I haven't posted for a while but have been skimming posts. I was thinking about the recent FDA comments about using external controls for approvals. As much as I dislike the persistently and unjustifiably negative comments of some posters, I do have to admit that I also have some concerns about the FDA saying patient-level data are needed for approvals based on ECAs. What NWBO did in their JAMA article in essence reproduced patient-level data as best they could from the group level data reported in prior publications. This is not true patient-level data and still could be biased despite the statisticians many efforts to fight against possible bias. The fact that JAMA reviewers thought this was good enough for publication gives me at least some hope for FDA approval despite the recently published FDA comments. The fact that GBM is an FDA-designated orphan condition with no new interventions in years, that trials for orphan conditions have more lenient rules for approval, that DCVAX has a very good safety profile (so relatively little benefit has to be shown for a cost:benefit analysis to favor approval), and that the FDA Orphan Condition Office should have an incentive to try to find a way to approve DCVAX for GBM also all give me hope for eventual approval. That said, I wonder if the company has been trying to obtain patient-level data from prior trials to use in their approval package? Might this have contributed to how long it has taken them to get the MAA submitted? There has been a move in recent years both here and in Europe to make full trial datasets publicly available to qualified researchers (NIH now mandates this for NIH funded studies). Here are some ways that I think NWBO might obtain patient-level data from prior trials: 1) simply request it from the Principal Investigators of prior trials (if these trials received NIH funding, they might be inclined to comply), 2) If anyone who is a Co-Investigator on the DCVAX GBM trial or who is an NWBO consultant worked as an investigator on prior trials, maybe they could facilitate obtaining data from prior GBM trials, 3) If any prior trials were run by companies that NWBO is now pairing with for combo trials (and potentially considering partnership), maybe these companies would make their prior trial data available. I hope that NWBO has at least made an effort to obtain patient-level data because I think this would greatly facilitate FDA approval chances. Without this, I think the best we might hope for is a conditional DCVAX approval by the FDA with some requirement of an additional phase 4 study. I am long and hopeful....
