Biotech Values

[iwfal]

$IONS

Spinraza Reimbursement

How predictable is it for this to happen? I guess some just don't learn or they are just completely out of touch:

FWIW the Anthem letter says:

To date, there is no evaluable published data on the safety and efficacy of nusinersen in individuals with more than two copies of SMN2 or symptom onset after 6 months of age. There are 4 additional ongoing clinical trials evaluating nusinersen's safety and efficacy in SMA, they include the following: (1) a Phase III study of type III SMA (late-onset) know as CHERISH (NCT 02292537; ClinicalTrials.gov; expected completion: June 2017 ), (2) a phase II randomized, double-blind, sham-procedure controlled study known as EMBRACE (NCT 02462759) for children diagnosed with SMA consistent with type II (intermediate disease severity) who were not eligible for enrollment in either ENDEAR or CHERISH, (3) an Expanded Access Program trial in infantile-onset SMA(NCT 02865109) and (4) an open-label follow-up study of those enrolled in completed trials of nusinersen, known as SHINE (NCT 02594124; clinicaltrials.gov).

I.e. the justification for not covering SMA2 or 3 is that there is no "evaluable published data" - and personally, after the FDA abdicated their role in the case of Etep, that seems a completely reasonable response even when the FDA approves for an indication (as they did for SMA2 and 3). That said the Anthem letter is a little strange in several ways:

a) They ID the CHERISH trial as being for SMA3. It isn't - it is for SMA2.

b) The note that CHERISH isn't scheduled to complete until June of this year, but in fact it was halted in November for overwhelming efficacy (on a motor function endpoint).

All told - I'd bet they start reimbursing for SMA2 shortly after CHERISH is either published in a journal, or is somehow added to the FDA documents (I don't think I've ever seen the latter happen). The big question IMO is whether SMA3 will ever be reimbursed?