In the early 1990s, breast cancer advocates like Deborah Collyar spent much of their time demanding attention. "We made a lot of noise," said Collyar, who is now president of Patient Advocates In Research, a national organization based in San Francisco, Calif. As one of the early cancer advocates, Collyar was also persistent. She called investigators, wrote letters, demanded audiences, attended meetings and stood in line at the microphone when speakers invited questions.
Collyar and other activists wanted more funds for breast cancer research. But they also wanted a voice in how those funds were spent: "We wanted to participate in the research dialogue," she said in a recent interview.
As the decade progressed, the advocacy movement made definite progress in that direction. Advocates won new seats on research planning and review groups, including the National Cancer Institute's (NCI's) agenda-setting Progress Review Groups and the American Cancer Society's grant application review committees.
In 1995, the Department of Defense cancer research programs began to include advocates as voting members of review panels. The Food and Drug Administration (FDA) brought advocates onto its cancer drug advisory committee in 1995 and made them voting members in 1998. In 2000, the Susan G. Komen Breast Cancer Research Foundation increased the number of advocates on its review panels from 7 to 24 and started including advocate scores in the overall ratings of grant applications.
"Organizations are now reaching out to advocates as opposed to the advocates beating on the door," said Michael Katz of the International Myeloma Foundation. "There's been an explosion of demand for advocates."
But that doesn't mean the challenges are over, according to advocates and researchers who met in September 2000, at the NCI's Cancer Advocates In Research workshop in Rockville, Md. In fact, one current challenge stems directly from success. The movement has evolved, say leading advocates, to the point where it requires infrastructure -- processes to guide and support laypersons in the research organizations that are beginning to accept their presence.
"Now that we're winning seats at the table, the question is how to use these seats to best advantage," said Yvonne Andejeski, M.D., Program Director of NCI's Liaison Activities, whose office organized the workshop.
One of the most pressing needs, according to both advocates and researchers, is to clarify the roles and responsibilities of Advocates In Research organizations. This theme emerged clearly at the workshop as well as in a series of interviews that NCI conducted in early 2000 with both advocates and researchers at the institute.
"Over and over again, we heard that there was confusion and concern about advocate roles," said Andejeski, who is leading the effort to create an infrastructure for advocate participation at NCI. Dubbed CARRA, for Consumer Advocates In Research and Related Activities, the new system is now taking shape, based on the surveys and on recommendations that emerged from the September workshop.
Much of the discussion at this meeting focused on the issue of roles: Should advocates try to evaluate the design of a clinical trial in a grant application? Are they expected to make judgments on methodology? The answer from many advocates is that their principal role on research committees is not to assess the science directly -- though some do -- but to contribute a patient's, family member's or research participant's perspective.
That perspective can, for instance, remind scientists that proposed research has to have relevance for cancer, said David Ringer, a program director at the American Cancer Society (ACS). "Even though it's great science, it may not have a clear path to an outcome relevant to cancer patients."
The patient perspective can also influence a study's design. Collyar tells about a trial in which the investigators proposed that patients undergo biopsies of the uterus every three months. "The advocates on the committee said that if they wanted to get patients to enroll in the study it would be better to do biopsies less frequently," said Collyar. The committee took their advice, the trial went forward, and patients did enroll.
Another classic example, said Ringer, was an epidemiologic study in which investigators planned to give a quality-of-life questionnaire to people who had just received a diagnosis of cancer. "Scientifically it made sense," Ringer said. "But the stakeholder said 'you're not going to be able to do that, you won't get reliable data.' It took a cancer survivor to see the flaw in the design."
Not all review panels produce such defined instances of the patient perspective affecting research, but those who have participated say that the advocate's presence makes small as well as big differences. "The fact that we're there when a study is being discussed, rather than reacting to it after the fact, can make all the difference in the way a trial is designed," said Mary Lou Smith, an early breast cancer advocate and now director of patient, payer and government affairs for the Coalition of National Cancer Cooperative Groups, based in Philadelphia, Penn.
Orientation and Training
How does a patient advocate contribute a lay perspective to a highly technical discussion? "A good grasp on the language and literature of science is important," said Sandra Zook-Fischler, a veteran advocate member of the FDA drug advisory committee.
Other advocates agree that it is important to understand research and the research process, and that this is where a training and orientation process is needed.
"Putting a non-scientist into a complex bureaucracy with no sense of role or task or expectation is a recipe for disaster," said Susan Weiner, Ph.D., vice chair for advocacy for the North American Brain Tumor Coalition.
Several advocate training programs are under way or planned. The ACS started an on-site training program for its advocates in 1999. The Coalition of National Cancer Cooperative Groups is planning a program, the Department of Defense has a mentoring program for new advocates, and the Komen Foundation instituted formal orientation process for its advocate reviewers in 2000. At the NCI, training and orientation will be an integral part of the new CARRA program, Andejeski said.
Other Parts of the Infrastructure
While the need to develop orientation processes is a high priority, other processes also need attention, say research advocates. One is the system by which advocates become involved in research activities In the early days, it tended to be through informal arrangements that depended on whom investigators already knew.
Now organizations are developing systems to draw from the larger pool of advocates. The ACS, for instance, advertises "openings" through its local chapters and the media and puts all applications into a database. Program directors then select from among the nominees using established criteria. Part of NCI's plan for its CARRA program is to develop appropriate criteria for selection and establish a database of advocate participants, screened, trained, and willing to be matched up with activities at the institute.
Evaluation and feedback are also evolving aspects of research organizations that have advocate participation. Procedures for follow-up interviews with both researchers and advocates are either planned or already established in several places.
Like other parts of the infrastructure, evaluation processes are a sign of how established the advocates-in-research movement has become in the past decade and how its needs are changing as a result of success. "Those of us who started this have to switch gears," said Collyar. "Now our challenge is to develop advocates who can dialogue effectively with researchers."