Evaluating the Economic Burden of Polycythemia Vera Across the Disease Continuum: A Systematic Literature Review
Author(s)
Surabhi Aggarwal, MPharm1, Najiya Nahan, MPharm1, Geetank Kamboj, MPharm1, Francesca Torelli, MPhil2, Victoria G. Empson, MSc3, Claudia Serra Monraba, MSc3, Hemant Rathi, MSc1, Christen Lykkegaard Andersen, MD, DMSc, PhD4, Hans Carl Hasselbalch, MD, PhD5.
1Skyward Analytics Private Limited, Gurugram, India, 2AOP Orphan Pharmaceuticals GmbH, Milan, Italy, 3AOP Orphan Pharmaceuticals GmbH, Wien, Austria, 4Copenhagen University Hospital, Rigshospitalet, Copenhagen Ø, Denmark, 5University of Copenhagen, Sjællands Universitetshospital, Roskilde, Denmark.
1Skyward Analytics Private Limited, Gurugram, India, 2AOP Orphan Pharmaceuticals GmbH, Milan, Italy, 3AOP Orphan Pharmaceuticals GmbH, Wien, Austria, 4Copenhagen University Hospital, Rigshospitalet, Copenhagen Ø, Denmark, 5University of Copenhagen, Sjællands Universitetshospital, Roskilde, Denmark.
OBJECTIVES: Polycythemia vera (PV) is a rare, chronic myeloproliferative neoplasm initiated by somatic driver mutations in the JAK2 gene. A prediagnostic phase of low-allelic mutational burden may precede overt PV, which may even progress to post-PV myelofibrosis or secondary acute myeloid leukemia. The economic burden of PV, particularly across different disease stages and complications, is not well documented. This systematic review based on PRISMA guidelines aimed to identify and synthesize evidence on PV costs and healthcare resource utilization.
METHODS: Studies published between 2010-2025 through Medline, Embase, Cochrane Library, and grey literature reporting direct or indirect costs, resource use, cost drivers, or economic models were included. Data were extracted using standardized grid and synthesized narratively due to heterogeneity.
RESULTS: Of 2,534 records identified, 25 studies were included: 23 observational studies and two economic evaluations. Most studies were based in the USA (n=9), followed by Asia (n=7), Europe (n=4) and other regions (n=5). The total annual direct cost varied from $940 to over $65,000 per patient (cost years 2013-2019). Direct medical costs were higher in patients with thrombotic complications, disease progression, or hospitalization. Increasing costs were observed over time due to disease progression. The mean (SD) length of hospital stay ranged from 1.67 (7.94) to 35.1 (33.3) days. Fifteen studies reported productivity loss and disability, but did not quantify associated indirect costs. Work Productivity and Activity Impairment scores indicated substantial burden. Across three studies, activity impairment ranged from 19.7% to 40.3% and overall work impairment from 13.4% to 33.0%. Two economic evaluations identified ropeginterferon alfa-2b as a cost-effective intervention: one in low-risk PV due to delayed disease progression, and another across broader populations, low- and high-risk patients.
CONCLUSIONS: PV imposes a growing economic burden, largely driven by disease progression, complications, and treatment resistance. Earlier diagnosis and therapies with proven disease-modifying effect may reduce long-term healthcare costs.
METHODS: Studies published between 2010-2025 through Medline, Embase, Cochrane Library, and grey literature reporting direct or indirect costs, resource use, cost drivers, or economic models were included. Data were extracted using standardized grid and synthesized narratively due to heterogeneity.
RESULTS: Of 2,534 records identified, 25 studies were included: 23 observational studies and two economic evaluations. Most studies were based in the USA (n=9), followed by Asia (n=7), Europe (n=4) and other regions (n=5). The total annual direct cost varied from $940 to over $65,000 per patient (cost years 2013-2019). Direct medical costs were higher in patients with thrombotic complications, disease progression, or hospitalization. Increasing costs were observed over time due to disease progression. The mean (SD) length of hospital stay ranged from 1.67 (7.94) to 35.1 (33.3) days. Fifteen studies reported productivity loss and disability, but did not quantify associated indirect costs. Work Productivity and Activity Impairment scores indicated substantial burden. Across three studies, activity impairment ranged from 19.7% to 40.3% and overall work impairment from 13.4% to 33.0%. Two economic evaluations identified ropeginterferon alfa-2b as a cost-effective intervention: one in low-risk PV due to delayed disease progression, and another across broader populations, low- and high-risk patients.
CONCLUSIONS: PV imposes a growing economic burden, largely driven by disease progression, complications, and treatment resistance. Earlier diagnosis and therapies with proven disease-modifying effect may reduce long-term healthcare costs.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE441
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Trial-Based Economic Evaluation, Work & Home Productivity - Indirect Costs
Disease
Oncology, Rare & Orphan Diseases