Designing and Analysing 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