Managing hope for those with spinal muscular atrophy

In December 2016, the Food and Drug Administration (FDA) approved Spinraza (nusinersen) as the first therapy to treat SMA. Its manufacturer, Biogen, launched Spinraza in January 2017. The cost of front-loading treatment the first year is $700,000; maintenance treatment every year after, $350,000.

SMA is a genetic disease that affects motor nerve cells in the spinal cord, affecting the ability to move and breathe. It’s rare. Only 1 in 6,000 to 10,000 babies will be born with it. The majority are born with the most severe form – SMA Type I, which has a high mortality rate and is a leading genetic cause of death for infants in the United States. Other forms of the disease, SMA type II, III and IV, are less severe and can manifest later in life.¹ As many as 10,000 to 25,000 children and adults in the United States currently have SMA.

Prime studied the real-world use of Spinraza through claims

Among 15 million commercially insured members, Prime identified 973 patients that had one or more SMA diagnosis codes over the study period of January 2017 to September 2018. This equates to 6 members per every 100,000.

While the average age was 13 years, the age of SMA patients ranged widely – from under one year old to 63 years old.

Here at Prime, we saw use of Spinraza quickly ramp up: from $0.15 PMPM in 2017 to $0.23 in the first 9 months of 2018 – a 53 percent increase in one year.

For the purposes of the study, we also wanted to follow individual patient use. We found 49 patients that met our study criteria of diagnosis code, index claim and nine-month continuous enrollment.

Some Spinraza claims processed under the pharmacy benefit, others processed under the medical benefit. Prime’s experience with integrating medical and pharmacy claims data made it possible for us to undertake this kind of study.

For each member, we added up Spinraza costs and all other medical and pharmacy costs to calculate patients’ total cost of care. Nusinersen costs comprised 86 percent of the cost of care for a patient with SMA.

Spinraza has a loading treatment protocol its first year

Spinraza is given as an injection into the cerebrospinal fluid (the fluid in the spine) via a medical procedure called intrathecal injection. The initial uptake schedule doubles the cost the first year of treatment. The four loading doses fall on day 0, day 14, day 28, and day 63. Then regular injections occur every four months after that. The first five shots in the first year cost about $700,000. In subsequent years, the schedule is more regular; then the cost is approximately $350,000.²

Dosing schedule

We see a role for stepped up care management or value-based contracting

Based on Prime’s claims analysis, 40 percent of members studied received two treatments less than expected in a nine month period. That could negate the clinical benefit of the treatment they’ve had so far (plus the hundreds of thousands of dollars spent on that care.)

We don’t know why use drops off. More research needs to be done. These results are limited for several reasons:

• A lack of data for patients obtaining patient assistance
• Patients in early access programs
• Patients switching their insurer (e.g., commercial plan to Medicaid)

Prime researchers presented this silver ribbon-winning study at the Academy of Managed Care Pharmacy’s (AMCP) Managed Care & Specialty Pharmacy 31st Annual Meeting March 25-28 in San Diego.

We need to work together toward a sustainable price

The Institute for Clinical and Economic Review’s (ICER) assessment of Spinraza’s pricing is that it currently exceeds commonly accepted thresholds. Using a cost per quality-adjusted life year (QALY) basis for calculation, ICER’s value-based price benchmark for Spinraza is $65,000.³ That suggests a discount of 90 percent off the current annual cost of $350,000 (for year two and following). Prime agrees.

Gene therapy Zolgensma FDA approved May 24, 2019

Patients with SMA and their families had been watching the calendar for the late May approval of Zolgensma^® (onasemnogene abeparvovec).

SMA is caused by a mutation in a specific gene that controls muscles needed to move and breathe. The new gene therapy manufactured by Novartis called Zolg ensma, replaces the mutated gene. This one-time only, intravenuous infusion over one hour, fixes the underlying gene mutation that causes SMA.Infants and young children in the clinical trials achieved and have maintained significant motor development landmarks in the two years since the original Zolgensma trials.⁴

A concern in the marketplace is Novartis pricing the drug at $2,125,000. Novartis is offering insurers to ability to pay $425,000 a year for five years. Novartis also encourages comparisons of the drug’s one-time cost to that of several years of chronic SMA therapy.⁵

The Institute for Clinical and Economic Review (ICER) said the $2,125,000 price falls within the upper bound of their value-based price benchmark range when certain conditions are met, such as treatment before 2 years of age and the SMA Type 1 version of SMA.

The market could feel the impact of this price very quickly

We identified 22 members with any type of SMA diagnoses under two years old. If half of them receive the gene therapy in 2019 (at a cost of $2,125,000 each), the potential cost is $23 million or net new spend of $0.13 PMPM.

Balancing hope with access

The world of orphan drugs and gene therapy bring exciting cures that give new hope to patients and their families. It’s hard to imagine a marketplace that can make those cures broadly accessible at $2,125,000 each.

On Prime’s health outcomes team, we don’t work in laboratories with genes. We’re not in pharmacies with the drugs themselves. We work in offices with computers and data bases. But understanding how doctors and patients use these important drugs provides us with important information:

• We can work with health plans on utilization management programs that support prescribers identifying the best recommended treatment protocols.
• We can work with health plans and prescribers on care management programs that support patients to keep adherence high, eliminate gaps in care and help them manage side effects.
• We can work with pharmaceutical manufacturers to obtain discounts and contract with them such that therapy outcomes are met; in short, we work to get the therapy price to its value.

Together we can make health care work better. It’s going to take some flexibility and cooperation all around.

References

1. SMA Overview. © 2015 Spinal Muscular Atrophy Foundation. Accessed at: http://www.smafoundation.org/about-sma/
2. A well-established dosing schedule. © 2019 Biogen. Accessed at: https://www.spinraza.com/en_us/home/taking/dosing.html
3. A look at Spinraza and Zolgensma for spinal muscular atrophy. Report at a glance. © 2019 Institute for Clinical and Economic Review. Accessed at: https://icer-review.org/wp-content/uploads/2019/04/ICER_SMA_RAAG_040319.pdf
4. Pagliarulo N. Accessed on February 18, 2019. Available at: https://www.biopharmadive.com/news/gene-therapy-could-be-cost-effective-in-sma-but-not-at-4m/544969/.
5. AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families. Novartis. May 24, 2019. Accessed at: https://www.novartis.com/news/media-releases/avexis-announces-innovative-zolgensma-gene-therapy-access-programs-us-payers-and-families