting to use them? Might be harder to estimate than a normally released product though

hinkley 3y ago

Sounds a lot like Mythical Man Month territory as well. More patients means more techs and more doctors to offer advice/test for side effects, and you can’t just wave a wand and get those. You have to invest in people to roll out a product.

civilitty 3y ago
Exactly. Patient selection in clinical trials is already notoriously difficult even with phase I trials where it's a few dozen healthy people at most. Finding the right patients so your control and test groups don't have a lot of confounding factors is a field unto itself. Doing so with terminal patients, who often have a lot of things going on including just the psychology of dying, is even more so. Almost everyone hires out to contract research organizations that have entire departments dedicated to the process with years long relationships with major hospitals and patient groups that serve as "sales funnels" for their clinical trials.

spoonfeeder006 3y ago

Could they increase supply in anticipation of dying people wanting to use them? Might be harder to estimate than a normally released product though