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Your Research, Their Hope

Credit: Wikimedia Commons / Jorgejesus4

It is common for scientists -- especially those who don't often work with patients -- to view diseases from the outside looking in. They may be driven by a desire to find a treatment or a cure, but they usually approach their work as a series of intellectual puzzles. Science's dispassionate approach to medicine has proven very successful, but sometimes it can be useful for scientists to consider the perspective of those who live with disease. The stories and struggles of patients and their loved ones can help connect scientists to a disease's human side.

"There are lots of savvy advocates out there … who really do know what the best science is, but other advocates don't understand how difficult developing an actual, effective treatment is and how research works." -- Rebecca Susan Dresser

There are many ways researchers can reach out to patients and their families, but one way -- building a relationship with a patient advocacy group -- can be particularly helpful. The potential advantages are not only humanitarian; they are also intellectual and practical. A patient advocacy group can inspire new directions for research. It can help recruit research subjects, especially when those subjects have relatively rare disorders. It can foster collaborations between scientists who might otherwise be competitors. Sometimes it can even provide new research funds.

But partnering with such organizations can be challenging for researchers used to working with only scientifically minded peers. Researchers and patient advocacy groups share many goals and interests, but those goals and interests aren't exactly the same. As a result, advocacy groups and scientists often have differences of opinion over a research program's focus, agenda, and timeline. Managing those differences and bridging gaps in knowledge and trust are essential to a successful research partnership. Science Careers spoke to several scientists who have studied these partnerships. They offer practical tips for how to cultivate a mutually beneficial relationship with a patient advocacy group and avoid some of the common pitfalls.

Finding partners

The first step in building a relationship with a patient advocacy group is to find a group to work with. Often, many of the groups that support research for rare diseases are small, family-run operations. If you know researchers in your field who've worked with an appropriate group, they are your best source for making those connections, says Richard Sharp, director of bioethics research at the Cleveland Clinic in Ohio, who occasionally consults for patient advocacy groups.

Courtesy of Richard Sharp
Richard Sharp

"Probably the best thing to do is work with senior folks in your field to identify possible collaboration opportunities," he says. "Oftentimes these small organizations rely on personal connections and the relationships end up being brokered by single individuals who happen to know a family or know the leadership of an organization."

If you don't know anyone who can introduce you personally, turn to one of the larger consortiums of patient advocacy groups, such as the Genetic Alliance, a nonprofit organization that helps connect researchers with patients who have genetic diseases; the National Organization for Rare Disorders; and the National Institutes of Health's Office of Rare Diseases Research. These organizations compile lists of patient advocacy groups and can help you figure out which ones would be right to assist you with the studies or clinical trials you're considering, says Aaron Panofsky, a sociologist at the University of California, Los Angeles, Center for Society and Genetics.

If all else fails, Panofsky adds, you can search the Internet for groups involved in advocacy for the disease you're studying. You can often get an idea of how reputable a group is by reading their online materials and seeing with whom they've collaborated in the past.

Making it work

A number of factors influence the success of partnerships and the quality of the resulting research. Panofsky recently published a study in Social Studies of Science in which he interviewed scientists and advocacy group members working in nine such partnerships across the United States and analyzed their responses.

Courtesy of Aaron Panofsky
Aaron Panofsky

One trend that emerged was that smaller organizations frequently require scientists to educate them on how the process of medical research works. "Lots of times, they've never engaged with scientists before. They've never thought about what a research grant is," Panofsky says. "Often the patient groups don't know exactly what they want or what they can do, so scientists actually can do a lot by educating the groups on what kinds of things would be helpful."

Science Careers Contributing Editor for Europe Elisabeth Pain explores a related topic this week: researchers working with local communities to access data and methods that otherwise would be unavailable to scientists.

If you need research subjects, ask if they can reach out to their members to find people willing to participate, Panofsky suggests. If they have a budget, ask if they'd be willing to support your research. You may need to explain that there's a lot of uncertainty in science -- that dollars they spend to fund your experiments may not lead rapidly to clinical trials, let alone a cure. That's important information for people whose loved ones might be dying. "Scientists need to be honest sometimes and say, 'Look, there's a good chance this won't help your kid,' " Panofsky says.

Above all, be honest in how you describe the scope, potential, and limitations of what you're proposing, says Rebecca Susan Dresser, professor of ethics in medicine at Washington University School of Law in St. Louis, Missouri. "Researchers need to be very clear about the kinds of studies they're interested in doing and what [the] knowledge gained would be."

Some patient advocacy groups are more science-savvy than others, Dresser says, but it is the scientists' responsibility to make sure group members and patients are informed about the likely pace of the research and what kinds of results they can expect. "There are lots of savvy advocates out there … who really do know what the best science is, but other advocates don't understand how difficult developing an actual, effective treatment is and how research works," Dresser says. "They're living with a serious situation so you have to realize that your audience may want to hear the best-case scenario," even when that outcome is quite unlikely. "Without being brutal, you have to be very focused on truth-telling."

Bad behavior

CREDIT: Stephen Kennedy
Rebecca Susan Dresser

At times -- albeit rarely -- the intense desire for a treatment breakthrough can lead members of an advocacy group to pressure scientists to compromise their scientific integrity, Dresser says. She recalls a case she witnessed in which a mother working with a muscular dystrophy group "got very upset because they wouldn't let her son enter a phase II trial … because he wound up not being eligible." She had agreed to work for the group under the impression that doing so would get her son into the trial. When that didn't happen, she felt betrayed, Dresser says. Group members may also ask scientists to let patients have access to a treatment outside of a clinical trial or attempt to influence a study's findings.

To avoid such situations, Dresser and Panofsky recommend setting clear guidelines early in the partnership as to what should be expected from the scientists and the group members. Get it in writing if possible, Panofsky says, but try to avoid bringing lawyers into the mix, as wrangling over legal details can have a chilling effect on relationships.

Scientists aren't immune to ethical lapses, either, Panofsky says, though typical scientist misbehavior in these partnerships tends toward neglect rather than misconduct. One of the gravest sins you can commit as a scientist working with an advocacy group is to swoop in, do your studies, then leave without bothering to share your results, he says.

Greed is not good

Some scientists have a tendency to be territorial -- a mindset that is antithetical to the goals of a patient advocacy group. People whose loved ones have a rare disease don't care about your professional rivalries or that you have exclusive access to a rich vein of research participants and clinical subjects, he says. They just want the best science possible. In fact, he says, many of the more sophisticated groups have built relationships with many established scientists and are more than happy to help you collaborate. That can be helpful for young scientists looking to build their CVs and make their mark on a field. It can also be a great way to build a long-term relationship with an advocacy group.

In fact, young scientists are often at a special advantage when applying for small-foundation grants, Sharp says. "One of the things that they're looking for is to fund people at an earlier stage in their career, hoping that that person will then stay in the field and continue to work with them and study that disease over time," he says. "They're especially looking for people who may not have a demonstrated track record of NIH [National Institutes of Health] funding, who can leverage smaller grants in service of going after bigger NIH and [other] federal support. So postdocs and junior faculty are exactly the kinds of people they're trying to attract."

Michael Price is a staff writer for Science Careers.