In first of its kind study, Prime Therapeutics explores paroxysmal nocturnal hemoglobinuria (PNH) C5 inhibitor treatment rates

Real-world evidence shows opportunity to assess medication value and management decisions

October 14, 2021

EAGAN, Minn. – Leading pharmacy benefit manager (PBM) Prime Therapeutics LLC (Prime) used its integrated medical and pharmacy claims data to evaluate paroxysmal nocturnal hemoglobinuria (PNH) C5 inhibitor treatment rates among its commercially insured population. This first known analysis conducted by a large PBM with integrated pharmacy and medical claims data for this condition found members often have unmet needs, such as the absence of therapy and therapy discontinuation, clinical outcomes such as breakthrough hemolysis events and continued blood transfusion needs, and underutilization of optimal sites of care, that resulted in high health care costs.

PNH is a rare, potentially life-threatening chronic condition.¹’² With PNH, blood cells lack complement regulatory proteins, so the body mistakes healthy red blood cells as damaged, leading to the destruction of those cells (called hemolysis).¹’² The approved treatment options available for the treatment of PNH during the analysis period were two complement C5 inhibitors: eculizumab and ravulizumab.

During 2018 through 2019 Prime analyzed 15 million commercially insured members over a two-year period to identify individuals with PNH claims indicated by either the member seeking PNH C5 inhibitor treatment or with a PNH diagnosis. Of the 15 million members, 162 members were identified for analysis. These members were divided into three categories based on treatment approach: 1) eculizumab users (57 members); 2) ravulizumab users (6 members); and 3) those with a PNH diagnosis but without a C5 inhibitor prescription claim at time of identification (99 members).

Once the 162 individuals were identified, their health care claims were assessed for 12 months for five key areas of health care outcomes and costs and found the following results:

  • Standard treatment of care: The mean dose for eculizumab users was higher than the dose recommended by the U.S. Food & Drug Administration (FDA) with four in 10 members treated with eculizumab receiving higher than label dosing.
  • Transfusions: Nearly 40% of members on therapy had at least one transfusion while approximately 21% had four or more transfusions. A common treatment goal for PNH is to eliminate the need for transfusions.
  • Breakthrough hemolysis (BTH): Another common treatment goal for PNH is to eliminate BTH events. One in four individuals had medical claims indicating a likely BTH event.
  • Discontinuation: Nearly 40% of eculizumab users and 50% of ravulizumab users discontinued therapy within 12 months.
  • Health care utilization and site of care: While the office setting tends to be the least costly for administration of C5 inhibitor therapy via IV Infusion, just over half of members received their infusion in this setting
  • Health care costs: Eculizumab members had the highest total costs of care (averaging nearly $730,000 each), followed by ravulizumab users (averaging over $370,000 each) and those with only a PNH diagnosis (averaging nearly $80,000 each).

“This analysis shows the benefit and strength of having integrated medical and pharmacy claims combined with real-word evidence analytics to help Prime assess medication value and inform management decisions,” said Patrick Gleason, PharmD, assistant vice president, health outcomes at Prime. “To our knowledge, this is the first real-world claims based PNH C5 inhibitor treatment assessment conducted by a PBM. These findings indicate the PNH condition is expensive to manage and therapy outcomes such as transfusions and breakthrough hemolysis events are still occurring, that may indicate an opportunity to optimize therapy to improve outcomes. We always want our members to follow best practices and clinical guidance when it comes to their health care. Conducting this type of analysis helps us identify opportunities that will lead to better health care outcomes for members.”

This study will be presented at the Academy of Managed Care Pharmacy (AMCP) Nexus meeting Oct. 18-21, 2021 in Denver, Colo.


  1. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-11. https://doi.org/10.1182/blood-2014-02-522128.
  2. Schrezenmeier H, Roth A, Araten DJ, et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505-14. https://doi.org/10.1007/s00277-020-04052-z.
Download AMCP research poster

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