# Targeted, adaptive and time-variant strategies for bleed prevention across the lifespan for persons with hemophilia A

> **NIH NIH K01** · YALE UNIVERSITY · 2024 · $173,448

## Abstract

Project Summary/Abstract
Options to reduce bleeding risk in persons living with hemophilia A are expanding. The issue is important
because the expense of factor concentrates, the most expensive drugs per beneficiary under Medicare Part B,
account for >80-90% of discounted lifetime hemophilia treatment costs (-$12-18 million per person). Options
currently include i) standard half-life factor products, ii) extended half-life factor products, iii) non-factor
substitutes, and iv) gene therapy. Analyses of optimal prophylactic strategies for persons living with hemophilia
A are complicated by study heterogeneity, a result of different inclusion criteria and disease severity
categories. To date, most health economic analyses have been funded by industry. A public-facing, easilyupdatable,
transparent, and independent health economic model does not exist for treatment decisions faced
by persons living with hemophilia A. Accordingly, the overarching goal of this research is to quantify value- and
equity-informed resource allocation thresholds for subpopulations living with hemophilia A. The applicant will
achieve the proposed aims of this K01 award under the guidance of established researchers who span health
decision science, health policy, hematology, and pharmacoepidemiology. The applicant will create a publicfacing,
transparent, and independent microsimulation model to evaluate all existing prophylactic treatment
strategies for persons living with hemophilia A. He will use a combination of intermediate and advanced health
decision science and pharmacoepidemiologic methodological techniques to derive equity weight thresholds,
assess the value of collecting information with future studies, and quantify annualized bleed rates from realworld
data. The research will make available needed information on the risks, benefits, and costs of bleed
prevention strategies in persons living with hemophilia A. The proposed career development and training goals
will provide the applicant with training in microsimulation, budget, and equity impact analyses, value of
information research, and pharmacoepidemiology - and lay the groundwork for future research. The proposed
training, infrastructure, and Yale institutional support will ensure the applicant's transition to independence.

## Key facts

- **NIH application ID:** 10949401
- **Project number:** 1K01HL175220-01
- **Recipient organization:** YALE UNIVERSITY
- **Principal Investigator:** George Goshua
- **Activity code:** K01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $173,448
- **Award type:** 1
- **Project period:** 2024-09-17 → 2029-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10949401

## Citation

> US National Institutes of Health, RePORTER application 10949401, Targeted, adaptive and time-variant strategies for bleed prevention across the lifespan for persons with hemophilia A (1K01HL175220-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10949401. Licensed CC0.

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