[Music] What is a primary endpoint in a clinical trial? So when you are developing a clinical trial for statistical purity, you have to define the endpoint or the principal question you're looking at, and that's called the primary endpoint. You can only look at so many statistical questions, you can have secondary endpoints, but the primary endpoint is the one you're really focusing on and that's how you determine how many patients you want for the trial. And that really determines a lot about how the trial's set up. Usually for most trials that'll be something like a response rate or overall survival. In the future, primary endpoints may include a percentage of patients who achieve MRD negativity. When we design a trial, we define something called the primary endpoint. The primary endpoint is the single thing that is: what is the purpose of the trial, what is the trial's question. And that primary endpoint in a large part defines the statistics on how we're going to design the trial, how many patients we need on the trial, how long the trial needs to be. And so the primary endpoint is the single most important question on this trial and a large part. The primary endpoint in myeloma is how long do patients stay in remission or maybe in the future it's going to be what percentage of patients achieve already negativity at a certain period of time. Primary endpoint is the end point that you utilize for a clinical from a stats or statistical standpoint to define how many patients you need to answer that question. So you have the question, Okay, we are going to compare daralendex with lendex to prove that there is a higher chance of the three drug combination being better than the truth of the combination in terms of. And then there's a blank and in terms of if we put prime progression free survival, that becomes our primary endpoint or overall survival, that's becomes our primary endpoint. And then we define the sample size according to that question. And the sample size will be different based on the kind of difference you want to see. You know, what kind of improvement you want to see, you know, is between RD and DRD Is it six months, 12 months? So, so based on that, the sample size changes. What is a secondary endpoint? So secondary endpoints are, again, not the primary endpoint but something that would be considered of interest. And so those are all set up beforehand to be statistically valid. Often a primary endpoint is what is the overall survival of my new therapy? How many years? Well, it might also be of interest to know, well, how long is the average person stay in remission? So duration of response may be a secondary endpoint. You may be concerned about the certain safety issues and that may be a secondary endpoint. If a certain you're worried a certain number of patients may get secondary cancers, that may be a secondary endpoint. But again, from a statistical standpoint, you could only do so many of those or you have to keep expanding how many patients are in your trial. So you have to predefined those to be statistically valid. Secondary endpoints which aren't asking the primary question and it's not what the statistics are built on are things like overall survival, how long are patients living, what is the overall toxicity rate of the two different groups? What is the percentage of patients who are able to take full dose of the medications? Those are secondary endpoints that are all important but aren't the single question that the study is asking that are driving how the studies designed. [Music]
HealthTree • June 27, 2023
