MSB - a new dawn, page-1561

Hi again,

re " ...but im sure I read on a couple of the insurers notices of support for ryoncil, that the other (cheaper options) had to be tried before ryoncil could be used?"

you need to start at the beginning here, IMO. And it has nothing to do with cheapness.

The FDA's not the issue any longer with paediatric SR-aGVHD & may well have become RYONCIL's biggest supporter now (due in part, IMO, to Incyte's overreach & attempted interference in the initial paediatric SR-aGVHD approval). I work through the reasons in Post #: 78943068 . In a nutshell, the FDA itself has said in its public response to the Lassman/ Incyte petition that ultimately the choice of RYONCIL vs JAKAFI is a matter for the relevant physician, but at the same time it has strongly reasserted that JAKAFI is not approved in patients under 12 years of age - IMO this is both FDA protection vs improper competittion from JAKAFI (and other 2nd-line drugs which are also not approved in this space) for a huge part of RYONCIL's approved market, but also FDA endorsement of the ultimate decision on choice of RYONCIL (vis-a-vis JAKAFI) being made by the paediatric patient themself.

The issue now has moved on to the insurers, as you point out, & the NCCN Guidelines (which haven't yet been updated for RYONCIL). However, posters who think a particular insurer is potentially problematic simply because its relevant medical policy on RYONCIL refers to options that " had to be tried before ryoncil could be used " may be reading those policies wrongly, IMO. As I see it, those insurer medical policies on RYONCIL which do state that coverage is provided subject to trial & failure etc are actually guaranteeing RYONCIL 2nd-line coverage for patients older than 12 years of age (albeit subject to conditions). Let's not forget that.

For example, here's an extract from a restrictive BlueCross BlueShield BlueCare RYONCIL medical policy that focuses on JAKAFI & has been in force since February 2025:


Note that coverage condition (d) only applies to paediatric patientsolder than 12 years of age. JAKAFI is not approved for any younger paediatric patients. This is the significance of 'age-appropriate labelling'. Note also that this particular policy doesn't require failure of any other 2nd-line treatment e.g. tacrolimus, and this is no doubt insurer recognition of the FDA not having approved any other treatment which overlaps with RYONCIL's current approval. So condition (d) falls away for those younger patients.

But this is all missing the main point, IMO.

The point here that should be of great interest to MSB holders is that coverage IS expressly confirmed as provided for RYONCIL as 2nd-line treatment in patients older than 12 years of age, despite the availability of JAKAFI treatment (which may be cynically cheaper to use in $$$ terms in the short term). And all that would be required of an older patient is a contraindication or intolerance to JAKAFI.

Even if that older paediatric patient were not intolerant or contraindicated, there is still the case for medical exemption on a case-by-case basis e.g. because given the severity of the Grade of SR-aGVHD (e.g. III or IV) the time that a response to JAKAFI might take would imperil the patient's life beyond the ability of RYONCIL to treat the patient properly.

And here we come full circle. This is where I feel the FDA has done MSB and paediatric SR-aGVHD physicians a great service in pointing out to insurers and the world at large in its December response to Lassman/ Incyte just when a patient's physician might be able to make that case for medical exemption from condition (d)-like restrictions to a relevant insurer. I'll repeat it here in this post:

" ..... RYONCIL, a cellular therapy, may offer important benefits to patients, including those who may prefer not to use JAKAFI, and as Petitioner acknowledges, JAKAFI is not approved in individuals under 12 years of age.

Ultimately, the decision of whether to prescribe RYONCIL or JAKAFI is one that rests with the prescriber and patient and will depend on factors such as the patient’s history, goals for treatment, and tolerability."

Thanks FDA. Thanks Lassman.

Cheers
GLTALTH