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1 Priorities for Engaging the Public in the Clinical Research Enterprise WORKSHOP INTRODUCTION A recent editorial in the Journal of the American Medi- cal Association noted that the clinical research enterprise is in crisis (Rosenberg, 2003~. The accompanying article iden- tified enhancing public participation in the Clinical Research Enterprise as a top priority for ensuring that the Clinical Research Enterprise is functioning optimally (Sung et al., 2003~. Key concerns regarding public involvement in clini- cal research include conflicts of interest, diversity of partici- pation, community involvement, safety, and privacy. Be- cause participatory-based research methods involve members of the public in all stages of the design and conduct of research, there is greater opportunity to address diversity of participation, community involvement, privacy, and other concerns. Training for clinical researchers and participants about the ethical conduct of research, including policies and procedures to monitor financial conflicts of interest, also have been suggested as ways to address these concerns (IOM, 2003; Schwetz and Dobs, 2002; Sung et al., 2003~. For these reasons, enhancing the role of the public in the Clinical Research Enterprise was the focus of this workshop. Opening the workshop, Mary Woolley, M.A., President of Research!America, noted that members of the public- patients, healthy participants, family members, and others- are very willing to take part in the research process. Re- search, she said, offers hope, which is a powerful motivator for the public to work with the research community. Researchers, too, are committed to speeding up the re- search process and making it as safe as possible through ac- countability and a willingness to learn from the public and to answer questions about research. Ms. Woolley cautioned, though, that the research community must stay out of the lecture mode and out of the habit of thinking that researchers know what the public "needs to know." Instead, they should listen to and learn from members of the public, she said. Numerous polls have shown that the public believes that s supporting research is a very high national priority, as re- flected by the bipartisan commitment to double the National Institutes of Health (NIH) budget in five years. However, Ms. Woolley pointed out that only about half the members of the public can name a single place where research is con- ducted (Charlton Research Company, 2002a). In addition, only about 2 percent recognize the NIH and its purpose (Charlton Research Company, 2002b). Ms. Woolley stated that members of the public want to be involved and will listen and learn as quickly as possible, but they do not want to be patronized. The public will be respectful, but they will not be docile; they will help researchers because all of us want research to succeed. The research community has not been very successful in attracting patients to participate in clinical trials only 4 percent of adult cancer patients are enrolled in clinical trials, commented Ms. Woolley. Research is an enterprise that most people have had no contact with, and in the past, researchers appear to have liked it that way, thinking that people without scientific training could not understand, much less intellec- tually contribute to, their research. Patient engagement should inform and saturate every aspect of research, from formulating a research agenda to study design, to study review, to oversight at all levels, to dissemination and to translation to practice, she noted. This requires making a seat at the table for not just one but for several nonscientists. According to Ms. Woolley, the purpose of the work- shop was not to determine a rigid definition of what patient- centered research should or should not be, but rather to iden- tify and agree to act upon ways in which the research community can more actively and more productively engage the public. Ms. Woolley concluded by describing the ideal outcome of engaging the public in clinical research. At that point, she said, when a member of the public asks a researcher what she does, the researcher will respond, "I am a researcher. I

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6 work for you. I serve the public's interest." And the ques- tioner will not say, "What are you talking about?" but in- stead will reply, "Yes, I know how research works for me; tell me how I can do more to support research." PRIORITIES FOR ENGAGING THE PUBLIC Since National Institutes of Health (NIH) Director Dr. Elias Zerhouni began his tenure at NIH in May 2002, he has quickly confronted a number of challenges facing the Insti- tutes. At the workshop, he made clear that engaging the pub- lic in the clinical research enterprise is a top priority. According to Dr. Zerhouni, engaging the public must be considered not as a nice gesture, but as a "strategic im- perative" for a number of reasons. First, translating basic advances into clinical reality is increasingly difficult. Sec- ond, discoveries and clinical validation must be accelerated to meet the rapid growth rate of healthcare needs and expen- ditures. As a percentage of GDP, the United States has the highest expenditure on health care in the world, and that rate is increasing (Levis et al., 2003~. Third, clinical approaches must be more efficient, by an order of magnitude, than cur- rent ones. Finally, public support and participation are es- sential, because these goals cannot be achieved without the public's help and understanding. Dr. Zerhouni pointed out that public participation in AIDS trials and coronary heart disease research has led to declines in the number of deaths from those diseases. "There is no doubt that if we just keep practicing medi- cine as we know it today, there is very little that we can see that will change the population dynamics of health and dis- ease in our country," Dr. Zerhouni noted. He listed three major priorities to further engage the public in the clinical research enterprise: trust, ongoing bi-directional communi- cation, and education. Trust While trust, defined by Dr. Zerhouni as "the ability to predict someone's behavior," has been an issue of concern for some time, it is now "the number one issue, and we need to tackle it and tackle it fully," he said. Retaining the trust of the public requires transparency, predictability, respect, qual- ity assurance, and a vibrant and respected national Clinical Research Enterprise. In Dr. Zerhouni's view, there are many aspects of the "There is no Doubt that if we just keep practicing medicine as we know it today, there is very little that we can see that will change the population Dynamics of health and Disease in our country." Elias Zerhouni ENGAGING THE PUBLIC IN THE CLINICAL RESEARCH ENTERPRISE relationship between the public and the Clinical Research Enterprise that are not sufficiently transparent. He cited con- flict of interest as one example, noting that conflicts must be addressed in a manner that is transparent both in reality and in the perception of the public. An actual lack of transpar- ency can foster distrust, but so can the perception that there is a lack of transparency. Dr. Zerhouni commented that maintaining a cadre of scientists and investigators who possess an invariant set of core values is important to foster predictability. Currently, in Dr. Zerhouni's view, the public believes that the core values are variable or not as strong as they should be within the Clinical Research Enterprise. He noted that a system cannot be built on regulations alone if it is to gain the trust of the public. ``lt is no longer possible for us to tell people what research we are going to co from the top. We need to have collaboration; other- wise, you will not get the results that you want from the Clinical Research Enterprise." Elias Zerhouni .......................................................................................................................................................................................................................................................... Respect also is essential if the Clinical Research Enter- prise is to retain the trust of the public, but the question is how to create the necessary respectful relationship. Privacy issues are one component of this relationship. Furthermore, with such a large Clinical Research Enterprise, there is a need for quality assurance mechanisms, including the credentialing of investigators, according to Dr. Zerhouni. Dr. Zerhouni noted that the current system is not work- ing properly. In his view, the problem cannot be fixed through unitary solutions, but will require a systems engi- neering approach, including an assessment of the relation- ships in the system between patient advocacy groups, the public, academic health centers, and community physicians. He noted that the ability of the United States to extract clini- cal research data from its investment in health care is not comparable to that of other countries, such as Sweden or England, because of a fragmented health care system in the United States and a lack of an interoperable information in- frastructure. Bi-Directional Communication Referring to bi-directional communication between the research community and the public, Dr. Zerhouni said, "It is no longer possible for us to tell people what research we are going to do from the top. We need to have collaboration; otherwise, you will not get the results that you want from the Clinical Research Enterprise." He noted that public input at NIH is protean, and currently relies primarily on one-way

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PRIORITIES FOR ENGAGING THE PUBLIC IN THE CLINICAL RESEARCH ENTERPRISE communication. The Council of Public Representatives (COPR) is an example of public input. Dr. Zerhouni believes that there is a need to ensure that patient advocacy groups are involved in collaboration early in the process, because they can accelerate the pace of research and can make it more effective and cogent. The United States public is diverse, and therefore, there is no one-size-fits-all solution. There are many, many ways to engage the public, but there has not been enough scientific study on the most effective ways to interact with the public, Dr. Zerhouni commented. Communication must be adapted to each segment of the public and be sensitive to trusted intermediaries such as doctors, patient advocacy groups, churches, and the media, he said. Dr. Zerhouni cited the media as a particularly important conduit for information. In addition, he noted that some members of the public, such as minorities and underserved people, are left out; they need to be included in the process. Physicians are essential for a vibrant Clinical Research Enterprise, but the proportion of academic physicians con- ducting and translating research has decreased since 1980 6 percent of doctors reported research as their primary career activity in 1980 versus 2 percent in 2000~ according to data 7 KEY ISSUES FACING THE CLINICAL RESEARCH ENTERPRISE Asked about the challenge of translating research dis- coveries into practice, Dr. Zerhouni responded that the prob- lem is complex, and that there is a need to address the eco- system the relationship of academic health centers and communities to each other. He added that there already is a deficit (of practitioners, funds, etc.) facing clinical practice, without the additional resources needed to translate research into practice. He noted that often the weak link in the Clinical Re- search Enterprise is translating findings into practice, in part because economic terms do not support this. Researchers make their discoveries and then move on to the next project, because this is where the funding is. Referring to the need to address clinical research as an enterprise, Dr. Zerhouni com- mented that the elements of the discussion will have to in- clude how to standardize and build a common infrastructure across the country and how to serve the country better in terms of spending on health care that is not effectively edu- cating doctors about what is right for patients. The public and private sectors should not be completely =~ separate but appropriate safeguards are essentialto earn from the Medical Marketing Service presented by Dr. ~ . ' Zerhouni. "So, no matter how you slice it, we are in a crisis mode," said Dr. Zerhouni. Education Dr. Zerhouni also commented that currently, there is a need to better educate the public about clinical research. He also noted that a well-informed public is supportive of clini- cal research and that public trust is based on education. He called upon clinical researchers to ensure that information given to the public is accurate and not misleading. He cau- tioned against creating false expectations in the public on the basis of incomplete information and called on NIH and oth- ers to "take the highest road possible on these issues." ``Engaging the public is a major priority, it is a national priority, it is not an option." Elias Zerhouni Dr. Zerhouni cited the Internet as one source to educate the public. Compared to other federal dot.gov websites, NIH is the most visited site in the country, with around 3.6-4.6 million users per month in 2002, according to NetRatings, Inc. "Engaging the public is a major priority, it is a national priority, it is not an option," said Dr. Zerhouni. To do this, there is a need to move from an institution-centric and inves- tigator-centric system to one that is patient-centric. public trust, according to Dr. zerhoun~. belt Is a symbiotic relationship, and we will have to do as well as we can to make sure that you have the ability to translate those discov- eries into reality," he said. Regarding the perceived bias in funding of clinical ver- sus basic research, Dr. Zerhouni countered, that the numbers do not indicate to him that there is a cultural bias. In fact, because study sections have been redesigned to facilitate the review of clinical research by a critical mass of peers, the success rate of clinical applicants is now similar to that of other applicants for funding. He further commented that he wants a balanced portfolio that looks at the impact of the research that is being done. Dr. Zerhouni, responding to a question about NIH fund- ing more outcomes research, commented that measuring to ensure that the modifications to healthcare patterns are more effective than those currently in use is very desirable. How- ever, he added that while this is important, it is not the mis- sion of the NIH to support a lot of outcomes research, unless there is potential to make a quantum change in the way clini- cal research is conducted.The clinical research system needs to be reengineered in a multidisciplinary fashion to best serve the country, he said. Referring to input from advocacy groups to the NIH, Dr. Zerhouni said that he is very impressed with the amount of interaction that occurs: The Institutes are very open. They have public representation on their Councils. "I don't have that sense of an ivory tower fortress from the Institutes," he commented. However, he noted that cross- Institute coordination is more complicated. Furthermore, he commented that earmarking congres- sional funds is detrimental, and the groups that focus solely

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8 "fThere is a need to] work with patient advocacy groups, especial Iy when you can have combinations of advocacy groups with a scien- tific agencathat really understand the spectrum of research activi- ties that need to occur for progress in any one Disease to happen." Elias Zerhouni on one disease or outcome are not necessarily helpful to the larger process. There is a need to "work with patient advo- cacy groups, especially when you can have combinations of advocacy groups with a scientific agenda that really under- stand the spectrum of research activities that need to occur for progress in any one disease to happen," he commented. REFERENCES Charlton Research Company for Research!America. 2002a. Public Doesn't Know Where Research Is Conducted. Data from Research!America polls 1998-2002. Charlton Research Company for Research!America. 2002b. Aggregate 2002: Most Do Not Recognize NIH. Data presented by Research!America. Institute of Medicine. 2003. Responsible Research: A Systems Approach to Protecting Research Participants. National Academies Press: Wash- ington, DC. Levit K, et al. 2003. Trends in U.S. health care spending, 2001. Health Affairs 22(1):154-164. Rosenberg RN.2003. Translating biomedical research to the bedside. JAMA 289: 1305-1306. Schwetz B. Dobs A. 2002. Streamlining the Clinical Research Enterprise. Journal of Investigative Medicine 50(5):22-26. Sung N. et al. 2003. Central challenges facing the national clinical research enterprise. JAMA 289:1278-1287. ENGAGING THE PUBLIC IN THE CLINICAL RESEARCH ENTERPRISE