Evaluation of Priority Setting and Programs of Research on Ethnic Minority and Medically Underserved Populations at the National Institutes of Health
As noted in Chapter 3, the National Institutes of Health (NIH) and the National Cancer Institute (NCI) sponsor a wide range of research and training programs relevant to cancer among ethnic minority and medically underserved populations. In addition to evaluating this research and program portfolio, the committee was asked to evaluate NIH's mechanisms for prioritizing research on cancer among ethnic minority and medically underserved groups, as stated in the committee's charge: "review … NIH's ability to prioritize its cancer research agenda for minorities and the role of minority scientists in decision making on research priorities."
This chapter summarizes the committee's assessment and includes an evaluation of opportunities for minority scientists and members of lay and community groups to provide input into the priority-setting process, the rationale by which policy decisions are made with regard to research priorities, and the adequacy of mechanisms for their implementation.
To understand the context for NIH's priority-setting processes and mechanisms for receiving input, it is necessary to understand how these processes are perceived by the scientific and lay communities. The first section of this chapter summarizes data that the committee collected with regard to these perceptions, including data from a survey of researchers interested in cancer among minority and medically underserved populations and a panel of individuals representing the perspectives of members of cancer survivor groups in ethnic minority and medically underserved communities. A review of NIH mechanisms for community and researcher
input follows. Finally, an analysis of the NIH priority-setting process for addressing cancer among minority and medically underserved communities is presented.
Survey of Research Stakeholders
The committee sought input both from ethnic minority researchers in the field of cancer research and from minority and nonminority investigators interested in cancer among minority and medically underserved populations. It sought this input as part of its attempt to understand the experiences of investigators across a broad range of disciplines who either have applied for NIH funding or have been involved in proposal review. These include individuals who either have some familiarity with the NIH portfolio of research on cancer among ethnic minority and medically underserved populations or may have benefited from NIH training programs designed to increase the level of representation of minority investigators in the field of cancer research.
It must be noted that the committee did not seek to identify a representative sample of the populations described above. Rather, the purpose of this survey was to identify some of the range of experiences and perceptions, both positive and negative, that minority investigators and those investigators interested in cancer among minority and medically underserved populations may have had with regard to NIH's priority-setting process and current program of research in this area. The survey results should therefore be interpreted with caution—they are likely not representative of any particular population of researchers, clinicians, or health policy analysts. They do, however, provide some insight into the kinds of perceptions that the extramural scientific community holds with regard to research on minority and medically underserved populations.
The pool of potential survey respondents was selected by obtaining the names and mailing addresses of 839 individuals who hold membership or who are otherwise affiliated with one of several organizations or cancer-related research programs. Specifically, names were obtained from the American Association of Cancer Research (minority research section), the Society for the Advancement of Chicanos and Native Americans in Science (members in cancer research-related fields), the Intercultural Cancer Council membership database, the Hispanic Cancer Control Network, the National Institute of General Medical Sciences (recent recipients of NIH minority training funds in cancer-related fields), and the Appalachian Leadership Initiative on Cancer (principal investigators). Whenever possible,
membership overlap was identified and duplication of survey solicitation was avoided. Data on the racial and ethnic distributions of the potential respondents are not available.
The committee received 220 completed responses to the survey. Among these, 33 identified their ethnicity as "white or Caucasian," 65 identified themselves as "African American," 90 identified themselves as "Hispanic," 12 identified themselves as "American Indian or Alaska Native,'' and 10 each identified themselves as "Asian American or Pacific Islander" and "other." Respondents were primarily doctoral degree holders; 92 reported their highest education level as the Ph.D. or other doctorate, and 74 reported holding M.D. degrees (several reported holding both the M.D. and the Ph.D.). The majority reported working in research or teaching settings, often in combination with clinical work. Forty percent of the respondents listed laboratory, clinical, or field-based research as their primary occupation, although many were also involved in clinical practice or teaching. Only 14 percent of respondents listed teaching alone as their primary activity, and only 16 percent listed clinical practice alone as their primary work. The majority (52 percent) reported working for a nonprofit educational or research institution.
An example of the survey is provided in Appendix D. Survey respondents were asked to provide demographic and educational information about themselves (race or ethnicity and highest degree obtained), as well as information about the nature of their current work (primarily research, teaching, clinical practice, or other) and employer (federal, state, or local government; nonprofit educational or research institution; health care service delivery; or other). In addition, respondents were asked whether they had submitted a research proposal to NIH for funding, whether they had responded to an NIH Request for Applications (RFA) or Program Announcements (PA), and whether they had received funding in the form of either a research or program grant or a training grant. Individuals who applied for funding were asked to rate and comment on their experience with the process.
In addition, respondents were asked how familiar they are with the NIH research priority-setting process and whether they had provided input into this process, either formally (via service on an NIH advisory body) or informally (e.g., ad hoc feedback to NIH staff).
Respondents were asked to rate and provide their perception of the receptivity of NIH to research proposals focusing on the health needs of minority and medically underserved populations, of the value that NIH places on research relevant to these groups, and of the value that NIH
places on the training of minority scientists. Subsequent questions asked respondents to provide examples of NIH programs that they feel have worked well in addressing the health needs of minority and medically underserved groups and to provide specific recommendations on how NIH might improve its approach to addressing research among these populations. Finally, respondents were asked to provide any additional comments in areas of the study charge.
The majority of respondents (74 percent) reported having submitted a research proposal to NIH or its institutes at some point in their careers. Nearly two-thirds (66 percent) reported having responded to an RFA or a PA from an NIH institute. More than half (58 percent) reported that they had received some form of research support from NIH. Only 16 percent reported having received research training funding from NIH or its institutes.
The survey questions tended to elicit widely varying responses. In some cases, responses appeared to be based upon a lack of information about NIH programs and resources, suggesting that outreach programs to targeted groups, such as underrepresented minority investigators, had failed to reach many of them. For example, several respondents expressed frustration that no research funds were available for minority investigators, especially young researchers transitioning from graduate or postdoctoral work to faculty positions. In fact, NCI sponsors several programs geared to young investigators and new faculty (see Chapter 3). For these survey respondents, however, the absence of such programs was viewed as detrimental to the development of ethnic minority cancer researchers, as noted in the comments of one respondent: "NIH seeks to train minority scientists yet fails to ensure adequate support once scientists reach a faculty position. A transition period of three years could make a big difference."
Peer Review and Priority Setting
With regard to the experiences of investigators applying for NIH research funds, many comments appeared to be critical of the process generally, irrespective of whether the proposal was specific to minority and medically underserved populations. "Deadlines are (after PAs) not realistic. Redundant paperwork," wrote one respondent, whereas another respondent noted, "The criticisms by and large focused on trivial details and often reflect ignorance of the reviewer in a particular field." Others found NIH staff to be unresponsive in the process. "Study section administrations
generally [are] not helpful," wrote one respondent. This sentiment was echoed by others: "The peer review process is increasingly idiosyncratic, with the review being conducted by junior investigators who have little experience in research," and "No real helpful input as to how to make the proposal fundable, and a very impersonal experience. No direct communication or discussion." Another respondent described proposal review as a "triage process" whereby staff make decisions regarding whether proposals fit RFAs, thereby limiting the number of proposals that advance.
Many others, however, offered praise for the peer-review process. "Excellent program directors," wrote one respondent, who continued: "Weakness of numerous staff changes over [the past] six years but quality of support is excellent." "Comments were helpful," wrote another and the, "Executive Secretary was helpful."
Significantly, several respondents pointed to aspects of peer review and the priority-setting process that are particularly problematic for minority researchers or those studying issues relevant to minority and medically underserved populations. Among the comments that reflected this point: "I believe in the peer review process for the most part. In times of budget constraints, funding decisions are sometimes skewed toward the more established scientists. This is not good for budding investigators." "[The] process [is] not supportive of community-based and community-driven research." "Big drawback [of the peer review process] is focus on researchers who have already done this—NIH reinforces success, [making it] very hard to get started." "Program staff and contracting staff were helpful…. However, few at the top understand the need to fund research adequately or address the resource issues that (minority) researchers face in conducting cancer research in minority and medically underserved communities." ''Good in general. Sometimes [there exists] a perception that connections and old school ties are more important than science." Several others felt that it was particularly difficult for investigators at historically black colleges and universities (HBCUs) to successfully compete for funding.
Few respondents reported attempting to provide input into the NIH research priority-setting process, but several commented on the process. Some comments reflected a theme similar to that described above: that minority investigators feel "shut out" of the process. "This is an 'old boys' network [and] it is difficult for a minority woman to…approach," wrote one respondent. "Have more of the review[ers] coming from HBCUs and breaking 'good-old-boy networks' among large white institutions," wrote another. Still others advocated that the level of representation of researchers from minority and medically underserved communities on NIH advisory and policy boards should be increased. "Appoint more minority investigators and administrators to key advisory committees and review
committees or study sections," "Include more qualified minorities in goal setting and identification of priority areas as they pertain to minorities," and "Include more minorities on review panels" were among the comments supporting this concern.
NIH Receptivity to Special Populations Research
Few respondents felt that NIH was receptive to research proposals focusing on the needs of minority and medically underserved populations. One respondent noted that "they [NIH] seem not to understand minority health issues" and added that there exists a critical need for researchers to work in partnership with community groups. The following comments reflect this sentiment: "NIH appears to be receptive, but funding priorities often place these [minority-and underserved-oriented] proposals at the end of the funding chain…. [T]he funding is often inadequate. Programs are funded at a level to fail." Research on minority and medically underserved populations "appear[s] to be a low priority or not a primary concern of many institutes.'' "Depending on politics—sometimes the need [for minority and underserved research] is not well received. [The lack of research in this area] is rationalized through issues of 'no earmarks' or [poor] quality of proposals submitted." A minority of respondents, however, were more positive about NIH's receptivity to special populations research: "I think it is improving thanks to a center [sic] overseeing minority research," wrote one respondent, whereas another respondent noted that NIH is "flexible enough to be reactive [to minority health research needs] within constraints of research-based programs."
Similarly, many respondents felt that research on minority and medically underserved communities was not a high priority for NIH. Priorities are "biased toward academic institutions," wrote one respondent from a community-based group. Among the comments of other respondents: "NIH recognizes the need for research relevant to minority and medically underserved communities, but does not invest in building the appropriate mechanisms to fund such research. Investment must be in the development of research training programs for minority researchers and … in the area of community-driven research." "Some specific programs [relevant to minority and medically underserved populations] have been terrific but then are ended." Special populations research is "a very recent concern [and] not a priority of Dr. Varmus."
Respondents were more positive regarding NIH training programs for minority scientists, and the value that NIH places on these. "I believe NIH
has made special efforts to provide training for minority graduate students and scientists," commented one respondent. Many were critical, however, of perceived gaps in funding and programs at two critical ends of the training "pipeline": outreach programs for promising high school students from minority and medically underserved communities and support for young researchers transitioning to faculty positions. Among the comments supporting this position: "Excellent options for training. Nothing specific for junior faculty." "NCI could improve the support of minority clinical scientists, especially during the transition period to independent investigation." "NIH seeks to train minority scientists yet fails to ensure adequate support once scientists reach a faculty position. A transition period of three years would make a big difference." Still others believe the problem of encouraging more individuals from underrepresented groups into the sciences is a problem beyond NIH's resources. Among the comments reflecting this view: ''There is a framework for training minority scientists; however, there is an insufficient critical mass of either applicants or mentors to make the process work," and "There is a great deal of effort going on into recruiting and training minorities and scientists [sic]. There is, however, a scarcity of candidates that hinders the best NIH efforts."
Many respondents listed minority training programs, however, as among the NIH programs that have worked well to address the research needs of minority and medically underserved communities. Several respondents pointed to NCI's Minority Supplement and Minority Access to Research Careers and Minority Biomedical Research Support training programs as highly effective in preparing well-trained minority scientists for work in cancer research fields. NCI programs that offer funds to assist young scholars—criticized by some in this survey as nonexistent—were praised by others. "The NCI program to support the transition from post-doctoral positions to faculty positions is excellent. It should be supported and extended to other NIH institutes," wrote one respondent. Many other respondents stated that the quantity and quality of cancer research among minority and medically underserved populations would increase as the number of scientists from these groups increased, suggesting that training programs are viewed as a critical link to improving research.
Other respondents pointed to NCI's Leadership Initiatives—the National Black Leadership Initiative on Cancer, the National Hispanic Leadership Initiative on Cancer, and the Appalachian Leadership Initiative on Cancer—as very effective in addressing the cancer control and research needs of these populations. Finally, other respondents pointed to specific
programs as having worked well to address the needs of special populations, including NCI-sponsored smoking cessation programs.
The respondents made recommendations in several general categories. These are summarized below. Representative comments related to each of the recommendations are presented in Appendix D.
Involve community members and community-based researchers as partners in the research process.
Many respondents felt strongly that community leaders and grassroots community health organizations offer invaluable expertise in assisting researchers who are planning community-based research. Community members are an underused resource who can assist in the design and implementation of research strategies, but only if they are involved as full partners in the process. NIH should encourage community collaboration, in the view of many respondents.
Improvements should be made in training and grant programs to increase the capacity for scientific research among minority and medically underserved populations.
Many respondents felt that the quality and quantity of proposals for health research among minority and medically underserved populations will improve only when the numbers of minority scientists are increased. Although most respondents praised NIH's minority scientist training programs, many urged greater attention to the needs of minority scientists at early career stages (i.e., at the postdoctoral and junior faculty stages of their careers). Others advocated for more outreach efforts to inform minority scientists of research funding opportunities and mentoring for successful grant competition.
Scientists from minority and medically underserved communities should be involved in the NIH priority-setting process and in staffing of NIH positions.
Several respondents pointed to a lack of cultural sensitivity and awareness of the needs of ethnic minority and medically underserved communities on the part of NIH scientific review groups and staff as a key impediment to increasing the number of programs targeted to these populations and funding research targeted to these populations.
Involve community members and community-based researchers as partners in research priority-setting.
- As in Recommendation 1 above, some respondents felt that the knowledge of community leaders and grassroots health advocates could be better used by NIH if these individuals were consulted in the research priority-setting process. Community members' knowledge of the health problems, resources, and needs of their communities would not only help to better inform NIH of the most pressing issues faced by minority and medically underserved communities, but such consultation and involvement in the priority-setting process would foster greater collaboration and community participation in research.
Research issues for minority and underserved populations must be integrated into a national cancer research agenda.
Some respondents questioned whether research on minority and medically underserved populations was a significant component of a national cancer plan. These respondents stressed that research among minority and medically underserved populations should not be an "afterthought" or compartmentalized within limited areas of NIH's research portfolio but should be integrated into areas of key focus.
Define special populations research more adequately.
Several respondents were critical of how "special populations" are defined at NIH, arguing that a better assessment of "underserved" groups is needed. Some argued that use of the term minority is pejorative and should be replaced in favor of terminology that more accurately describes cancer risk.
Involve institutions serving ethnic minority and medically underserved communities in cancer research.
Several respondents argued that institutes serving minority and medically underserved communities, such as HBCUs and institutions that serve the Hispanic populations, have the greatest ability to perform culturally appropriate research with minority and medically underserved populations. In addition, these institutions would have greater access to minority and medically underserved study populations and have greater opportunities to train ethnic minority scientists and researchers. Respondents urged NIH to make greater investments in these opportunities.
What are the Needs of Cancer Survivors in Ethnic Minority and Medically Underserved Communities?
To understand the needs of cancer survivors in ethnic minority and medically underserved communities, the committee received testimony from representatives of several community-based cancer prevention and
health promotion organizations, as well as testimony from an individual at an academic center who has spearheaded several community-based health efforts, primarily in low-income, rural communities. These individuals were asked to comment on how well the NIH research results are communicated and applied to cancer treatment and prevention programs for minority and medically underserved communities.
Many of these individuals spoke from personal experiences as cancer survivors who sought information for themselves and their communities and who would address the specific needs that they faced. Zora Kramer Brown is founder and chair of the Breast Cancer Resource Committee, which addresses the prevention and treatment needs of African American women at risk of or living with breast cancer. The organization plans future initiatives to address cancer among African American men. Venus Gines is a 6-year survivor of breast cancer, a community activist from Atlanta, Georgia, and a member of the NCI Director's Consumer Liaison Group. Lucy Young is a 10-year survivor of breast cancer. After recovery, she founded the Chinese American Cancer Association (CACA) in Flushing, New York, to support and meet the needs of immigrant cancer patients, especially those with language difficulties.
Finally, Barbara Clinton, director of the Center for Health Services at Vanderbilt University, spoke from the perspective of her 17 years of work with community-based health initiatives in Tennessee, Kentucky, Virginia, West Virginia, Florida, Louisiana, and Arkansas. Much of her work involves mobilization and linkage of community resources to address health needs, including the promotion of lay health advisers, the provision of technical assistance to communities, and other efforts to assist low-income and medically underserved populations. Two other individuals with expertise in the survivorship needs of ethnic minority and medically underserved communities, James Williams (executive director of US-TOO, International, an organization focused on the needs of prostate cancer survivors) and Yvette Joseph Fox (executive director of the National Indian Health Board), were invited to discuss their perspectives and work with the committee but were unable to attend.
Zora Kramer Brown noted that African Americans have a greater vulnerability to cancer than the general population. African Americans contract cancer at earlier ages than the majority population and suffer from higher cancer mortality rates. Data are needed, she stated, to understand why these disparities exist: "Is it genetically based? Is it environmentally based? Is it dietarily based? Is it the result of cultural values or belief systems, or is it economically based?" she asked.
Brown stated that "by and large, the National Institutes of Health and other federal agencies have been most supportive of these [community and advocacy] groups. The Breast Cancer Resource Committee, in fact,
has enjoyed a productive collaboration with NIH regarding breast cancer education and survival issues among African-American women … [F]ederal agencies, generally, have been particularly supportive in helping to communicate research data to minority communities, in supporting education and early detection in underserved populations, and in addressing to some extent other overall survival needs of cancer victims."
She added, however, that NIH must improve its record with regard to the accrual of minority populations in clinical trials, in establishing collaborations between comprehensive cancer centers and historically black medical schools, and in increasing the participation of minorities in cancer review panels (see below).
Venus Gines noted that in the Latino culture, feelings of fatalism regarding cancer are high: cancer is viewed as a "death sentence." This fatalism prompted Gines to seek more information regarding her breast cancer diagnosis of 6 years ago. She found very little information in Spanish, her native language. Much of the information was merely translated from English and contained little information about cancer in Latinos. "In order for me to get the important data that are necessary for scientists to find out about Latinos and breast cancer, we need people who are of the culture who can help."
Gines developed Mi Nueva Esperanza (My New Hope), which provides education regarding breast cancer to Latina women in the form of a picture book with writing in clear, simple Spanish. More than 3,000 copies have been distributed nationwide, including in migrant worker camps. This effort was supported in collaboration with the American Cancer Society.
Gines also organized a health fair in the Atlanta, Georgia, area, in collaboration with Hispanic community organizations. This health fair offered clinical breast examinations and mobile units to provide mammograms and Pap smears. Child care was provided, and other barriers to participation were addressed.
Lucy Young founded CACA after finding little information in her native language as she battled breast cancer. In addition to helping Chinese-American cancer survivors to develop support networks, the group sponsors educational fora and develops and translates educational materials. CACA also offers free home care and free transportation services to low-income cancer patients. CACA was the first of the 3,500 local units sponsored by the American Cancer Society to focus on the needs of Asian American populations.
"Through my experience, I realized one thing," she stated, "that in suffering, one can sometimes discover one's life potential, as well as how to develop it. Ten years ago when I learned I had breast cancer, I asked God, 'Why? Why me? Why now?' But today, I know the answer."
Barbara Clinton has worked to develop and support community-based health initiatives ranging from maternal and infant health promotion to cancer prevention and screening. She has been extensively involved in the development of models in which lay health advisers are used for health intervention purposes. Much of this work takes place in rural communities in Appalachia. Clinton noted the link between cancer and environmental carcinogens, noting that "the South is the nation's biggest hazardous waste dump," especially in low-income communities in the South. She also addressed the need for greater provision of information from NIH to managed care organization, and for research on the use of lay health advisers.
The panel highlighted several themes and recommendations, as summarized below.
1. Inclusion of minorities in clinical trial research must be a high priority at NIH.
Zora Kramer Brown provided an example of how a lack of inclusion of minority women in breast cancer screening trials led to recommendations from an NIH Consensus Panel that may be appropriate for many women but not minority women. The Panel concluded that evidence was lacking to recommend breast cancer screening among women ages 40 to 49, but Brown stated that these data ignored the fact that the greatest increase in breast cancer is among African American women under the age of 40. The Panel's recommendation was later reversed, but the initial recommendation was based on studies from Sweden, Canada, and England that did not include African-American women.
"We have not done a good job of convincing the African-American community that clinical trial research actually does benefit them," said Brown. "And in order to do that there has to be education that is coupled with it. And I do believe that education is research." Brown added that minority patients and their providers need to better understand how to gain access to clinical trials.
Brown draws a distinction between poverty, illiteracy, and cultural beliefs. Poverty and illiteracy may pose barriers to clinical trial accrual, but these are not cultural factors, she indicated. NIH needs to attend to poverty and illiteracy, but it should not ignore cultural factors that may pose barriers to accrual in clinical trials.
2. Collaboration between comprehensive medical centers and historically black medical schools should be fostered.
Most clinical research is conducted in large research hospitals that do not serve African Americans, according to Brown. African Americans do, however, frequent hospitals associated with historically black colleges and medical schools. This limits NIH's ability to access black populations and
incorporate findings from African-American researchers and institutions. "A teaming of representatives from our majority comprehensive medical centers with representatives from our HBCU institutions and facilities has the potential to greatly advance our understanding of cancer incidence and mortality among African Americans," she stated.
Brown pointed to the success of National Human Genome Research Institute-Howard University collaboration on genetically based risk among African Americans as an example of such a collaboration.
3. Greater participation of minorities in cancer review panels is needed.
Brown noted that NCI has simple nondiscrimination provisions in its requirements for the selection of cancer review panels. The U.S. Department of Defense, in contrast, actively recruits minorities for scientific review panels, with minority population representation on some panels being up to one-third or more of the total. Inclusion of minorities on panels can increase NIH's sensitivity to proposals from scientists, particularly minority scientists, who study minority populations, she noted.
4. The portfolio of "special populations" research at NIH must adequately address the range of possible etiologic factors for cancer among racial and ethnic groups.
Brown criticized the NIH portfolio of research on cancer etiology among racial and ethnic groups as too narrow and failing to examine possible biological or genetic differences. "The argument that the results of basic research can be extrapolated to all populations simply does not apply," she stated. "In fact, it is entirely inconceivable that the results of studies [with] African Americans, upon expanded scrutiny, might be legitimately extrapolated to one or more other ethnic groups."
"I have a concern that, in the absence of definitive results from other factors," she continued, "the standard explanation for cancer discrepancies in African Americans might become too comfortably and conveniently couched in terms of culture and economics…. [W]e owe it to the African-American population and to the nation to either rule out or to identify definitively the other factors to which I have alluded."
5. NIH staff must reflect the diversity of the U.S. population.
Brown stated that staff at NIH need to include minorities so that staff are representative of the populations that they study.
6. NIH must improve educational and outreach efforts to minority communities.
Venus Gines criticized NIH outreach efforts as inadequate. "I think
what we need is more visibility of NCI people," she stated, "because in Atlanta, for instance, you mention NCI, nobody knows the National Cancer Institute [in] our community. They're up there in an ivory tower maybe, but they really don't know what the National Cancer Institute does. They hear [of] the American Cancer Society because of all the [information about] Relay for Life, and all this has been in the news. But NCI needs to be a little more visible in the communities, I think, for one thing."
Zora Kramer Brown added that health providers in minority communities are often overburdened. She also stated that NIH needs to be sensitive to the special circumstances and outreach needs faced by health providers and health educators in these communities.
Barbara Clinton stated that issues of understanding and addressing a diagnosis of cancer for many ethnic minority and medically underserved individuals are complex and require the patient to negotiate culturally and institutionally imposed barriers to the retrieval of information. Information should therefore be tailored to these needs, she indicated.
7. Greater sensitivity to culturally appropriate outreach efforts is needed.
Venus Gines noted that cancer prevention and control messages must be tailored to ethnic minority communities, a task that is often more complicated than it appears. "We should try to stay away from just straight translating from English to Spanish. We need to really focus on the culture, and I think that's very important."
Both Venus Gines and Lucy Young also noted the need for cancer prevention and outreach messages to address potential cancer stigma and "taboo" issues among minority communities. In the absence of having cancer survivors from these communities as visible role models, they stated, many ethnic minorities with cancer may not want to be publicly identified. Young added that many Chinese Americans living with cancer may not want others in the community to know of their condition.
8. Follow-up services should be made available for individuals after cancer screening.
The need to provide follow-up services, especially for ethnic minority patients for whom late diagnosis is a problem, is very important. Venus Gines described her frustration in trying to find appropriate follow-up services for women screened at the Atlanta community health fair with abnormal Pap smear results.
9. A strategic plan to address the survivorship needs of ethnic minority communities should be developed.
Lucy Young recommended that NCI form a diversity team to develop ongoing task force committees to develop strategic plans for different ethnic
groups. "These committees should collect and gather information on cancer-related grassroots organizations to identify their needs," Young said. "Then, submit a proposal of suggestions to NIH or NCI to better serve the different ethnic groups." Population groups such as "Asians" should not be lumped together, because Chinese, Korean, Japanese, and other groups are culturally different.
10. Community-based groups need assistance in grant writing to be competitive for NIH grants.
Lucy Young, Barbara Clinton, and others agreed that community-based organizations with innovative cancer control or research proposals should be encouraged to submit proposals but that they often lack the time, resources, or skills to do so. NIH should provide technical assistance to these groups to improve their ability to compete for grants.
11. Environmental risk factors disproportionately affect ethnic minority and medically underserved groups, and therefore should be prominent in the NIH portfolio.
"At the community level, cancer prevention is inextricably linked to environmental degradation," noted Barbara Clinton. Scientists should move "beyond the notion that personal health behavior alone explains the increased mortality in low-income populations." NCI should "investigate the relationship between environmental exposures that occur early in life and the development of adult diseases as well as transgeneration effects that occur in the child of the person who was exposed to the environmental toxin."
Clinton also recommended that NIH "support leadership exchanges between staff of the NCI and national and local environmental organizations." Finally, she noted, cancer control research should support existing community-based efforts to address environmental issues.
12. NCI should expand investigations of lay community health workers, who may be especially effective in addressing the needs of cancer survivors in minority and medically underserved communities.
Lay community health workers, according to Barbara Clinton, provide locally tailored and culturally sensitive information to patients on subjects that are confusing and sometimes taboo. "These workers help people access available resources in cost-effective ways," she noted. NCI should also expand research on health navigators, who help patients understand treatment options, providers, and other choices. Research questions should investigate how and under what conditions these providers work best.
13. NCI should provide information to managed care organizations
to provide them with up-to-date information regarding best practice outcomes and cost data.
Clinton noted that "HMO [health maintenance organization] decision guidelines often deny payment for any treatment that is not approved by the FDA [Food and Drug Administration] on the grounds that the treatment is experimental, even though trial treatment may be a better and even more cost effective option for the patient." HMO coverage of clinical trial treatment may improve the accrual of minority patients.
Mechanisms for Ethnic Minority Community and Researcher Input into the Cancer Agenda
The perceptions and experiences of community representatives and grassroots leaders, as well as those of researchers interested in cancer among minority and medically underserved groups, are important in helping to provide an understanding of NCI's mechanisms for the involvement of these constituencies in research priority setting. NCI's response to minority community and researcher input is led by the recently established Director's Consumer Liaison Group (DCLG) and the Office of Special Populations Research (OSPR). At the NIH level, the effort is led by the Office of Research on Minority Health (ORMH).
Office of Research on Minority Health, NIH
As noted in Chapter 3, ORMH, established by the director of NIH in 1990, was authorized by the U.S. Congress in 1993 (P.L. 103-43). Its mission is to improve the health status of minority Americans through biomedical research and to expand the participation of minorities in all aspects of biomedical and behavioral research. Part of its responsibilities includes an ongoing consultative process with outside organizations, a process that ORMH initiated in 1991 with the establishment of a 53-member fact-finding team. More recently, Congress has mandated that an Advisory Committee on Research on Minority Health be established to advise ORMH and NIH (this Advisory Committee is described in Chapter 3). ORMH reports that of the 9 members currently impaneled on the Advisory Committee, 6 are ethnic minorities (12 members are ultimately expected to be appointed).
Prior to passage of the congressional mandate for the establishment of an ORMH advisory panel, however, ORMH's research priority-setting processes appeared to be based largely on the research agendas of collaborating NIH institutes and centers (ICs) and the views of an ad hoc review panel, as noted in Chapter 3. Although a broad framework of 13 minority health research priorities was established by the advisory fact-finding team
and was published in 1992 (National Institutes of Health, Office of Research on Minority Health, 1992), and six ''priority areas" for funding minority health and training initiatives were established on the basis of "continuing consultation with the minority community and experts within the ICs" (Ruffin, 1996), criteria for the prioritization and funding of research on specific diseases such as cancer are not explicit. Further, it is apparent that ORMH and its newly established advisory body will be able to more effectively leverage NCI's resources to support minority cancer research programs if its NCI analogue, OSPR, is afforded greater authority and responsibility in establishing and implementing a minority cancer research agenda, as will be discussed below.
Office of Special Populations Research, NCI
OSPR grew out of a report of an NCI Special Action Committee, an internal group of program, planning, and management staff from all NCI divisions formed to ensure that the cancer research needs of special populations were being adequately addressed (National Cancer Institute, 1996a). Reports were presented in 1990, 1992, 1994, and 1996, with the last report recommending the abolishment of the committee and the establishment of a focal point within NCI's Office of Program Operations and Planning to provide leadership and to coordinate activities addressing the cancer research needs of special populations, including a multiethnic advisory committee made up of NCI staff that would replace the Special Action Committee. OSPR is also establishing an external liaison committee consisting of scientists, medical professionals, civil rights advocates, and others involved in minority health issues (O. Brawley, National Cancer Institute, personal communication, May 29, 1998). OSPR also collaborates with ORMH to establish research priorities, but OSPR lacks the appropriate funding mechanisms or program authority to exert influence over research priorities at NCI. OSPR and its advisory body therefore provides guidance within NCI, but without program funding or authority, it is relatively powerless in working with ORMH or acting independently to effect meaningful changes in the research agenda at NCI.
Director's Consumer Liaison Group, NCI
In 1997, the NCI director established DCLG to provide advice and make recommendations to the Advisory Committee to the Director of NCI. (The Advisory Committee is chaired by the NCI director and includes the chair of the National Cancer Advisory Board and the chairs and co-chairs of the NCI Board of Scientific Counselors and Board of Scientific Advisors.) NCI's Office of Liaison Activities (OLA) coordinates and supports
the activities of DCLG. OLA's goals are to create and maintain ongoing communications and information exchange between the national cancer advocacy organizations and NCI and to cooperate and collaborate with these groups in areas of mutual interest (National Cancer Institute, 1998f).
The purposes of DCLG are (1) to help develop and establish processes, mechanisms, and criteria for identifying appropriate consumer advocates to serve on a variety of program and policy advisory committees responsible for advancing the mission of NCI; (2) to serve as a primary forum for discussing issues and concerns and exchanging viewpoints that are important to the broad development of NCI programmatic and research priorities; and (3) to establish and maintain strong collaborations between NCI and the cancer advocacy community to reach common goals (National Cancer Institute, 1998f). DCLG is also described in greater detail in Chapter 5.
The Planning Group that led to the implementation of DCLG specifically addressed the means of achieving appropriate diversity within DCLG and wanted to ensure multicultural representation among DCLG's 15 members, along with other important characteristics among its members, such as individuals of both genders and of various ages, individuals with cancer at different sites, and individuals from various types of organizations. The Planning Group would have stipulated that at least one-third of DCLG members belong to a racial and ethnic minority but was precluded from doing so by a federal law prohibiting the selection of individuals on the basis of race. Thus, the Planning Group's final recommendation was that DCLG's membership: (1) be culturally diverse, (2) include individuals with cancer at different sites, (3) include medically underserved individuals, (4) include men and women, (5) include individuals from a range of organizations (local, regional, and national), (6) include individuals of various ages, and (7) include individuals from diverse geographic locations (including individuals from rural and urban areas). The initial DCLG includes African-American, Asian-American/Pacific Islander, Hispanic, Native American, and non-Hispanic white members (National Cancer Institute, 1998f).
One of the express purposes of DCLG was "to help NCI widen the pool of qualified consumer advocates who can be called upon to serve on NCI advisory committees and other groups" (emphasis added; National Cancer Institute, 1998f). Thus, at least the stage is set for participation of a more diverse and expansive array of minority community representatives in NCI's policy-making and priority-setting processes.
Other NCI Advisory Panels
No strategy for increasing the pool of qualified minority scientists to
serve on NCI's advisory committees and other groups comparable to that established for consumers through DCLG currently exists. NCI's senior management turns to seven different advisory groups for advice and counsel: (1) the President's Cancer Panel, (2) the National Cancer Advisory Board, (3) the Board of Scientific Counselors, (4) the Board of Scientific Advisors, (5) the Intramural Advisory Board, (6) the Extramural Advisory Board, and (7) the DCLG. Currently, 19 percent (21 of 113) of the people serving on these groups are members of minority groups—9 Asian Americans/Pacific Islanders, 7 African Americans, 4 Hispanics, and 1 American Indian/Alaska Native—whereas that proportion was 14 percent (19 of 134) in 1992. There is wide variability, however, in ethnic minority representation among the various advisory groups. More than one-third (35 percent) of the National Cancer Advisory Board members (6 of 17) were ethnic minorities as of December 1997, yet among NCI's two most powerful advisory bodies, the Board of Scientific Advisors and the Board of Scientific Counselors, only 11 percent (3 of 26) and 17 percent (7 of 42) are ethnic minorities, respectively. NCI reports that consumers are represented on the majority of NCI advisory panels, but the numbers of these individuals on each panel were not specified.
It therefore appears that significant representation on advisory panels by members of ethnic minorities takes place primarily when NCI embraces a conscious plan for diversity: witness the original intent of the Planning Group for DCLG to have one third of its 15 members be ethnic minorities and the final makeup of the group, which includes representatives from five different minority groups.
Concern regarding ethnic minority representation on NCI advisory panels was also salient in the input to the committee from ethnic minority researchers in the field of cancer research, as well as from minority and nonminority investigators interested in cancer among minority and medically underserved populations. As indicated above, opinions about the ways of increasing the level of representation included (1) increased participation by researchers from minority and medically underserved communities in goal setting and identification of priority areas pertaining to minorities, (2) appointment of more minority investigators and administrators to key advisory committees and review committees or study sections, and (3) development of an advisory board to address the research needs of minority and medically underserved communities. Thus, a comparable strategy for widening the pool of minority investigators (and administrators) who serve on policy-making and priority-setting groups within NCI, as has been developed for consumers through DCLG, is an apparent need.
Recommendation 4-1: NCI should develop a process to increase the representation of ethnically diverse researchers and public representatives
serving on all advisory and program review committees so that the makeup of these committees reflects the changing diversity of the U.S. population. NCI should develop an evaluation plan to assess the effect of increased and more diversified ethnic minority community and researcher input on changes in NCI policies and priorities toward ethnic minority cancer issues.
Such increased and more widely based minority community and researcher input is not the desired end result but is one of many means of marshaling the considerable resources of NCI in reducing the burden of cancer among minorities and medically underserved individuals in terms of fewer deaths, fewer new cases, increased lengths of survival, and increased quality of life among cancer survivors. Such input at least ensures that ethnic minority and medically underserved populations will have a (substantial) voice at all levels of discussion in NCI's agenda. The committee believes that the impact of such a policy shift should be assessed to determine whether it will ultimately have a significant effect on NCI's policies and priorities.
Recommendation 4-2: The research needs of ethnic minority and medically underserved groups should be identified on the basis of the burden of cancer in these populations, with an assessment of the most appropriate areas of research (i.e., behavioral and social sciences, biology, epidemiology and genetics, prevention and control, treatment, etc.).
Another aspect of the potential influence of minority community and researcher input into NCI is the environment within which such input operates. NIH and NCI operate within a primary milieu of "greatest scientific opportunity." In the words of NCI's director: "The NCI's cancer research funding strategy is to assure that there is sufficient funding to enable scientists to pursue those research areas with the greatest scientific opportunity—that is, the greatest opportunity to increase our knowledge of cancer" (National Cancer Institute, 1998d, p. 26).
However, the director also acknowledges that "a second critical factor that guides program direction, in addition to scientific opportunity, is the burden of specific cancers" (National Cancer Institute, 1998d, p. 26). This "second critical factor" is essential for addressing the cancer problems of minority and medically underserved populations because NCI has acknowledged that ''while we have seen a decline in overall cancer prevalence and mortality rates in the last five years, the decline did not occur in all American populations. Overall rates do reflect a story of success based on statistical averages, but when we examine specific ethnic and racial groups, and
those in underserved situations, we find that declines in cancer incidence and death have not occurred for all populations" (National Cancer Institute, 1998b, p. 1).
Acknowledgment of the role of "burden of disease" in guiding NCI program direction and the acknowledgment that declines in cancer incidence and death have not occurred for many minorities are significant in view of a recommendation of the NCI Special Action Committee's 1996 report, which led to the creation of OSPR. In that report, the committee also recommended that "the NCI research portfolio needs to be [data driven and] reviewed with a focus on the cancers noted as significant in the minority cancer statistics monograph. Cancers that disproportionately affect special populations should be carefully examined to identify research opportunities…. [T]he results of such a review should be discussed in the broader context of NCI's mission under the auspices of the National Cancer Advisory Board and the Boards of Scientific Advisors/Counselors" (National Cancer Institute, 1996a, p. 11). The committee concurs with this recommendation, as will be discussed below in greater detail in a review of NCI and NIH mechanisms for research priority setting.
Recommendation 4-3: For NCI to address the needs of ethnically diverse and medically underserved populations effectively, the Office of Special Populations Research (or some other designated entity or entities) must possess the authority to coordinate and leverage programs and resources across the divisions and branches of NCI to stimulate research on ethnic minority and medically underserved populations. This authority can be established by providing such an office with:
- a leadership role in major NCI-wide priority-setting bodies and
- special resources to fund programs specifically targeted to these populations, or
- accountability for the institution-wide allocation of program resources.
Special Populations and Priority Setting at NCI
The evaluation of priority setting for research on cancer among special populations must be undertaken against the backdrop of three basic considerations. First, the fundamental missions of NCI and NIH frame the larger public policy context within which issues such as the types of research and levels of funding commitment for special populations should be judged. Second, a clear and persuasive rationale for any special research
focus on specific subpopulations must be articulated, and the efforts to address the needs of special populations should conform to that rationale. Third, the criteria and procedures used to set research priorities for the general population must be examined with an eye to their adequacy to priority setting for special populations. This section discusses these three issues.
The mission statement of NIH states that its primary aim is research designed "to uncover new knowledge that will lead to better health for everyone." The mission statement of NCI embodies similar institutional goals tailored to its specific disease focus. The ultimate aim of NCI, according to a 1998 NCI report, Priority Setting at the National Cancer Institute (National Cancer Institute, 1998g) is "to prevent or cure cancer." Given the quite general language of the NIH and NCI mandates, a wide array of policy options and research priorities is compatible with fulfillment of their missions. Accordingly, the NCI report emphasizes that priority setting is necessary "because there are more mission-related things that could and should be done than there are resources available to do them'' (National Cancer Institute, 1998g, p. 7).
A three-pronged approach is outlined in NCI's fiscal year 1999 bypass budget:
Sustain at full measure the proven research programs that have enabled NCI to come this far.
Seize extraordinary opportunities to further progress made possible by previous research discoveries.
Create and sustain mechanisms that will enable NCI to rapidly translate findings from the laboratory to practical applications that will benefit everyone.
NCI's approach reveals three especially noteworthy principles relevant to this committee's charge. As indicated in more detail below, these principles themselves offer no definitive guide to priority setting, but they do point the way toward reducing some common misunderstandings that sometimes color the public debates about the relation between NCI's mission and its choice among the many policy options in service of its mission.
First, the central aim of NIH and its institutes, including NCI, is the production of new knowledge about the causes, prevention, and cure of cancer. Both NCI and NIH emphasized the acute need to focus on the production of knowledge that might not otherwise be generated without the support of public agencies. This rationale is often articulated as the
basis of a preponderance of emphasis on funding basic research that would not find sufficient support in the marketplace when no immediate financial rewards are expected. However, this principle has particular importance for setting research priorities for special populations as well.
For example, if knowledge about the etiology of cancer among low-income populations with greater than average exposures to suspected environmental contributors to an increased cancer incidence or increased mortality from cancer is known but studies are unlikely to be conducted within the private sector, then there is at least an initial rationale for giving research of this type the same careful consideration in the priority-setting process as other research that lacks commercial incentives. Indeed, not only may such research lack commercial incentives but there may also be commercial disincentives such that without sufficient commitment by public agencies, many scientific opportunities to learn about the fundamental causes and etiology of cancer may be missed.
Second, because the ultimate aim of publicly supported research is the improvement of the public's health, any and all types of research—whether it falls within traditional contrasting categories, such as basic or applied research, behavioral or molecular research, or fundamental or translational research—are appropriate candidates for consideration in the priority-setting process. From the perspective of a purely public health approach (again, this is not included as the general perspective that NCI should adopt), all types of research that may ultimately contribute to improved health outcomes are consistent with NCI's mission. In addition, if scientific opportunities in those areas are identified, fulfillment of NCI's mission dictates their careful consideration.
If, for example, as Richard Klausner, the director of NCI, has testified, more than 50 percent of the burden of cancer is due to behavioral and lifestyle factors; and if these differences frequently appear to be positively associated with differences in socioeconomic, educational, cultural, or other discernible characteristics of identifiable subpopulations, then their consideration within the priority-setting process is warranted. Moreover, if some portion of the increased burden of cancer appears to be attributable to the failure of some subpopulations to adopt proven behavioral modifications that would reduce their cancer burden, then research into effective mechanisms of communication is itself a scientific opportunity that merits thoughtful consideration within the priority-setting process.
Third, as the language of NCI and NIH mandates make clear, the obligations of the public agencies extend to all segments of the public. In the language of the NCI mandate, the aim is to develop "practical applications that will benefit everyone." A consequence of this commitment is an institutional obligation to monitor and revise the research portfolio on an ongoing basis with an eye to those populations whose health needs are
underserved, especially when the differential health burden is attributable, at least in part, to a differential in the advancement of scientific knowledge regarding the causes and prevention of cancer among those subpopulations.
Alternative interpretations of the responsibilities and obligations of publicly supported research agencies exist, however. A narrowly construed focus on public health would dictate priority setting such that the aggregate public health is maximized, even if such policies do not allow some subpopulations to receive the expected benefits of such research. However, the committee rejects this interpretation of NCI's mission for several reasons. First, it is contrary to the plain language of NCI's own mission statement and incompatible with its institutional history and long-standing commitment to using data regarding differential health burdens as one factor among others pertinent to the priority-setting process.
Second, such an interpretation is incongruous with recent expressions of congressional policy with respect to the NIH priority-setting process. For example, the NIH Revitalization Act of 1993 (P.L. 103–43) mandated that women and minorities and their subpopulations be included in all human subject research, and federal regulations implementing this mandate for all NIH-supported research became effective in September 1994. The rationale for greater inclusion in clinical trials is the aspiration that "the manner in which scientific knowledge is acquired be generalizable to the entire population of the United States" (National Institutes of Health, 1994).
Rationale for Focus on Subpopulations
A proponent of crosscutting research as the sole means of addressing the needs of special populations might reject the use of targeted or special population-based approaches on the basis of the assumption that all issues of fairness and comprehensiveness of research policy can be accommodated by a combination of research strategies that are expected to yield information about causes and treatment of cancer within the general population and compliance with existing mandates for inclusion of members of those populations within ongoing clinical studies. Thus, it might be argued that the goal of obtaining broad generalizable knowledge is best met by inclusionary crosscutting studies. However, the committee rejects the adequacy of this approach.
Behind the desire for research that yields broad generalizable scientific knowledge is the more fundamental aspiration to interrupt a chain of research policy decisions that can result in some segments of the overall population receiving demonstrably fewer of the fruits of publicly supported research than others. The crux of the rationale for modification of
clinical research guidelines is articulated with great clarity in a 1993 Institute of Medicine (IOM) report discussing issues of inclusion of women in clinical trials (Institute of Medicine, 1993). The report expressed its concern that steps be taken to avoid situations in which subpopulations bear differential health burdens as a partial consequence of disparities in clinical benefit, which are in turn due to disparities in scientific research applicable to conditions affecting them. Although the IOM report did not attempt to resolve the issue of whether or how much of the differential health burden experienced by women could be attributable to exclusionary policies toward women in research, the IOM committee that wrote the report opposed institutional policies that magnify the potential for leaving subpopulations systematically disfavored. In addition to inclusionary policies, the IOM report on the inclusion of women in federally funded research made clear that its underlying rationale supported the consideration of additional, targeted research into conditions that uniquely or differentially affect women.
The present IOM committee endorses the reasoning of the earlier IOM committee and finds that it is applicable to the need for vigilance in identifying and responding to the cancer burden disparities experienced by subpopulations as a fundamental requirement of fairness in the administration of publicly supported programs meant to benefit all segments of U.S. society. The message of the earlier report is one that the present committee reiterates: NIH should be on the lookout for differential health burdens among subpopulations, and when there are scientific opportunities to study the causes of such differential burdens or to reduce their magnitude, such research focused on these subpopulations is a legitimate option for consideration by a publicly accountable agency. In instances in which the differential health burdens are considerable, such options should be given the highest level of consideration within the NCI priority-setting process.
One important difference between the rationale for targeted research among women and the rationale for targeted research among groups defined as special populations in this report should be emphasized. That is, an additional rationale offered as partial support for targeted research among women is the assumption that some diseases are physiologically, hormonally, or genetically mediated by biologically based gender differences. As stated in Chapter 2, however, the committee rejects the view of race as a biologically based concept. Thus, no part of the committee's support for the appropriateness of cancer research targeted to subpopulations rests upon assumptions about the basis of group differences in disease expression similar to those for research targeted to women. Indeed, no analogous biologically based assumption is necessary. As documented in Chapter 2, the existence of differences in the occurrence of disease
among subpopulations may be attributable to numerous causes including environmental exposures, and cultural and behavioral differences, among other factors. Instead, the reasons for such differences are open research question rather than guiding assumptions meant to support targeted research.
Criteria and Priority-Setting Procedures
Thus far, the committee has argued that the statements of mission of NIH and NCI support and in some instances necessitate an important role for consideration of differential health status among subpopulations in the priority-setting process. Moreover, the committee has articulated a more fundamental rationale for the need for special vigilance and attention to the needs of those subpopulations. This rationale rejects the adequacy of approaches that merely seek to address the needs of those subpopulations in the routine portfolio of research initiatives in favor of supplementary strategies that focus directly on subpopulations. This section considers further whether the criteria and procedures used for priority setting are generally compatible with and sufficient for priority setting for the study of cancer among special populations.
The most detailed official discussion of criteria for priority setting used across the 21 institutes that make up NIH are elaborated in its recent pamphlet, Setting Research Priorities at the National Institutes of Health (National Institutes of Health, 1997). In defending what might be called an organic view of scientific discovery, the document states that "science is by nature structured and self-correcting" and that "science itself sets its own priorities" (p. 6). NIH thereby rejects the view that the growth of scientific knowledge can be managed with precision through the use of some topdown planning model or that any simple algorithm can be used to rank those areas of inquiry most requiring funding. Many factors must be taken into account in the priority-setting process, according to the NIH pamphlet; chief among them are the public health needs of the nation and the scientific opportunities or most promising avenues of research available given the state of scientific knowledge developed thus far. Thus, the ultimate choice of which among the many avenues should be explored is a complex one requiring a global appreciation of the current status of scientific research across all major areas of inquiry, the most promising theoretical approaches with crosscutting potential, and an awareness of the many conditions that impose large health burdens in the form of premature mortality, excess morbidity, loss or restriction of functioning, and pain.
The priority-setting policy of NCI (National Cancer Institute, 1998g) tracks that of NIH, often relying on language identical to that used in the
NIH pamphlet. NCI maintains that the decision as to what to study rests primarily on two guiding principles:
Scientific opportunity. What research areas and projects have the greatest potential to expand knowledge? The answer to this question comes primarily from the scientists themselves, working in hundreds of academic, medical, public, and private research institutions around the country.
The burden of cancer. Research funding is likewise guided by the degree to which specific cancers affect Americans. For example, lung and colon cancer, which account for high numbers of cancer deaths each year, are among NCI's priorities.
Although both the NCI and the NIH statements reject any simple algorithm for priority setting, both place great weight on the need for priority setting to be driven largely by scientific opportunities. The aim is to generate new knowledge along a broad frontier, and an underlying assumption behind the desire for a broad and diverse portfolio is the claim that the growth of scientific knowledge is unpredictable. Thus, the ability of science policy experts to develop a systematic plan is inevitably limited and is subject to frequent revision. Incorporating language borrowed from the NIH pamphlet (National Institutes of Health, 1997, p. 6), NCI argues that "the precision with which investigators and administrators describe the targets and outcomes of research … cannot alter the inescapable truth that many of the results of research are unpredictable … Thus, science has the ability to refresh its own priorities in order to seek opportunities that are ripe for pursuit and capture" (National Cancer Institute, 1998g, p. 7).
A cautionary observation contained in the NIH pamphlet points to the difficulty of relying too heavily on disease burden. One worry is that there are many incompatible but equally relevant ways of measuring disease burden. They include the number of people with the disease, the number of deaths caused by a disease, the economic and social costs of a disease, the number of premature deaths caused by a disease, or the extent of pain, disability, or suffering caused by a disease. No single agreed-upon measure of disease burden exists, nor should one exist when research priorities are being set. All measures of disease burden have some moral claim, and the adoption of none is plausible if it means the exclusion of the others.
Moreover, a recent IOM report on research priority setting at NIH cautions against using cost data as a proxy for ranking funding decisions (Institute of Medicine, 1998). The fact that funding for research on one disease is several times greater than funding for research on another disease on a per capita or per death basis may seem to disease-specific advocates
or their friends in the U.S. Congress to involve some unfairness or unjustifiable inequality. However, the disease receiving greater research funding may differ from the less richly funded disease receiving less research funding in a number of relevant aspects. One disease may be more painful and debilitating, may result in more premature deaths, or may be a communicable disease that threatens the community as a whole; or given the current state of scientific understanding about a particular disease, investments in research on that disease may offer greater prospects for cure or prevention than a similar investment is likely to yield if it were devoted to research on another disease. Thus, such disparities in health outcomes, whether they track groups defined by disease or other criteria (e.g., ethnicity, geography, or occupation), provide the initial reasons for greater attentiveness to issues of health differentials within the research priority-setting process. Such differences alone, however, do not provide decisive reasons for altering funding priorities.
The inherent difficulties of assessing the relative health needs and the ineffectiveness of setting priorities on the basis of any of the available measures of disease burden tend to elevate the importance of scientific opportunities. Without an assessment of scientific opportunities, public funds are likely to be squandered with little or no demonstrable health benefits, and the obligation to pursue the well-being of the community as a whole will not be met. As a consequence of treating scientific opportunity as a necessary but not sufficient condition for according high priority to those areas in which advances are most likely, the primary emphasis of NCI and the other institutes within NIH is placed on individual investigator-initiated research proposals that are peer reviewed for scientific merit (National Institutes of Health, 1997).
The heavy emphasis on scientific opportunity has been endorsed by NCI Director Richard Klausner in his congressional testimony regarding priority setting at NCI. In addition, Klausner's testimony in hearings conducted by the present IOM committee further reflect a general preference for addressing the health needs of special populations through crosscutting research designed to lead to discoveries benefiting everyone. The value of the crosscutting approach is additionally supported by the claim of unpredictability in the growth of scientific knowledge. The pursuit of promising scientific leads in one area often reveals unexpected knowledge about the cause, prevention, or treatment of other medical conditions. Thus, the confidence in policies that concentrate efforts wherever the most promising leads emerge is reinforced by the reasonable expectation that approaches believed to yield the greatest anticipated gains in knowledge may be expected to yield the greatest serendipitous gains in knowledge.
Neither the importance of scientific opportunity nor the difficulties in assessing and weighing relative need, however, obviate the importance of
taking account of differential health burdens in the priority-setting process. Nor does the expectation of serendipitous gains in knowledge undermine the justification for allocating some portion of the diversified research portfolio to the study of those conditions or populations differentially burdened by cancer. For several reasons, on the basis of considerations of both fairness and good science, such targeted approaches may be useful complements to the mainstream approach.
First, just as there are reasonable limits to the ability to plan science, there are similar limits to how much one can rely on the organic model of the growth of scientific knowledge. The maxim that "science is self-correcting" is true only within limits. Although parallel and subsequent inquiry can confirm, refute, or modify the state of knowledge gleaned from one avenue of inquiry, there is no guarantee that the organic growth of knowledge will be uniform and free of lumps and gaps. Indeed, the organic model assumes that its trajectory will be unpredictable, and just as its unpredictability can yield serendipitous benefits, it can also result in large gaps differentially affecting some portions of the population.
In fact, a substantial body of evidence in the history of science supports the claim that developments in scientific knowledge have been instigated and aided by exogenous forces that redirect scientists' attention toward the unexpected and away from the linear, highly focused approach that concentrates on well-defined problems with ready hypotheses to be tested. Thus, the fact that a large measure of unpredictability exists in science does not uniquely favor building exclusively on what is known but favors as well some attention to questions not readily answerable from within the conventional scientific consensus. For example, research devoted to a better understanding of medical conditions among those most burdened by a disease may yield insights into causes and prevention not readily obtainable through study of the overall population, and this in turn may yield a better understanding of the etiology and nature of disease in others as well. Such principles of focused, targeted research are mainstream operational assumptions guiding much of current genetic research, in which it is hypothesized that understanding of causal mechanisms in smaller populations at greater than average risk will lead to improved understanding of similar conditions in others who are not the direct focus of inquiry.
In short, if uncertainty is a persistent feature of the growth of scientific knowledge, then it would be unreasonable to sacrifice all targeted research for the sake of deliberate crosscutting research, and the serendipitous character of science ensures that pursuit of the most promising leads from the current baseline of knowledge in heavily studied areas can be expected to produce unanticipated gaps no less than unanticipated gains in knowledge. The prospect of the existence of such gaps in which substantial
differential health burdens exist therefore warrants vigilant monitoring of differentials within subpopulations and attention to the need for research on the causes and mitigation of those differences as a supplement to the other parts of the research portfolio.
Moreover, a predictable consequence of the heavy reliance on researcher-initiated proposals is the increased likelihood that important questions regarding disease among those most heavily burdened will go unasked, especially if environmental and behavioral factors play an important mediating role and the incentives of the marketplace make their study less likely. Accordingly, various strategies designed to direct, motivate, and focus researcher imagination may be necessary as a supplement to the investigator-initiated approach if gaps in scientific knowledge contributing to differential health burdens are to be taken seriously.
Although NCI has provided the committee with many additional documents outlining its past and ongoing initiatives for special populations and its plans for the expansion of its Division of Cancer Control and Population Sciences, some unanswered questions remain. In oral testimony before the committee, Director Klausner emphasized the importance of crosscutting research and the fact that the differential cancer burdens experienced by some subpopulations will derive benefits from the knowledge gained from research on cancer generally. According to Klausner, much of the research not attributed to budgetary codes specific to research on cancer among special populations also produces secondary benefits for special populations, and thus, data regarding special initiatives alone fail to provide a full accounting of the level of NCI's commitment to meeting the health needs of those populations. The committee acknowledges the importance of these ancillary contributions that some critics may fail to appreciate.
However, the committee believes that Klausner's remarks emphasizing the secondary benefits derived from crosscutting research leave the status of NCI's commitment to special population research initiatives somewhat uncertain. In supplementary written testimony provided to the committee by Barbara Rimer in response to committee questioning (Rimer, communication with the study committee, 1998), NCI indicated that it intends to pursue both the traditional investigator-initiated approaches and RFAs targeted to special populations. In addition, Rimer shared budget proposals for future initiatives affecting many of the groups that NCI defines as special populations.
The committee applauds Rimer's statement of commitment to the continuation and expansion of such initiatives. However, important unresolved issues of both the process and the criteria used for priority setting for research among special populations remain. For example, the future role of the Director of Special Populations remains unclear. Klausner has
indicated that he views the role of the Director of Special Populations as "the eyes and ears" on these matters. The committee believes that the director also needs to have a strong voice in priority-setting activities affecting special populations, as well as clear institutional guidance for identifying high-priority objectives for research on such populations.
Moreover, the Office of Special Populations Research currently lacks many of the institutional advantages that would ensure that an NCI commitment to research among special populations has a chance to be successful. It has no independent resources to fund a separate portfolio of initiatives for special populations research, has no clear criteria for recommending the priorities for such initiatives that are dependent upon the resources of other parts of NCI or means of guiding such initiatives, and holds no official position on any of the NCI advisory committees responsible for setting major intramural or extramural priorities.
In response to committee questions regarding the appropriateness of focusing research on special populations on the basis of burden of disease, Klausner indicated that the recommendations of the Special Action Committee on Minority/Special Populations did not represent current NCI policy. Although no specific recommendation of the Special Action Committee was repudiated in Klausner's testimony, the committee believes that many of the recommendations merit careful consideration. Among its useful recommendations were proposals to improve the input of staff and other persons knowledgeable about the needs of special populations, an acknowledgment of the importance of better incidence and mortality data among these groups, and the need for support for a data driven research agenda taking into account the top five cancers affecting each minority population (National Cancer Institute, 1996a).
The committee generally agrees, however, with the conclusion of the NIH priority-setting pamphlet (National Institutes of Health, 1997) regarding the difficulty and multiple ambiguities of the concept of burden of disease and how it is measured. The recommendations of the Special Action Committee Reports of 1992 and 1996 were silent on the issue of how those top five most burdensome cancers should be conceptualized, but this omission need not be fatal to NCI efforts to focus on neglected areas of research on cancers affecting each of the special populations. An increased burden can be detected in many ways, ranging from increased mortality to an increased incidence of particular cancers. No one-size-fits-all approach should be applied to each population. A more fruitful strategy will require the assessment of the unmet research needs of each population with whatever data are available regarding the type of burden specific to that subpopulation, in combination with an appraisal of the scientific opportunities. In short, no simple formula for addressing each group's top
five most burdensome cancers will be adequate, and no uniform measure of burden across all subpopulations will be feasible.
The committee agrees with many of the conclusions of the recent IOM Committee on Priority Setting at NIH (Institute of Medicine, 1998), especially its general endorsement of the peer-review process and the central importance of scientific opportunity as a necessary condition for the allocation of scarce resources. However, lying beyond the scope of its charge are issues that this committee was asked to evaluate, and the committee concludes that in setting priorities for research among special populations, additional weight must be given to burden of disease. This does not mean that the demand for scientific opportunity should be sacrificed, however; it means only that unless NCI takes steps to initiate and stimulate research among special populations, the scientific opportunities from research within those communities will continue to lag behind those from research in areas where more effort has been expended in the past. Moreover, the committee concludes that as a supplement to the regular mechanisms for investigator-initiated research, additional steps are necessary to ensure that research questions for all of the subpopulations that NCI serves are adequately addressed. The committee concludes this chapter with the following recommendations.
Recommendation 4-4: Investigator-initiated research must be supplemented to ensure that the cancer research needs of ethnic minority and medically underserved populations are addressed.
The committee finds that the research priority-setting process at NCI and NIH fails to serve the needs of minority and medically underserved populations. Although the processes for receiving input from interested scientists (e.g., ethnic minority scientists and those interested in research among ethnic minority and medically underserved populations), community groups, and consumers are improving (e.g., with the conception of DCLG), the level of representation of these groups on key NCI advisory panels should be improved. In addition, their impact on the priority-setting process should be evaluated to ensure that policy changes follow from increased representation. Assessment of the burden of cancer among minority and medically underserved populations and consideration of the burden of cancer within the large framework of scientific opportunity should be key aspects of the research priority-setting process. To stimulate research on cancer among minority and medically underserved populations, NCI must expand RFAs and other mechanisms, especially in areas
where critical research gaps exist. The committee's specific recommendations are as follows:
Recommendation 4-1: NCI should develop a process to increase the representation of ethnically diverse researchers and public representatives serving on all advisory and program review committees so that the makeup of these committees reflects the changing diversity of the U.S. population. NCI should develop an evaluation plan assess the effect of increased and more diversified ethnic minority community and researcher input on changes in NCI policies and priorities toward ethnic minority cancer issues.
Recommendation 4-2: The research needs of ethnic minority and medically underserved groups should be identified on the basis of the burden of cancer in these populations, with an assessment of the most appropriate areas of research (i.e., behavioral and social sciences, biology, epidemiology and genetics, prevention and control, treatment, etc.).
Recommendation 4-3: For NCI to address needs of ethnically diverse and medically underserved populations effectively, the Office of Special Populations Research (or some other designated entity or entities) must possess the authority to coordinate and leverage programs and resources across divisions and branches of NCI to stimulate research on ethnic minority and medically underserved populations. This authority can be established by providing such an office with
- a leadership role in major NCI-wide priority-setting bodies, and
- special resources to fund programs specifically targeted to these populations, or
- accountability for the institution-wide allocation of program resources.
Recommendation 4-4: Investigator-initiated research must be supplemented to ensure that the cancer research needs of ethnic minority and medically underserved populations are addressed.