Skip to Main Content

RTI uses cookies to offer you the best experience online. By and clicking “accept” on this website, you opt in and you agree to the use of cookies. If you would like to know more about how RTI uses cookies and how to manage them please view our Privacy Policy here. You can “opt out” or change your mind by visiting: http://optout.aboutads.info/. Click “accept” to agree.

Accept
RTI International
  • About
    • Office Locations
    • Executive Leadership
    • Corporate Governance
    • Partner with Us
      • U.S. Government
      • Clients and Funding Agencies
      • Industry and Commercial Clients
      • Foundations and Associations
      • Bilateral Agencies and Multilateral Banks
      • Universities and Academic Research Institutions
      • Suppliers and Small Businesses
    • Commitment to Quality
      • RTI's Client Listening Program
    • Ethics and Human Research Protection
    • Living Our Mission
    • Veteran Opportunities at RTI

    About

  • Practice Areas
    • Health
      • Public Health and Well-Being
      • Health Care Transformation
      • Behavioral Health
      • Health Behavior Change
      • Precision Medicine
      • RTI Health Solutions (RTI-HS)
      • RTI Center for Community Health Evaluation and Economics Research
      • Health Equity
      • RTI Health Advance
    • Transformative Research Unit for Equity​
      • Equity Capacity Building Hub
      • Social and Economic Justice Research Collaborative
      • Narrative Research and Community Engagement Lab
    • Education and Workforce Development
      • Early Childhood
      • K-12 Education
      • Postsecondary Education
      • Career and Adult Education and Workforce Development
      • Education Policy, Systems, and Governance
      • Education Research Methodologies
      • Education Technologies
    • International Development
      • Energy for Development
      • Environment
      • Global Food Security, Agriculture, and Nutrition
      • Global Health
      • International Education
      • Monitoring, Evaluation, Research, Learning, and Adapting (MERLA)
      • Youth and Economic Opportunity
      • Building Resilience Against COVID-19 in Developing Countries
      • Water, Sanitation, and Hygiene (WASH)
      • RTI Center for Governance
    • Climate Change
      • Clean Energy Technology and Renewables
      • Climate Finance
      • Climate Justice and Equity
      • Climate Planning, Preparedness and Resilience
      • Climate Policy
      • Climate Vulnerability, Adaptation, and Mitigation
      • Economic Impacts of Climate Change
    • Water
      • Food-Energy-Water Nexus
      • Water Quality
      • WASH (Water, Sanitation, Hygiene)
      • Water Resources Management
    • Energy Research
      • Carbon Capture and Utilization
      • Biomass Conversion
      • Natural Gas
      • Energy Efficiency
      • Industrial Water
      • Syngas Processing
    • Environmental Sciences
      • Air Quality
      • RTI Center for Water Resources
      • Urban Sustainability
      • Toxics
      • Building Resiliency in the FEW Nexus
      • Climate Change Sciences and Analysis
      • Environmental Policy
      • Environmental Justice
      • Sustainable Materials & Waste Management Solutions
    • Justice Research and Policy
      • RTI Center for Community Safety and Crime Prevention
      • RTI Center for Policing Research and Investigative Science
      • Child Well-Being and Family Strengthening
      • RTI Center for Forensic Sciences
    • Food Security and Agriculture
      • Market Systems Strengthening
      • Food Safety
      • Food and Nutrition
      • Global Food Security, Agriculture, and Nutrition
      • Climate-Smart Agriculture
      • Agricultural Innovation
      • Obesity Prevention
    • Innovation Ecosystems
      • Innovation Advising
      • Innovation for Economic Growth
      • Innovation for Emerging and Developing Economies
      • Innovation for Organizations
      • Research, Technology, and Innovation Policy
      • Technology Acceleration
    • Military Support
      • Military Behavioral Health
      • Military Health and Human Performance
      • Military Sexual Assault, Harassment, and Domestic Violence Prevention
      • Wearable Sensor Technologies
      • Military Health System Transformation

    Practice Areas

  • Services + Capabilities
    • Surveys and Data Collection
      • Survey Design
      • Instrument Development
      • Survey Methodologies
      • Data Collection
      • Establishment Surveys
      • Health Registries
      • Data Analysis and Reporting
      • Research Operations Center
    • Statistics and Data Science
      • Survey Statistics
      • Environmental Statistics
      • Coordinating Centers for Multisite Studies
      • Analysis and Design of Complex Data
      • Biostatistics
      • RTI Center for Data Science
    • Evaluation, Assessment and Analysis
      • Evaluation Design and Execution
      • Advanced Qualitative, Quantitative, and Mixed Methods
      • Evaluation, Monitoring, and Assessment
      • Economic Analysis
      • Evaluating Communication Interventions and Campaigns
      • Evidence Synthesis for Policy and Practice
      • Risk Assessment and Prediction
    • Program Design and Implementation
      • Systems Strengthening and Scaling
      • Capacity Assessment and Building
      • Policy Reform Support
      • Curriculum and Teacher Professional Development
      • Interventions and Prevention Programs
      • Implementation Science
    • Digital Solutions for Social Impact
      • Human-Centered Design of Digital Solutions
      • Digital Product Development
      • Digital Communication Campaigns
      • Digital Data Analytics
    • Research Technologies
      • Survey Technologies
      • Data Management and Decision Support Systems
      • Geospatial Science, Technology, and Visualization
      • ICT for Limited-Resource Settings
      • Mobile Applications
      • Web Applications
      • Bioinformatics
      • Interactive Computing
    • Drug Discovery and Development
      • Medicinal Chemistry
      • Molecular Design and Cheminformatics
      • Behavioral Pharmacology
      • Drug Metabolism and Pharmacokinetics (DMPK)
      • In Vitro Pharmacology, Bioassay Development, and High-Throughput Screening (HTS)
      • Isotope Labeling
      • Regulatory Consulting and Support for Medical Products
    • Analytical Laboratory Sciences
      • Bioanalytical and Toxicology Research
      • Forensic Sciences
      • Physicochemical Characterizations
      • Metabolomics
      • Proficiency Testing and Reference Materials
      • Microbiology
      • Analytical Chemistry and Pharmaceutics
    • Engineering & Technology R&D
      • Biomedical Technologies
      • Decarbonization Sciences
      • Environmental Exposure & Protection
      • Materials & Environment
      • Sustainable Energy Solutions

    Services + Capabilities

  • Centers
    • RTI Center for Advanced Methods Development
    • RTI Center for Communication Science
      • Communication Research
      • Communication Design
      • Communication Delivery
    • RTI Center for Data Science
    • RTI Center for Education Services
      • Teaching and Learning
      • Education Leadership
      • Peer Learning Networks
      • Strategic Consulting
    • RTI Center for Forensic Sciences
    • RTI Center for Global Noncommunicable Diseases
      • Program Financing & Economics for NCDs
      • Health Systems Strengthening for NCDs
      • Communication Science and Behavior Change for NCDs
      • Implementation Science for NCDs
    • RTI GenOmics, Bioinformatics, and Translational Research Center
      • Disability Studies
      • Ethics
      • Newborn Screening
    • RTI Center for Water Resources
      • Water Resources Sectors
      • Water Resources Services
      • Water Resources Tools
    • RTI Center for Governance
    • RTI Global Gender Center
    • North Carolina Center for Optimizing Military Performance
    • NCCU-RTI Center for Applied Research in Environmental Sciences
    • RTI Center for Climate Solutions

    Centers

  • Impact
    • Newsroom
    • Insights Blog
    • Events
    • Publications
    • RTI Press
      • About the RTI Press
      • Instructions for Authors
      • RTI Press Collections
    • Projects
    • Global Reach
      • Asia
      • Eastern Europe and Central Asia
      • RTI International India
      • Africa
      • Middle East and North Africa (MENA)
      • Latin America and the Caribbean (LAC)

    Impact

  • Experts
    • Our Experts
    • In-Depth With Our Experts
    • Related News
    • Experts In the Media
    • RTI Fellow Program

    Experts

  • Emerging Issues
    • COVID-19 Research
    • Artificial Intelligence
    • Global Health Security
    • Cannabis Research
    • Opioid Research
      • Interventions for Opioid Use Disorders
      • Preventing Opioid Misuse and Overdose
      • Treating Opioid Use Disorders
    • Policing Research and Investigative Science
    • Drone Research and Application
    • E-cigarette Research
    • Zika Virus Research
    • Integrated Governance

    Emerging Issues

  • COVID-19 Research + Response
  • Global Reach
  • Insights Blog
  • Newsroom
  • RTI Press
  • Publications
  • Partner With Us
  • Careers
  • Facebook IconTwitter IconInstagram IconYouTube IconLinkedin Icon
  • Home
  • Impact
  • Publications
  • Subcutaneous C1-inhibitor prophylactic treatment for hereditary angioedema: a budget impact model

Subcutaneous C1-inhibitor prophylactic treatment for hereditary angioedema: a budget impact model

Krishnarajah, G., Knox, H., Graham, C., Christiansen, S., & Zuraw, B. (2018). Subcutaneous C1-inhibitor prophylactic treatment for hereditary angioedema: a budget impact model. Journal of Managed Care and Specialty Pharmacy, 24(4-a Suppl), S37. [D20]. https://doi.org/10.18553/jmcp.2018.24.4-a.s1

Copy citation

Abstract

BACKGROUND: Hereditary angioedema (HAE) is characterized byrecurrent, debilitating attacks of edema of the face, trunk, limbs,abdomen, and genitourinary and respiratory tracts, which generallyrequire immediate treatment with acute medications (on-demandtherapy). In June 2017, the U.S. FDA approved C1 esterase inhibitorsubcutaneous (human; C1-INH [SC], HAEGARDA, CSL Behring) asroutine prophylaxis to prevent attacks in adolescent and adult patientswith HAE, based on the phase 3 COMPACT clinical trial. This studydemonstrated a 95% reduction in the median attack rate with C1-INH(SC) 60 IU/kg and a >99% median reduction in the use of on-demandtherapy relative to placebo.OBJECTIVE: The purpose of this analysis was to assess the use of ondemandtherapy before and during use of prophylactic treatment withC1-INH (SC) in the COMPACT trial.METHODS: The COMPACT trial included a screening period of up to4 weeks and a run-in period of up to 8 weeks, followed by 2 consecutive16-week treatment periods in which patients self-administeredC1-INH (SC) and placebo in a crossover manner. Use of on-demandtherapy in the pre-randomization period (ie, screening and run-inperiods) was compared with use during prophylaxis with C1-INH(SC) 60 IU/kg in the COMPACT study.RESULTS: Data on use of on-demand therapy was available for 43patients randomized to receive C1-INH (SC) 60 IU/kg. During the prerandomizationperiod (up to 12 weeks), 78 uses (58%) of C1-INH (IV)therapy (Berinert, CSL Behring), 56 uses (42%) of icatibant, and 1 useof other C1-INH (IV) therapy were reported. During prophylaxis withC1-INH (SC) 60 IU/kg (up to 16 weeks), 47 uses of C1-INH (IV, Berinert)therapy (84%) and 9 uses of icatibant (16%) were reported. The mediannumber of uses of on-demand medications decreased from 3 during thepre-randomization period to 1 during prophylaxis with C1-INH (SC) 60IU/kg, a median reduction of 75% (95% CI: 50 to 100%, P<0.0001). Inthe pre-randomization period, 5/43 patients (12%) used no on-demandtherapy, whereas during prophylaxis with C1-INH (SC) 60 IU/kg, 21/43patients (49%) used no on-demand therapy.CONCLUSIONS: Patients with HAE used less on-demand therapyduring prophylactic treatment with C1-INH (SC) than during thepre-randomization period (ie, no prophylaxis). The reduction in useof on-demand therapy for HAE attacks will have cost implications.

Share
  • Share on Facebook
  • Share on Twitter
  • Share on Linkedin
  • Email

Publications Info

To contact an RTI author, request a report, or for additional information about publications by our experts, send us your request.

  • +1 919 541 8787
  • publications@rti.org

Recent Publications

OCCASIONAL PAPER

Bringing an equity-centered framework to research

OCCASIONAL PAPER

The Preschool Entitlement

OCCASIONAL PAPER

Culturally informed community engagement

Article

Does the relationship between alcohol retail environment and alcohol outcomes vary by depressive symptoms? Findings from a US Survey of Black, Hispanic and White drinkers

RTI Logo
Partner With Us
  • US Government
  • Commercial
  • Foundations & Associations
  • Multilateral Donors
  • Universities
  • Suppliers
Site
  • Privacy Policy
  • Security Policy
  • Site Map
  • Terms of Use
  • Accessibility
  • Contact Us
Contact Us
Facebook Icon Twitter Icon Instagram Icon YouTube Icon Linkedin Icon
delivering the promise of science
for global good
RTI Health Solutions RTI Innovation Advisors RTI Health Advance

© 2023 RTI International. RTI International is a trade name of Research Triangle Institute. RTI and the RTI logo are U.S. registered trademarks of Research Triangle Institute.