| > One doesn't need elaborate phase 3 RCTs to figure out if there's a good shot that a treatment is working....The existing system raises costs and causes people to die while waiting a decade or more for exciting treatments The FDA often approves cancer drugs without a phase 3 randomized trial. In fact, most new cancer drugs are approved without a phase 3 trial. Just taking a random cancer drug from this list: https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-dru... "The efficacy of IMDELLTRA was evaluated in Study DeLLphi-301 [NCT05060016], an
open-label, multicenter, multi-cohort clinical trial....A total of 99 patients received IMDELLTRA..." This is a new small cell lung cancer drug approved via a phase 2 study that didn't have a control arm and wasn't blinded. This is pretty typical. > one can see it in tumor response and comparison to known KMCs. Anything measured by a human can be biased by knowledge that a patient received a treatment, including tumor response (often blobs on a screen from a FDG PET/CT scan.) RCTs are the gold standard. We don't need to start chipping away at the rigorous standards we have in place to accurately measure the value that a medicine offers. What we can do - and are doing right now - is do a risk-benefit analysis and allow drugs to be approved with a weaker set of data so that patients with a life-threatening illness can get access earlier. |