Cost-effectiveness analysis of low-dose prophylaxis versus on-demand treatment for moderate-to-severe hemophilia A in India.

Abstract

AIM:

Hemophilia A (HA) is an inherited bleeding disorder caused by a deficiency of clotting factor VIII in the blood. In resource-limited settings like India, affordability is a significant challenge in managing patients with severe HA. This study aims to assess the cost-effectiveness of intermediate-dose prophylaxis versus on-demand factor therapy in adult and pediatric populations with moderate-to-severe congenital HA without inhibitors in India.

METHOD:

We conducted a prospective cost-effectiveness analysis from a societal perspective, categorizing patients into a base state and a joint disease state (patients with Hemophilia suffering extensive bleeds leading to chronic joint disease). Using targeted literature search and primary market research, we developed a Markov model measuring the total cost of Hemophilia treatment and health outcomes, including life-years (LYs), quality-adjusted life-years (QALYs), incremental cost-utility ratio (ICUR), and incremental cost-effectiveness ratio (ICER). The model extended over a lifetime horizon of 70 years with a one-year cycle length. Sensitivity analyses assessed study robustness.

RESULTS:

Low-dose prophylactic therapy was cost-effective for adults (>18 years) and pediatric populations (<18 years), yielding better health outcomes (adults: 0.15 LYs and 2.43 QALYs gained; pediatric: 0.40 LYs and 3.12 QALYs gained). Intermediate-dose prophylaxis showed positive net monetary benefits in terms of Quality-Adjusted Life Years (QALYs) for both adult and pediatric populations, with dominant ICER and ICUR values in both cases.

CONCLUSION:

Using intermediate-dose prophylactic factor VIII therapy is a cost-effective approach that improves clinical outcomes compared to on-demand therapy in the Indian adult and pediatric HA populations without inhibitors.

• Authors: Tulika Seth, Kapil Garg, Prakas Kumar Mandal, Anupam Datta, Shailendra Verma, Suresh Hanagavadi, Usha Rani Thota
• Journal: Hematology (Amsterdam, Netherlands) (Hematology) Vol. 28 Issue 1 Pg. 2277497 (Dec 2023) ISSN: 1607-8454 [Electronic] England
• PMID: 37933875 (Publication Type: Journal Article, Review)

Chemical References

• Factor VIII

Topics

• Adult
• Humans
• Child
• Hemophilia A
• Cost-Effectiveness Analysis
• Prospective Studies
• Cost-Benefit Analysis
• Factor VIII (therapeutic use)
• Joint Diseases (drug therapy)