Annual AMCP conference includes Prime clinical research

Poster presentation topics include total cost of care and utilization trends for spinal muscular atrophy (SMA) and paroxysmal nocturnal hemoglobinuria (PNH)

March 25, 2022

The Academy of Managed Care Pharmacy (AMCP) is a professional association of pharmacists, physicians, nurses and professionals in life sciences and biopharmaceutical companies that helps patients get the medications they need at a cost they can afford by supporting evidence-based approaches to benefit design and population health management.

At this spring’s annual AMCP event hosted March 29-April 1 in Chicago, members of Prime’s health outcomes team will present two research posters. Research poster presentations provide an opportunity for authors to share their insights and outcomes to advance managed care practice, both through presentations at AMCP spring and fall annual meetings and through publication in AMCP’s Journal of Managed Care & Specialty Pharmacy (JMCP).

“Presenting our research in front of our peers is an incredible opportunity to be challenged, supported and exposed to diverse thinking,” said Patrick Gleason, PharmD, assistant vice president, health outcomes at Prime and AMCP board member. “We appreciate AMCP for supporting evidence-based managed care pharmacy research, and we are proud to consistently share our research findings year over year.”

Prime’s spring 2022 research

Spinal Muscular Atrophy Drugs: Expenditure, Utilization, and Impact of Prior Authorization Criteria Among 15 Million Commercially Insured Members

Overview: Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by survival motor neuron gene mutation with an incidence of 1 in 10,000 live births.¹ Nusinersen (Spinraza®) and risdiplam Evrysdi®) are the two non-gene therapy treatments for SMA. Using integrated pharmacy and medical claims data from more than 15 million commercially insured members, this research show utilization and trends, examines the proportion of total SMA health care costs are associated with the drug, and how real-world utilization and spend are impacted by differences in SMA drug prior authorization (PA) criteria and uptake/switch to the newer product risdiplam.

Major findings: In the analysis period from January 2018 through March 2021, Prime found:

  1. Utilization, spend and trend: Nusinersen utilizers remained relatively flat, risdiplam utilizers increased 33%, nusinersen total paid PMPM remained relatively stable, risdiplam total paid PMPM increased from $0.002 to $0.07.
  2. Utilizer characteristics and total cost of care (TCC): 57.7% risdiplam utilizers had previous nusinersen use, nusinersen utilizers were on average 6 years younger than risdiplam utilizers and median total cost of care per utilizer per month were similar for nusinersen and risdiplam utilizers.
  3. Nusinersen or risdiplam (i.e., drug cost) was more than $4 of every $5 health care dollars spent to care for SMA members, in this study. Annualized median SMA health care total cost of care was $489,000 to $520,000.
  4. Utilization and spend trend stratified by narrow and broad PA criteria: There was a 1.8 times per 1,000,000 lower utilization rate in the narrow PA group for nusinersen. Risdiplam utilization per 1,000,000 was 3 in the broad PA group compared to 4 in the narrow PA group while nusinersen total paid PMPM was 2.1 times higher in the broad PA group compared to the narrow PA group, and risdiplam total paid PMPM in the broad and narrow PA groups was $0.01.

Overall, narrow PA criteria may be impacting nusinersen utilization and spend. However, it does not appear to be impacting risdiplam utilization and spend, although this finding is limited by only 32 members evaluated due to risdiplam newly being available on the market

Real-World Analysis of C5 Inhibitor Administration Characteristics, Resource Utilization and Total Cost of Care Among Ravulizumab-Treated PNH Patients Using Integrated Medical and Pharmacy Claims

Overview: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, potentially life-threatening, chronic condition.² There are multiple treatments for PNH including complement C5 inhibitors (C5I) eculizumab and ravulizumab, and newer therapy pegcetacoplan. PNH treatment goals include reduction in number of transfusions, improvement in hemoglobin levels, and reduction in debilitating symptoms. In 2021, eculizumab was identified as the 10th most expensive U.S. drug at $678,392 per year. Since ravulizumab approval in 2018, some clinicians have switched PNH patients from eculizumab to ravulizumab, which also costs more than $400,000 per year dependent upon patient weight, frequently over $450,000 per year for an adult. This research sought to understand real-world dosing patterns, break through hemolysis, transfusion rates, and PNH total cost of care, especially among those switching from eculizumab to ravulizumab therapy.

Major findings: Among 15 million commercially insured members meeting analytic criteria, Prime found 1 in 5 members appears to be receiving a dosage higher than approved by the Food & Drug Administration (FDA), and ravulizumab therapy was associated with a lower discontinuation rate than eculizumab while ravulizumab utilizers also have a 12 percent lower total cost of care compared to eculizumab. This integrated medical and pharmacy claims data may help inform drug management decisions and optimize drug therapy and outcomes for members receiving these extremely expensive medications.


  1. Spinraza® and Zolgensma® for Spinal Muscular Atrophy: Effectiveness and Value. Evidence Report from the Institute for Clinical and Economic Review. February 22, 2019. doi: https://icer.org/wpcontent/uploads/2020/10/ICER_SMA_Response_to_Comments_022219.pdf.
  2. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-11. https://doi.org/10.1182/blood-2014-02-522128.

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