Intravenous Treatment Workflow for Paroxysmal Nocturnal Hemoglobinuria from the Perspective of Nurses and Pharmacists in US Clinical Practices

INTRODUCTION:

This study aims to describe the steps involved in the intravenous (IV) ravulizumab treatment workflow for paroxysmal nocturnal hemoglobinuria (PNH) within the US healthcare system. By examining the perspectives of nurses and pharmacists, this research aims to better understand the time and resource needs associated with PNH IV therapy and help identify potential areas for optimization in medical centers with the adoption of an oral therapy alternative.

METHODS:

Building on the previous phase of the study with five hematologists in the US clinical practice, in-depth semi-structured qualitative interviews were conducted with additional six nurses and six pharmacists from different US medical centers, between May and July 2024. Participating nurses and pharmacists were experienced with IV ravulizumab treatment for patients with PNH. Data from these interviews were analyzed to update the process map for IV ravulizumab treatment from the hematologist's perspective. In the updated map, detailed multiple steps and associated time for ravulizumab preparation in the pharmacy setting and treatment administration in outpatient settings for PNH were added. Nurse's and pharmacist's perspectives on process complexities related to IV treatment were also collected.

RESULTS:

Participating nurses and pharmacists were evenly distributed between academic- and community-based settings. The capacity of the infusion centers where they practice varied between 16-60 infusion chairs. The number of years of professional experience ranged between 6-35 years for nurses and 14-30 years for pharmacists. The participants reported seeing between 2-30 patients with PNH and having treated between 2-15 patients with PNH with IV ravulizumab in the past 12 months.

Nurses reported an average patient chair time between 3.0-3.5 hours for the loading dose and 2.0-2.5 hours for the maintenance dose of IV ravulizumab, with a post-medication observation period of 0.5-1.0 hour. The average total clinic time from patient check-in to check-out, including time for laboratory tests, pre-medications, treatment administration, and post-medication observation, ranged between 3.0-4.5 hours to 5.5-6.0 hours when including a physician visit on that day. Nurses reported that, when necessary, patients would typically receive supportive care, such as blood transfusions, on a different day and usually in a separate department within the same medical center.

Pharmacists did not report specific complexities. For doses within the recommended schedule, processes are reportedly in place for the coordination of treatment ordering, release, preparation, and administration to avoid any delays on the day of treatment. Overall, the total time for preparation plus all verification steps ranged between 30-60 mins, including 8-15 minutes for the treatment preparation itself. Pharmacists reported that the number and type of vials of ravulizumab are ordered per patient based on their required dose to minimize wastage. Some also reported that electronic medical record systems allow for a difference of 5-10% between the ordered and administered dose. Since the vials are single use, the full vials can be claimed for reimbursement even if not completely used.

Reported challenges were mainly related to obtaining insurance approvals for treatment initiation and scheduling issues, as infusion centers often operate at high capacity, sometimes resulting in dose delays. In addition, due to the complexity of insurance approvals and lack of emergency stocks of ravulizumab in most medical centers, breakthrough hemolysis is typically managed with off-cycle doses of any available complement inhibitor in inpatient settings.

CONCLUSIONS:

The results of this study suggest that IV ravulizumab treatment for PNH present complexities within the healthcare system. The time and resources required for IV treatment can be burdensome for providers, and with infusion centers operating at full capacity, adopting treatments with alternative routes of administration for PNH, such as oral therapy, could free up space and enhance scheduling flexibility for other competing conditions. Further studies are needed to explore the time commitment required for IV treatment versus alternative treatment routes from the patients' perspective.

Winokur:Novartis Pharmaceuticals Corporation: Membership on an entity's Board of Directors or advisory committees. Mulherin:Pharmacosmos: Honoraria; Apellis: Consultancy, Honoraria, Research Funding, Speakers Bureau; Novartis: Consultancy, Research Funding; Daiichi Sankyo: Consultancy. Yacoub:PharmaEssentia: Consultancy; Pfizer: Consultancy; Incyte: Consultancy; CTI Pharma: Consultancy; Notable Labs: Consultancy; Servier: Consultancy; Apellis: Consultancy; Acceleron: Consultancy; Gilead: Consultancy; Novartis: Consultancy; BMS: Consultancy; AbbVie: Consultancy; Celgene: Consultancy. Lee:Novartis: Current Employment, Current holder of stock options in a privately-held company. Bilano:Novartis Pharmaceuticals Corporation: Current Employment. Yen:Novartis Pharmaceuticals Corporation: Current Employment, Current holder of stock options in a privately-held company. Geevarghese:Novartis Pharmaceuticals Corporation: Current Employment. Paulose:Novartis Pharmaceuticals Corporation: Current Employment, Current holder of stock options in a privately-held company. Guerin:Analysis Group: Other: Annie Guerin is an employee of Analysis Group, Inc. (AG), an economic consulting firm. AG receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis. Latremouille-Viau:Analysis Group: Other: Dominick Latremouille-Viau is an employee of Analysis Group, Inc. (AG), an economic consulting firm. AG receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis. Bungay:Novartis Pharmaceuticals Corporation: Other: Rebecca Bungay is an employee of Analysis Group, Inc, an economic consulting firm that receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis Pharmaceuticals Corporation. Marathe:Novartis Pharmaceuticals Corporation: Consultancy, Other: Gayatri Marathe is an employee of Analysis Group, Inc, an economic consulting firm that receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis Pharmaceuticals Corporation. Waheed:Novartis Pharmaceuticals Corporation: Consultancy; Alexion: Consultancy.