Unprecedented 100% of First 14 Patients with Cancer Respond to Dostarlimab
For cases like this, <i>exponentially scaled studies</i> should be allowed.<p>Current practice is to enroll 14 participants (and some control participants) and wait many months for the results to roll in, which, if the treatment is effective lets people not on the treatment die unnecessarily.<p>A better approach is to have a  rolling approach.   Ie. Every time a potential participant is found, they are allowed to join the study if the results from all other participants show that it is statistically beneficial, minus a <i>risk budget</i>.<p>So if a treatment does really well like this, then it will quickly expand to a large number of participants.   There is no need to wait for results - as soon as the current cohort of participants survives a few extra days, or as soon as someone in the control group dies, you can enroll a few more participants.<p>And if a treatment is marginal, then the group size will only expand very slowly, or not at all, as more and more data is collected from existing participants.   If the data starts to show a detrimental effect of the same size or larger than the risk budget, then the study can take on no new participants, and if the collected data is not enough to approve the treatment then the result is a failure.