Payer Diagnostic Criteria and Prior Authorization of SMA

Surya Singh, MD: The timeliness of therapy and how payers ensure that patients get access early to treatment for SMA [spinal muscular atrophy] is 1 of the thorniest questions that everyone, all stakeholders, are wrestling with. It’s not only payers too. I’ll broaden it for a second and then zero in on the payer perspective and how managed care deals with it. But, timeliness is incredibly important here because we’re trying to prevent deterioration. You’re trying to allow these patients, these infants, to continue to march on toward motor milestones they wouldn’t otherwise achieve without treatment. That’s the point of all of this, and I don’t think any of us want to lose sight of that. Earlier treatment from the day that it’s available and conferring with experts is really important.

Two complications have emerged that I think everyone is still grappling with. One is that the differential diagnosis can be very broad. Until there’s either a mandated screening that takes place, so we know when people have the deletions that lead to the disease to start off with, we’re left with when these symptoms start to show up and patients are missing motor milestones. It can mimic lots of other things, then what happens in that diagnostic process? I would say just before I zero in on the payer issues that that time to diagnosis is still probably the most important thing. Just looking at it globally, that dictates the timing of treatment. This is because, is the diagnosis suspected, or does the person say, “This is just a baby who is a little floppy and hasn’t hit their milestones and is still in the range of normal or has a different sort of clinical presentation”?

That aside for a second, once the request has been made now, the diagnosis is in site, and the payer is involved, the payers are trying to do a couple of things…these are activities that are relevant to SMA but also more probably for a prior authorization [PA].

One is that there are a lot of effort and resources going into the electronic extraction of the necessary data to fulfill the PA criteria behind the scenes. The doctor submits their request or is notified that a PA is required, and all of the data that’s needed to confirm that this patient is appropriate for treatment—according to the medical policy that the payer has in place—is fulfilled without the treating physician and his or her staff having to do anything else. That’s the future; that is where a lot of resources are going. I think payers are increasingly making progress on that path to be able to get that stage.

That said, that’s not the norm right now, especially when there are specific laboratory values that are required and so on. Rare disorders get an expedited review, in most cases from payers, because obviously timeliness is of the essence here. However, the delays are typically when there’s “additional information required,” and most often clinical documentation is the additional information they’re looking for. There is a lag when that additional information is part of the policy and the person doing the review says, “I can’t make a rendered decision until I get that,” and then we can see delays of up to 10 days. That is like an eon when you’re talking about families dealing with it.