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[Free Whitepaper]

Designing And Analyzing Hemophilia Clinical Trials

Hemophilia is a condition which affects the blood’s ability to clot, thus causing a person to bleed (both internally and externally) more frequently and for longer, have painful localized swellings, cause deformities in joints, have a strikingly reduced quality of life and ultimately reduced life expectancy. There are several types of hemophilia, the most common of which are hemophilia A (effecting ~ 1 in 5,000 males) and hemophilia B (effecting ~ 1 in 30,000 males).

This whitepaper focuses on considerations for designing and analyzing clinical trials for treatment of Hemophilia A.

Key areas explored within this White Paper include:

  • An Introduction to Hemophilia
  • Current treatments for Hemophilia
  • Challenges involved with studying Hemophilia Clinical Trials 
    • Large amount of information for statistically meaningful results
    • Competition for patients in trials
    • Inhibitors Development
    • Factors that result in lengthy and ethically questionable design  
    • Quantifying endpoints
    • Pharmacokinetics
  • Good Practices
    • Recruitment
    • Safety Information
    • Selecting the correct patients

Designing and Analyzing Hemophilia Clinical Trials

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