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
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