3 ms·
The big hope for people who suffer from ME/CFS is an off-label use of an existing drug such as Rituximab. Under the current system, drugs go through phase I tr
by alan-crowe 8y ago
The big hope for people who suffer from ME/CFS is an off-label use of an existing drug such as Rituximab.
Under the current system, drugs go through phase I trials to make sure that they are safe enough for doctors to prescribe. Then they go through phase II trials to get a feel for dosage and effect size for the intended indication. Then they fail at phase III because they compare poorly with an existing drug for that indication.
What determines whether ME/CFS patients get to try a drug, to see if it does anything for them? Phase I and II results. Fair enough.
But also Phase III. So the efficacy of the drug for a different indication plays a positive roll. If Rituximab were ineffective for leukemia, it would be forbidden to explore it for ME/CFS in a way that might later justify a big trial to check for efficacy. Given the side-effects of Rituximab, that is fair enough, but the law is equally restrictive on all that failed in Phase III. So drugs with mild side-effects that did nothing for the intended indication cannot be explored for ME/CFS.
That is sad. Those who believe that ME/CFS is due to a viral infection would love to try a failed anti-viral. Those who believe that ME/CFS is an auto-immune disease would love to try a failed treatment for Irritable Bowel Syndrome. Meanwhile, researchers tend to stay away from diseases with such unclear etiology.
But the efficacy requirement has worse implications. As wikipedia puts it
> Phase III studies are randomized controlled multicenter trials on large patient groups (300–3,000 or more depending upon the disease/medical condition studied) and are aimed at being the definitive assessment of how effective the drug is, in comparison with current 'gold standard' treatment.
So a drug that passes Phase I and Phase II can be eligible or ineligible for off label exploration because of the efficacy of a different drug for a different indication.