Who We Are
OUR MISSION AND INSPIRATION
As a 501(c)(3) non-profit corporation, our mission is to help prepare patient advocacy groups for clinical trials. We are committed to connecting with rare disease focused patient advocacy groups to guide them towards readiness for natural history studies or the clinical trial phase. It’s our heart’s passion to help these groups achieve their vision.
FOUNDER'S MESSAGE
Patient Organizations can play an important role in the development of treatments for rare diseases. They can help in the design of the right study, increasing its likelihood of success, as well as expediting the drug development process.
When a Pharmaceutical, Biotechnology, or Academic group (“Sponsor”) embarks on a clinical development program for a common disease, there is typically a wealth of information in the literature and trials conducted previously to use in designing a trial. Information is readily available about disease progression, appropriate endpoints, timing of endpoints, etc.
With a rare disease, and even more so with an ultra-rare disease, there is often very little information available to aid in study design. Sponsors may have two options: 1) running natural history studies to gather sufficient information to adequately design the trials, which can add several years to the clinical development program, or 2) Proceeding with a clinical trial based on limited data, increasing the chance that the studies will fail. Patient organizations that are able to collect data from patients over time prior to the start of a clinical development program can significantly increase the chances of success of a drug development program.
BOARD OF DIRECTORS





SCIENTIFIC AND MEDICAL ADVISORY BOARD



THE PROS TEAM


