Soft Tissue Sarcoma Update, Issue 1, 2017 (Video Program)FDA approval of trabectedin for patients with unresectable or metastatic liposarcoma or leiomyosarcoma who experience disease progression after receiving an anthracycline regimen
2:53 minutes.
TRANSCRIPTION:
DR LOVE: Anything else you want to say about trabectedin and also the clinical research that led to it being approved? DR POLLACK: Yes. I mean, trabectedin has been around for a long time. It’s been really frustrating that it hasn’t been available in the US sooner. DR LOVE: It’s been available in Europe for a while, right? DR POLLACK: It was available in Europe for over a decade. And I’m not really well versed in the politics that led to it not being approved in the States. But the trial that was relatively recently published by George Demetri and colleagues showed a progression-free survival benefit of 4.2 months versus 1.5 months. And that was highly statistically significant, and that’s the basis for approval. That trial actually didn’t see an overall survival benefit, but I have no doubt that in patients like this, they’re living longer because of trabectedin. DR LOVE: And I guess also, what’s the difference in approval in the United States and Europe? DR POLLACK: I think things have changed. I think that for a long time — this is just me looking at it. I’m not an expert on the regulatory process or things like that. But sarcoma is a rare disease. And it’s really frustrating if you try to hold it to the same trial standards that you hold diseases where it’s really easy to accrue trials that have hundreds of patients on each arm. It’s really tough in sarcoma to have that kind of big trial. It takes a real national effort to do a trial like the trabectedin trial that George Demetri or any of these big randomized trials. It takes a real concerted effort by the community. For a really long time there was nothing approved for sarcoma. Then pazopanib was actually the thing that changed that. Pazopanib was tested in a randomized trial of multiple different sarcoma subtypes. That trial actually excluded liposarcoma because of some Phase II data. And they saw a progression-free survival benefit without an overall survival benefit. And I think because there was so much uproar in the sarcoma community because trabectedin hadn’t been approved, I think the FDA took a closer look at approving it based on this progression-free survival benefit, really with the thought that, geez. These patients don’t have anything that we can offer to them. And this drug really seems to be working, at least in terms of stabilizing patients’ disease for a period of time. And that led to the approval. And I think that’s kind of opened the doors to approve based on a PFS benefit, without an OS benefit, for drugs like trabectedin. |