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Appendix D
The Learning Health System Series:
Workshop Common Themes
1. VISION
The Learning Healthcare System
Adaptation to the pace of change: continuous
learning and a much more dynamic approach
to evidence development and application, tak-
ing full advantage of developing information
technology to match the rate at which new
interventions are developed and new insights
emerge about individual variation in response
to those interventions.
Stronger synchrony of efforts: better consis-
tency and coordination of efforts to gener-
ate, assess, and advise on the results of new
knowledge in a way that does not produce
conflict or confusion.
Culture of shared responsibility: to enable the evolution of the learning
environment as a common cause of patients, providers, and researchers and
better engage all in improved communication about the importance of the
nature of evidence and its evolution.
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284 PATIENTS CHARTING THE COURSE
New clinical research paradigm: drawing clinical research closer to the
experience of clinical practice, including the development of new study
methodologies adapted to the practice environment and a better under-
standing of when RCTs are most practical and desirable.
Clinical decision support systems: to accommodate the reality that although
professional judgment will always be vital to shaping care, the amount of
information required for any given decision is moving beyond unassisted
human capacity.
Universal electronic health records: comprehensive deployment and effec-
tive application of the full capabilities available in EHRs as an essential
prerequisite for the evolution of the learning healthcare system.
Tools for database linkage, mining, and use: advancing the potential for
structured, large databases as new sources of evidence, including issues in
fostering interoperable platforms and in developing new means of ongoing
searching of those databases for patterns and clinical insights.
Notion of clinical data as a public good: advancement of the notion of the
use of clinical data as a central common resource for advancing knowl-
edge and evidence for effective care—including directly addressing cur-
rent challenges related to the treatment of data as a proprietary good and
interpretations of the Health Insurance Portability and Accountability Act
(HIPAA) and other patient privacy issues that currently present barriers to
knowledge development.
Incentives aligned for practice-based evidence: encouraging the develop-
ment and use of evidence by drawing research and practice closer together,
and developing the patient records and interoperable platforms necessary
to foster more rapid learning and improve care.
Public engagement: improved communication about the nature of evidence
and its development, and the active roles of both patients and healthcare
professionals in evidence development and dissemination.
Trusted scientific broker: an agent or entity with the public and scientific
confidence to provide guidance, shape priorities, and foster the shift in the
clinical research paradigm.
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APPENDIX D
Leadership: to marshal the vision, strategy, and actions necessary to create
a learning healthcare system.
2. CARE COMPLEXITY
Evidence-Based Medicine and the Changing Nature of Health Care
Increasing complexity of health care: New
pharmaceuticals, medical devices, technologies,
and predictive data offer much promise for im-
proving health care, but they also introduce
high levels of complexity, requiring changes on
the parts of both caregivers and their patients.
Unjustified discrepancies in care patterns: The
intensity of healthcare service delivered for
similar conditions varies significantly across
geographic regions, particularly in areas that
require discretionary decision making. How-
ever, the higher-spending regions often do not
deliver better-quality care, hence offering sub-
stantial opportunity for reduced spending with-
out sacrificing health outcomes.
Importance of better value from health care: The current healthcare system
is not designed to deliver value, and the nation’s long-term fiscal challenges
are serious and are being driven predominately by excessive medical spend-
ing, often on interventions of no clinical benefit. Opportunities exist to
eliminate wasteful spending with no reduction in health care, as well as
to improve the overall health outcomes, but agreement is needed both on
what constitutes best care and on what constitutes value in health care.
Uncertainty exposed by the information environment: An irony of the
information-rich environment is that information important to clinical
decision making is often not available, or is provided in forms that are not
relevant to the broad spectrum of patients—with differing levels of health,
socioeconomic circumstances, and preferences—and the issues encountered
in clinical practice. This is due to too little clinical effectiveness research,
too poor dissemination of the evidence that is available, and too few incen-
tives and decision supports for evidence-based care.
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286 PATIENTS CHARTING THE COURSE
Pressing need for evidence development: More and better evidence—
including comparative and longitudinal data—is needed to determine the
effectiveness and usefulness of new medical interventions, treatments, drugs,
devices, and genetic information. There is an untapped potential to reduce
healthcare costs and improve quality by developing evidence not only for
specific medical interventions, but also for the way health care is delivered.
Promise of health information technology: Electronic medical records (EMRs)
and clinical data registries offer tremendous potential both to generate new
evidence and to augment randomized clinical trials. Addressing privacy and
proprietary issues that limit data access and sharing would help to support a
system in which electronic medical records, clinical registries, and other types
of electronic data could contribute to building a more robust evidence base.
Need for more practice-based research: How might the system better sup-
port the notion of a “living textbook of medicine” in which the experi-
ence of healthcare diagnosis and treatment is routinely captured in order
to better care for those in the future. To develop best evidence for the
delivery of medicine that is geared toward the needs of individual patients,
investment is needed into infrastructure for the gathering and analysis of
healthcare data and information, and standards and protocols to ensure
their accuracy and reliability.
Shift to a culture of care that learns: This changing role will require health-
care providers and patients to adopt a culture that supports the generation
and application of evidence. Effective culture change must also be accom-
panied by insurance and reimbursement system reform that encourages
development and application of the systems necessary.
New model of patient-provider partnership: With the increasing complexity
of care, and the need and demand for more patient involvement, the tra-
ditional “physician-as-sole-authority” model will need to adapt to support
patients as integral partners in medical decisions.
Leadership that stems from every quarter: Adapting to and taking advan-
tage of, the changes in the healthcare environment will take broad leader-
ship. A strategic focus on the development and application of evidence will
require the involvement of both the public and private sectors working
together, and with policy makers, providers, patients, insurers, and other
stakeholders in the steps toward change.
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APPENDIX D
3. EFFECTIVENESS RESEARCH
Redesigning the Clinical Effectiveness Research Paradigm:
Innovation and Practice-Based Approaches
Address current limitations in applicability
of research results: Because clinical condi-
tions and their interventions have complex
and varying circumstances, there are different
implications for the evidence needed, study
designs, and the ways lessons are applied:
the internal and external validity challenge.
In addition, although our assessment of can-
didate interventions is primarily through
pre-market studies, the opportunity for dis-
covery extends throughout the lifecycle of an
intervention—development, approval, cover-
age, and the full period of implementation.
Counter inefficiencies in timeliness, costs, and
volume: Much of current clinical effective-
ness research has inherent limits and inefficiencies related to time, cost and
volume. Small studies may have insufficient reliability or follow-up. Large
experimental studies may be expensive and lengthy, but have limited applica-
bility to practice circumstances. Studies sponsored by product manufacturers
have to overcome perceived conflicts and may not be fully used. There is a
strong need for more systematic approaches to better defining how, when,
for whom, and in what setting an intervention is best used.
Define a more strategic use to the clinical experimental model: Just as there
are limits and challenges to observational data, there are limits to the use
of experimental data. Challenges related to the scope of inferences possible,
to discrepancies in the ability to detect near-term vs. long-term events, to
the timeliness of our insights and our ability to keep pace with changes in
technology and procedures, all must be managed. For the future of clinical
effectiveness research, the important issues relate not to whether random-
ized experimental studies are better than observational studies, or vice
versa, but to what’s right for the circumstances (clinical and economic), and
how the capacity can be systematically improved.
Provide stimulus to new research designs, tools, and analytics: An exciting
part of the advancement process has been the development of new tools
and resources that may quicken the pace of our learning and add real value
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288 PATIENTS CHARTING THE COURSE
by helping to better target, tailor, and refine approaches. Use of innovative
research designs, statistical techniques, probability and other models may
accelerate the timeliness and level of research insights. Some interesting ap-
proaches using modeling for virtual intervention studies may hold prospects
for revolutionary change in certain clinical outcomes research.
Encourage innovation in clinical effectiveness research conduct: The kinds
of “safe harbor” opportunities that exist in various fields for developing
and testing innovative methodologies for addressing complex problems are
rarely found in clinical research. Initiative is needed for the research com-
munity to challenge and assess its approaches—a sort of meta-experimental
strategy—including those related to analyzing large datasets, in order to
learn about the purposes best served by different approaches. Innovation
is also needed to counter the inefficiencies related to the volume of studies
conducted. How might existing research be more systematically summa-
rized; or, different research methods organized, phased, or coordinated to
add incremental value to existing evidence?
Promote the notion of effectiveness research as a routine part of practice:
Taking full advantage of each clinical experience is the theoretical goal of
a learning healthcare system. But for the theory to move closer to the prac-
tice, tools and incentives are needed for caregiver engagement. A starting
point is with the anchoring of the focus of clinical effectiveness research
planning and priority setting on the point of service—the patient–provider
interface—as the source of attention, guidance, and involvement on the key
questions to engage. The work with patient registries by many specialty
groups is an indication of the promise in this respect, but additional em-
phasis is necessary in anticipation of the access and use of the technology
that opens new possibilities.
Improve access and use of clinical data as a knowledge resource: With the
development of bigger and more numerous clinical data sets, the potential
exists for larger scale data mining for new insights on the effectiveness of
interventions. Taking advantage of the prospects will require improve-
ments in data sharing arrangements and platform compatibilities, address-
ing issues related to real and perceived barriers from interpretation of
privacy and patient protection rules, enhanced access for secondary analysis
to federally sponsored clinical data (e.g., Medicare part D, pharmaceutical,
clinical trials), the necessary expertise, and stronger capacity to use clinical
data for post-market surveillance.
Foster the transformational research potential of information technology:
Broad application and linkage of electronic health records holds the poten-
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APPENDIX D
tial to foster movement toward real-time clinical effectiveness research that
can generate vastly enhanced insights into the performance of interventions,
caregivers, institutions, and systems—and how they vary by patient needs
and circumstances. Capturing that potential requires working to better
understand and foster the progress possible, through full application of
electronic health records, development and application of standards that
facilitate interoperability, agreement on and adherence to research data col-
lection standards by researchers, developing new search strategies for data
mining, and investing patients and caregivers as key supporters in learning.
Engage patients as full partners in the learning culture: Access to up-to-date
information by both caregiver and patient changes the state of play in sev-
eral ways. The patient sometimes has greater time and motivation to access
relevant information than the caregiver, and a sharing partnership is to the
advantage of both. The more patients understand and communicate with
their caregivers about the evolving nature of evidence, the less disruptive
will be the frequency and amplitude of public response to research results
that find themselves prematurely, or without appropriate interpretative
guidance, in the headlines and short-term consciousness of Americans.
Build toward continuous learning in all aspects of care: This foundational
principle of a learning healthcare system will depend on system and culture
change in each element of the care process with the potential to promote in-
terest, activity, and involvement in the knowledge and evidence development
process, from health professions education to care delivery and payment.
4. DATA UTILITY
Clinical Data as the Basic Staple of Health Learning:
Creating and Protecting a Public Good
Clarity on the basic principles of clinical data stewardship: The starting
point for expanded access and use of clinical data for knowledge develop-
ment is agreement on some of the fundamental notions to guide the activi-
ties for all individuals and organizations with responsibility for managing
clinical data. Workshop participants repeatedly mentioned the need for
consensus on approaches to such issues as data structure, standards, report-
ing requirements, quality assurance, timeliness, de-identification or security
measures, access and use procedures—all of which will determine the pace
and nature of evidence development.
Incentives for real-time use of clinical data in evidence development: Current
barriers to the real-time use of clinical data for new knowledge discussed
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at the workshop ranged from regulatory and
commercial issues to cost and quality issues.
Participants suggested the need for a dedi-
cated program of activities, incentives, and
strategies to improve the methods and ap-
proaches, their testing and demonstration,
the cooperative decision making on priorities
and programs, and the collective approach to
regulatory barriers.
Transparency to the patient when data are
applied for research: Patient acceptance is key
to use of clinical data for knowledge develop-
ment, and patient engagement and control are
key to acceptance. In this respect, clarity to
individual patients on the structure, risks, and
benefits of access to data for knowledge development was noted by partici-
pants as particularly important. Patient confidence and system accountabil-
ity may be enhanced through transparent notification and audit processes
in which patients are informed of when and by whom their information has
been accessed for knowledge development.
Addressing the market failure for expanding electronic health records:
Currently, market incentives are inadequate to bring about the expansion
of use of electronic health records necessary to make the point of care a
locus for the development, sharing, and application of knowledge about
what works best for individual patients. Shortfalls noted by participants
included demand by providers or patients that is not sufficient to counter
the expense to small organizations, competing platforms and asynchronous
reporting requirements that work against their utility for broad quality
and outcome determinations, and that even the larger payers—apart from
government—do not possess the critical mass necessary to drive broader
scale applicability and complementarity. It will likely take a deeper, more
directed and coordinated strategy involving Medicare leadership to foster
such changes.
Personal records and portals that center patients in the learning process:
Patient demand could be instrumental in spreading the availability of elec-
tronic health records for improving patient care and knowledge develop-
ment. Such demand will depend upon much greater patient access to,
comfort with, and regular use of programs that allow either the mainte-
nance of personal electronic health records or access through a dedicated
portal to their provider-maintained electronic medical record. As noted
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APPENDIX D
during the workshop, many consumer-oriented products currently under
development give patients and consumers more active roles in managing
personal clinical information, and may help to demonstrate value in the
speed and ease of personal access to the information, better accommodate
patient preference in care, and foster a partnership spirit conducive to the
broader EHR application.
Coordinated EHR user organization evidence development work: The devel-
opment of a vehicle to enhance collaboration among larger EHR users of dif-
ferent vendors was raised during the workshop as a means to accelerate the
emergence of more standardized agreements and approaches to integrating
and sharing data across multiple platforms, common query strategies, virtual
data warehousing rules and strategies, relational standards, and engagement
of ways to reduce misperceptions on regulatory compliance issues.
The business case for expanded data sharing in a distributed network:
Demonstrating the net benefits of data sharing could promote its use.
Benefits suggested by participants included cost savings or avoidance from
facilitated feedback to providers on quality and outcomes; quick, continu-
ous improvement information; and improved management, coordination,
and assessment of patient care.
Assuring publicly funded data for the public benefit: Federal and state funds
that support medical care, as well as support insights into medical care
through clinical research grant funding, are the source of substantial clinical
data; yet, many participants observed that these resources are not yet ef-
fectively applied to the generation of new knowledge for the common good.
Broader semantic strategies for data mining: Platform incompatibilities for
clinical data substantially limit the spread of electronic health records and
their use for knowledge development. Yet discussion identified strategies
using alternative semantic approaches for mining clinical data for health
insights, which may warrant dedicated cooperative efforts to develop and
apply them.
Public engagement in evidence development strategies: Generating a base of
support for and shared emphasis on developing a healthcare ecosystem in
which all stakeholders play a contributory role was noted by many partici-
pants as important for progress. Ultimately, the public will determine the
broad acceptance and applicability of clinical data for knowledge develop-
ment, underscoring the importance of keeping the public closely involved
and informed on all relevant activities to use clinical data to generate new
knowledge.
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5. EVIDENCE
Learning What Works: Infrastructure Required
for Comparative Effectiveness Research
Coordinating work and ensuring standards
are key components of the evidence infra-
structure: Infrastructure for evidence devel-
opment includes the capacity for greater
coordination in the setting of study priori-
ties; the development of systematic decisions
for the conduct of comparative effectiveness
research, systematic reviews, and guideline
development; and ensuring the consistent
translation of developed information.
Learning about effectiveness must continue
beyond the transition from testing to practice:
Pre-market testing for the safety and effective-
ness of various interventions cannot assess
the results for all populations or the circum-
stances of use and differences in practice patterns, so gathering information
as interventions are applied in practice settings should represent a key focus
in designing the infrastructure to learn which care is best.
Timely and dynamic evidence of clinical effectiveness requires bridging
research and practice: Although historical insulation of clinical research
from the regular delivery of healthcare services evolved to facilitate data
capture and control for confounding factors, it may not adequately inform
the real-world setting of clinical practice. With the prospect of enhanced
data capture electronically at the point of care, on real-world patient popu-
lations, and statistical approaches to improve analysis, as well as increasing
demand to keep pace with technologic innovation, the divide of clinical
research from care practice increasingly limits the utility of research results.
Current infrastructure planning must build to future needs and oppor-
tunities: Emerging questions include those related to the management of
multiple co-occurring chronic diseases of increasing prevalence in an aging
population, the improved insights into individual variation relevant to both
treatments and diagnostics, and the impact of innovation in shortening the
lifecycle of any particular intervention. Emerging tools include innovations
in trial design, the development of new statistical approaches to data analy-
sis, and the development of electronic medical and personal health records.
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APPENDIX D
Keeping pace with technological innovation compels more than a head-to-
head and time-to-time focus: With the rapid pace of change in the nature
of interventions and the difficulty, expense, and time required to develop
studies—and the challenges of ensuring the generalizability of results in
the face of limitations of traditional approach to randomized controlled
trials (RCTs)—a first-order priority for the effectiveness research is the
establishment of infrastructure for a more dynamic, real-time approach
to learning. Leveraging new tools such as health IT should allow for a
more networked and distributed approach to information sharing and
evidence creation.
Real-time learning depends on health information technology investment: It
was noted that collecting data is the most time-intensive part of trials and
studies, and IT is critical to streamlining this work. The increasing complex-
ity of the factors involved in understanding the effectiveness of clinical op-
tions under different circumstances requires a blend of database access and
computing power that can only be provided from broadly applied health
information technology. A policy framework for privacy and security will
be necessary to build and maintain public trust that information will be
protected as it is shared.
Developing and applying tools that foster real-time data analysis is an
important element: The scope and scale of evidence needs suggests that
innovation is needed across the range of research methods, from making
clinical trials faster and less expensive, to moving beyond randomized trials
to better address practical circumstances. To take advantage of health in-
formation technology, statistical tools, and analytic algorithms that can be
embedded in databases to allow real-time insights will be important. Simi-
larly, tools are needed that will allow findings to be drawn from databases
built on different vendor platforms, using semantic technology to integrate
currently disparate medical data, and for developing the next generation
of statistical tools for the analysis of clinical data, including the building of
models that allow insights to be generated by virtual studies.
A trained workforce is a vital link in the chain of evidence stewardship:
Given the pace of change in the number and variety of clinical interventions
as well as in the tools and approaches to assessing them, there is a need to
ensure that these developing opportunities are matched by the skills of the
workforce. This includes training and education in the methodologies of
research design, translating research, guideline development, and maintain-
ing and mining clinical records. It also includes attention to re-orienting the
education of front-line caregivers around their emerging responsibilities for
access, interpretation, and discussion with patients of a dynamic evidence
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APPENDIX D
problems, which may result in treatment errors and duplicative services.
Participants described how team-based care offers the potential to rectify
this disconnected care and limit human error. Effective teams are aided by
an appropriate information technology infrastructure, which facilitates
efficient and effective communication of health information, as well as by
collaborative organizational systems, from medical homes to accountable
care organizations.
Efficient: Patients, their families, and clinicians should expect care to be
appropriate to need, resources, and time required. Participants underscored
the fact that currently, much of the care that is delivered is neither neces-
sary nor efficient. Among the chief complaints of patients are increasing
out-of-pocket costs and lost time in the care process. This finding is not
surprising given that the current incentive structure, focused on volume
over value, encourages overuse and waste. As multiple participants noted,
the United States spent roughly 17 percent of its gross domestic product on
health care last year, yet this investment did not yield the health outcomes
commensurate with the costs. To gain greater value, participants stressed,
the costs and outcomes of care must be more transparent to patients, and
new payment models—ranging from bundling payments for an entire epi-
sode of care, to pay-for-performance systems, to global payment—must be
implemented.
Accountable: All relevant aspects of the clinical experience, including patient
perspectives, should be captured and routinely assessed against expectations.
This information is vital not only to achieving effective patient management,
but also to judging whether experiments with new delivery system models,
payment incentives, or standards of care are having their intended effect on
improving patient health and promoting efficiency. Measuring performance
and disseminating innovations that work (and eliminating those that do not)
constitute a systematic way of improving healthcare delivery. One presenta-
tion highlighted how this systematic approach to improvement allowed the
speaker’s organization to enhance care by conducting comprehensive reviews
of interventions for different conditions, adopting the best practices identi-
fied by that review, and measuring the performance of the revised standard
of care.
Transparent: Information on the outcomes of care—both effectiveness and
efficiency—should be readily accessible and understandable to patients
and their families. Several speakers mentioned the frustration felt by
patients regarding the lack of understandable information on the costs,
quality, and outcomes of care, especially in light of reports about medical
errors and the increasing personal burden of costs and inefficiencies of
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302 PATIENTS CHARTING THE COURSE
care. It was noted that, when offered a choice, patients do not routinely
choose more costly or more intensive interventions. However, choice and
information about alternatives are rarely available. It is clear that action
to improve value—better outcomes at lower cost—requires transparent
information on the costs and outcomes of care.
Trustworthy: Patients should expect a strong and secure trust fabric on all
dimensions—safety, quality, security, efficiency, accountability, and equity.
In few areas of human endeavor is trust on each of these dimensions more
important. Yet one presenter noted that, even in the face of information that
50,000 to 90,000 deaths per year are caused by medical errors, health care
lacks the basic trust elements of transparency and accountability needed to
drive improvements in quality and safety. In a learning system that draws
lessons from each care experience, public trust must be bolstered in all
aspects of the healthcare enterprise: equitable access to reliable clinician
knowledge and skills, safeguards on clinical processes, privacy and security
of medical records, and validity and safety of clinical trials.
Learning: In a learning health system, the patient is an active contributor
to, and supporter of, the learning process. Each patient experience offers
the potential to deepen the knowledge base that drives care quality and
outcomes—at the individual, practice, and societal levels. A focus of the
workshop was the stake of the patient in fostering a digital health utility
that provides needed information to patients and their clinicians, ensures
synchronization among providers, and generates knowledge for progress—
for example, for comparative effectiveness insights, public health activities,
or postmarket monitoring of approved technologies and drugs. Reference
was made, for example, to the need for a common core data set for EHR-
based data that would allow reliable, platform-independent research across
large patient populations. These are issues in which patients have a strong
stake, and they must have confidence in the system’s functionality for the
generation of timely and reliable new insights.
9. COST AND OUTCOMES
The Healthcare Imperative: Lowering Costs and Improving Outcomes
Challenges for health costs and outcomes
Health cost excesses with personal, institutional, and national conse -
quences. In the past decade, U.S. health costs have increased by 92 percent,
representing approximately four times the inflation rate for the economy as
a whole. Out-of-pocket costs for individuals have increased by 40 percent.
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APPENDIX D
Overall, Medicaid now takes almost 20 per-
cent of state budgets, crowding out other
state priorities such as education.
Health outcomes far short of expectations.
Despite health spending double the average
for other developed nations, U.S. health out-
comes rank below two to three dozen other
countries on indices such as life expectancy,
care for chronic disease, and persistent dis-
parities in the access and outcomes of care.
Fragmented decision points, inconsistent
principles, and political distortions. Barriers
to appropriate care include poor care coor-
dination, lack of consistent evidence-based
guidelines, payment systems that encourage volume over value, and politi-
cal influences that sometimes overturn scientific determinations.
Domains of waste and inefficiency in healthcare spending
Unnecessary services: services reflecting choices or levels beyond those sup-
ported by evidence or benchmarks.
Inefficiently delivered services: inefficient labor use, time-flow discontinui-
ties, duplicate services, medical errors.
Excess administrative costs: billing and insurance-related costs for payers
and providers, inefficient reporting requirements.
Prices that are too high: for medical services, pharmaceuticals, products and
devices, relative to benchmarks.
Missed prevention opportunities: for preventable obesity, diabetes, heart
disease, stroke, pulmonary disease, cancers, infections.
Medical fraud: systemic over-billing, billing for undelivered services, use of
unlicensed providers.
Drivers of the problems
Scientific uncertainty. Clinical evidence development is not keeping pace
with new diagnostics, treatments, and insights into individual variation.
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Perverse economic and practice incentives. The fee-for-service reimburse-
ment system rewards service volume rather than value.
System fragmentation. Multiple, disconnected, and uncoordinated decision
points in healthcare delivery and finance are fundamental challenges to
efficient and effective care.
Opacity as to cost, quality, and outcomes. Without meaningful and trust-
worthy sources of information on costs and outcomes of care, neither
patients nor their clinicians can make fully informed decisions.
Changes in health status. An aging population, the growing prevalence of
obesity, and increases in multiple co-occurring, complex chronic diseases
are accelerating the need for health services.
Lack of patient involvement. The culture of care is not yet conducive to
active patient participation in care decisions, despite growing use of web-
accessible information and evidence of the positive effect of shared decision
making on health outcomes.
Under-investment in population health. Because health status is impor-
tantly influenced by behavioral, social, and environmental factors, progress
depends on a stronger commitment to population-wide health programs.
Corrective levers
Streamlined and harmonized health insurance regulation. Reduce com-
plexities and inconsistencies in coverage standards and requirements often
unique to a jurisdiction.
Administrative simplification and consistency. Streamline and harmonize
inconsistent payment and reporting requirements that create unnecessary
and excessive administrative costs.
Focus incentives on results and value. Focus payments on outcomes and
value, and increase targeting those at highest risk of poor outcomes.
Quality and consistency in treatment. Establish treatment guidelines as the
starting point for effective care, tailoring as indicated, and capturing the care
experience for continuous improvement.
Evidence that is timely, independent, and understandable. Foster effective
care through a dedicated, unified program that provides reliable guidance;
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APPENDIX D
keeps up with innovation and changing science; and improves practice reli-
ability, consistency, and impact.
Clinical records that are reliable, sharable, and secure. Use electronic health
records to enhance the effectiveness and efficiency of care, facilitate patient
handoffs, provide clinical decision guidance, and foster patient involvement.
Data that are protected but accessible for continuous learning. Create a
digital utility with clinical data as a resource for real-time monitoring of
the results of treatment, ongoing generation of new evidence for effective
care, and continuous care improvement.
Transparency requirements on cost, quality, and outcomes. Build an ac-
cessible information resource, with transparency as to cost, outcomes, and
value serving as a critical element of system change.
Culture and activities framed by patient perspective. Position patient perspec-
tives and needs as primary—and convenience and interest as secondary—for
the design and execution of healthcare organization and delivery.
Medical liability reform. Diminish defensive medicine as a detrimental,
significant driver of unnecessary services and procedures—e.g., through
harbors for best evidence practices, caps on non-economic damages, spe-
cialized tribunals.
Prevention. Elevate the focus on prevention, ranging from clinical preven-
tive services to community health and wellness.
10. VALUE
Value in Health Care: Accounting for Cost,
Quality, Safety, Outcomes, and Innovation
Urgency: Increasing healthcare costs consistently outpace inflation, squeez-
ing out employer coverage, adding to the uninsured, and doubling out-
of-pocket payments—without commensurate health improvements. The
long-term consequences for federal budget obligations, driven by the
growth in Medicare costs, amount to an estimated $34 trillion in unfunded
obligations.
Perceptions: We have heard that for patients perceived value in health
care is often described in terms of the quality of their relationship with
their physicians. Clinicians discussed value as diagnostic and treatment ap-
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306 PATIENTS CHARTING THE COURSE
proaches that offer increased confidence in
the effectiveness of services offered. Employ-
ers viewed value improvement in terms of
keeping workers and their families healthier
and more productive at lower costs. For
health insurers, we heard that value improve-
ment means emphasizing interventions that
are crisply defined and supported by a high
level of evidence. Health product innovators
spoke of value improvement as a product
that is better for the individual patient, more
profitable, and contributes to product differ-
entiation and innovation.
Elements: Value from health care has dimen-
sions beyond the nature, cost, and effective-
ness of a particular intervention, including those related to elements such
as preference, satisfaction, and appropriateness to circumstance. Value
determination also means determining the right price, and we heard that,
from the demand side, the right price is a function of perspective. From the
supply side, the right price is a function of the cost of production, the cost
of delivery, and the incentive to innovation.
Gain: Because reliable information is the starting point for improving value,
discussants underscored the importance of adequate transparency and con-
tinuous improvement of insights on the safety, efficacy, effectiveness, and
comparative effectiveness of interventions.
Decisions: Currently, decision rules seem to many stakeholders to be vague,
inconsistent, and poorly tailored to the evidence.
Information: Because the quality of evidence varies, as do the methods used
to evaluate it, transparency as to source and process, care as to interpreta-
tion, and clarity in communication were noted as key.
Incentives: Often noted in the workshop discussions was that the incentives
prevalent in the American healthcare system are poorly aligned to effec-
tiveness and efficiency, encouraging care that is procedure- and specialty-
intensive and discouraging primary care and prevention.
Limits: We have heard that obtaining the value needed will continue to be
elusive until better means are available to draw broadly on information
as to services’ efficiency and effectiveness, to set priorities and streamline
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APPENDIX D
approaches to filling the evidence gaps, to ensure consistency in the ways
evidence is interpreted and applied, and to marshal incentives to improve
the delivery of high-value services while discouraging those of limited value.
Communication: Patients and providers do not communicate well with
each other about diagnosis and treatment options or cost implications.
Communication is often absent between multiple providers for a single
patient, increasing the prospect of service gaps, duplications, confusion, and
harm, according to discussants. Further, communication between scientific
and professional organizations producing and evaluating evidence is often
limited, resulting in inefficiencies, missed opportunities, and contradictions
in the production of guidance.
Providers: We heard that the clearest barriers to provider-level value im-
provement appear to lie in the lack of economic incentives for a focus on
outcomes and also in cultural and structural disincentives to tend to the
critical interfaces of the care process—the quality of the links in the chain
of care elements.
Patients: It was noted that patients most often think of value in terms of
their relationship with their provider but ultimately the practical results
of that relationship, in terms of costs and outcomes, hinge on the suc-
cess of programs that improve practical, ongoing, and seamless access to
information on best practices and costs and of payment structures that
reward accordingly. Workshop discussants offered insights into the use of
various financial approaches to sensitize and orient patient decisions on
healthcare prices according to the evidence of the value delivered.
Manufacturers: Health product manufacturers and innovators naturally
focus on their profitability but because product demand is also derived from
the ability to demonstrate advantage with respect to outcomes and efficiency,
manufacturers expressed an interest in regulatory and payment approaches
that enhance performance on outcomes resulting from product use.
Tools: Despite the broad agreement on their importance, we heard that the
analytical tools and capacity to evaluate, in either absolute or comparative
terms, the basic elements of value—outcomes and costs—are substantially
underdeveloped and will need greater attention.
Opportunities: Although attaining better value in health care depends on
reducing the fragmentation that is its central barrier, we heard a number
of examples of measures that might be taken at different levels, both to
achieve better value now and to set the stage for future progress.
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308 PATIENTS CHARTING THE COURSE
11. LEADERSHIP
Leadership Commitments to Improve Value in Health Care:
Finding Common Ground
Build trust and collaboration: Health care
depends for its effectiveness on the close co-
operation of all parties involved. Building
trust and facilitating transformative change
will require broader-based collaboration and
cooperative stakeholder engagement.
Foster agreement on “value” in health care:
Although all participants agreed on the cen-
trality and importance of the value achieved
from health care, different groups often think
of value in different ways. A multistakeholder
effort might drive clarity and consensus on
those principles and elements of value com-
mon to all stakeholders.
Improve public understanding of evidence: Too often, people perceive that
certain common terms such as “evidence based,” “research,” “medical
necessity,” and “risk” suggest a restrictive or experimental element to their
care. It will take systematic and coordinated communication strategy to
better convey the central concepts that medical evidence is dynamic, that
evidence-based medicine is the provision of care that the evidence suggests
is best for any given patient at any given point in time, and that health care
is a joint patient–provider endeavor.
Characterize the impact of shortfalls in the evidence: Documenting the
consequences of provision of care on the basis of too little evidence or
the potential benefits of providing care on the basis of the right evidence
is a prerequisite to obtaining an improved understanding of and demand
for evidence-based care and stakeholder activation.
Identify the priorities for evidence development: The first step to a system-
atic and coordinated effort to conduct the most important assessments is the
identification of the priorities as a sort of consensus national problem list
and research agenda of the most pressing issues for medical care decisions.
Improve the level, quality, and efficiency of the research: Policies that
facilitate the ability to use clinical data to monitor the effectiveness of in-
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APPENDIX D
terventions are needed. Novel approaches to the conduct of clinical trials
are needed. A more structured lexicon for “best practices” in undertaking
observational studies may be necessary.
Clarify and promote transparency: Consensus is needed to establish com-
mon principles of transparency and standards for how they should be
applied in each sector. One starting point might be with principles for
evidence interpretation.
Establish principles for the interpretation and use of evidence: Decisions on
market approval, insurance coverage, provider use, and patient acceptance
are all informed by some interpretation of the evidence. Clarity of the guid-
ing principles is important.
Improve engagement in the full lifecycle of interventions: Many factors are
at play for each intervention—for example, similarity to previously tested
interventions, the safety and effectiveness of an intervention for some popu-
lations but not others, the availability of biomarkers predictive of efficacy,
and costs that vary by scale and stage of application or by the need for later
services. Facilitating innovation, access, and effective information gathering
while emphasizing patient safety, appropriate application, improved out-
comes, and efficiency will require a set of lifecycle-oriented decision-making
rules that are more carefully considered than they are at present.
Focus on frontline providers: Accelerating the translation of clinical re-
search into practice involves addressing matters of professional education,
credentialing, licensure, practice support, economic incentives, patient ac-
ceptance, and the culture of care. It will require the central and coordinated
involvement of the organizations that represent those providers.
Foster a trusted intermediary for evidence: In this information age, health-
related information is constantly presented through news reports, market-
ing, professional organizations, journals, and the Internet, but it is often
confusing and even contradictory. A trusted information source—one that
is independent but that engages all stakeholders—is needed to identify gaps;
set priorities; establish standards; and guide the development, interpreta-
tion, and dissemination of evidence on clinical effectiveness.
Build the capacity to meet the demand: Currently, the combined resources
of the various public and private organizations involved in studying com-
parative clinical effectiveness meet but a small and scattered fraction of
the demand. The centrality of the problem to the quality and efficiency—
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310 PATIENTS CHARTING THE COURSE
the viability, according to some—of the nation’s healthcare system may
require the creation of a new independent entity devoted to the work.
Create incentives for change: Economic and policy incentives to engage the
use of the best available evidence and more fully engage patients in the clini-
cal decision-making process include the alignment of purchasing incentives
accordingly when value is determined; use of the reimbursement power
of insurers and other financial incentives to generate new insights from
medical care (e.g., coverage with evidence development); and the linkage of
purchaser and payer decisions to performance incentives for best practices,
outcomes, and the better secondary use of routinely collected data.
Accelerate advances in health information technology: Health informa-
tion technology can facilitate the development of learning networks and
accelerate the generation of evidence, enable data aggregation and utiliza-
tion, deliver evidence to the point of care, and expand research capacities.
Coordinated stakeholder action—and financial incentives—should be able
to speed the progress toward universal application of electronic health re-
cords and access to information both on basic interoperability issues (e.g.,
standards and vocabulary) and, possibly, the development of more radical
data search innovations.
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