banter and General Discussion, page-8267

Hi @irenekwshiu,

thanks for your interest & the link. That's a good spotlight session - Dr Verdun seemed to me at pains to get straight into it, and I was impressed that she was willing to start by admitting the FDA's inefficiencies and OTP's understaffing right off the top.

On Operation 'Warp Speed for Rare Diseases', Dr Verdun appeared really enthused. Of course, RYONCIL has Orphan drug designation already, & Orphan drugs are intended for the treatment, prevention, or diagnosis of a rare disease or condition (by definition). So, this seeming change of 'heart' from FDA may benefit RYONCIL's chances of more supportive treatment next time round. Who knows, we might even be in with a chance for the pilot program there - for CBER, the focus is on cellular or gene therapy products addressing an unmet medical need for a rare or serious condition, particularly those with the potential for significant disability or death within the first decade of life. Sounds like RYONCIL to me.

But I thought more important - as you noted - was Dr Verdun's response when asked about SATs. In her own words (any errors in the transcript are mine):

" (ARM interviewer) ...One of the big barriers for rare diseases, thinking about trial design requirements, is because you'd love to do randomized pacebo-controlled studies & that's kind of the gold standard but oftentimes that's not feasible or possible, and how do you think about the benefit-risk in offering..how do you think about trial flexibility in these diseases?

( Dr Verdun) I think it's extremely important, as you said. Not everything can be a randomized controlled trial. We have INDs open for diseases where there are 11 patients in the United States. Regulatory flexibility is key. I'm certainly open to that....yes, there has to be a consideration for the disease, the benefit-risk, how rare it is, and we have to do what we can to partner to get more of these therapies to patients that need them. And so, I agree - everything cannot be a randomized controlled trial.

..but I think that my overall goal is to increase and foster development in this space - if it's not accelerated approval, then a traditional approval & look at more innovative ways to sort of get trials done & get them completed...so I think that the regulatory flexibility has to occur in all of our pathways.

So, I'm committed to that. "

Okay then.

Also, having been Director of the Office of Blood Research & Review, I believe Dr Verdun will have a more than casual acquaintance with & interest in Dr Joanne Kurtzberg's seminal work on cord blood and HSCT, as well. Mother of 4, too.

All of which IMO will not hurt RYONCIL's BLA chances for the paediatric indication one little bit.

All good IMO
Cheers