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 collect data and biological samples 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.
PROS supports rare disease patient organizations with: