The Effect of Trial Design on Approval of GVHD Therapies


Panelists consider the importance of studying trial drugs in a blinded, randomized fashion to mitigate physician and patient bias regarding patient-reported outcomes for GVHD therapies.


John F. DiPersio, MD, PhD: I just want to throw in 1 comment that we have 3 drugs approved for chronic GVHD [graft-vs-host disease]: ibrutinib, Rezurock, and ruxolitinib. The first 2 drugs were approved with a single-arm open-label design in small studies. The only study where there was a randomized trial was the ruxolitinib study. It’s so hard to measure responses, as Yi-Bin was saying. Sometimes the responses are meaningful, but many times they aren’t meaningful and they’re hard to measure. For chronic GVHD approval in the future, drugs should be tested in a randomized fashion. The ruxolitinib study was against the standard-of-care, best-available therapy, but it’s more important that it's done.

If you want to know if a drug is effective, you’ve got to do it in a randomized prospective trial that’s blinded. Blinded to not only the patient but also the physician. I come into the room when I put somebody on a new drug, and I say, “How are you feeling?” They say, “Ugh.” I say, “You look a little better.” They say, “I guess I am a little better.” There’s a lot of physician and patient bias when they start a drug. It’s very important. I’d like to know for sure that they work, but I don’t know if they work and how well they work because they were never tested in a randomized fashion. That’s my point. I guess the only drug that passed that low bar is ruxolitinib and even though it was an open-label study, it was a randomized trial.

Pashna N. Munshi, MD: I agree, John. Even at the recent ASH [American Society of Hematology] Annual Meeting, Dr Stephanie Lee presented patient-reported outcomes from the REACH3 trial, showing that there was improved quality of life.

John F. DiPersio, MD, PhD: Quality of life is absolutely key as well. The patient has to be blinded. If you come in and you say, “How is the new drug going?” and they say, “I guess I’m a little better. I think I’m a little better,” then you know exactly what I’m talking about. There’s confusion from both the physician side and the patient side.

Transcript edited for clarity.

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