"One small step for man, one giant leap for mankind," were Neil Armstrong's famous words when he became the first man to set foot on the moon. That small first step became the jumping-off point for a whole new world of discovery and space exploration. Similarly, the first humble steps of the patient advocacy movement started decades ago with a few hopefuls treading lightly onto uncharted territory. Their efforts have paved the way for those after them, leading to new legislation, a more patient-centric approach to pipeline development, and new strategic partnerships between those that run the industry and those they serve.
The first "giant leap" in giving patients a voice in industry came in the 1980s, with the AIDS epidemic as the catalyst. "Prior to the AIDS epidemic, patient advocacy groups were supporting traditional research and timelines, and were still in the March of Dimes mindset—family members raising money to give to a particular cause for typical academic research," says Greg Simon, senior vice president of the Patient Engagement Group at Pfizer. "But when people are dying so rapidly—that causes a crisis that's very different from diseases such as Alzheimer's, for instance."
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Because of the shock of the epidemic and the nature of the disease, Simon says, a new way of getting things done—getting drugs pushed through the FDA review process and getting legislation passed—was crucial. Advocacy groups such as the AIDS Coalition to Unleash Power (ACT UP) rose to the occasion. "They just walked in and broke all the rules," says Simon. "They occupied offices. They wrote inflammatory letters calling the senior people at NIH like Tony Fauci inhuman idiots. And when Fauci was asked how these groups got his attention, he said, 'Well, when they keep writing articles with headlines that say Tony Fauci is an idiot, you start paying attention.'"
And it worked. Fauci invited the ACT UP advocates into his office, and from those conversations, according to Simon, Fauci "realized that he had to change the way we go about doing research." Part of that change was allowing AIDS patients who were not enrolled in clinical trials to try the new, pre-approved drugs anyway, because they might not have the time to wait for approval (a process now fully institutionalized as the Expanded Access Programs). Though the method was unprecedented, says Simon, "it was about accelerating the effort and breaking the rules to allow desperate people to try things we wouldn't let them try if they were not terminal." Pressure from the AIDS community also helped strengthen the FDA Fast Track initiative, in which the FDA defines therapeutically important new drugs and gives them special treatment in moving them through the registration process. This form of accelerated approval was formally endorsed in congressional legislation in 2007, and is seen as a major victory for patients with diseases representing a serious unmet medical need.
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According to Simon: "The lesson of the AIDS advocates isn't that you have to go around breaking windows. It's that you have to bring about change—you have to break the paradigm."