Evaluation of the Healthcare System Burden Associated With Intravenous Treatment for Paroxysmal Nocturnal Hemoglobinuria

Speaker(s)

Mulherin BP1, Winokur D2, Yacoub A3, Lee S4, Bilano V5, Yen G4, Geevarghese A4, Paulose J4, Guérin A6, Latremouille-Viau D6, Marathe G6, Waheed A7
1Hematology Oncology of Indiana, Indianapolis, IN, USA, 2UMass Memorial Health Care, University of Massachusetts Medical School, Worcester, MA, USA, 3KU Medical Center, The University of Kansas, Kansas City, KS, USA, 4Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA, 5Novartis Pharmaceuticals UK Ltd, London, UK, 6Analysis Group, Inc., Montreal, QC, Canada, 7Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA

Presentation Documents

OBJECTIVES: To delineate steps in the intravenous (IV) treatment workflow with ravulizumab for paroxysmal nocturnal hemoglobinuria (PNH) from a healthcare system perspective and identify steps potentially simplified with an oral therapy.

METHODS: Individual in-depth semi-structured qualitative interviews were conducted with five US hematologists between October and December 2023. Participating physicians were from large medical centers (academic and community-based), experienced in treating PNH patients with IV ravulizumab. The interview data were analyzed to identify steps in the IV treatment workflow, leading to the development of a process map, covering treatment decision/planning to outpatient administration. Physicians’ perspectives on process complexities related to IV treatment and steps that could be simplified through the adoption of an oral therapy were collected.

RESULTS: The process map delineated multiple steps from treatment decision to administration, inclusively. The approximate time from treatment decision to administration ranged between a minimum of 2-4 weeks. Delays were primarily attributed to limited vaccination access in community settings and insurance approvals. Additional delays occurred, particularly off-label dosing schedule, for example for breakthrough hemolysis, due to added complexity of obtaining insurance approvals. Patients spent an average 3-4 hours for treatment administration at the infusion center. The lack of flexibility in scheduling, given infusion centers operating at full capacity, was noted. Hypothetically, with an oral treatment, alleviated steps would include treatment scheduling and administration at infusion centers.

CONCLUSIONS: The study revealed complexities associated with IV treatment with ravulizumab for PNH from a healthcare system perspective. Given IV treatment is resource- and time-intensive for both patients and providers and that infusion centers are operating at full capacity, adopting an oral treatment could potentially enhance infusion center capacity and improve scheduling flexibility for other competing conditions. Further studies are warranted to understand the IV treatment burden from additional healthcare personnel (nurses, pharmacists) and the patient perspective.

Code

HSD51

Topic

Study Approaches

Topic Subcategory

Surveys & Expert Panels

Disease

Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)