The Rise of Patient Advocacy

The effects of rigorous patient advocacy are easily seen today throughout the pharmaceutical industry. Patients who suffer the debilitating effects of rare diseases are banding together to be heard in an attempt to have their conditions studied to further the development of treatments and cures. However, the influence of patient advocacy groups has not always been as it is today. Premier Research’s own Juliet Moritz, executive director of the Strategic Development Department of Rare Diseases, was recently featured on Applied Clinical Trials with an article discussing this very topic.

The Influence of the AIDS Crisis

The year 1988 marked the first time a group of activists took physical action in order to be heard. At a Columbus Day rally at the Department of Health and Human Services in Washington, D.C., AIDS activists set the stage to seize control of the FDA headquarters the next day, focusing the nation’s attention, including that of the FDA, on the thousands of AIDS patients who were dying from the disease. Within three years, the FDA began recruiting patient advocates who had direct experience with that and other diseases. This program, known today as the Patient Representative Program, has more than 300 diseases and conditions represented, with patient advocates sitting on 47 FDA advisory committees and panels serving as voting members.

Patient Advocacy Continues to Expand

In 2015, guidance issued from the FDA marked the first time that recommendations were published that had originated within the disease community. The advocacy group Parent Project Muscular Dystrophy drew on patient, parent, caregiver, clinician and expert experience as well as that of the pharmaceutical industry to draft a document that today frames the way the FDA thinks about Duchenne muscular dystrophy. What was once perceived as a disconnect between the pharmaceutical industry, the FDA and the patient is becoming a cooperative partnership as the role of patient advocacy continues to expand. Especially in the case of rare diseases in which quantitative information is limited by the number of patients affected by the disease or condition, qualitative, patient-based input is necessary to the drug-development success rate.

Read Juliet Moritz’s complete article here.