Thanks Pyrr for sharing another perspective to the data on DCVax-Direct that was presented.
A few additional considerations ... you probably already considered but not highlighted in your post:
1. Stable Disease also implies that the primary tumor has decreased up to 30% in size. Partial Response requires a >30% response.
2. Based on the 5-Year survival rate % for each cancer that I shared a few days back, I find it highly improbable that the clinical trial would've recruited a number of "Kris Carr". It is possible Alan Butler was one of these cancer survivors, but I don't think it is applicable to the entire group. Even with selective criteria, the probability of being able to find "Kris Carr" similars is non-existent.
3. I fully agree on the failure of future PIII trials that you've stated in your write up below. As this was Phase I, I appreciate the fact that the company used this opportunity to understand a bit more about their product AND variables that they can influence for the next trial. Yes, the trial was selective, but it might have been done to ensure they could reasonably separate groups of folks to understand the impact of Method A vs Method B, different dosage schedules, and different dosage volume.
Now that they have collected more information about DCVax-Direct, the Phase II trial can be more targeted and leverage to gain more information than a Phase III.
Though for a successful PIII trial, I do hope the company definitely considers some of the points you've made below.
Additional Questions:
1. With regards to your suggestion about measuring effectiveness of the DCVax-Direct treatment by providing a detailed analysis of each patient's life expectancy, would the information be accurate enough to share? I don't know how I feel about providing life expectancy numbers considering all of these patients have gone thru a number of different treatments (average 3) ... thus confounding any kind of life expectancy estimates. In addition, life expectancy estimate vary so greatly considering the point they joined the trial that not sure even if the numbers shared would be accurate. Let me know if you share the same perspective.
I agree that what is necessary is the need for a controlled group, but that is something that will happen in Phase II and PIII
2. To clarify on the DCVax-L modeling, based on the German paper of only 49 randomized patients, is the new suggestion that the most recent Trial re-sizing due to an over performing control group rather than an over performing treatment group?
3. For trial participants, even though only survival is mentioned wouldn't that be enough considering how sick these patients are. The stage IV pancreatic cancer mOS = 3.9. From the DCVax-Direct trial's 7 patients, the mOS = ~7.5. I know n = 7, but I mean that would be the most apples to apples comparison, I believe. Unfortunately, I am not a Bio Stats guy so I will defer to you in this. I know the above result is not significant as the sample size is too small.
But the question is, do you believe with all of the information gathered about DCVax-Direct (dosage, volume, activation, etc) and the 3 Phase II trials determined that future trials have a higher probability of success?
