Study participant discusses risks, benefits
Study participant discusses risks, benefits
She was diagnosed with rare, progressive disease
Weighing risks and benefits in human subjects research can be an objective, clinical process, unless the person who is measuring is also a study participant whose life is at stake.
Amy Farber, PhD, an instructor in social medicine at Harvard Medical School in Boston, MA, has served as an IRB member and a research advocate, and she has weighed risks and benefits from these perspectives. But it is her recent role as a study participant that has given her a unique perspective when speaking to IRB members and others in the research industry, as she did in Washington, DC, at the 2006 Annual Human Research Protection Program Conference of the Public Responsibility In Medicine & Research (PRIM&R), held Nov. 15-18.
"There is an urge to have a neat formula for balancing risks and benefits," Farber says. "You also want to be able to take into account the potential harms and the potential dangers and benefits, and having a formula that would help you balance these is perhaps a useful aspiration, but difficult to attain."
Farber's role as a research participant was in response to her diagnosis in 2005 with a very rare, progressive, and fatal disease called lymphangioleiomyomatosis (LAM).
LAM is a multi-system disorder that leads to respiratory failure in women of childbearing years, as abnormal cells invade normal lung tissue and overtake surfaces normally dedicated to oxygen exchange. Patients frequently experience shortness of breath and lung collapses. The disease is commonly misdiagnosed as asthma, bronchitis, and emphysema. There is no effective treatment for the disease.
Farber was part of an observational study about LAM conducted at the National Institutes of Health Heart, Lung, and Blood Institute. Her initial participation was based on the promise of a definitive diagnosis of LAM.
"The first time I participated in a study it was as if I was exchanging participation for a diagnosis," Farber says.
"I was torn," Farber adds. "Partly, I resisted an official or definitive diagnosis because I didn't see the point of having one if there was no treatment, and then certainly, once you have the diagnosis there are risks attached to the stigma of it, or, more concretely, the threats of penalties or inability to get or maintain insurance."
Now, as an advocate for an effective treatment for LAM and as the executive director of the LAM Treatment Alliance in Cambridge, MA, Farber meets with scientists and others to support increased research on LAM and to advocate for further clinical trials involving LAM patients. Her efforts and success in garnering attention for the disease by pre-eminent scientists and clinicians were highlighted in a feature story in the Wall Street Journal on June 29, 2006.
"In my case, I am still pining for a well-designed trial," Farber says. "We have recently had some success in creating a partnership with a drug company to get a trial moving."
There are monthly meetings about LAM and a related disease called tuberous sclerosis at Harvard Medical School, and more than 60 researchers gather regularly to discuss research that would leverage what is known about common diseases to find out more about LAM, Farber explains.
"We mobilize problem-solving," she says. "We create and foster collaboration to work on very targeted research projects that are prioritized based on problem-solving, state of science conversations, and finding a treatment."
This approach cuts through the bureaucracy and facilitates researcher access, shepherding researchers past barriers on their way to find a treatment in a highly targeted way, Farber adds.
LAM only affects women of childbearing years, and this fact has made Farber sensitive to obligations IRBs have in each protocol to ask about the gender specificity of the data being collected, she notes.
For instance, to what degree is LAM estrogen-driven or estrogen-dependent? As Farber has learned more about the disease, she's experienced a rude awakening at how little researchers have discovered about how estrogen works in the female body, she says.
"Sadly, we don't have the very best grasp of the body, in terms of the menstrual cycle and other complex hormonal ebbs and flows, for instance," Farber says. "There are a lot of elephants in the room when you talk about naturally occurring processes in women's bodies."
Farber's IRB experience has taught her that any discussion of research ethics or a study's risks and benefits must consider the community of people who have an interest in the research, including participants, researchers, community members, IRB members, etc.
"Everyone brings different things to the table, and we'd be naïve to think researchers and clinicians and managers don't have various health issues affecting their loved ones," Farber says. "For me, that's one of the important lessons of being on an IRB: that some of the most concerned, ethically-attuned comments and insights can come from researchers and clinicians steeped in actual practice and sometimes not from ethicists."
When Farber was involved in the observational study, she weighed the risks and benefits to her own participation in this way:
- There was arguably no direct patient benefit, and so there was, perhaps, no possibility of therapeutic misconception, she says.
- It was an observational protocol, and she wondered if it was misleading to refer to it as a clinical trial when there was nothing being tried.
- Farber recognized some potential benefits for herself, such as being able to interact with highly skilled and trained health care professionals who took an interest in her case.
- There was potential benefit for the LAM community, but this created the question of whether knowledge that was beneficial to the LAM community as a whole was also beneficial to her. "It's knowledge and action that helps me cope with the disease," Farber says. "What do you do with the knowledge? Do you just go on with your life?"
In Farber's case, the risk-benefit analysis led to an interesting conclusion. She decided she would not be able to just go on with her business as usual when there were so many resources available to her to mobilize into action that would benefit the LAM community.
"Through networks and the tremendous generosity of so many clinicians, researchers, friends, family members, colleagues, volunteers, and fellow patients, I realized I potentially had access to what might be a fast track to human research, with an approach that would support, foster, and fund that approach in some way," Farber says.
Farber's role as facilitator of research is balanced between her career in science, her work as an IRB member, and her most recent experience as a research participant.
"I feel like my IRB membership complements my experience as a research participant and advocate, and it helped me to think critically about the process of research," Farber says. "My experience has returned me to the question of what's fair to ask of patients, and what do I want as a trial subject/advocate."
Her goal in speaking to IRB members is to encourage them to think about how IRBs do or don't think about issues of trust and how to balance risks and benefits, she says.
"And how should they address the fact that some research participants want not just protection from research but access to research, and is having access enough?" Farber says.
Weighing risks and benefits in human subjects research can be an objective, clinical process, unless the person who is measuring is also a study participant whose life is at stake.Subscribe Now for Access
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