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Considerations for Payers Covering Combination Treatment of SMA and the Impact of Utilization Management


John Brandsema, MD, and Julie Parsons, MD, discuss considerations for combination SMA treatment and factors impacting SMA care pathways, such as prior authorization and multistep therapies.

John Brandsema, MD: The consideration of combination patient therapy in this disease needs to appreciate the nuance of the decision we’re trying to make. We’re balancing an irreversible consequence of undertreatment—loss of motor neurons that we can’t get back—with the clear goal of not overtreating somebody without extra benefit or exposing them to more adverse effects than they would have experienced otherwise. We need clear data about whether these things in combination have a different effect overall on the body and whether that impacts their experience with the disease.

The trouble is that it can take time to develop this, and we’ve been doing it in the real world for only a few years. As was brought up earlier in the conversation, we had our first treatment in 2016. Relative to a lifetime with SMA [spinal muscular atrophy], that isn’t a lot of time to make these decisions. We’re still actively collecting all these data. There isn’t a lot in the literature to guide somebody making this decision, whether it’s a provider or a family trying to process what would be the right thing for their individual living with SMA to base this on. In some senses, it’s a leap of faith based on some anchoring of preclinical and scientific thinking and what we’ve seen in our real-world experience using these medications in research trials and in the clinic.

At this time, we’re still actively in data accumulation mode, and it isn’t a clearly delineated algorithm to care. All of us feel that in the clinic every time we have a very complex discussion with one of these patients that navigates a lot of issues, and we try to come to a mutual decision of what might be reasonable and discuss that with our collaborators in the payer system to figure out what’s of the best interest of this person in terms of their life with SMA.

Julie Parsons, MD: Management utilization can be a challenge for these very complicated medications and drugs. In Colorado, we’ve established a complex-drugs team and program to try to take that burden off patients and to create a system where we’re able to get a single-payer agreement and insurance authorization, and have Medicaid instituted to provide therapies and care for these patients fairly quickly.

As we’ve reiterated several times during the conversation, we consider treating newborns diagnosed with SMA a medical emergency. Across the country, the majority of us try to treat these babies as quickly as possible. Our goal in Colorado is to treat them in under 21 days, and we’ve been fairly successful with that. But that means we have to have all the systems in place and ready to go to get insurance authorizations, to get the pharmacy and ordering in line, and to get our clinicians in line and available to do either the [spinal] taps or the IV [intravenous] therapy. It requires a tremendous amount of work up front to create a system that works efficiently and effectively for patients. If a patient had to manage this on their own and go through insurance, get authorization, and set things up in terms of appointments and everything, it would be impossible for them to be effectively and efficiently treated with the disease-modifying therapies.

The majority of the pharmaceutical companies have some assistance programs that have been helpful for patients, but it isn’t necessarily helpful in terms of the medical system and getting everything in place, set up, and going so we can efficiently and effectively treat patients. They still have a tremendous burden in terms of the prevalent population of patients who have more progressive disease with durable medical equipment and respiratory equipment. Some of our patients have tracheostomies, ventilators, and gastrostomy tubes. There’s a tremendous amount of medical equipment and burden that families deal with. But we try to offload the prior authorization and medical management as much as we can for patients. That helps them, so at least this isn’t something that they have to always navigate on their own with their very complicated lives.

John Brandsema, MD: I sometimes find it difficult to have the words to express how these targeted treatments in the clinic have changed care in our care teams and for the families that we serve in the clinic. The experience of SMA in the past was of mourning loss. Every clinic visit would be: “What’s harder? What are you not able to do that you were able to do before? What’s a new challenge for you? How can we figure out how to address this with our various providers on the team who are hoping to make your function and quality of life as good as possible?”

Now we’re at least talking about stability. But usually—especially in the pediatric clinic, where we work—we’re talking about development happening superimposed on this disease, and we’re seeing improvements that people are able to celebrate. “This is a new thing I can do. Here’s a video of me doing something I never did before.” Or they show us something in the clinic that we’ve never seen them do before, and we can all celebrate that together.

That impact on quality of life is massive when you’re facing a disease that’s going to be progressive with no possible treatment. That was our messaging when we [initially] met these families in the clinic. Unfortunately, in the most common forms, there was progression to death at a very young age, and that loss was tragic to go through over and over with our patients and their families. Now they’re having much longer and fuller lives with SMA on treatment, and we’re working on finessing how we can best do standard of care in that new treated world. That’s a very different place to be in from when we were trying our best to hold back a tide that was relentlessly surging against us with this disease in terms of loss.

Transcript edited for clarity.

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