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Informing the Future: Critical Issues in Health, Sixth Edition (2011)

Chapter: Shaping Research Priorities

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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Shaping Research Priorities

The late Paul G. Rogers, known as “Mr. Health” when he served in the U.S. Congress, and who later chaired Research!America, used to say, “Without research, there is no hope.” To get the most out of every research dollar, researchers must focus on critically important health priorities and proceed in the most efficient manner. Prioritizing research is especially critical in tight economic times when even fewer dollars may be available to fund valuable scientific investigations.

The Institute of Medicine (IOM) has a long history of helping governments at all levels and private groups in many quarters shape their health research programs. IOM studies have offered research blueprints for tackling stubborn challenges that call for innovative approaches as well as new questions that demand immediate attention.

Assessing the oil spill in the Gulf of Mexico

When the Deepwater Horizon offshore drilling rig in the Gulf of Mexico exploded on April 20, 2010, it killed 11 workers and unleashed one of the largest oil spills in U.S. history. The resulting cleanup efforts came to mark the nation’s largest and most demanding on-water response ever, with many thousands of commercial workers and volunteers often working under harsh conditions for days and weeks on end. The spewing oil raised both environmental and health concerns.

The potential effects on human health were both immediate and long term. Worries arose about exposure to the oil itself, to the chemical dispersants used to break up the oil, and to the fumes generated by fires set to burn off oil slicks, among other physical threats. Adverse social, economic,

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

and psychological effects also may threaten the mental health of diverse Gulf Coast populations, which includes uniquely at-risk groups, including such groups as those in the fishing community who may temporarily or permanently lose their livelihoods.

Adverse social, economic, and psychological effects also may threaten the mental health of diverse Gulf Coast populations, which includes uniquely at-risk groups.

Even before the ruptured oil well was capped in July, the federal government initiated a number of efforts to protect the health and well-being of individuals and communities in the affected regions. Officials recognized early on that monitoring and surveillance would be key in detecting the spill’s physical and behavioral health effects on individuals and in identifying and assessing appropriate preventive and healthcare services. Accordingly, the Department of Health and Human Services (HHS) asked the IOM to convene an urgent workshop to examine the role and possible organization of monitoring and surveillance programs.

The IOM convened the first workshop in June 2010, bringing together more than 350 federal, state, and local government officials; scientists from varied disciplines; policy experts; healthcare providers; public health advocates; and community representatives and residents from affected areas. Presenters reviewed current knowledge and identified gaps regarding the human health effects of exposure to oil and chemical dispersants. Presenters considered information about the specific populations that might be at increased risks for adverse health effects. Presenters discussed communication strategies to convey information about health risks to at-risk populations, accounting for culture, health literacy, language, technology, and geographic barriers. They explored research methodologies and data collection needs. And as a final objective, they discussed the potential components of a framework for short- and long-term surveillance to monitor the spill’s potential adverse health effects.

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Many participants observed that assessing the effects on human health of oil spills and response activities is complex, involving such factors as the chemical composition and environmental degradation of the oil and

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

dispersants, as well as the unique characteristics of affected populations. Similarly, human health is multidimensional: physical, psychological, and socioeconomic factors influence the overall well-being of individuals and communities. A number of workshop participants predicted that the oil spill disaster will likely have an even greater effect on the psychological health of affected communities because of serious and prolonged disruptions to the social environment and local economies. They noted that community involvement is essential when designing surveillance systems and related activities. Local residents and communities have unique experience and expertise that can improve surveillance-related activities, especially if community engagement begins early. Speakers also described how coordination between and among all interested parties—public and private—can strengthen existing and developing surveillance and monitoring systems.

As called for in its commitment to HHS, the IOM also has conducted several other related studies, under the auspices of a specially appointed Committee to Review the Federal Response to the Health Effects Associated with the Gulf of Mexico Oil Spill. In one study, the committee examined the proposed plans for a new program to be run by the National Institute of Environmental Health Sciences, which is part of the National Institutes of Health (NIH). Called the Gulf Long-Term Follow-Up Study for oil spill clean-up workers and volunteers—or the GuLF Study—its aim is to fill some fundamental gaps in knowledge about the health effects of oil spills. Such new information may lead to improved understanding of the Gulf oil spill and help in identifying ways to prevent adverse health outcomes today and in any similar disasters in the future.

Local residents and communities have unique experience and expertise that can improve surveillance-related activities, especially if community engagement begins early.

The IOM convened a daylong workshop in September 2010 to gather scientific and community comments on the design and methodology to be used in the GuLF study. As explained by its principal investigator, the study will follow two groups. One group will include adults 18 years of age or older who worked or volunteered for 1 or more days in any cleanup task and therefore were exposed to potential health threats; the other group will include adults who completed safety training but did not perform cleanup work and therefore were not directly exposed, along with other unexposed community members, such as friends and relatives of the work-

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

ers, if needed. These groups will be examined or monitored in various ways for a range of physical and psychological health symptoms that affect or may yet affect these populations.

Discussions at the workshop are presented in Review of the Proposal for the Gulf Long-Term Follow-Up Study: Highlights from the September 2010 Workshop: Workshop Report (2010). Participants explored a number of suggested approaches for improving the study, while at the same time recognizing the time, legal, and resource limitations that may impede the improvements from being made. For example, study officials can make greater use of resources and expertise available both at the federal level and locally, and they can provide more specific, focused outcomes or concrete hypotheses that can be used to guide decisions about the data to be collected. In addition, the study would benefit by including more health outcomes, such as additional psychosocial measurements and gathering of data from pregnant women affected by the oil spill.

Other suggestions focused on recruitment and outreach. For example, study officials can give careful consideration of how to maximize the enrollment and retention of study participants, while also planning for

image

Levels of population exposures and anxiety or concern as defined by proximity to the oil leak.

SOURCE: Assessing the Effects of the Gulf of Mexico Oil Spill on Human Health, p. 32.

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

enrollment lower than predicted in the protocol. Fostering collaboration with the community will be important as well, and communications with all members of the community should be culturally sensitive and take health literacy into account.

The IOM’s recommendations go beyond the GuLF study alone. Based on its information-gathering process, which included the daylong workshop in September 2010, the IOM committee tasked with this work identified a number of potentially useful areas of research. In Research Priorities for Assessing Health Effects from the Gulf of Mexico Oil Spill: A Letter Report (2010), the committee singled out five areas that are most promising:

• Research to generate evidence about the psychological and behavioral effects of the oil spill. Policymakers and health officials can use such evidence to guide efforts to improve the health status of individuals affected by the spill, as well as to contribute to the prevention and treatment of similar health outcomes in future disasters.

• Research to obtain information that is as comprehensive as possible about exposure to the oil, dispersants, and by-products of the controlled burns.

• Research on assessing seafood safety in both the near term and long term. The findings should be clearly communicated to the affected communities.

• Research to evaluate and compare communication and engagement methods to determine which would be most effective in disaster-preparedness efforts.

• Research to determine the framework needed to deploy a rapid research response for future oil spills and other potential disasters.

Evaluating federal worker safety programs

In an ongoing commitment, the federal government supports a variety of research efforts to protect and improve the health and safety of the nation’s millions of workers. The National Institute for Occupational Safety and Health (NIOSH) is the lead agency for such research. NIOSH has research programs in eight main areas—hearing loss; mining; agriculture, forestry, and fishing; respiratory diseases; personal protective technology; traumatic injury; construction; and health hazard evaluation.

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

At the request of NIOSH, the IOM and the National Research Council (NRC) jointly have conducted a series of studies since 2004 to evaluate the relevance and impact of the research programs. As a first step, a lead IOM/NRC committee developed a common framework to be used in the evaluations. The framework is based on a model that is widely used in program evaluation and planning. Called the logic model, it organizes program efforts into four basic categories: inputs, activities, outputs, and outcomes. Eight separately appointed committees then used the framework over several years to assess the individual programs, and each committee issued a report on its findings.

At the conclusion of the studies, the IOM/NRC framework committee held a public workshop where discussions focused on the experiences gained in the evaluation process. From these discussions and other deliberations, the committee produced Evaluating Occupational Health and Safety Research Programs: Framework and Next Steps (2009). The report details the evaluation framework developed and used for the evaluations; summarizes lessons learned along the way; and presents a revised framework, along with a related set of recommendations, for NIOSH and other federal agencies to use in future research evaluation efforts.

Among its recommendations, the committee says that NIOSH should establish a system for periodic external evaluation complemented by internal self-assessments on a regular basis. NIOSH also should continue to build and improve research translation to effectively move research into practice. As part of the translation process, the institute should listen to people in the workplace and beyond to identify intervention needs that should be targeted with additional research.

In other steps, NIOSH should increase and improve surveillance of work-related injuries, illnesses, exposures, and working conditions so that information needed to assess program relevance and impact will be available for future evaluations. Enhanced surveillance should prove informative in balancing research priorities. Also, future evaluations should systematically consider intramural and extramural research activities, in terms of both evaluating the impact and relevance of each type of research and assessing the extent to which intramural and extramural research are integrated in strategic planning.

In addition to these wide-ranging evaluations of research on worker safety, the IOM has reviewed NIOSH’s research plans for addressing health concerns raised by a single class of workplace hazards: asbestos fibers and similar mineral particles. Prior and ongoing exposures to asbestos con-

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

tinue to contribute to respiratory diseases—including mesothelioma, lung cancer, and asbestosis—despite the fact that asbestos is no longer mined in the United States.

To help in responding to ongoing questions and concerns in this field, NIOSH released a detailed research plan in January 2009, Asbestos Fibers and Other Elongated Mineral Particles: State of the Science and Roadmap for Research. At the request of NIOSH, the IOM and the NRC jointly appointed a committee to review the scientific and technical quality of the proposed plans. In A Review of the NIOSH Roadmap for Research on Asbestos Fibers and Other Elongate Mineral Particles (2009), the committee reports that overall, NIOSH put together a comprehensive plan that likely would yield important results. The committee also offers recommendations that could boost the expected knowledge and practical returns from the research programs.

Among recommended actions, NIOSH should clarify the vision and rationale for the roadmap. The vision statement should point toward research that will differentiate effects from exposure to a range of elongate mineral particles and help determine the influence of size, shape, and other physical and chemical characteristics of these particles on human health. The rationale should clearly articulate the influence that ongoing and future research can have on improving public and occupational health. In addition, NIOSH should revise the roadmap to emphasize the need for collaboration and integration of research among the mineralogical, toxicological, epidemiological, and exposure-assessment disciplines.

On a broader scale, NIOSH should continue to work with other federal agencies, as well as with private-sector groups and nonprofit organizations, in developing an overarching strategy for research. Such a strategy might include several elements in addition to the framework and goals set forth in the proposed roadmap, among them an interdisciplinary system for prioritizing research activities to ensure maximum efficiency in an environment of limited funding; an approximation of the resources needed to carry out high- and middle-priority efforts; and a plan for review, evaluation, and accountability for individuals and institutions receiving support for research.

The National Institute for Occupational Safety and Health should continue to work with other federal agencies, as well as with private-sector groups and nonprofit organizations, in developing an overarching strategy for research.

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

Advancing women’s health research

Among other populations that the federal government targets for research, women comprise more than 50 percent of the U.S. population but historically have gotten short shrift as subjects of biomedical research. Over the past 2 decades, however, there have been major changes in government support of women’s health research—in policies, regulations, and the organization of research efforts. To assess the impact of these changes, Congress in 2008 directed HHS to ask the IOM to examine what has been learned from that research and how well it has been put into practice and communicated to both providers and women.

Women comprise more than 50 percent of the U.S. population but historically have gotten short shrift as subjects of biomedical research.

The IOM committee appointed for the task defined women’s health broadly, encompassing health conditions that are specific to women, are more common or more serious in women, have distinct causes or manifestations in women, have different outcomes or treatments in women, or have high morbidity or mortality in women. In Women’s Health Research: Progress, Pitfalls, and Promise (2010), the committee reports that research has contributed to significant progress in reducing the morbidity and mortality associated with some conditions, while other conditions have seen only moderate progress at best. Gaps remain, both in research and in the application of results to benefit women in general and across multiple population groups, and the committee proposes a number of recommendations for improvements.

The NIH should emphasize research on common determinants and risk factors that underlie multiple diseases that affect women. Research to date has paid inadequate attention to the social and environmental factors that, along with biologic risk factors, influence women’s health. In addition, researchers should pay greater attention to assessing quality-of-life issues, such as mobility or presence of pain, and promoting wellness.

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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

In addition to conducting women-only research as appropriate, the research community should strive to integrate women’s health considerations into all health research, such that sex- and gender-based differences between men and women are routinely and consistently assessed. Toward this end, the government and other funding agencies should ensure adequate participation of women and reporting of sex-stratified analyses in health research. More attention also needs to be paid to finding the most effective ways of moving research findings into clinical practice and public health policies.

Improved public communications are needed as well. Many people often are confused by conflicting findings and opposing recommendations emerging from health research, including women’s health research. The committee calls on HHS to appoint a task force to develop strategies to effectively communicate research-based health messages to women. The federal government can support improved communications by requiring all federally funded studies to include a plan for disseminating findings to the public, providers, and policymakers.

Conditions Discussed by Committee, Categorized by Extent of Progress

Conditions on Which Research Has Contributed to Major Progress
Breast Cancer
Cardiovascular Disease
Cervical Cancer
Conditions on Which Research Has Contributed to Some Progress
Depression
HIV/AIDS
Osteoporosis
Conditions on Which There Has Been Little Progress
Unintended Pregnancy
Maternal Morbidity and Mortality
Autoimmune Diseases
Alcohol and Drug Addiction
Lung Cancer
Gynecological Cancers Other than Cervical Cancer
Non-Malignant Gynecological Disorders
Alzheimer’s Disease

SOURCE: Women’s Health Research: Progress, Pitfalls, and Promise, p. 4.

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

Boosting research on rare diseases

While many diseases affect large numbers of people and attract research attention, some diseases strike relatively few individuals and often go neglected. As a result, researchers lack even a basic understanding of many rare—or “orphan”—diseases. Also, effective therapeutics are available for only a small fraction them, and even when available, some are extraordinarily expensive. In light of these realities, the NIH, with support from the Food and Drug Administration (FDA), asked the IOM to appoint a committee of experts to examine the opportunities and obstacles in developing drugs and medical devices for treating rare diseases.

In Rare Diseases and Orphan Products: Accelerating Research and Development (2010), the committee calls for implementing an integrated national strategy that would include seven key elements. In simplified form, these elements are:

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1.     active involvement and collaboration by a range of public and private interests;

2.     timely application of advances in science and technology;

3.     creative strategies for sharing research resources and infrastructure;

4.     appropriate use and further development of trial design and analytic methods for conducting research on small populations;

5.     reasonable rewards and incentives for private-sector innovation, coupled with prudent use of public resources for product development when necessary to respond to important unmet needs;

6.     adequate support for public agencies that fund research on rare diseases and regulate drugs and medical devices; and

7.     mechanisms for weighing priorities for rare diseases research and product development, establishing collaborative as well as organization-specific goals, and assessing progress toward these goals.

Components of these elements already exist, some more robust than others, and the committee recommends a number of specific steps to foster

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

their further development and implementation. Steps for the NIH include developing a comprehensive action plan for rare diseases research that covers all institutes and centers and working with the FDA to ensure that NIH-funded drug studies are designed to meet FDA standards for product approval. Other steps include identifying unmet needs for medical devices to treat rare conditions and improving understanding of how public and private insurance programs have an effect on access to drugs and medical devices for such conditions.

To help build on the recommendations and existing activities, HHS should establish a national task force on accelerating rare diseases research and product development. To operate for perhaps 4 to 8 years, the task force would bring together leaders from government, industry, academic and other research institutions, and advocacy groups. Its aim would be to promote, coordinate, monitor, and assess the implementation of public and private initiatives on rare diseases and orphan products and to support additional opportunities for public-private collaboration.

Ethical issues in studying drug safety

Although federal regulations call for drugs under development to be tested for safety and kept off the market if problems are detected, adverse health effects often are discovered only after drugs have been in widespread use. Historically, most of the FDA’s authority and activity related to drugs has centered on approval prior to entering the market. In 2007, Congress expanded the FDA’s authorities and responsibilities over drugs during the postmarketing period. The new authorities, many of which were recommended by the IOM in The Future of Drug Safety: Promoting and Protecting the Health of the Public (2007), provided the FDA with a range of additional regulatory tools.

With its expanded powers, the FDA recognized that it faced a number of new challenges and questions, both ethical and scientific, regarding the study of drugs after approval. It turned to the IOM for help, and the IOM appointed a committee of experts to evaluate a number of questions submitted by the FDA on how best to conduct studies of the safety of approved drugs.

As a first task, the committee was directed to undertake only one of the FDA’s questions—What are the ethical and informed consent questions that must be considered when designing randomized clinical trials to eval-

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×

uate potential safety risks?—and to submit a report by July 2010, in time for a joint meeting of two scientific advisory committees to the FDA examining the testing and safety of a particular drug.

In that preliminary report, Ethical Issues in Studying the Safety of Approved Drugs: A Letter Report (2010), the IOM committee describes a conceptual framework that the FDA should use for evaluating ethical and informed consent issues related to its postmarketing evaluations. The framework consists of four classes of considerations: the public health context of drug safety, regulatory science and public accountability, design considerations, and additional ethical obligations to research participants. Within each category, the committee explores a number of key issues that the FDA might be expected to face in determining whether and how to conduct postmarketing studies. The committee will issue a subsequent report on the remaining questions in late 2011.

Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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Suggested Citation:"Shaping Research Priorities." Institute of Medicine. 2011. Informing the Future: Critical Issues in Health, Sixth Edition. Washington, DC: The National Academies Press. doi: 10.17226/13180.
×
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This report illustrates the work of IOM committees in selected, major areas in recent years, followed by a description of IOM's convening and collaborative activities and fellowship programs. The last section provides a comprehensive bibliography of IOM reports published since 2007.

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