Challenges with Payor Systems for the Diagnosis of SMA

EP: 1.An Overview of the Epidemiology of SMA

EP: 2.Clinical Burden of Spinal Muscular Atrophy

EP: 3.A Multidisciplinary Approach to Diagnosis and Treatment

EP: 4.Screening and Diagnosis of SMA in Children and Adults

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EP: 5.Challenges with Payor Systems for the Diagnosis of SMA

EP: 6.Adapting to Rapid Advancement in Treatments in SMA

EP: 7.New DMTs in SMA: Nusinersen

EP: 8.New DMTs in SMA: Onasemnogene and Risdiplam

EP: 9.Payor Perspective on Coverage Decisions for DMTs in SMA

EP: 10.Shared Decision Making When Selecting a DMT in SMA

EP: 11.Unmet Needs and the Future of Treatment in SMA

Dennis Scanlon, PhD: Is there a role for the payer in trying to facilitate and expedite early access to screening [for spinal muscular atrophy], or at least not inhibit it? For many clinical conditions, people sometimes complain about the need for prior authorization. Or is the payer just providing awareness that there may be availability to get somebody in quickly?

Kevin U. Stephens Sr., MD, JD: The payer is very important for screening. The key is to activate the health care delivery system once you have a positive screen. Remember that this is a game changer for the mom and their family. This is a severe illness that requires a lot of attention and resources. Many times, they are not prepared to expend the time and resources needed to take care of these children. Navigating through the health care delivery system with a child who has this illness can be very difficult, particularly if the mom and the family are young, with the wraparound services you have to do. From the payer perspective, we have to really launch our resources to help case management, disease management, and multidisciplinary teams. We have to help introduce the family to how to access these services, as opposed to resorting to emergency departments and repeated admissions. Once these milestones are lost, the prognosis and everything starts to decline. It’s so important to start as early as you can with the screening and put these services on the line with the mom and the families.

Emma Ciafaloni, MD: I have a comment. As a clinician, I think it’s very important that payers become strong partners of ours. As you know, there are published guidelines on what to do in the setting of a positive newborn screening depending on the number of copies. The uniform recommendation is the sooner, the better. Based on all the trials, acting before 6 weeks of age is the best. But from everything we know, even postcommercially, if you treat at 12 days vs 6 weeks, it’s going to be much better, especially for the 2 copies. That clinic with the multidisciplinary expertise includes a full-time person whose job is to talk with the payers to get the preauthorization as quickly as possible. Time is motor neurons. Payers need to understand that when a clinic calls you with a 7-day-old baby and we are asking you for a specific prescription of drug, time is of the essence. Sometimes, the baby has to go on an insurance: the mom’s, the dad’s, or Medicaid. There is a lot of complexity. For clinicians and the families, those days are precious. This is not something that should sit on your desk for a week or 2. The community is learning the urgency. That’s obviously where we can partner best with the payers to make it happen quickly.

Dennis Scanlon, PhD: I sense in your response that there’s variation across payers in terms of their responsiveness to these requests, and maybe even a little frustration at some delays. Is that accurate? Are there models so we can make sure these delays don’t happen? Are there some cases where the payers, Medicaid programs, or others are doing a good job that we can learn from and try to replicate across multiple payers?

Emma Ciafaloni, MD: It’s education. At my institution [University of Rochester Medical Center], and I’m sure at many others, we started working with our financial leadership, pharmacists, and the liaison with the payers at least 6 months before the first FDA-approved drug because we knew they were coming. This is a relatively rare disease for a big payer. It’s not as common as seizures or headaches. There was a lot of preemptive work that went into partnering with all the different partners, including the payers, to say, “This is coming. These are going to be the guidelines. This is going to be the time.” In the end, everybody wins. If we delay treatment, the outcome is going to be worse. They’re going to need more hospitalizations and services. This is a win-win. The key is education. Preemptively talk with your partners at your institution and with the payer and say, “This is coming.” Usually, we pick up the phone and have a direct line with whomever is in charge of making the decision. That’s the way we can make it work best for our families.

Maria Lopes, MD, MS: Dr Ciafaloni is bringing up a great point. Sometimes payers don’t know what they don’t know. This ability to not only partner but assist in understanding what may be going on helps in terms of that average delay. Many payers may think they’re streamlined. They’ll even have the prior authorization criteria online. If this is causing delays, and a week matters, what are some of the opportunities to facilitate that? What’s the role for care management or your specialty pharmacy area? What’s the role in getting patients to that center of excellence early on that has an approach to the management and more capabilities in dealing with multiple payers that understand the authorization process? I totally agree. It’s an opportunity for us to look at the cost of failure. The failure is in the delay. These are still high-cost drugs that are going to be used. By causing that delay, we’re not optimizing the benefit of the potential for these treatments.

Dennis Scanlon, PhD: Medicaid oftentimes manifests in contracts with various specific payers. I imagine that Medicaid ultimately is a payer for many of these cases. Of course, Medicaid is a federal program that’s administered at the state level, so there’s quite a bit of variation. Are there any Medicaid policy changes or awareness at the Medicaid program within states or at the federal level that might be important here?

Maria Lopes, MD, MS: Yes. Most Medicaid plans will have a process. The process usually involves medical policy review, technology assessment, and the prior authorization on the specific therapy. It’s important to understand where the holdup is. Is it that the prior authorization is too cumbersome? Is there just a delay in the authorization? Are there denials being issued that are inappropriate? To answer the question, we need to understand what the problem is. Usually, every state is tasked with developing policy criteria. There are forums in which medical directors meet with the state legislature. This happens on a regular basis. If this gets on the agenda and a case is presented or the best practice is presented at a high level, there’s usually visibility for other medical directors who are responsible for these topics across the entire state. That’s often a great way of assisting with opportunities around gaps or disseminating best practices around what the care delivery model can look like.

Transcript edited for clarity.