We ask for your support of the PPA, not only because it may save lives and refocus our efforts in the fight against brain cancer, but may actually reduce the costs involved with treating patients.
Under the current compassionate access system, once the treatment is approved, there exists no centralized, third-party tracking system on patient responses or side effects. This is both reckless and short-sighted. Through the PPA, third-party registries are required both for communication to the patient population and researchers, but also to eventually assist in the full drug approval determination. This is also critical in the design of combinational therapy options as it will demonstrate future considerations of care. The FDA approval pathways must be continually rethought and redesigned to balance faster access to therapies with increased post-market surveillance requirements. The PPA provides the framework to protect patient safety, while also providing patients with the opportunity to access potentially life-saving treatments the current system prevents them from accessing.
As Congress continues to consider legislative solutions to support patients with rare and life-threatening diseases, and in light of the current congressional efforts to reauthorize the FDA user fee program, we urge you support and pass the PPA. The PPA is not only a way to advance trials and protect patients, but it may be the most effective way to save lives. It is for this reason that over 50 foundations and chapters in 21 states and 3 countries support this measure. We need your help in making it a priority.