Outcomes Research in Cardiovascular Disease
Report of the NHLBI Working Group
The objective of the National Heart, Lung, and Blood Institute
(NHLBI) Working Group on Outcomes Research in Cardiovascular Disease
(CVD) is to advise NHLBI on opportunities in Outcomes Research
related to CVD, and to provide guidance on priorities in allocating
resources for Institute-sponsored and investigator-initiated CVD
The definition of CV Outcomes Research. For the purposes
of this Working Group, "Outcomes Research" is defined as applied
clinical research that generates knowledge to improve clinical
decision-making and health care delivery to optimize patient outcomes. It
is the study of the delivery and consequences of health care on
outcomes from the perspective of patients, providers, and the
health care system. Outcomes Research focuses particularly on
providing evidence to assess and promote the effectiveness of
therapeutic interventions, enhancing the migration of best practice
to clinical practice, elevating the quality of care, and supporting
the optimal allocation of resources for all patients. The
research has direct, proximate, and immediate relationships to
patient care and health-care delivery. Research addresses
not only patterns of care, but also elucidates factors contributing
to those patterns, and determines their consequences. The
research also is directed toward developing strategies to address
current deficiencies in knowledge or implementation directly relevant
to clinical practice.
Outcomes Research is not defined by a particular methodology, but
rather its objective – the study and eventual improvement of the
end results of health care. Outcomes Research can employ methodology
of non-experimental (observational) designs, as well as quasi-experimental
or clinical trial approaches to test interventions, whichever
is most suited to the specific research question. Outcomes Research
draws from the disciplines of clinical epidemiology, statistics,
health services research, psychology, sociology, anthropology,
economics and management sciences.
The need for CV Outcomes Research. Despite the accelerating
pace of basic and traditional clinical research in CVD, clinicians
and policy-makers often lack knowledge about how best to promote
CV health and health care delivery in clinical practice. Despite
all our advances we lack basic information about contemporary
patterns of care. We have a limited array of valid tools to quantify
and improve clinical performance. We often define efficacy but
commonly do not know the effectiveness of different clinical strategies
in actual practice as well as the impediments to achieving the
true potential of the best interventions. We need information
concerning the impact of clinical and policy strategies on a broad
range of patient outcomes, not just mortality and events. We need
to know how to best involve patients in decision making and the
best approaches to translating evidence into clinical practice
so that outstanding quality of care is universally available.
Outcomes Research generates knowledge about the effectiveness of
clinical strategies; the health and health care experiences of
patients (including measures of health status); quality of care;
the balance of costs and effectiveness; and studies of clinical
decision-making. Outcomes Research can be descriptive and prescriptive,
focusing on current deficiencies in knowledge or interventions
to improve clinical practice. Thus, Outcomes Research aims to
inform patients, providers, health care systems, payers, and policy
makers of the effects of health care and provide data to support
the development of evidence-based strategies (at the patient,
provider, and system levels) that will best serve the health needs
of the nation. CV Outcomes Research applies these aims and approaches
specifically to the prevention or treatment of CVD.
CV Outcomes Research can support national initiatives to improve
health care. The goals of CV Outcomes Research are closely
aligned with a number of existing federal initiatives.
- Healthy People 2010 sets forth ambitious goals to reduce
CVD risk, improve CVD outcomes, and eliminate health disparities.
Attaining these goals will require effective programs that actively
support the integration of existing knowledge and discoveries
into clinical practice.
- The Agency for Healthcare Research and Quality, the Centers for
Disease Control and Prevention, the Centers for Medicare &
Medicaid Services, the Department of Veterans Affairs, and the
Department of Defense are all seeking to promote the best "real-world"
strategies for the prevention and treatment of disease; to determine
the success of health care strategies by assessing patient-centered
outcomes; to measure adherence to "best practices" (usually representing
those embodied in clinical practice guidelines); and to develop
interventions to improve the performance of the health care system.
Success in these endeavors will depend on research that can identify
best clinical practices, evaluate different clinical strategies,
identify wasteful resource utilization, explore the involvement
of patients in decision-making and study the effects of clinical
and policy-related decisions related to CV health.
- The National Academy of Sciences’ Institute of Medicine (IOM)
has published a blueprint for improving health care delivery in
a document entitled, ‘Crossing The Quality Chasm: A New Health
System For The 21st Century.’ This report identified
six core goals for the future of American health care: safety,
effectiveness, equity, efficiency, timeliness and patient-centeredness.
These domains, which are not currently a focus of traditional
CV clinical research, are important topics for investigation and
there is an urgent need for research into how best to attain the
IOM’s goals in patients with CVD.
- Many federal organizations, including the NHLBI, have strategic
plans to reduce health disparities and the newly created National
Center for Minority Health and Health Disparities is championing
national efforts to identify and remediate disparities in CV care.
Central to the elimination of health disparities is research that
investigates where disparities exist, assesses their consequences,
and explores the causes of differences in outcomes between racial,
ethnic and vulnerable socioeconomic groups and develops and evaluates
the effectiveness of interventions to eliminate these disparities.
The Working Group believes that the NHLBI can take a leading role
in fostering research to support these and other efforts related
to CVD care and outcomes, ensuring that health care provided at
the patient, provider, and health system level is supported by
a strong scientific foundation built on contemporary, patient-centered,
III. Methods Used to Generate this Report
Working Group members, whose backgrounds cover a broad spectrum
of experience within Outcomes Research, considered a range of
possible research areas, current gaps in knowledge, and the expected
CV trends in the nation. The group first reviewed ongoing programs
and funded projects within the NHLBI. Related initiatives and
priorities set by other National Institutes of Health and federal
agencies were also considered. The group had conference calls
and an in-person meeting to reach consensus about the research
The Working Group sought to identify high-priority areas in CV
outcomes research that deserve support and to provide examples
of important areas for future investigation. The objective was
not to provide a comprehensive list of topics in CV outcomes research,
but rather to identify research directions that the Working Group
believes require the most immediate action, with particular attention
given to the stated missions of the NHLBI and the NIH. The examples
listed below are not intended to exclude the consideration of
other meritorious research areas or investigator-identified topic
Having described specific priority research domains and specific
topics, the Working Group concluded that directed programmatic
commitments are needed to support the growth and development of
CVD Outcomes Research.
IV. Working Group Recommendations
The Working Group identified several priority areas, including
the development of national surveillance projects for high prevalence
CV conditions, the promotion of patient-centered care, definition
and implementation of best practice, and expansion of studies
that consider the cost of interventions in the context of their
effectiveness. Within each of these topics, the Working Group
described examples of initiatives that could serve the Institute
and the public. In addition, the Group identified several areas,
including promoting the use of existing data, augmenting the methodology
of Outcomes Research, training of new investigators, and increasing
study section support, that are critical to the success of CV
Top Tier Priorities
National Surveillance of CV Care and Outcomes
Large-scale NHLBI-funded population-based epidemiological studies,
such as the Framingham Heart Study and the Cardiovascular Health
Study, have helped define the prevalence, determinants, incidence,
and consequences of CVD. However, there are no analogous national
resources directed to assess the quality of CV care and health
outcomes of community-based populations with or at risk for CVD.
For example, the Worcester Heart Study has provided important
insights, but is geographically limited and does not include long-term
outcomes aside from mortality. The Atherosclerosis Risk in Communities
(ARIC) surveillance identifies myocardial infarction (MI) and
coronary heart disease (CHD) death in four communities (Minneapolis
suburbs; Forsyth County, NC; Washington County, MD; and Jackson,
MS) by review of hospital discharge diagnosis codes, medical record
abstraction, and death certificate reviews. It has no follow-up
component except for linking, when appropriate, of events for
patients occurring within 30 days of one another. The Minnesota
Heart Survey identifies MI and CHD mortality in the Minneapolis/St.
Paul area by review of hospital discharge diagnosis codes, medical
record abstraction, and death certificate reviews. Non-fatal
cases are not followed longitudinally. This study also has
an out-of-hospital sudden cardiac death autopsy component. It
does not involve any patient interviews.
Other agencies also have databases and projects that are relevant
to surveillance, but they also have their limitations. The administrative
databases of the Agency for Healthcare Research and Quality provide
information about hospitalizations, but lack clinical detail and
long-term outcomes. The Centers for Disease Control and Prevention
conduct national surveys of risk factors and behaviors, but do
not focus on clinical CV conditions. Similarly, the surveys by
the Agency for Healthcare Research and Quality provide important
insights about access and health behaviors, but do not focus on
clinical conditions. The National Health and Nutrition Examination
Survey (NHANES), conducted by the Centers for Disease Control
and Prevention’s National Center for Health Statistics (NCHS),
collects information concerning general population health and
diet, but does not address CV-specific quality of care and a wide
range of patient outcomes.
Thus, despite rapid changes in CV knowledge, efficacious treatments,
and technical capabilities, we have no established national capacity
to assess how technologies and treatments are used or to measure
their performance when implemented in real world conditions. The
generalizability of randomized controlled trials and other efficacy
studies is rarely formally assessed but often presumed. Answers
to questions concerning population and patient-level impacts of
health care across a broad spectrum of outcomes, including mortality,
events, quality of life, and cost, are not available. For example,
how will drug-eluting stents be adopted and perform in practice?
What determines the adoption of internal defibrillators, are there
disparities in their use, and what is their impact on patients’
lives? Similarly, the identification of problems with medications
or devices often comes through the fortuitous reporting of individual
cases rather than an existing systematic process of evaluation.
Given the substantial commitment of resources to improving CV health
and health care, a national CV surveillance system could provide
the infrastructure to develop new insights into improving practice
and target future efforts to improve care and outcomes. The Centers
for Medicare & Medicaid Services’ (CMS) Cooperative Cardiovascular
Project (CCP) was a limited example of such a system. The CCP
was developed as a large, one time cross-sectional evaluation
of acute myocardial infarction care in elderly patients. This
clinically rich database has supported many studies addressing
the clinical management of acute myocardial infarction in the
elderly. The CCP was able to produce such research because it
generated a database that included a representative national sample
and detailed medical record abstraction of information about processes
of care and clinical events, supplemented by additional physician
and hospital data. Despite the novel value of CCP, the study had
notable limitations, including its limited time frame (1994-96),
the lack of data on quality of life or functional status, the
omission of patients younger than 65 years of age, and the absence
of information on outpatient treatment patterns.
Other groups have also sought to provide data on CV care and outcomes.
The Veterans Administration has studied the relationship between
patterns of health care delivery and outcomes through its Quality
Enhancement Research Initiative (QUERI) groups. For example, the
heart failure QUERI has identified a national cohort of veterans
with heart failure using administrative data, and efforts are
underway to link this resource to clinical and outcomes data.
Professional medical societies have also initiated CV procedure
registries, including the Society for Thoracic Surgeons’ national
database for cardiac surgery and the American College of Cardiology’s
National CV Disease Registry for percutaneous coronary interventions.
Finally, there are clinical registries funded by pharmaceutical
and medical device companies. These condition- or procedure-specific
registries have added substantially to the medical literature,
but most of these current efforts are limited in that they focus
on the treatment of hospitalized patients, frequently lack follow-up
information, do not have information on post-hospitalization outcomes
or utilization of care, lack reliable assessment of detailed patient-centered
outcomes, and are often restricted to limited geographic areas,
age groups, or other selected patient populations. These also
collect information only from hospitals that volunteer to participate.
In addition, health services researchers often have limited access
to these proprietary commercial databases and their use is aligned
with company interests.
Another example of such a proposed surveillance system has been
developed in cancer via the NCI Cancer Care Outcomes Research
and Surveillance (CanCORS) Consortium (http://healthservices.cancer.gov/cancors/).
The CanCORS Consortium is collecting detailed primary data from
medical records and surveys of patients and providers to understand
variations in the treatment and outcomes of lung cancer and colorectal
cancer in 5 states and regions, 5 large health plans, and the
Veterans Administration system.
Establish a national surveillance program for CV care and outcomes.
We recommend a series of national or multi-regional clinical cohort
studies that would bridge the gap between evidence and practice
and provide information on practice, care and outcomes. These
studies should be undertaken for several key CVD conditions, designed
to describe, with longitudinal data and adequate clinical detail,
population-based patterns of care across the US, and their short-
and long-term consequences. We recommend that initial priority
conditions focus on acute coronary syndromes, heart failure, and
atrial fibrillation. We also recommend that selected CV procedures
(e.g., percutaneous coronary intervention, coronary artery bypass
surgery, and implantable cardioverter-defibrillators) be studied
based on their high prevalence, impact on patients and the health
care system, and variations in clinical strategies and outcomes.
These studies will provide descriptions of current practice patterns
and their associations with patient outcomes, support the development
of performance measures for quality, allow the identification
of ‘best practices’ from which quality improvement initiatives
may be developed, stimulate discussion about best practice, generate
evidence about the effect of national health care policies, and
identify topics for future research. Those that enroll patients
by condition (rather than procedures) can also provide the foundation
for risk-stratification models and the ability to appreciate which
patient subsets benefit most from procedures and other such interventions.
Finally, such registries also lay the foundation for the existence
of an established network of investigators to describe, refine
and test novel strategies that will accelerate the translation
of new scientific knowledge into clinical practice.
Because of the urgency in addressing known and suspected health
care disparities, we suggest that racial and ethnic minority groups,
individuals in poverty, older people and/or other vulnerable subgroups
should be well represented, in order to permit appropriate comparisons
Promoting ‘Patient-Centered’ Care
Recently, the IOM emphasized the need for more ‘patient-centered’
care as a critical step towards improving the quality of American
health care. Patient-centered care is that which helps clinicians,
"in attending to their patients’ physical and emotional needs,
and maintaining or improving their quality of life," and gives
patients the opportunity to be "the locus of control" in medical
decision-making. Key components of patient-centered care include
the assessment of patient-centered health status outcomes (i.e.
symptom burden, functional limitation, and health-related quality
of life), and the incorporation of patients’ views about treatment
into clinical decision-making.
Although clinical research has facilitated tremendous advances
in the care of CV patients, surprisingly little research has focused
on patient-centered health status and on patient preferences.
Studies are needed to improve our understanding of the impact
of various CV diseases and therapies on patient health status
and to evaluate interventions designed to improve health status
outcomes for patients with CVD. Furthermore, studies are needed
to assess the utility of health status measurement and elicitation
of patient preferences in clinical practice to maximize patient
1. Identify determinants of patient-centered health status.
We recommend studies to identify the key determinants of patient-centered
health status in populations with CVD. These studies will require
the collection of longitudinal clinical and health status outcomes.
The knowledge generated from these studies can lead to the development
of interventions to improve health status for patients with CVD,
and to the development of studies focused on the use of health
status data to guide clinical decision-making. This recommendation
overlaps with the call above for "National Surveillance of CV
Care and Outcomes." Thus, as above, we recommend a focus on high
prevalence conditions such as acute and chronic ischemic heart
disease, heart failure, and atrial fibrillation. In addition,
such studies should address high risk and under-served populations
such as older persons, women, racial/ethnic minorities, patients
in poverty, patients with multiple comorbid conditions (e.g. diabetes,
chronic obstructive pulmonary disease, depression), and patients
in high-risk settings (e.g. nursing homes).
2. Evaluate patient-centered tools and interventions. We
recommend studies to evaluate the utility of patient-centered
tools or interventions in clinical care. The measurement and explicit
use of patient preferences to promote shared decision-making and
the measurement and integration of health status information to
guide management of CV disease are promising approaches to increase
the appropriateness of interventions and optimize the impact of
clinical interventions. For example, CV health status measures
may be provided to clinicians to help identify patients at particularly
high risk for subsequent adverse outcomes, and who may thus benefit
from more intensive management. As another example, potentially
modifiable aspects of health status (e.g. depression) may be targeted
for intervention. Patient-level interventions, such as patient
activation and/or patient training, and interventions aimed at
the hospital, health-care system, or community are also important.
Where possible, studies should include explicit evaluation of
the impact of these interventions on a broad range of patient
outcomes (e.g. mortality, morbidity, satisfaction, and health
status). Longitudinal studies will be necessary to demonstrate
the value of these interventions over time. A focus on high-risk
and/or under-served populations should be encouraged. Studies
that include a focus on chronic disease management, coordination
and integration of care, access to care, and/or transitions between
health care environments would be of particular value. Studies
are needed in all CV populations, but those with marked health
status deficits, such as heart failure, ischemic heart disease,
atrial fibrillation, peripheral arterial disease, and cerebrovascular
disease, may be of particular interest.
3. Include patient-centered health status outcomes in clinical
trials. The NHLBI has been committed to the inclusion of health
status outcomes in clinical trials. We recommend NHLBI continue
this support and continue considering the inclusion of CV health
status measures (i.e. formal measures of symptom burden, functional
status, and health-related quality of life) in all relevant CV
clinical trials sponsored by the NHLBI. A workshop on integrating
health status into CV studies might be an appropriate approach
to pursue this recommendation.
Defining "Best Practices"
Many clinical decisions lack a strong evidence base, resulting
in uncertainty regarding what constitutes optimal care. Thus,
data are needed concerning the comparative effectiveness of different
Also critical to improving care is defining best practice based
on the medical literature. This effort requires the impartial,
methodologically rigorous synthesis of the research literature
into evidence-based guidelines for care. Importantly, this effort
should also include rapid integration of new evidence on a "real-time"
basis. What is currently lacking is a professionally-recognized
core group of experts, such as the Agency for Healthcare Research
and Quality Evidence-Based Practice Centers, to provide a rigorous
synthesis of the evidence to support the continuous integration
of new evidence. The effort could directly support the development
and revision of CV clinical practice guidelines, conducted by
professional societies such as the American College of Cardiology
and the American Heart Association.
1. Evaluate effectiveness of CV treatments and strategies.
We propose that the NHLBI enhance its current portfolio of studies
to evaluate further the effectiveness of CV treatments and clinical
strategies. An effectiveness study is one that is testing an intervention
in an applied ("real-world") setting, where the effects of the
intervention are affected by both efficacy and adherence/delivery.
Studies consistent with this aim may be observational or experimental.
Randomized trials have the particular strength of allocating measured
and unmeasured factors among the various study groups. Observational
study designs can provide important evidence to guide practice
where experimental designs are not feasible or forthcoming. They
also may have particular utility in quantifying the risks of interventions
as they are applied beyond the original efficacy studies.
2. Support evidence-based reviews of CV treatments. We propose
the NHLBI provide support for CV Evidence-Based Centers, perhaps
in collaboration with AHRQ and the professional societies, which
would be specifically charged to provide research and analytic
support for the development of empiric, unbiased evidence reviews
using accepted evidence synthesis and meta-analytic methods. The
Evidence-Based Centers would provide adequate resources to conduct
systematic literature searches needed to support scientifically-based
national CV care guidelines that are being conducted by professional
societies. They would also continuously monitor the published
literature and provide ‘real-time’ updates to specific guidelines
as needed. The topics identified through the systematic reviews
as having insufficient evidence for clinical care recommendations
should then be referred back to the NHLBI and disseminated to
potential investigators as topics for future research. This effort
should be synergistic, not redundant, with current AHRQ efforts.
Implementing "Best Practices"
Numerous studies have documented a large gap between evidence-based
care recommendations and the actual care delivered in community
practice. These ‘lost opportunities’ have real consequences for
patients. Thus, there is a need to generate knowledge about the
means to improve physicians’ adoption of optimal therapies in
routine CV care. To date, intervention studies designed to increase
physician compliance with practice guidelines have often lacked
adequate controls and have tended to assess single, rather than
multi-modal interventions. Such assessments also have generally
focused on hospital-based treatment rather than the outpatient
setting. Furthermore, nearly all of these studies have assessed
changes in provider behavior over a limited time period, creating
uncertainty about the durability of these approaches. Finally,
these studies also have been limited in terms of power and scope,
with limited patient and provider samples, and have been applied
in selected clinical settings and populations. While these early
demonstration projects were important first steps for the field,
there remains much to do in terms of identifying optimal strategies
for implementing best practices.
1. Identify factors associated with provider delivery of effective
therapies. We propose a call for observational qualitative
and quantitative research to identify strategies to evaluate and
promote physicians’ adherence to evidence-based guidelines as
well as to characterize key structure and process features of
successful implementation of "best practices". These efforts would
investigate potential causes for the wide national variation in
guideline adherence. Studies may employ existing data sources,
utilize the surveillance initiative proposed above, or alternatively
gather de novo additional data on structure and/or process
variables that may explain variations in treatment. The goals
of this research would be to identify successful practices and/or
organizational features that can be applied widely. Study of existing
‘leading centers’ could generate novel potential approaches for
improving provider adherence that could then be rigorously tested.
2. Test interventions to improve provider delivery of effective
therapies. We recommend the continuation and expansion of
programs such as NHLBI-RFA HL-01-011 (Trials Assessing Innovative
Strategies To Improve Clinical Practice Through Guidelines In
Heart, Lung, And Blood Diseases), which was a one-time call for
research "to evaluate interventions that represent innovative
strategies that can be employed in clinical practice to improve
implementation of national, evidence-based clinical practice guidelines."
Areas of highest impact would include primary and secondary prevention
interventions, acute coronary syndromes, and heart failure. Particular
interest should be focused on the transition from inpatient to
outpatient settings and longitudinal care. While unique interventions
in a particular setting are of interest, priority should be focused
on those interventions that can be applied broadly, ideally on
a regional or national level.
Patient Involvement in Care
Advances in the prevention and treatment of CVD are often not fully
realized because of limited involvement by patients in their own
care. Rates of non-adherence with prescribed regimens of pharmacotherapy,
exercise, diet, smoking cessation and self-monitoring often range
from 30% to 50%. The issue of patient adherence becomes even more
salient as management guidelines advocate extensive combination
therapy for many conditions. Discontinuities in the long-term
management of therapeutic regimens, including the paucity of systems
to facilitate feedback to patients regarding their progress in
achieving therapeutic goals, also diminish patient adherence.
Previous research has predominantly focused on hospitals and primary
care practices. Critical opportunities for improvement in patient
adherence exist in home self-management at the transition from
outpatient setting to hospital care, and at the transition from
hospital care to outpatient care. Maximizing the benefits of pharmacotherapy
and lifestyle approaches will require a sustained and substantial
commitment to new research initiatives on patient adherence. Future
research on patient adherence should focus not only on the patient,
but also on the provider and the health care environment.
1. Study approaches to improving patient adherence. We propose
calls for research that enhances patients’ adherence to medication
regimens, with the consideration of incorporating proven models
of behavioral change. Research on patient adherence has tended
to be disease-specific, without sufficient attention to individual
behavioral factors and cultural factors that may differ among
racial, ethnic, and socioeconomic groups that affect the effectiveness
of various preventive and treatment regimens. Strategies that
mobilize not only the resources and capabilities of individual
patients, but the network of family, friends, and others with
whom they interact should be developed. As part of this research,
there is a need for improved approaches to measuring adherence.
These efforts would build on recent NHLBI initiatives that have
sought to understand and promote patient adherence. For example,
HL-01-005 (Overcoming Barriers To Treatment Adherence In Minorities
And Persons Living In Poverty) sought to evaluate interventions
in clinical care settings designed to improve adherence to medically
prescribed lifestyle and medical regimens but was limited to specific
groups. Future initiatives could build on this effort and be less
restrictive of the population studied. Also, HL-01-005 required
a randomized trial study design, whereas other study designs might
be very informative in addressing this topic.
2. Study novel approaches to enhance patient self-management.
We propose that the NHLBI foster research on the development and
clinical implementation of technologies and systems that enhance
patients’ participation in monitoring and management of chronic
CV conditions. Devices for self-monitoring parameters such as
symptoms, health status, blood pressure, anticoagulation and blood
glucose in the home environment are available, yet these devices
are not well integrated into treatment practice. Likewise, interactive
technologies such as web-based instruction and feedback that can
support patient responses to these data are infrequently utilized.
Innovative ways of organizing health care personnel and systems
of care to empower patients and enhance their involvement in care
are also needed. Research support should be directed to evaluate
the effectiveness of advanced interactive technologies and systems
that incorporate not only instruction but also ongoing monitoring
and corrective feedback.
In an era in which health care costs are rapidly increasing, society
is demanding that health care offer good value, providing good
outcomes at a fair price. Thus, it is no longer sufficient to
show that a test predicts outcome, that a therapy is efficacious
or that a pattern of practice can be shown to be effective. It
is also critically important to show that these services are worth
the costs associated with them.
Study cost-effectiveness of clinical strategies. We recommend
NHLBI continue including cost-effectiveness analyses in relevant
CV clinical trials that it sponsors. Establishing cost-effectiveness
in real world settings is equally important to the efficacy evaluations
in clinical trials. We recommend that the NHLBI consider fostering
investigations of the economic consequences of alternative clinical
strategies to reduce risk and diagnose and treat disease in real
world settings. These investigations, funded by non-industry sources,
are important to guiding reimbursement policies and resource allocation.
V. Additional Recommendations
Promote research using existing databases.
The investment in studies by the NHLBI and other federal agencies
has produced several detailed datasets that contain information
that can be used to yield important insights even after the specific
aims of the project are addressed. The datasets are a national
resource and have tremendous potential to yield important insights
that can advance clinical science. The NHLBI has done an excellent
job making datasets available to investigators. Support for such
access to databases should be continued. In addition, we propose
that mechanisms be expanded to provide funding support for proposals
that make appropriate use of existing data to address important
research questions. This mechanism ideally would have a rapid
evaluation cycle and be directed specifically for data analysis.
Develop and refine analytic tools in Outcomes Research.
We need to promote the development of advanced analytic tools from
other disciplines that can be used in Outcomes Research. Such
disciplines include biostatistics, econometrics, and the behavioral
sciences. Examples of powerful tools used in these disciplines
but generally not supported in CV research efforts are: hierarchical
linear models (for nested study designs such as group-randomized
trials of physician-level interventions); propensity scores and
instrumental variables (for causal inferences from observational
evaluation), and structural equation modeling (for the study of
unobservable categories underlying observed phenomena such as
components of CV quality of care). Basic methodological work also
needs to be undertaken to quantify potential differences among
providers in their quality of care, to develop and refine tools
to assess patient-centered data (i.e. preferences and health status)
including strategies to overcome barriers to the use of these
data in clinical practice, and to advance the methodology of the
handling of missing data. Finally, research methods are needed
to enhance appropriate interpretation of data on health status
and resource use. If CV researchers are to use these advanced
tools, methodological research to further advance the tools should
also be supported. To support the interdisciplinary work in this
area the NHLBI could consider Program Project grants in Outcomes
Support trainees/young investigators.
Ultimately, CV Outcomes Research will be defined by the investigators
who are dedicated to this field. As the field grows and matures,
an essential component will be the specific training and career
development of young investigators. This training needs to include
the development of skills necessary to conduct high-level Outcomes
Research and provision of strong mentorship from established outcomes
researchers. To this end, we recommend the formation of career
development awards (KO8/K23) within NHLBI specifically dedicated
to CV Outcomes Research. The review panel for these applications
should consist of established outcomes investigators with multidisciplinary
In addition to the standard requirements for KO8/K23 applicants,
key components to career development awards in CV Outcomes Research
A. Requirement of a formal educational plan with emphasis on the
attainment of an advanced degree (e.g. PhD, MPH) in an appropriate
field (e.g. health services research, epidemiology, statistics,
public health), unless already attained. Career development awardees
would be required to obtain or document prior formal didactic
training in topics such as clinical epidemiology, biostatistics,
health services research methods, health policy, health economics,
and health systems.
B. Strong local and national mentorship including:
- Demonstration of strong local support for the development of the
awardee, including commitment of protected clinical time and administrative
- Local mentorship in Outcomes Research by one or more investigators
who are established independent outcomes researchers (e.g. active
funding and a publication track record in the field as defined
in this document) and can offer significant time commitment to
the development of the awardee.
- National collaborative mentorship with established outcomes researchers
in areas that will complement the applicant’s research interests
and promote further career development.
- Multidisciplinary mentorship and collaboration.
- An Outcomes Research application by the awardee that addresses
one or more important Outcomes Research domains (e.g., the priorities
outlined in this document or one or more of the IOM domains of
quality), and demonstrates the applicant’s increasing independence
as an investigator.
- A detailed career plan from the awardee demonstrating strong commitment
to an academic career as an independent outcomes researcher and
clear short- and long-term career goals.
The formation of dedicated career development awards in CV Outcomes
Research would directly support many of the key recommendations
of the NHLBI Research Training and Career Development report (November
1999), including expansion of existing training programs to address
new/emerging areas, the inclusion and emphasis on advanced education
of the awardees, a strong emphasis on mentorship plans, and a
multidisciplinary approach to research and mentorship.
There is also a need for support for mid-career awards. The NIH
has K18 awards (1- 2 years of support for "re-tooling and gaining
lab experience" for mid-career investigators who want to broaden
or redirect their research). These awards are usually targeted
towards a specific area of NIH interest (e.g. genetics, stem cells,
etc.). This might be a good mechanism for the mid-career clinician
or scientist who wants to learn Outcomes Research methodology.
We believe that the implementation of career development awards
in CV Outcomes Research will help ensure that the NHLBI trains
the leaders in this field for the future.
Expand study sections’ expertise in Outcomes Research.
The Working Group recommends efforts be undertaken to ensure that
study sections that will review Outcomes Research applications
include experts who appreciate the role of Outcomes Research,
understand the methods employed, and understand the strengths
and weaknesses of different types of research designs employed
in this type of work. Ensuring that study sections have appropriate
representation of Outcomes Research investigators is crucial to
the fair evaluation of applications responsive to the research
priorities described in this report.
CV Outcomes Research holds great promise for providing evidence
that will guide the evolution of health care delivery and promote
a health care system that is safe, effective, efficient, timely,
equitable and patient-centered. CV Outcomes Research seeks to
discover new knowledge about the prevention, diagnosis and treatment
of CVD through a focus on the end results of health care; and
seeks to ensure the translation of ‘best practice’ into clinical
practice. The focus is on the patient and ‘real world’ settings
with the intention of addressing the practical needs of
clinicians, clinical policy makers, purchasers and patients/consumers.
This document provides a roadmap to support investigations that
are directly relevant to clinical care and complement the longer-term
investments of the Institute. We believe that a dedicated investment
in CV Outcomes Research will directly improve the quality of care
delivered in the United States.
Posted: June 2004