Patient advocacy is a vital component in assuring that roadblocks to optimal treatment do not prevent cancer patients from obtaining the help they deserve.
Recently, a group from Parent Project Muscular Dystrophy presented the FDA with a set of patient advocacy initiated guidance for a rare disease to help expedite development and review of potential therapies for Duchenne’s muscular 
dystrophy.
Over the course of six months, more than 80 representatives, including parents and patients, medical experts, academics and biopharmaceutical industry representatives, worked with the FDA to help shape the guidance of this disease.
“By working closely with FDA to provide industry and other clinical trial sponsors with clearer guidance from the patient perspective, we will increase the likelihood that clinical trials will be designed to better match the unique needs of Duchenne patients,” said PPMD president Pat Furlong.
For rare diseases like Duchenne’s and GIST, clinical trials are difficult to design and implement because of challenges like small study populations and the challenges faced with understanding rare diseases. Utilizing the experience of actual patients, caregivers and medical professionals can help to minimize these challenges.
