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Patient Activism

Researchers Are Admonished To Stay Focused

by Rick Mullin
June 19, 2006 | A version of this story appeared in Volume 84, Issue 25


Patient advocacy has played an important role in setting the course of drug development over the past 20 years. With the groundwork laid by AIDS activists beginning in the 1980s, advocacy groups for breast cancer, Alzheimer's disease, multiple sclerosis, and other diseases have gotten into the mix on drug company research advisory panels, on Capitol Hill, and within patient communities. They are credited with increasing access to therapies, accelerating drug approvals, improving the protocol for clinical trials, and raising funds for research. They even have had an effect on keeping a lid on pricing in some cases.

With genomics and biotechnology taking hold in drug research and the likelihood of breakthrough drugs hitting the market in the short-term future, these groups say they may have to gear up to fight some of the same battles again.

A new class of AIDS drugs, for example, has advocates concerned about pricing. Keeping the public up to date and focused will be an increasing challenge given the big wave of new information accessible through the Internet. Their biggest challenge, some say, will be in keeping drugmakers focused on the target of cures for patients as researchers move into new scientific territory.

Paula Kim, president of Translating Research Across Communities, believes that patients began truly asserting themselves in the health care system—specifically in the development, availability, and pricing of drugs—late in the game. "It wasn't until the late 1970s that advocates and researchers really started getting to know each other, and that really was jump-started and fueled by the AIDS movement," she says. "They understood the research issues, got on Congress' case, and freed up dollars."

And the research community understood the power of the voices of patients and advocates when it came to making things happen on Capitol Hill. "The cancer community took notice of the AIDS movement and, following suit, developed even stronger models of how to interface with the research community," Kim says. "In many ways, the AIDS and breast cancer community had a lot to do with laying very important groundwork."

Bob Huff, editor of GMHC Treatment Issues, a newsletter published by Gay Men's Health Crisis in New York City, says patient activism began with groups in New York City and San Francisco, notably ACT UP (AIDS Coalition To Unleash Power), that took it upon themselves "to study the whole system," by talking to regulators, scientists, and finally drug companies, which, he says, weren't initially receptive.

"The biostatisticians adopted us first," says Huff, who was active with ACT UP in the 1980s. "They worked through statistical methods to show that things could happen, that things could change." The most significant early success was in gaining access to drugs. The parallel-track mechanism, which allowed patients access to drugs that were still in Phase III clinical trials, was a major victory, as was the introduction of surrogate markers in trials, he says.

In the late 1990s, a new group, the AIDS Treatment Activists Coalition, was formed, partly to act as a "one-stop" liaison with the big drug companies and also as a means of keeping patient groups from becoming too cozy with the industry, a trap that AIDS advocates never fell into, according to Huff. "We wouldn't have had a Vioxx in HIV," Huff claims, "because we see all the data. We go to the FDA advisory committee hearings and look at as much data as we can from the very beginning. We know the investigators. On the rumor mill, we will hear about an unusual adverse event very early. There is a large network, and activists are always communicating. The Internet has made this possible."

After 20 years, Huff says, a "cordial and periodically open" relationship has developed between patient advocates and big drug companies, which "are kind of set in their ways." Biotech companies, on the other hand, are more consistently open to interaction, Huff says. "The biotechs take big risks in general, and they take risks in how they interact with the community," he says. But in both cases, the place of patient groups at the table is ensured. Most recently, advocacy groups have gotten involved with safety data monitoring boards, he says, and patient community members spend a lot of time generally educating new people at the drug companies. "We're being defined as expert consultants."

Being in this position presents several challenges, according to Kim. One is vetting information for the patient community. With so much unfiltered information on the Internet, readers are left "to be their own filters, and that makes it really hard to make sure the right message goes to the right people," she says. And what is the right message? Everyone, she says, has an opinion.

Kim says she is also frustrated with how medical and academic research works. "Many researchers have as their goal to do things for patients, but really a lot of it is about getting published, getting academic tenure, and advancing their careers. That is the be-all and end-all. But drug research doesn't end with publishing a paper. It just doesn't."

This problem figured prominently in a panel discussion on translational medicine at the BIO 2006 conference in Chicago in April. Art Mellor, president of Accelerated Cure Project for Multiple Sclerosis, a patients' advocacy group, lamented the lack of collaborative effort in MS. "We haven't learned anything from all this research," Mellor says. "The incentive structure is broken. We have all these surrogate milestones and publications that further careers, but we're drifting farther from the real goal." Mellor, who suffers from MS, says there need to be results that directly impact patients. "I want to see how all your cool findings connect to helping me."

An engineer by training, Mellor says what he first recognized upon investigating the state of research on MS was the lack of a central plan and a shortsightedness of vision. "You have house carpenters, and we need skyscrapers," he says. "We need a different set of disciplines, not just more money for carpenters." Developing new approaches requires better collaboration and a better communal store of data. Mellor says his group is creating such a repository. Access to the data, he says, requires that researchers share their findings.

Kim says that drugmakers and consumers are poised at a crucial moment for the first forays into personalized medicine. "Delivering the right medicine to the right person at the right time," Kim says, "would be a wonderful thing. But we're limited by the pace at which we can get the biomarkers and diagnostics in place and the pace at which we can get things out and in place commercially in order to really make it happen." The primary limiting factor, according to Kim, is the extent to which the industry's focus can be kept on the patient.

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