5 ms·

One of the big impediments against more companies engaging in compassionate use is required adverse event reporting to the FDA. To my knowledge (clinicians / cl

One of the big impediments against more companies engaging in compassionate use is required adverse event reporting to the FDA. To my knowledge (clinicians / clinical research specialists please correct me here if I'm wrong) companies are required to report any adverse events experienced by patients granted early access to a drug under compassionate use. Any adverse event reported may make it harder for them to bring a drug to market. In addition, it is very difficult for companies to screen these patients or guarantee that they are adhering to regimens as they would in a clinical trial. This biases companies against providing the drugs, even though many would want to. In my opinion, the FDA should drop this requirement and allow for universal access to medications for terminal patients under compassionate use.

JoshTriplett 11y ago
As I understand it, when you hear a long list of possible side effects ("nausea, vomiting, upset stomach..."), that's derived from those same trials; if a thousand people try it, and two report those symptoms, they go on the list. (I don't know the actual threshold below which you can discount it as unrelated, but I'd be surprised if it was the same as a threshold for statistical significance, and I'd be somewhat surprised if there's any effort to seek causation.)
- DanBC 11y ago
  Patient information leaflets in the UK classify side-effects by frequency reported in clinical trials and, I think, yellow card reports. "Very common" affects 1 user in 10 "Common" affects 1 to 10 users in 100 "Uncommon" affects 1 to 10 users in 1,000 "Rare" affects 1 to 10 users in 10,000 "Very rare" affects less than 1 user in 10,000 "Not known" cannot be estimated from available data. This says nothing about the severity of the side effect.
DanBC 11y ago
What's the advantage of stopping reporting of adverse events? Doesn't that just make it easier for dangerous / ineffective (but fantastically expensive) drugs to come to market?
- Thriptic 11y ago
  In very sick patients (those generally filing for compassionate use) it's sometimes difficult to pin down whether the drug actually caused a problem or death. Were the patients properly adherent? Did they have comorbidities? Were they simply too sick and died? A few deaths might cause the FDA to order additional trials which may actually not be necessary, to the tune of millions of dollars. If you were a company that spent years and hundreds of millions trying to bring a product to market, would you want to take that risk?
- Thriptic 11y ago
  In addition (adding on to my comment below), companies don't want the FDA to try to use the outcomes of patients taking the drug for compassionate use as a metric of efficacy before a proper phase 2 or 3 clinical trial has been run, for the exact same reasons.

JoshTriplett 11y ago

As I understand it, when you hear a long list of possible side effects ("nausea, vomiting, upset stomach..."), that's derived from those same trials; if a thousand people try it, and two report those symptoms, they go on the list. (I don't know the actual threshold below which you can discount it as unrelated, but I'd be surprised if it was the same as a threshold for statistical significance, and I'd be somewhat surprised if there's any effort to seek causation.)

DanBC 11y ago
Patient information leaflets in the UK classify side-effects by frequency reported in clinical trials and, I think, yellow card reports. "Very common" affects 1 user in 10 "Common" affects 1 to 10 users in 100 "Uncommon" affects 1 to 10 users in 1,000 "Rare" affects 1 to 10 users in 10,000 "Very rare" affects less than 1 user in 10,000 "Not known" cannot be estimated from available data. This says nothing about the severity of the side effect.

DanBC 11y ago

What's the advantage of stopping reporting of adverse events? Doesn't that just make it easier for dangerous / ineffective (but fantastically expensive) drugs to come to market?

Thriptic 11y ago
In very sick patients (those generally filing for compassionate use) it's sometimes difficult to pin down whether the drug actually caused a problem or death. Were the patients properly adherent? Did they have comorbidities? Were they simply too sick and died? A few deaths might cause the FDA to order additional trials which may actually not be necessary, to the tune of millions of dollars. If you were a company that spent years and hundreds of millions trying to bring a product to market, would you want to take that risk?
Thriptic 11y ago
In addition (adding on to my comment below), companies don't want the FDA to try to use the outcomes of patients taking the drug for compassionate use as a metric of efficacy before a proper phase 2 or 3 clinical trial has been run, for the exact same reasons.