After a long break, I just posted another blog:
Unfortunately, the news is not good, but that’s the thing about research. Some will succeed some will not, and we just need one to succeed…
3 Likes
Phase 1 trials are designed to test the safety of new drugs, so I would not conclude that the treatment doesn’t work. A larger phase 2 study is needed to determine whether it works.
This is a common misconception (that phase-I trials are only for safety), which the FDA encourages with some of the text on their web site. But the truth is that almost all phase-I clinical trials aimed at curing type-1 diabetes collect data on both effectiveness and safety. I know that some web pages talk about phase-I trials being for safety, and maybe they are for other diseases, but for type-1, phase-I trials cover both safety and effectiveness. This one certainly did: the primary outcome was safety and the secondary outcome was effectiveness.
Since the effectiveness results of the trial were unsuccessful, I don’t see that the safety results of the trial matter much. Sure, the safety part was successful, but that does not mean you should ignore that the effectiveness part was unsuccessful.
Joshua
4 Likes
Actually, that’s no misconception at all. Though the borders between the phases may be fuzzy, the primary purpose of phase 1 trials is testing safety and that’s what they’re designed for. Efficacy may be included, but is secondary to that. In phase 1, any firm conclusions about the efficacy, whether positive or negative, are premature.
1 Like