A Systematic Literature Review on Economic Evaluations and Cost-Effectiveness of Second-Line Treatment Options for Immune Thrombocytopenia
Author(s)
Isabelle Lundqvist, MBA1, Kalitsa Filioussi, MD2, Shaun Walsh, MSc.3, Aditi Kataria, M. Pharm4, Lovneet Saini, M. Pharm4.
1Novartis Sverige AB, Kista, Sweden, 2Novartis Farma S.p.A, Milan, Italy, 3Novartis Ireland Ltd, Dublin, Ireland, 4Novartis Healthcare Pvt. Ltd., Hyderabad, India.
1Novartis Sverige AB, Kista, Sweden, 2Novartis Farma S.p.A, Milan, Italy, 3Novartis Ireland Ltd, Dublin, Ireland, 4Novartis Healthcare Pvt. Ltd., Hyderabad, India.
OBJECTIVES: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet counts, leading to increased bleeding and bruising. The costs associated with ITP treatment can vary significantly. This systematic literature review aimed to identify and critically evaluate the published evidence on cost-effectiveness of treatment options for adult patients with ITP.
METHODS: Scientific databases including Embase®, MEDLINE®, Cochrane library, and PubMed were searched for relevant publications from inception till 10th February 2025. Data on patient characteristics, model specifications, utility and cost inputs, Quality Adjusted Life Years, Life Years and Incremental Cost-effectiveness Ratios were extracted from the included studies.
RESULTS: A total of 28 studies and 22 Health Technology Assessments were included. Most studies reported cost-effectiveness analyses (n=18) followed by cost utility analyses (n=15) and commonly evaluated interventions included romiplostim (n=30), eltrombopag (n=26), and rituximab (n=13). Markov models were commonly used, with time horizon varying from 49 days to lifetime and cycle-length from 28 days to one year. Several studies found thrombopoietin receptor agonists (TPO-RAs), particularly eltrombopag and romiplostim, to be cost-effective compared to standard of care or alternatives. Patient monitoring costs (n=14) were identified as the key cost-driver, followed by costs associated with management of bleeding events (n=9), drug administration (n=8) and hospitalizations (n=8). Severe bleeding events incurred costs up to €23,795.57 in Europe, with utility values well below population norms and disutility values ranging from -0.766 to -0.072.
CONCLUSIONS: TPO-RA emerged as the most evaluated and cost-effective treatments for ITP with respect to second-line settings, though costs varied substantially. While bleeding is an inherent symptom of ITP, its inclusion in modelled health states and utility decrements highlights its contribution to the overall economic and quality of life (QoL) burden. Treatment strategies that reduce bleeding and improve QoL may offer both clinical and economic benefits in ITP management.
METHODS: Scientific databases including Embase®, MEDLINE®, Cochrane library, and PubMed were searched for relevant publications from inception till 10th February 2025. Data on patient characteristics, model specifications, utility and cost inputs, Quality Adjusted Life Years, Life Years and Incremental Cost-effectiveness Ratios were extracted from the included studies.
RESULTS: A total of 28 studies and 22 Health Technology Assessments were included. Most studies reported cost-effectiveness analyses (n=18) followed by cost utility analyses (n=15) and commonly evaluated interventions included romiplostim (n=30), eltrombopag (n=26), and rituximab (n=13). Markov models were commonly used, with time horizon varying from 49 days to lifetime and cycle-length from 28 days to one year. Several studies found thrombopoietin receptor agonists (TPO-RAs), particularly eltrombopag and romiplostim, to be cost-effective compared to standard of care or alternatives. Patient monitoring costs (n=14) were identified as the key cost-driver, followed by costs associated with management of bleeding events (n=9), drug administration (n=8) and hospitalizations (n=8). Severe bleeding events incurred costs up to €23,795.57 in Europe, with utility values well below population norms and disutility values ranging from -0.766 to -0.072.
CONCLUSIONS: TPO-RA emerged as the most evaluated and cost-effective treatments for ITP with respect to second-line settings, though costs varied substantially. While bleeding is an inherent symptom of ITP, its inclusion in modelled health states and utility decrements highlights its contribution to the overall economic and quality of life (QoL) burden. Treatment strategies that reduce bleeding and improve QoL may offer both clinical and economic benefits in ITP management.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
SA5
Topic
Study Approaches
Topic Subcategory
Literature Review & Synthesis
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)