Ryoncil is being positioned as tertiary care, ie a "medicine of last resort".
Rough numbers: 200 patients receive an allogenic bone marrow transplant. 50% (100) develop aGvHD. 50% of those fail to respond to steroids
We now have 50 SR-aGvHD patients who move on to secondary therapy (ruxilitinib/Jakafi). Let's grant that 80-85% respond to rux.
We're left with 7-10 RR-SR-aGvHD patients That's the group that is being targeted for tertiary therapy with Ryoncil.. Note that some of these may be treated with the kinase inhibitor du jour or other FDA-approved immune modulator used off label. Perhaps we're down to 5 - 8 patients out of the original 200 who are in dire straits at high risk of death and who move on to treatment with Ryoncil.
Positioning Ryoncil as a medicine of last resort is a decision that has been made by SI/management. They had/have the option to do a head-to-head phase 3 trial pitting Ryoncil against Jakafi. They've chosen not to do that so far and I can see merit in this approach given Ryoncil's price tag and the time it would take to do a phase 3. Should a label extension to adults be granted, clinicians interacting with insurance companies and looking out for their patients will ultimately determine when any individual patient receives Ryoncil. If it's highly effective with minimal SAE's, I would expect the pool of 5 - 8 to expand.
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